I’ve never used a fax machine. In fact, I hardly know how they work. So it’s been difficult for me to imagine my dad, Jack, sending his doctor a fax about his need for regular testing of his very long chain fatty acid levels in the early 1990s, years before I was born.
In the months before that fax was sent, my dad was diagnosed with adrenomyeloneuropathy (AMN), the later onset form of the rare neurological disease adrenoleukodystrophy (ALD). AMN causes dysfunction of the spinal cord, which results in walking problems, bladder and bowel issues, neuropathy and more. This diagnosis explained why my dad had been particularly clumsy and walked with a strange gait since his 20s.
What we know about AMN and ALD has changed a lot since the early 1990s. For starters, very long chain fatty acid levels do not necessarily correlate to disease severity as was once thought. More importantly, it’s now well known that over 40% of men with AMN experience cerebral involvement — meaning their brain is also affected by the disease — and in 20% it is severely progressive, leading to complete disability and death. When my dad was diagnosed with AMN, this was not as clearly defined.
My mom remembers learning about my dad’s AMN diagnosis when they began their relationship, but does not recall any doctors communicating risk of cerebral disease. In fact, she says he was told he may need a cane when he was older, but that was really all he needed to worry about. So when he began experiencing behavioral changes in 2001, no one connected this to his AMN. It wasn’t until months later that a counselor suggested he get an MRI of his brain to rule out a brain tumor. Only then did my family discover my dad’s brain had demyelination consistent with cerebral ALD. Two years later, he was gone.
Since I was in kindergarten when he died, most of what I know about my dad’s experience with ALD comes from the stories I have been told, mostly from my mom. A few years ago when my mom was cleaning out her office, she came across a folder with a plethora of documents that she used to keep track of everything ALD-related when my dad was still alive. There are many types of documents in this folder — faxes from my dad to his doctors, handwritten notes my mom kept detailing my dad’s symptoms, letters from medical professionals about my dad’s prognosis, information about bone marrow transplants printed from early 2000s search engines.
While I have always been eager to learn more about what my dad’s losing battle with ALD was really like, I didn’t open this folder until about a year ago. I knew there would be things difficult for me to read, especially about my dad’s dementia which made him act in ways that were extremely out of character. I also knew I would learn things I didn’t know before, or find out things that would challenge my long-held understanding of my dad’s experience with this disease. All of these were true.
It’s been almost 20 years since I lost my dad, and while time does not heal, it does make grief feel more like a dull ache than a stabbing pain. I don’t often get outwardly emotional when thinking or talking about my dad, but reading through these pages made me feel the weight of his loss that I haven’t felt in a long time. It also helped me paint a much clearer picture of what my family went through all those years ago.
Through these letters and notes I was able to imagine him as he was before ALD took everything from him, like hearing his voice in the way he wrote the fax to his doctor. Now, I understand my dad in a way I never have before.
As I got older I became highly involved in the ALD community — attending annual conferences, participating in Facebook groups, staying updated on the latest research and clinical trials. I have a vested interest in staying involved given the fact that ALD is an X-linked disease, meaning I inherited the gene variant from my dad. ALD most severely affects boys and men, but many women with the gene (traditionally referred to as “carriers”) eventually experience AMN-like symptoms. I have also become an advocate for ALD and all rare diseases, particularly on social media.
And about a year ago, I joined the team at AllStripes to help aid in their mission to unlock new treatments for people affected by rare disease. I’m grateful to work for a company that aims to help people in the rare disease community piece together the details of their journeys. I know firsthand how meaningful this information can be.
I’ve never used a fax machine. In fact, I hardly know how they work. So it’s been difficult for me to imagine my dad, Jack, sending his doctor a fax about his need for regular testing of his very long chain fatty acid levels in the early 1990s, years before I was born.
In the months before that fax was sent, my dad was diagnosed with adrenomyeloneuropathy (AMN), the later onset form of the rare neurological disease adrenoleukodystrophy (ALD). AMN causes dysfunction of the spinal cord, which results in walking problems, bladder and bowel issues, neuropathy and more. This diagnosis explained why my dad had been particularly clumsy and walked with a strange gait since his 20s.
What we know about AMN and ALD has changed a lot since the early 1990s. For starters, very long chain fatty acid levels do not necessarily correlate to disease severity as was once thought. More importantly, it’s now well known that over 40% of men with AMN experience cerebral involvement — meaning their brain is also affected by the disease — and in 20% it is severely progressive, leading to complete disability and death. When my dad was diagnosed with AMN, this was not as clearly defined.
My mom remembers learning about my dad’s AMN diagnosis when they began their relationship, but does not recall any doctors communicating risk of cerebral disease. In fact, she says he was told he may need a cane when he was older, but that was really all he needed to worry about. So when he began experiencing behavioral changes in 2001, no one connected this to his AMN. It wasn’t until months later that a counselor suggested he get an MRI of his brain to rule out a brain tumor. Only then did my family discover my dad’s brain had demyelination consistent with cerebral ALD. Two years later, he was gone.
Since I was in kindergarten when he died, most of what I know about my dad’s experience with ALD comes from the stories I have been told, mostly from my mom. A few years ago when my mom was cleaning out her office, she came across a folder with a plethora of documents that she used to keep track of everything ALD-related when my dad was still alive. There are many types of documents in this folder — faxes from my dad to his doctors, handwritten notes my mom kept detailing my dad’s symptoms, letters from medical professionals about my dad’s prognosis, information about bone marrow transplants printed from early 2000s search engines.
While I have always been eager to learn more about what my dad’s losing battle with ALD was really like, I didn’t open this folder until about a year ago. I knew there would be things difficult for me to read, especially about my dad’s dementia which made him act in ways that were extremely out of character. I also knew I would learn things I didn’t know before, or find out things that would challenge my long-held understanding of my dad’s experience with this disease. All of these were true.
It’s been almost 20 years since I lost my dad, and while time does not heal, it does make grief feel more like a dull ache than a stabbing pain. I don’t often get outwardly emotional when thinking or talking about my dad, but reading through these pages made me feel the weight of his loss that I haven’t felt in a long time. It also helped me paint a much clearer picture of what my family went through all those years ago.
Through these letters and notes I was able to imagine him as he was before ALD took everything from him, like hearing his voice in the way he wrote the fax to his doctor. Now, I understand my dad in a way I never have before.
As I got older I became highly involved in the ALD community — attending annual conferences, participating in Facebook groups, staying updated on the latest research and clinical trials. I have a vested interest in staying involved given the fact that ALD is an X-linked disease, meaning I inherited the gene variant from my dad. ALD most severely affects boys and men, but many women with the gene (traditionally referred to as “carriers”) eventually experience AMN-like symptoms. I have also become an advocate for ALD and all rare diseases, particularly on social media.
And about a year ago, I joined the team at AllStripes to help aid in their mission to unlock new treatments for people affected by rare disease. I’m grateful to work for a company that aims to help people in the rare disease community piece together the details of their journeys. I know firsthand how meaningful this information can be.
I’ve never used a fax machine. In fact, I hardly know how they work. So it’s been difficult for me to imagine my dad, Jack, sending his doctor a fax about his need for regular testing of his very long chain fatty acid levels in the early 1990s, years before I was born.
In the months before that fax was sent, my dad was diagnosed with adrenomyeloneuropathy (AMN), the later onset form of the rare neurological disease adrenoleukodystrophy (ALD). AMN causes dysfunction of the spinal cord, which results in walking problems, bladder and bowel issues, neuropathy and more. This diagnosis explained why my dad had been particularly clumsy and walked with a strange gait since his 20s.
What we know about AMN and ALD has changed a lot since the early 1990s. For starters, very long chain fatty acid levels do not necessarily correlate to disease severity as was once thought. More importantly, it’s now well known that over 40% of men with AMN experience cerebral involvement — meaning their brain is also affected by the disease — and in 20% it is severely progressive, leading to complete disability and death. When my dad was diagnosed with AMN, this was not as clearly defined.
My mom remembers learning about my dad’s AMN diagnosis when they began their relationship, but does not recall any doctors communicating risk of cerebral disease. In fact, she says he was told he may need a cane when he was older, but that was really all he needed to worry about. So when he began experiencing behavioral changes in 2001, no one connected this to his AMN. It wasn’t until months later that a counselor suggested he get an MRI of his brain to rule out a brain tumor. Only then did my family discover my dad’s brain had demyelination consistent with cerebral ALD. Two years later, he was gone.
Since I was in kindergarten when he died, most of what I know about my dad’s experience with ALD comes from the stories I have been told, mostly from my mom. A few years ago when my mom was cleaning out her office, she came across a folder with a plethora of documents that she used to keep track of everything ALD-related when my dad was still alive. There are many types of documents in this folder — faxes from my dad to his doctors, handwritten notes my mom kept detailing my dad’s symptoms, letters from medical professionals about my dad’s prognosis, information about bone marrow transplants printed from early 2000s search engines.
While I have always been eager to learn more about what my dad’s losing battle with ALD was really like, I didn’t open this folder until about a year ago. I knew there would be things difficult for me to read, especially about my dad’s dementia which made him act in ways that were extremely out of character. I also knew I would learn things I didn’t know before, or find out things that would challenge my long-held understanding of my dad’s experience with this disease. All of these were true.
It’s been almost 20 years since I lost my dad, and while time does not heal, it does make grief feel more like a dull ache than a stabbing pain. I don’t often get outwardly emotional when thinking or talking about my dad, but reading through these pages made me feel the weight of his loss that I haven’t felt in a long time. It also helped me paint a much clearer picture of what my family went through all those years ago.
Through these letters and notes I was able to imagine him as he was before ALD took everything from him, like hearing his voice in the way he wrote the fax to his doctor. Now, I understand my dad in a way I never have before.
As I got older I became highly involved in the ALD community — attending annual conferences, participating in Facebook groups, staying updated on the latest research and clinical trials. I have a vested interest in staying involved given the fact that ALD is an X-linked disease, meaning I inherited the gene variant from my dad. ALD most severely affects boys and men, but many women with the gene (traditionally referred to as “carriers”) eventually experience AMN-like symptoms. I have also become an advocate for ALD and all rare diseases, particularly on social media.
And about a year ago, I joined the team at AllStripes to help aid in their mission to unlock new treatments for people affected by rare disease. I’m grateful to work for a company that aims to help people in the rare disease community piece together the details of their journeys. I know firsthand how meaningful this information can be.
[Sydney] Good day to everyone joining us and welcome to today's X talks webinar. Today's talk is entitled the RWE ROI Series: The Transformational Value of Real World Data for Drug Development and Regulatory Decision-Making. My name is Sydney Perelmutter and I'll be your Xtalks host for today.
Today's webinar will run for approximately 60 minutes. This presentation includes a Q&A session with our speakers. This webinar is designed to be interactive and webinars work best when you're involved, so please feel free to submit questions and comments for our speakers throughout the presentation using the questions chat box and we'll try to attend to your questions during the Q&A session. This chat box is located in the control panel on the right hand side of your screen. If you require any assistance please contact me at any time by sending a message using this chat panel. At this time all participants are in listen-only mode.
Please note that this event will be recorded and made available for streaming on xtalks.com.
At this point I'd like to hand the mic over to Evelyn Pyper who will introduce our speakers for today's event. So Evelyn, you may begin when ready.
[Evelyn] Great! Thank you so much Sydney and good morning to everyone in North America attending and good afternoon to anyone joining from Europe or the EMEA region. I'm Evelyn Pyper and I lead Evidence Strategy at PicnicHealth and I am really thrilled to launch our picnic Health 2023 Webinar Series, The RWE ROI.
This overarching theme really came about from the fact that even though real world evidence has become well-recognized as part an important part of the drug lifecycle, many of the conversations that we've overheard happening about RWE, whether at conferences or webinars, still felt very surface-level and didn't necessarily convey the critical return on investment or risk of an action regarding real world evidence. Throughout the rest of this webinar series, each session will convene a unique panel of global stakeholders to explore the RWE ROI from a variety of perspectives.
[Evelyn] So today to launch things off we have a very distinguished panel of speakers to launch this webinar series. Our focus today is on the emerging and really exciting space of real world data for drug development and regulatory decision making.
To introduce our speakers I'll start with Jesper Kjaer. Jesper is Director of The Data Analytics Centre at the Danish Medicines Agency, and Co-Chair for HMA / EMA Big Data Steering Group. Jesper has 20+ years of experience in data management, analyses and data visualization, having previously worked in academia and the pharmaceutical industry.
He has headed up activities in EU Framework Programmes, TransCelerate Biopharma. Jesper is Co-PI of PHAIR: Pharmacovigilance by AI Real-time analyses, applying FHIR resources to healthcare data in DK for real-time safety surveillance. He is involved in EHDS pilot work and is an EMA DARWIN EU advisory board member.
Welcome Jesper we're really really pleased to have you. Thank you so much.
We also have with us today Noga Leviner. Noga is the Co-founder and CEO of PicnicHealth, a digital health company founded on the belief that empowering patients to gain control of their medical data is the critical first step to improving lives and health outcomes and driving scientific advances. PicnicHealth has pioneered the use of advanced human-in-the-loop machine learning algorithms to abstract key endpoints from medical records at scale with improved accuracy and efficiency. Noga is a vocal advocate for patients’ rights to control their own health data, having founded PicnicHealth in 2014 to help patients manage their medical records after personally facing the challenges of being a Crohn’s disease patient in the US medical system. She has spoken widely on the subject including at the White House. Welcome Noga.
[Noga] Good morning.
[Evelyn] Thanks so much. We will also have joining us today shortly Najat Khan, but I will save her bio for when she's on our call and will keep you on the edge of your seat for that one. So to kick things off, we want to you know, before we get into a deeper Q&A to really understand the perspective that each of our speakers are coming from, because we've intentionally convened folks that are coming from slightly different angles of this topic. So, a common thread that connects all of our panelists is really a dedication to ensuring that real world data is living up to its potential.
Historically we know that real world evidence has largely focused on more downstream post-authorization used but in line with today's theme each of you are contributing to important upstream applications of real-world data for pharmaceutical Innovation. To start off, I'll just ask each of you to share briefly, a less than five minute overview of the perspective you're bringing to this topic. And second, what your priorities have been over the past year. I will start with Jesper to provide your regulatory perspective
[Jesper] Well thank you. So I think it's important for me to start with saying that the randomized controlled trials or clinical trials are the gold standard right. We're not from a regulatory perspective stepping away from that. But we're realizing that real world data and evidence is really a very important supplement, and sometimes even we can observe it is probably the only real possibility. You often find yourself in situations where small populations, unethical situations that are not really suitable for randomization will lead you to using real world data to generate your evidence. But it's very important for us to look at this not as a binary situation, but something where each of them brings their own value.
Now there's a long history of actually using real world evidence. We may have called it something else in the past, like observational studies. Some of them more prospectively than secondary prospective data collection, more than secondary use of existing data. We have a reality around us that is changing where more and more health care data is born in an electronic way and that gives new opportunities and it would be an absolute waste not to use those opportunities to get better insights into the effects and side effects of treatments and also the value of medical devices. There's in particular the use around side effects obviously, for very good reasons. You will not be able to uncover every single possible side effect in the clinical trial. We can do as much as we possibly can, within the limits and reasonable design of those clinical trials to uncover that-but there will be situations where actually just running the clinical trials will put us in a situation where we're looking with a certain focus lens into the population that will ultimately be treated by the new medication. And for that purpose, over the years we've had the instruments of post approval effectiveness and safety studies that, in many cases, have been the real world data so to speak that have been the foundation for us to make further decisions and evaluations.
Now with these new opportunities, we are strengthening those capabilities by using that data for that. And we're starting to see examples where real-time surveillance after approval actually can be a real thing in the data pools that are available. But it's very important for these data sources that we're able to declare them, that we're able to describe them. I would assume that most data sources can actually be used, but they may not all be well-suited for the scientific questions you're going to look at with them. And-not going into the details of what distinguishes one data source from another-the whole understanding about that not every data source is equally useful, it's just very important to have at hand. And even with the best possible data sources that we know of that have been extensively used and demonstrated their validity and robustness over the years, it’s still very important we actually have the meter data in place to understand these data sources.
We basically need to understand what happened with the data as it was collected, stored, and handled so we can truly understand that what we're looking at is not an effect of circumstances with data handling or new policies but the real situation with the treatments we're looking at. And the real situation we would like to understand is in a real world setting: what are the situations-in all sorts of populations that are now getting the treatment-whether that's by choice of the treating physician, whether that's new indications, whether that is in some cases what we see that research is leading to an off-label use. We would like to understand that continuously, but more importantly, in the real situation of comorbidity and polypharmacy. Really understanding what we couldn't have-all possible outcomes in the clinical trials that we need the real world data to understand.
We'll also need the real world data up front to truly understand disease progression, standard of care, and all of that really is underneath of developing new medicines that can inform us in our decision process. Then I think what we are aiming at with some of the initiatives in Europe with the Big Data Steering group of building both a DARWIN EU Data Quality Framework, the training we're doing and also the data standardization strategy, is really to think about data quality and a standardized approach to this. One thing I think we still need to develop and think about is how can we actually not only make passive secondary use of the data, but really look at how do we move ahead to a Learning Healthcare System where prospective data collection is something that is going to be even more helpful so we ultimately can avoid some of the challenges with secondary use. In clinical trials we have the protocols to really define what to do and then describe whenever we deviate, and why do we deviate, so we can truly understand what is cause and effect in the data we're looking at, but we are not left without opportunity with the secondary use of the data.
That's why we are proposing the Data Quality Framework to better describe that. To have the metadata to understand the ability to use the data and the ability to have missing and wrong data and understand why did that originate. [This is] something we can actually deal with in a clinical trial and prospective protocol-related data collection as well. So that's something I hope will continue down that path, and I'm confident with the initiatives we have right now in Europe, we're going to see a lot of real world evidence being used in the decision processes and primarily fueled by the fact that as you establish the DARWIN EU system, you build the quality framework, and then you expand over the years with European health data space the infrastructure is there. The notion that this data will also be used for developing medicines, both in the pre-approval and post-approval phases, will lead to a different way of collecting that data and using that data for that purpose as well, which is something I'm very much looking forward to happening.
[Evelyn] Thank you so much. I think hopefully I can speak for everyone when I say that the model that you have developed is something I think many other regions would be smart to adopt. And certainly as I sit here in Canada, with our, you know, fragmented data ecosystem, it's encouraging when I hear of others trying to follow in the footsteps of the EMEA when it comes to this sort of data infrastructure. Before I pass things over to Noga to introduce her solutions provider perspective, I do want to introduce Najat who has joined us.
[Evelyn] Welcome to Najat.
[Najat] Thank you for having me.
[Evelyn] Thank you so much. We're really pleased to have you. So for everyone, Najat is the Chief Data Science Officer and Global Head of Strategy & Operations for R&D at the Janssen Pharmaceutical Companies of Johnson & Johnson. In this combination of leadership roles — unique in the healthcare industry — Najat is responsible for overseeing the Janssen R&D strategy, pipeline and portfolio optimization and investment, and for fully integrating data science and digital health end-to-end across the pharmaceutical pipeline to drive transformational innovation for patients.
Welcome to Najat. We're looking forward to hearing a bit more about the perspective you bring to this particular topic in a moment.
[Najat] That's great.
[Evelyn] Great. So Noga, I will pass things over to you, to tell us a little bit about the perspective you bring in terms of what PicnicHealth is and what your priorities have been over the past year.
[Noga] Sure, thanks Evelyn. I'll start with a quick introduction to the company. PicnicHealth is a digital health company that builds deep real world datasets. We are able to get this deeper data in two ways. The first, which I'm sure I'll end up talking about quite a bit, is just working directly with consenting patients. We take a very patient-centric approach, meaning we have a direct relationship with the patient, so we can really go get all the data that surrounds that individual, including from all of their medical providers, but also incorporating, you know, both historical and longitudinally going forward, but also incorporating their reported outcomes and points of view. So that's one key part.
And then the other side is actually from a technology perspective. Having the human-in-the-loop machine learning model that you alluded to earlier in the introduction, which combines the state-of-the-art machine learning with human curation. So while the patient relationship gives us that deep complete view, our machine learning combined with human curation allows us to then go really deep within the records and extract those key clinical endpoints, that really key information, that's in unstructured text in a way that's both high quality and meeting the quality standards-that I'm sure we'll continue to hear a lot about-but also that's possible to scale. That's scalable thanks to the help of the machine learning, so what you end up with is: a very complete, clinically-rich view of the patient, that's longitudinal both retrospective and then of course prospectively following the patient and being able to get at their patient reported outcomes. And because the data doesn't come from a particular site or type of facility (medical facility) we actually get really quite good representativeness because we are able to include diverse patients who have a variety of comorbidities, disease subtypes, and treatment histories. Because the process to consent and to participate is meeting the patients where they are, like in their own communities at home, it's a much lower bar to participate and sort of lower requirement than what you would see in a clinical trial.
So I'll comment and say that this data, in light of the comments we just heard Jesper make, I'll say this data doesn't replace broad data for something like surveillance. It doesn't replace that really broad, kind of shallower data for something like surveillance, but we see a lot of demand for this richer data, richer real-world data, as well coming from Pharma functions across the product lifecycle from R&D to Market Access, Med Affairs, Commercial teams. And of course relevant today through our work with our life sciences partners, we are increasingly seeing real world data leveraged to understand disease earlier in the value chain and optimize clinical trials.
And you asked about priorities so I'll touch on that quickly. In the past year we raised our $60 million dollar Series C funding which is fantastic. Going into the current environment, we are growing our research program portfolio, connecting with new patient populations, and always growing and continuing to build our industry partnerships.
[Evelyn] Fantastic! Thanks so much, Noga.
[Evelyn] And finally from the pharmaceutical perspective we have Najat. We heard from your introduction that there's quite a bit that falls under the umbrella of data science and R&D strategy. I'd love to hear about where your priorities have been in terms of real world data specifically and where you're coming from for this topic.
[Najat] Sure, that's great, Evelyn. Thank you so much again and to PicnicHealth for having me here. As you can see I'm trying to get better lighting, but hopefully you can hear me fine, which is what matters the most.
First of all, I want to say, both panelists, the comments are really spot on. You know, maybe just taking a step back from a pharmaceutical perspective and the perspective really focused on how do we make better medicines for patients, right? I mean that's, at the end of the day, the insights: things we can do, like leveraging verbal data, that we couldn't do before, or things we can do better. All are driven to that common purpose of translating it into insights and then a medicine that's for patients. I would say the way I think about real world data is not even just in clinical development. Starting from the very crux of the question that we're trying to answer, what's driving the disease, right?
I mean I think that's where it's really challenging. Like you know even today how much we understand about disease biology. If you think about so many diseases, Alzheimer's, Parkinson's, they're not named after the driver or the causal effect of whatever is driving the disease, but much more so based on the physician that identified the syndrome. So that in itself tells you that there needs to be a deeper understanding of disease biology. So the way I define real world data is of course claims and EHR, but you also have omics, like transcriptomics, genomics, and images, everything that's not clinical trial data. So if you think of the totality of understanding a person's journey, what drives disease, you need to look at the totality of data. And so we are actually leveraging that from the very first part, which is better understanding the drivers of disease and redefining disease, as a result of it.
So as an example, a lot of the audience probably knows about these longitudinal data sets-for instance, Noga was mentioning too-called UK Biobank or Our Future Health, I mean there's so many. But essentially it's longitudinal. You have all of these different data points collected well, good data quality, and what that has allowed us to do is to be able to stratify people that have severe depression to patients that have severe depression based on their sort of genotypic and phenotypic architecture. So really driving precision medicine more into an area like depression, which let's face it, the medicines that we have today work but we could do better. I think we can all agree that we could do better and that insight is then translating into how we design our programs for depression. I wanted to use depression as an example because we talk a lot about precision medicine and oncology but we need to bring that in Immunology and Neuro and so that's just one example.
Another way that we're using real world data a lot is, let's say you figure out what's the target that's causing that protein and you figure out the molecule you need to actually modulate it. The next thing that comes up is how do you run the right trial and design it the right way. And Noga spoke a little bit about this as well, which is, instead of relying on publications or instead of relying on multi-center sort of analyses of the patient's journey, you could now use real world data across the board. Across all of the patients that actually have a disease, then go back and say, "What were the risk factors that were driving them?", and that actually helps you design the protocol in a much more effective way. So you can do certain things such as inclusion/exclusion criteria, you can simulate it using real world data to say, "Okay, is this actually reflective of the patient journey in the real world?", and "Why is that important?".
It’s actually because it's important when you start recruiting the patients. You can't operationalize something if it's not pragmatic in terms of what's truly happening in the real world. And we've done that, like for one of our vaccine programs. For instance, we used 100 million patient lives-de-identified of course-working with external partners and then really understanding what are the risk factors that they'll get this certain infection. And that actually was then translated into the protocol design itself, working with our clinicians and now that study is running in Phase 3. It helps helped us enrich the trial a lot more with the right patient that would be impacted, which is the patients, but it also helped us ensure that a trial is much more efficient so we can get to the data that we need and then hopefully the therapy that we need, knock on wood, for patients as quick as quickly as possible.
And maybe the last example I'll mention is also just a lot of the viewers here probably saw some of the guidance on external control arms that came out yesterday from the FDA. Super helpful, because the way we think about real world studies broadly-and ECA is a part of it (external control arms)-is you can leverage that to really contextualize what's the right standard of care, how are patients doing in the real world with the physician's choice versus the active arm of a trial.
We're not just thinking about it, we've actually leveraged this for our regulatory submissions, working very closely with the FDA. Early pre-specification conversations are super important, so you're getting feedback on your protocol, on the data elements being super transparent about the pros and cons, all of that. The thing I want to emphasize is no data is perfect and no guidance is perfect. There's always a case-by-case discussion, but this is where early discussions and collaborations and transparency and rigor in what's done becomes supremely important and that's something I want to underscore. Like in my organization, we have the highest highest bar because there's a lot of real world studies, but to do it well takes a totally different level of talent and we'll talk more about that and capabilities later.
And then last thing, but not the least, is also the space around what I would say is clinical trial recruitment. We're using real world data to understand where the eligible patients are and then go to the patients, open our sites where the patients are verses the other way around. And we've seen tremendous impact in terms of both how effectively we can recruit, but then also ensuring that our trials are representative of the patients, that we will treat from a demographic perspective, from a diversity perspective, etc. Using decentralized trials and so forth.
So as I have this monologue for the last couple of minutes-and thank you everyone for bearing with me-the point to underscore every single aspect of how we figure out what was causing the disease, to designing that all-important trial, to executing that all-important trial, to generating that all-important evidence would becomes critical for access that ultimately helps to get our medicines to patients. Every single aspect of it is being disrupted positively using real world data.
[Evelyn] Thanks so much. Yeah, all really salient examples. And I know you mentioned that it felt like a real treat yesterday, that the day right before our webinar, the FDA released the guidance on external control arms. I think it's just reflective of month-by-month, things are developed. Like over the past year, the amount of guidance that's come out, the amount of real action around providing industry guidance, but also guidance to companies like ours, like PicnicHealth, where it's really showing this is not just a nice to have anymore, it really is something that the expectation is high quality real world data as part of an evidence submission.
[Najat] And Evelyn if I can just maybe underscore one thing that you mentioned. You know all the examples I mentioned, it's not something that we just do internally, right. Yes, we build the algorithms, the methodologies internally, but the datasets and the partnerships, we have accelerated a lot because of the work that, you know, companies, young companies like PicnicHealth, and others are doing. It's a lot of hard work. Let's just put it this way. To take a lot of the data that's fragmented, not always very clean, not standardized as guidance is coming and pulling that together, creating the cohorts-Noga was saying curating a lot of the work in the structured and unstructured datasets-that all becomes extremely important. So, it's a partnership across regulators, startup companies, and also pharma and biotech companies. It all has to come together. We couldn't do it without each other. It's exciting to hear the examples, but the foundation of it is the work that's being done across the board. So I just want to acknowledge that.
[Evelyn] Thanks so much. Really appreciate that call out. And maybe we'll shift gears from our big picture, all the things that we're all tackling, into a bit more of a slightly personal fun question. So a career in this space of data strategy, data science, applications of advanced analytics really requires a unique set of strengths and capabilities that each of our panelists possesses. Arguably these skills are valued and transferable across virtually every sector today. So question for each of you: Why healthcare? Why you do you find yourself here in healthcare using your skills for this type of mission versus you know financial sector or other technology sectors?
I'll start things off with Najat. We'll bounce back to you.
[Najat] Sure. Why not healthcare? I mean, listen, I'm a bit biased maybe. A quick backstory, not to bore anyone, but you know I grew up in Bangladesh and then I grew up in the UK. I came to the US when I was 17. So really lots of continents in a short period of time. And the thing that always moved me from early on, and it helped that my parents were both physicians, is the fact that when you see somebody get better, person, animal (I'm a big animal lover), that feeling is unmatched.
Like I even get goosebumps as I think about when somebody actually, it's not just the patient, you see the impact on the family. Early on, I recognized that was something that would just get me out of bed, like that's my passion. The other thing I also recognized was that the pain and suffering when you actually have a disease, whether it's for the family or the patient, that's common, across any country, whether it's a developing country like Bangladesh, or you're in the UK, in the harshest area, or in the US and some of the communities that are underprivileged.
And the equity in terms of health care is something that we're not where we need to be. So I think I knew early on just based on that. It's just something that's unwritten. I also growing up did a lot of work around non-profit work going to the fields that I remember I was excited about it. My dad was like if you're excited about something just go to the entry level and just experience it right and it was tough and I didn't know how to speak the language and there's so much, but the barrier and the kindness and the empathy that comes from it. I just think it's not something, anything I could feel in any different sector. And then COVID just puts it on another level right. Like the reality of it, as we saw, that was the most important thing. Health became the most important thing and so just fast forwarding.
Then the next question was how do you impact and you mentioned data is so important but I mean-I don't know Jesper, Noga maybe can comment on it too-like 20 years ago this wasn't even a very well established field. I remember when I was doing my undergrad as an international student which means you have to keep your GPA super high or else it's a problem because I was on a scholarship and stuff anyways. I did all of the sciences like physics, chemistry, bio. I also did computer science and I also did econ. So it was just this weird plethora of different disciplines and I remember so many people asked me “Najat, why? Do you just like pain? Why are you doing all this stuff?” And I was like well, you know, I could see the computational aspect would become more important as more data is generated and just like things that we cannot compute in our minds right and science was at the core of what I cared about: science and medicine of course.
The reason I went to a liberal arts college and did econ, I recognized when I was growing up that unless you take all those great ideas and make something out of it like a business that's sustainable you can't really have an impact in a sustained way for patients. So that was the confluence of it. It came like a very organic way and when I talk about it retrospectively it sounds so cool, but honestly at that point I was gonna say, "I'm interested. I think this is going to be interesting". So, then when I did my PhD at Penn I was very lucky. I had a crossroads. I could pick an advisor that was a very core organic chemistry, like you know publish, publish, publish, and then I had another advisor who was new not, tenured, so higher risk but was much more into interdisciplinary approaches. Long story short, my PhD was a confluence of working on physical modeling on what molecules might work and there are so many colleagues in the computer science department that helped me. I learned from them. Then actually coming to lab and making it with my own hands as an organic chemist. So I was developing molecules for detecting early cancer. Early detection of cancer and also therapeutics and then actually working with folks in the Penn med school to translate that into in vitro and in vivo work and that was a very sort of non-traditional PhD but it was very application-focused and had a PI that actually was like, "okay if you do good work, if you don't we're gonna have a conversation." So the reason I'm saying all this for folks that are watching is it's not something I decided on but I think as you progress, now thinking about data science and AI, machine learning, RWE is great, but the question is what do you apply it to. So understanding multiple disciplines of what you applied to that is going to actually have an impact and not just doing it because it's a cool algorithm or a cool methodology is super important if your core mission is to have impact on patients. So I'll just say I could do it in finance but I don't think I'd be this excited if I did it.
[Evelyn] Well thanks so much. I’ll pass things over to Noga. Why healthcare for you? I know Najat just mentioned creating sustainable companies and that felt like a really good segue to pop it over to you.
[Noga] I think in some ways I think my experience parallels yours, Najat, but actually it's kind of the opposite because I got into this a little bit later in life. I actually also will just share my personal story. I got into Healthcare and started and founded PicnicHealth because I'm a patient myself and until I had the experience of being a patient, I wouldn't have thought of applying the skills that I had built over my career to healthcare. I think it's like so trite, like so obvious that it goes without saying, people always say like if you don't have your health you don't have anything, but when you're in the situation where you're actually dealing with that there's just like no substitute. There’s nothing more obvious than if you don't have your physical, your mental well-being, you don't really have anything else. I was actually a patient and I was diagnosed with Crohn's disease in my 20s and it was just looking around me at the landscape. It was just so obvious that if we could act like from the experience of being a patient and how divorced I was from the research space, how hard it was for me and frustrating it was for me to get a hold of my own data that if we could just bring patients, that there was a sustainable business to be built that would benefit patient and so clearly benefit other parts of the healthcare sector.
I think the bar for improving things in terms of the healthcare experience for patients and ability to participate is sadly so low. There's so little that's happened. The world is changing I think things are getting a bit better, but I think at the end of the day being here in Silicon Valley, you feel like there's people who are like spending their whole careers optimizing l some little thing, like a little button and then you kind of look at the experience patients are having in healthcare and the way we're using data in our system and it's like these broad swaths of opportunity. For anyone who's had any kind of personal experience you can just see that there's like massive massive opportunity for really big shifts in impacts that you can have. That's really kind of palpable.
[Evelyn] Thanks so much, Noga. Jesper, how about you? How did you find your way into healthcare?
[Jesper] By failure actually. I failed studying veterinary medicine back in the 90s and then I realized while I couldn't really learn another anatomy textbook by heart, it was during the whole internet development and taking the IT route was just interesting. Just by chance, one of my colleagues said I have this database at one of the hospitals that needs a little bit of help, "Can you go and meet up with them?" And then ended up in a research group for the next 13 years. It turned out what we did there was really impactful. There was observational data on a global scale. The DID study was actually funded by industry through the FDA to look at adverse drug reactions for HIV treatment. Obviously that suddenly became really really meaningful as you had that opportunity it also gave you an opportunity to suddenly realize how closely connected the world is in this.
I remember some of the work we did. We actually went to the NIH in Washington and presented to Anthony Fauci about data models and what to do on a global scale for a network that still exists today. And then getting that interest of industry, and now the regulatory world. I just got soaked into it and couldn't let go of it. To me observational data, the real world data, has been part of my work for the past more than 20 years now, so not really getting out of it. It just seems to be coming back in new wrapping. And more of it by the way. So a lot more data than in the past.
[Evelyn] Well I think we're all glad that you're here, that the three of you are in healthcare…And probably we won't let you leave because there's more work to be done! Jumping back to you Jasper,I saw you speak about DARWIN EU at a conference last fall. It was fantastic. Since then, I saw that the EMA announced the first eight data partners selected as part of the Phase I of DARWIN. These seem to include both public and private institutions from across Europe including Spain, Netherlands, Estonia,Finland, France and the UK. From your perspective, if you could distill down, in the time that we have, what are the most important considerations for assessing or selecting a data source or partner whether it be for the purpose of DARWIN or more broadly?
[Jesper] I mean DARWIN in particular actually has a connection office that proposes this to EMA and The Advisory Board as well. There are lots of factors to be considered, maturity of the data source, the quality management system of that, the whole procedures, and ultimately, at the end of the day, also that you can demonstrate scientific value from that data.
Then this is a network of data sources, your capability to connect with the common data model and then interact in a collaboration around this is obviously also important. I think there's a number of factors, but if you would take one down, is really the scientific question driving the selection of the data sources because we need the relevant data sources to the scientific questions we actually see in the regulatory work. So, you'll see a bit of dynamic around this as we expand into primary or secondary particular disease registries as well. We're talking really rare disease. We have some of the reference networks in Europe we can utilize in a different fashion but it's really true to make sure we get the appropriate data sources to the scientific questions and then obviously data quality and Quality Management Systems around that matter.
But ultimately we are fortunate to have many data sources where we can pick from. So, I think the choice is really to give everyone room and then see it develop over the years to come.
[Evelyn] Great, thanks so much! Najat, given you have global oversight, certainly at any given time I imagine you're thinking a lot about what's happening in Europe, what's happening in the U.S and elsewhere. In terms of data systems, historically we're often very confined to these geographic barriers and I'm curious if you're seeing promising progress in how real world data can be used or is being used today across diverse geographic contexts? Any examples of that?
[Najat] I mean, look there's definitely progress being made with different approaches in different places. Maybe I'll break it up into two ways. One is true regulatory grade real-world data and then there is real-world data non-regularly grade. I think for the number two bucket there's quite a bit of progress being made, especially in regions like Brazil, in the LATAM region, in China, and others as well. Sometimes people ask me, "Is that even important?? It actually is. It depends on what question you're trying to answer. If you're trying to answer the questions around-Noga mentioned this, Jesper mentioned-understanding the patient journey. Understanding it early on. What's the standard of care there? So it helps you for the design. I was saying the protocol in a much more thoughtful way with a global mindset, which is definitely critical for us. Also if you're trying to recruit patients, just understanding which sites patients are going to. From a site collection perspective there's a lot of opportunity. I think Noga said, in terms of the bar there's a lot to be done. I always say the flip of it is only 10% of what we make has success. So, there's huge room for improvement. I think from that perspective, especially with LATAM, also some of the Gulf Countries, we are starting to see that they're actually trying to aggregate a lot of their data sets, connect it, link it and so forth. Maybe not at a national level, but much more at least regional levels or key hospitals. So, it's a start.
Let me think about more globalization and having more diversity in our trial, that's definitely helping, so that's one example. I would say from a regulatory grade perspective it's still really…. like I was mentioning before, a lot of the work in the U.S. I think a lot of the funding that's really gone on from VCs and private equity funds, etc. Congratulations Noga on the recent raise. It's really helping us, in the last few years, push to having higher regulatory grade rich data. Because at the end of the day a lot of it comes down to the exact question you're trying to answer.
I think in Europe, Jesper can say more than I can, but with DARWIN and a lot of the other networks it's definitely getting there as well. Ex-U.S, some of the other regions, I don't think they're at that regulatory grade yet. If I can be just really frank, there are some changes, like in Singapore, there's some really good data sets from Taiwan, but large countries because I think the step one is just getting everything together. Then next step two is that next layer of maturity of actually making sure that your data quality, privacy considerations…. Alot of the guidance is also still evolving in some of those regions as well. So try to be practical about the answer but it's definitely moving in the right direction. But what I want to emphasize is even what's happened today, it can be really really useful in terms of how you develop it.
[Evelyn] Definitely. Shifting gears to Noga. I think you might have a unique perspective on this coming from PicnicHealth. Are you seeing anything today as someone who's working with life sciences partners that suggests that these attitudes around data crossing borders are changing or should change?
[Noga] I'll leave the whether they should change aside for the moment, but I will say that I think we're starting to see some encouraging signals of progress that there is for openness to using real world data across regions. At the core I think people recognize that, more and more, every data point counts and you just have to find patients where they are.
Among our broader portfolio of therapeutic areas we work in we really see this the most in rare and ultra rare diseases, where every data point, every patient counts. It’s particularly important and what we've found is that what's most important is the ability to be flexible to adjust to what matters in a particular region or context, to be able to get that really complete picture. Then we found that being able to, with low activation energy, go back in and change the data model to meet what's important in a particular region or context, customize it to align with what they're looking for, it really does a lot to give comfort rather than just having a static data set that has to be used in the way it was designed for another region.
Ultimately I think sponsors are using our data to support evidence packages and regulatory submissions both in the US and increasingly across borders as well.
[Evelyn] Thanks, and from this PicnicHealth perspective, as Noga knows, we're often working with real-world data and evidence champions from their respective organizations as opposed to you know the naysayers or maybe traditional trialists. The challenge I think in PicnicHealth is less about convincing folks the value of real world data and these various novel applications, but more aboutarming our main champions with the information. That'll help them have broader internal buy-in.
[Evelyn] So a question, we'll start with Jesper and then move on to Najat. You both champion initiatives that can be considered innovative in their use of data and data sciences. What's your experience been with navigating and securing internal buy-in for real-world data projects and fighting that good fight for real-world data?
[Jesper] Often when it's maybe outside the status quo, I think what has really been the driver with us has been a strategic approach from the top and then downwards. There's been something that we decided to put into the European strategies, in the Danish medicines obviously also for the past 10 years in our strategy and then I think it obviously helps if you're in an environment like the Danish, where doing registry-based research to inform how you do healthcare and treatment and so forth is just baked into the way you do things.
That has been a major factor in making that change and then through demonstration projects, step-by-step to actually do this in our procedures and our processes. To bake in the use of data in a different way. That's why we established the Data Analytics Center, to have a core function of expertise that would collaborate with the other skills in our agency to use real world data in decision making processes. Whether that's in our pricing department, whether that's in our safety surveillance, whether that's in our approvals, even to the point of our HR system, we can actually do stuff with data analytics matters. Just bake that in everywhere, that starts to change the paradigm. That's the same mechanism with the other agencies in Europe and it's the same mechanism within EMA. It's really something that we from a strategic point of view, have decided to do and that really makes the change.
[Evelyn] That's great. Najat, you've probably seen changes over time but today, are you still needing to convince the value of real world evidence? Are we past that stage? Can you push the boundaries a bit more around what we're doing then with that real world data and evidence?
[Najat] What I would say is, right now, where we are there are a couple of things. I think it's very similar to what Jesper said. It started very much with, will real world data actually replace RCT? That was the big thing if you think like four or five years ago. I think that was almost as over ambitious an aspiration which was not based in reality. The reality is clinical trial data is extremely important, but we can always say there's more data that you need to have more holistic evidence generation. It’s an “and,” it’s not an "or".
So step one was just to shift that at least, in my experience and like Jasper said, it started from the top. Our head of R&D just had great foresight in terms of the impact of data science that can have end-to-end. Then our CEO, Joaquin Duato, I mean he has said the combination of science and data science technology is going to be what's really propelling us in the future. So, I think having that foresight investment support from the top. Then the other thing we also did which was really important is building a team because to know what good looks like and to actually do great work and to play in a professional arena, you have to have professionals at the end of the day. So, we built it as a function, as a new business unit that actually reports through me to the EC eventually.
The thing that was important as I built out the team, was to get that grassroots because you don't want just a top-down for a large organization. You want the grassroots support as well. You need to have demonstration projects, back to what Jasper was saying, but focus in areas that are actually going to make a difference and in a time frame that's not five years. That's why we started in Clinical Dev and not Discovery because they're taking so long, just being super practical. Everybody can get behind that question, like all different groups, but the other part was actually having bilingual talent that understands clinical science. A lot of them have backgrounds in oncology, neuroscience, and so forth, but also folks that are appointed experts in real world data, AI, machine learning, and so forth. That creates a common bridge in languages so that you can actually have a discussion at the grassroots level. How would you solve a lot of these problems? For where we are right now, tremendous progress. We started building this team two and a half years ago. It feels like much longer than that. We have data science fit-for-purpose use cases across every single clinical program, so it's totally done at scale today. You'll get the odd questions, "Do we need real world data?", but we have enough demonstration projects where it started off as, "hey let's keep it for an optionality perspective," but then it becomes really important as you go towards regulatory submissions.
The other thing is it's been done with great care and rigor, not just internally but with our external partners and also with great SMEs. I also always have the team actually review it with third-party SME, so it's like you never want to be drinking too much of your own Kool-Aid. I always say you always want people to look at it. Like maybe this is a grad student in me and I’ll always poke holes into everything because that makes the ultimate output better.
There's a lot of support, but there's always priorities in an organization so it's continuous. It's not ever done. There's always priorities and if there's one bad real-world study that comes up, everybody regresses and that's what we have to be really careful about. If it's not done the right way, same thing with AI algorithms and so forth. That's why I always say we have to work together as a team, internal and external.
[Evelyn] Well thanks so much. I imagine, too, as the suite of capabilities that real world data can be applied to is ever-growing, I'm imagining that there's always going to be that iterative conversation around, "Five years ago we weren't using real world data for X, but now we are." So, how do we make sure it's the quality and the output is what we need to see?
On that note, because we're coming down for time, and we do want to save time for questions from the audience at the end, I'll ask for maybe a rapid fire round of responses from our panelists on this last one. With all these advancements in data science and digital health, it's transforming how we're using real world data as all of you have spoken about today in clinical research and in clinical care. If you had to pick one data or digital development that has you really really excited for the year ahead because of its potential to transform our understanding of a disease what would that be? Noga, I will start with you.
[Noga] That's a great question. I will say having now worked in the sector for 10 years, I think almost every single year actually when I got into this…. I was re-watching a video the other day that I had put together when we were just starting 10 years ago and my co-founder and I were saying, "there's been a regulatory change and within a matter of a year, patients are all going to have access to all their data through APIs." This complete transformation is happening and that was basically the last time that I was optimistic about that. I learned my lesson very quickly, but there have been these glimmers of hope along the way that were actually happening, that there was progress. That regulatory change is going to make a difference and I think the reality is, we have learned not to be overly optimistic. And yet I actually think what's happening right now and the changes that 21st Century Cures Act is bringing on is incredibly powerful and exciting. It's really like we're going to be shifting the balance of power to ensure not only that patients can get their data but also that they're really empowered to choose how to share it.
Ultimately, what that means is fewer barriers to access for patients and much more real-time data, which I think not only matters for patients and their own direct experience navigating the healthcare system, but also for the kind of life-saving clinical research that they want to participate in and choose to contribute to.
[Evelyn] Great and I think a good segue to Jesper, in terms of a regulatory example from Noga. What has you excited as a regulator yourself about this year ahead?
[Jesper] Oh no doubt, the scaling of DARWIN EI into our regulatory processes is the biggest. But what I'm also seeing is that in our own little country, we are taking learnings from Covid-19 and the use of real-time surveillance to the next level together with computer scientists and building artificial intelligence machine learning techniques on top of that because ultimately the next pandemic may unfortunately just be around the corner. So, if there's an opportunity to learn from that. I'll see some of that effect this year already and we're looking forward to having a kind of a new tool available in our toolbox.
[Evelyn] Great! thanks! And Najat, how about you? I'm sure there's lots that has you excited for the year ahead, but what would be the standout for you?
[Najat] What I'm really excited about is the fact that thanks to ChatGPT, which still needs a lot of work, AI is actually becoming much more mainstream and people are starting to get that this is here to stay, which I think is actually, if done the right way, is really good for everything we're talking about. Because I think one challenge that comes up is sometimes-I'm just thinking for the broader audience-it's hard to understand how all of this is going to change healthcare and the details and the data quality and everything that we rightfully talk about.
I think once you can actually use it and you democratize the use of something and you see how interesting and cool it can be, then the underpinnings of it which is generative AI, generating new thoughts, images, videos, whatever….Think about the future, even happening right now, you can generate many different structures, protein structures, biology. You can generate you know different chemical structures. Because at the end of the day, the English language, or whichever language it might be that ChatGPT uses, in chemistry and biology you also have a language. Whether it's the amino acids in biology and in chemistry it's the different atoms. At the end of the day, I think you're going to see an arc. It's going to take time, to Noga's point, it's going to take time because there's going to be a lot of ups and downs but that gets me excited because that's new novel insights of molecules and structures that we can create and ultimately think about. Even protocols we write, you can actually generate that if you have the right data set so you make things faster so that our clinicians or data scientists can focus on the right areas. That gets me excited because I think it's just making it a little bit more tangible for people and if done the right way, I think it can also progress a deeper understanding and appreciation for what we are all collectively trying to do in healthcare
[Evelyn] Thank you and yes it feels like there certainly hasn't been a time where there's been more dinner table conversations around AI and certainly Covid helped with that. I think everyone now understands…
[Najat] Yeah can I just say Evelyn, it's so funny my dad, who you is pretty engaged, he tells me the other day, "Hey I'm doing this project proposal for this non-profit,:- he does a lot of non-profit work-and in that one of the core focus was AI machine learning. I was like I'm so happy to see that. This is women in underprivileged areas ensuring that they get the right care early on and Telehealth and all the stuff. Then I said to him, "I'm so glad to see that." He's like, "Why, it's mainstream. Why are you surprised?" And I was like whoa that's somebody that would have not said that a year ago. That was a personal moment for me when I heard him say that.
[Evelyn] Amazing! Yeah, valuable data point for us to have as we're all in it 24/7 to get that like oh actually there's diffusion into everyone's lives at this point.
[Evelyn] I know we're at time. I would love to keep talking to our panel for another hour or more but we are at time and I do want to just still give the opportunity for any attendees who are able to stay on the line that wanted to ask questions or hear the responses to some of the questions you have. I will pass things over to Sydney at Xtalks to help us wrap up with that session.
[Sydney] Thank you very much, Evelyn and thank you all for that very insightful presentation. We have time for maybe a question or two.I've already received a few from our audience, so I will start with those.
Our first question is “How to evaluate the method to deal with missing data from RWD?”
[Najat] I will have to drop, but I can quickly answer that question. There are many different approaches in terms of looking at missing data, for automating. First thing is definitely take a training data set and then you look through it and say what are the parameters or elements I'm looking for. There's quite a few methodologies that have been already published, for instance by Sebastian Schneeweiss at Harvard and others. There's actually an approach that we use that framework to say, okay what are the key elements, whether it's demographic or some of the things, endpoints that you're looking at for that question. Always remember the intended use and then we see what is the missing data. Then there's a threshold of how much missingness you can have. Generally we don't go with one dataset, we look at two, three different datasets. A lot of the time, what we do, it's an iteration. We work with our partners back and forth in terms of where we need to curate more, where we need to fill gaps and in parallel we're also having conversations with regulators just to say, okay we have ORR but progression-free survival, there's this XYZ is lacking.
So, it's not like a static approach and I think that's the beauty of it. With almost anything in science, it's pretty iterative but that's what I would say. It’s a lot of what we're doing, there are frameworks and methodologies. I don't think there's one that everybody uses, you can also use different ones, specifically for oncology versus some of the other disease areas. I'm sure Noga and Jesper will have many more great insights. Great to see everyone! I will have to drop. Thank you so much for having me, again!
[Evelyn] Thanks, Najat! Thanks for coming. Anything to add from Noga or Jesper?
[Jesper] A quick one from the regulatory perspective. I appreciate the references to Sebastian's name and others and assimilation of how to do that. That's definitely a useful approach to consider. I would just like to point to the fact of the Data Quality Framework we have released where the implementation of that towards real-world data is something we'll do as a demonstration project over the next half year. So, maybe a space to watch to better understand the regulatory perspective around data quality and missing data. Something to watch out for.
[Noga] And I'll just say very briefly that I think because we work directly with the patient, our approach here is pretty different. We actually make an effort to look at the individual patient and, using claims data, understand every interaction they've actually had in the healthcare system. Then we can literally go chase those down one by one and actually ensure that we have that really complete-I think people are calling it "past complete" picture. You're not going to do that on a national scale or continental scale I should say, but I think when you're working with smaller patient-centered cohorts it is actually possible to chase that information down and get to completeness, at least in set windows of time that you can define.
[Sydney] All right thank you all for those answers. We'll get to one last question. So, earlier the panel mentioned the importance of representative RWD. This audience member would like to know if any of the panelists could speak to promising ways that they are seeing RWD being used to study diverse, often underrepresented patient groups and sub-populations.
[Noga] Yeah, I can speak to that. I think…oh man I hate to say this….but as I mentioned earlier in another context that the bar is so low and I think this is another place where we see things like, at PicnicHealth, the cohorts that we build, we have this very deep patient-centered data compared to running an observational study that you have to recruit into or compared to a clinical trial population. You are asking some effort from patients to sort of sign up and make a contribution but it's a five minute sign up that patients can do from their home. So you're not getting that same barrier that you would see for example in clinical trial participation. I think when we see those representative, more diverse datasets, that the reality is that sometimes I think, for example our work in MS, it's just kind of shocking how little people understand about what the disease even looks like in non-white populations. We know black women are more affected than other populations. We know their disease looks really different, has different outcomes, but I think we're just starting to piece together what that even looks like. For better or worse, I think the reality is that sometimes datasets that give access to a look at diverse populations end up just being about the baseline of getting to the core understanding you have about a disease for a broader population and making sure that extends across the reality of the whole population.
[Sydney] All right well thank you so very much for those answers and for this presentation. We've reached the end of the Q&A portion of the webinar. If we couldn't attend to your questions, the team at PicnicHealth may follow up with you or if you have any further questions you can direct them to the email address on your screen. So thank you everyone for participating in today's webinar. You'll be receiving a follow-up email from Xtalks with access to the recorded archive for this event. The survey window will be popping up on your screen and your participation is appreciated as it will help us to improve our webinars.
Now please join us in thanking our speakers Evelyn Pyper, Noga Leviner, Najat Khan and Jesper Kjaer. We hope you found this webinar informative. Have a great day, everyone.
Sydney Perelmutter:
Good day to everyone joining us and welcome to today's Xtalks webinar. Today's talk is entitled Real-World Data By Design - Incorporating Different Data Types Into Clinical Trials. My name is Sydney Perelmutter and I'll be your Xtalks host for today. Today's webinar will run for approximately 60 minutes. This presentation includes a Q&A session with our speakers. This webinar is designed to be interactive and webinars work best when you're involved, so please feel free to submit questions and comments for our speakers throughout the presentation using the questions chat box, and we'll try to attend to your questions during the Q&A session. This chat box is located in the control panel on the right hand side of your screen. If you require any assistance, please contact me at any time by sending a message using this chat panel. At this point, all participants are in listen only mode. Please note that this event will be recorded and made available for streaming on xtalks.com.
At this point, I'd like to thank PicnicHealth who developed the content for this presentation. PicnicHealth is a healthcare technology company that partners directly with patients to build deep real-world data sets. The company leverages state-of-the-art machine learning combined with human curation to port complete medical records into an easy to use online application. The platform gives patients unprecedented access to and control over their medical records and, with their consent, the opportunity to contribute this valuable data to further scientific research. Now I'd like to introduce our speakers for today's event. As the head of Analytics Innovation and Digital Health, Gaelan and his team are responsible for creating and developing innovations across R&D. He co-leads the BMS digital innovation pillar for global drug development, which is enabling a spectrum of digital solutions, including several types of digital trial capabilities. In past roles, Gaelan has led and developed strategic partnerships with large academic medical centers and networks. He has also supported trial design and startup for the BMS oncology and immunology programs.
Next I'd like to introduce Sneha. Sneha is a certified PMP with over 15 years of industry-specific project management, risk planning and mitigation and regulatory experience spanning all phases of the drug development life cycle across a variety of therapeutic areas. As the Global Lead of the RWE Innovation Pillar of Decentralized Studies, her focus is to define the corporate innovation strategy for this pillar through strategic review and landscaping of industry trends and customer needs, engage key stakeholders and guide the ideation process to define and refine the existing innovative and strategic portfolio for decentralized studies. In her nearly decade-long tenure at IQVIA, Sneha has been responsible for providing senior oversight to cross-functional teams across a wide variety of therapeutic areas and study designs with a special focus on rare disease registries and post authorization safety studies.
Andrew Larsen is the VP of Partnerships at PicnicHealth and an industry leader in creating fit-for-purpose real-world data solutions. At PicnicHealth, he works with partners across the life sciences ecosystem to help advance disease understanding and support the development and access to innovative medicine for patients. Prior to joining PicnicHealth, Andrew worked with partners across the life sciences ecosystem to help advance disease understanding to develop portfolio and asset strategies across all stages of development with a specialization in evidence generation needs.
Lastly, Evelyn Pyper is Evidence Strategy Lead at PicnicHealth, a patient-centric real-world data company. Her career in real-world evidence spans the public and private sector as well as regional and global markets. Prior to PicnicHealth, she worked as Associate Director of Market Access at J&J Global Public Health, focused on securing access to HIV treatments in Sub-Saharan Africa, and as RWE Manager of a diverse portfolio of partnerships and research projects at Janssen Canada. Evelyn has a Bachelors of Health Sciences with a minor in Psychology from McMaster University and a Master of Public Health degree from Queen’s University. Now without further ado, I'd like to hand the presentation over to our speakers so you all may begin when ready.
Evelyn Pyper:
Wonderful. Thank you so much, Sydney. Good morning or good afternoon from wherever you're joining us today. We are really pleased to welcome you to this second webinar in the PicnicHealth 2023 webinar series. When the vision for this series was coming to life, it centered around two core ideas. The first was recognition that the conversations around real-world evidence really needed to move past these very didactic sessions on what is real-world data, what are its challenges and opportunities into more kind of topic and context specific conversations. Second, the question of who are the people from across different organizations and perspectives that we'd want to ask to sit down for a coffee and pick their brain on these nuanced topics. With those ideas, the webinar series was born and I'm really excited to have with us here today Sneha, Gaelan, and Andrew to share their unique perspectives on the use of real-world data and innovative approaches in clinical trials.
Evelyn Pyper:
With that, we'll dive right in. As all of you know, the overall theme for this webinar series is “RWE ROI”, which is really about going beyond the return on investment of RWE to really considering what's the risk of inaction, the risk of not doing something, when it comes to use of real-world evidence. On that note, if we reflect back on how clinical trials for years have been done in the past versus how they're starting to evolve to be done today, we can all start to appreciate how much might not have been possible if trials had not evolved to incorporate new designs or new types of data. My question for all of you, and I wouldn't say it's a softball question to start off by any means, is what comes to mind for you when you think about how has the use of real-world data or decentralized approaches opened up new possibilities for clinical trials? I'll start with you, Sneha. What comes to mind when you hear that question?
Sneha Kishnani:
Thanks, Evelyn. It's a very interesting question. What comes to my mind is access to additional patient populations. When we think about decentralization of different data collection approaches–patients that may be very ill, patients that may be in rural areas, not necessarily having access to care–allowing and enabling data collection to happen in their workplace or in their home enables additional patients to be part of that research, and we can then strive for representation and diverse representativeness in the clinical trials that we're after.
Evelyn Pyper:
Great. Thanks so much. Gaelan, what about you? What comes to mind when you think about what's possible today?
Gaelan Ritter:
Yeah. I mean, Sneha's right. I think a lot of it is that kind of the flexibility has opened up access, so clinical trials were a very closed world in the past, and you needed to be in a very specific place in your life and in your finances and in your disease state to be able to participate and making things more flexible, adding the opportunities to participate in the kind of ways that work for you as a patient has really changed that dynamic and opened the aperture of who's able to actually participate and how they're able to participate and the amount of effort that it is to be in a clinical trial. I mean, clinical trials have always been and probably will always be more difficult than standard of care to be a participant, but at the same time, that barrier is coming down a bit over time with these new technologies, which is something that makes it more palatable to be a part of a trial. It's really been a nice kind of, it's leading to nice opportunities for patients coming in. Lot of work still to be done, but it's heading in the right direction.
Evelyn Pyper:
Certainly sounds hopeful – feels like the theme there. Andrew, what about you? Anything else to add?
Andrew Larsen:
Yeah. Well, first off, very excited to be on a panel with yourself, Gaelan and Sneha, and looking forward to the conversation today. I think both the points they brought up are absolutely critical. How do we get more patients involved in trials where the actual data is then going to be much more meaningful to really all downstream stakeholders, regulators, payers, providers, and ultimately patients where it's going to be more reflective of the populations that are going to receive the treatment at the end of the day? I think there's another sort of component when you think about the challenges that have existed for trials. They take a lot of resources, a lot of time to pull them off and there's always been this bit of a bottleneck where it sort of comes to inflexibility where every...you know I'm sure if there are ClinOps people on this call, I'm sure they all have their own horror stories with a dozen amendments involved and it's really hard to do, but it's often critical because the understanding of diseases change, the understanding of the population, the treatment, what you're trying to actually answer.
A big part of real-world data is how do you actually create a system with these trials which allows for a lot more ability to evolve what the data can be pulled in to answer new research questions as it changes. I think there was just a JAMA article or publication last week that basically said one out of five phase III trials in oncology changes their primary endpoint. I'm sure we could spend time discussing that data point the rest of this call, but I think it really does speak to the fact that research questions change–and how you bring in additional data sources, without trying to pivot the Titanic, to actually answer those questions and really optimize your trial from the start, to have that flexibility baked in.
Evelyn Pyper:
Thanks so much. I'm all ready to dive right into things, guys. We're off to a great start. Thinking about all the different ways that real-world data may now be leveraged for clinical trials–from early informing study designs to capturing additional outcomes to even serving as an external control–I'm wondering, Gaelan, where are you seeing the greatest need or opportunity for real-world data today, if you had to prioritize?
Gaelan Ritter:
Yeah. I think in terms of need and opportunity, it really goes back to what Andrew's saying, so I think data acquisition. We've done a lot of work in patient finding and enrollment and trial design. I think that data acquisition is the next kind of frontier for real-world data. The way we collect most of the data and studies today using EDC and capabilities like that is something that's 25 years old at this point, in terms of a technique, and it leaves enormous burden and gaps on patients and sites to be able to participate in that activity.
To Andrew's point, there's no flexibility built into that system. So you need to–I think with real-world data–taking that next step, working on the acquisition side, incorporating more complete medical records into the clinical trial record, images, scans, genomics, wearables and other data sources that we see coming through, that are honestly becoming standard practice in certain diseases, and clinical trials are taking a longer time to catch up unfortunately. Rather than leading new technologies for data acquisition, trials are lagging in a lot of instances, and so I think that's the space where we really need to start accelerating the work in real-world data to be able to bring trials to parity and then kind of push beyond the techniques that you're seeing in standardized practice. It's going to lead to a lot of opportunities to get more information and more learnings out of the studies we run and also make them more efficient for everybody that's involved.
Evelyn Pyper:
Right. Thanks. Yeah, certainly sounds like many of these approaches, they're innovative in the context of clinical trials but not innovative in their own right necessarily. Sneha, from your perspective at IQVIA–which we know is a large organization, with a wide variety of solutions and teams working in this space–does what Gaelan shared align with what you're hearing from customers across the board? And given your title, which is Global Lead, RWE Decentralized Studies, can you speak to some maybe emerging solutions that are increasingly needed in today's trial world?
Sneha Kishnani:
Yeah. I am hearing what Gaelan is saying. The lack of flexibility is creating burden across the whole system in essence. Not only at your sites with your patients, but with your sponsors. With respect to the emergent solutions, I think participant centricity is critical. Optionality is critical. I just attended the DTRA decentralized clinical trials conference back in mid-April up in Boston, and the consensus was unanimous. There needs to be simplicity in what we are doing and it's really complicated and confusing for all of the stakeholders involved. It doesn't need to be, so I think we need to get back to our roots. We look at what is the core of what we're looking to collect. How do we simplify that for our sites and patients through these methods of different types of data acquisition?
It could be, as Gaelan mentioned, wearables and other devices, but also with respect to the point that sites are not going away, so how do we simplify things for our sites? Really putting our stakeholder needs first. There's more and more use of technology. What we're seeing at the sites now, what we're hearing from the sites is that, "Hey, you're throwing six different platforms at me. I've got a different login for each one." There's a movement in the industry towards things like single sign-on and leveraging things that other industries are doing so well. It's finally coming into our space. But I think we need to continue to move in that direction and bring that kind of rich customer experience to healthcare.
Evelyn Pyper:
Yeah, absolutely. Similarly, Andrew, as VP of Partnerships at PicnicHealth, I'd say you have your finger on the pulse of pharma customer needs and how they might be evolving. Can you share a little bit more about the work specifically that PicnicHealth does for those that aren't aware, the types of use cases that it might support, and what sort of interest you're hearing from customers related to real-world data use in clinical trials?
Andrew Larsen:
Yeah, absolutely. To start at the highest level, PicnicHealth is a patient-centric real-world data platform where we work with consenting patients to create longitudinal complete real-world data across their journey, and that means operating in a site-agnostic manner. So wherever they've received care in the U.S., we're able to procure the full set of medical information from that facility, including medical records, physician notes, labs, imaging, and create a harmonized data set that's deidentified for researchers and also present that back to the patient for their own benefit and really empowering them to be part of their care journey as well. That same connection that we have with the patient is used to also collect primary data in the form of primary or patient reported outcomes where we can actually create a much more holistic view of the patient experience and what they're going through. I think to Sneha's point, a huge part of this is really you can't add 15 different options for 15 different data modalities, and really how do we streamline this process to collect the information that we need that's critical for the study with the least burden on all the stakeholders in the process?
That's really been something that PicnicHealth has strived to do – starting with the patient, where essentially it's really just the sign-up that's the lift on their part to actually create these data sets for researchers and how that's evolved to actually apply to the trial space, so there's obviously a suite of needs, but maybe [I’ll give] just to do two examples. From day one when a participant enrolls, we're able to collect a deep longitudinal history on that patient, such that you can really have a nuanced view of really what is the stratification between all the different patients in the study and tie that back to what the variations and outcomes that you see are, and this is nice to have for a lot of studies, but really critical when you think about the ultra [rare] or orphan space where there are places where you may not be able to actually find enough patients to run a comparator arm and there's no actual natural history that exists. So actually using that either directly, or as supporting evidence for the comparator of those trials when they actually take an investigational therapy, is critical.
Then on the back end it's all about following patients, whether it be due to the burden of the study, they are a potential risk of lost to follow-up, or additionally, it's something where it's like after the site component has ended, how do you continue to capture what happens in the real world? This is both, I think, incredibly important when you think about both regulatory and especially payer applications. When we think about the wealth of accelerated approvals, we've seen that tied to surrogate endpoints. Really, how do we capture the outcomes that are happening in the real world later on? Then additionally, I think there's an aspect here where when you think about physician assessments that don't occur in the real world. Having a single data set that ties together the physician assessments that you were able to administer as part of this study and actually tying that to the real-world outcomes you can see, to extrapolate what these implications are–in ways that HTA bodies or payers may understand the value that this therapy brings to patients in a way that’s more apples-to-apples that they're used to seeing.
Evelyn Pyper:
Thanks so much. I'm a big fan of clarifying terminology, especially in this space when we have lots of solutions, lots of solutions providers, and we hear a lot of terms like patient-centric, patient generated, patient mediated. Andrew, can you clarify what these mean and how you would look at the differences between each term?
Andrew Larsen:
Yeah. Good luck getting a clear definition of patient-centric out of anyone, so I'll tackle that last because that's probably the “squishiest”. But starting with patient-mediated. Patient-mediated is really about when you're working with a consenting patient and that ability to unlock the medical information that they can contribute to research. That's sort of the gateway into that category, though I think the actual spectrum of outcomes from a patient-mediated approach really is contingent on what is the infrastructure and technology that you're using to unlock the information once you have that patient opting in. And I think really PicnicHealth has been built around from day one: how do we take this burden off the patient of capturing this longitudinal journey, and both giving that back to the patient but also enabling researchers as well for that information? Then, patient-generated data, probably the most straightforward of all these. Data that you're generating through engaging a patient, whether that be wearables or patient-reported outcomes.
Then lastly, for the patient-centricity, I think this is something where, as I sort of alluded to, I'm not going to create the dictionary definition, but I can at least talk a little bit about how we think about it internally, where we are an organization of patients as well, so we try to abide by a system that is really built [around] putting them first, and that means starting with them consenting into all of our studies and they have that control over their information. It's also something where we try to–for all the information we collect–we present back to them for their own benefit. Then, also the fact that really our ability to engage and work with patients to make sure that we are capturing what's needed to advance their disease condition, it layers in their input as well through that ability to engage them and collect information, whether it has to do with symptoms, outcomes, quality of life. That's a key component of our operating system.
Evelyn Pyper:
Awesome. Thanks. Maybe that can become the definition? We'll see. We'll try. We'll see what we can do about putting that in the dictionary.
Andrew Larsen:
Best of luck.
Evelyn Pyper:
Yeah, thanks. Whether we're talking about patient mediated data or other approaches, I think it's clear that having these options and solutions come into the trial space, it's kind of like a double-edged sword because you have people trying to make decisions on the best approach, but with lots of choice comes lots of questions around like, ‘when is the right fit for each of these things?’. It can be overwhelming, especially for those that are not sitting on the solution side, but really having to make a call with a suite of options in front of them. If we think about patient-mediated approaches, tokenization approaches, or traditional site-based approaches–starting with Gaelan, from your perspective, when do each of these make the most sense or not make the most sense?
Gaelan Ritter:
Yeah. I mean, you make a good point about it kind of being part of the planning exercise, so it really is looking at—we're kind of building out decision trees of when you use these and you kind of maximize the robustness of the data that you acquire, for the least burden on patients and sites that you can acquire it. So when you look at things like patient-mediated and some of the work with PicnicHealth, it's really been a nice opportunity to get large data sets with significant medical history, significant other physician interactions and patient journey without huge burden on patients and sites, and so using capabilities like that primarily is going to be the future of clinical trial data acquisition. Then you go to: okay, I can't get everything from that, so now I'm going to need some things where it's patient input into–whether it's a wearable or an ePRO, eCOA, whatever else–I'll need patient interaction for the next step of things, which reduces some of my burden on the site, but the patient's going to have to be involved.
And so in large studies with hard outcomes that sort of exist in claims data sets, that's usually still sufficient, but there's a lot of places where I think patient-mediated becomes more relevant when you're considering places like needing coverage across the population. And that coverage is very important to incorporate things that are in the unstructured notes or deep within the clinical text, including things that may have to do with imaging, that may have to do with patient-reported outcomes, or may have to do with those sets of information. And then linking to other things as well that may exist in like cost information, like claims for economic analysis. To say probably the most overused phrase on these calls, it has to be “fit-for-purpose” at the end of the day. So the decision trees you sort of teed up earlier, it's like figuring out for what you need, what is the right suite of solutions.
Then your final step is going to be the more traditional approach, where there's just no other way for me to get this, so the patient's going to have to go to a clinical site, the site's going to have to perform the activity and record the information, and that kind of is the one you want to use as sparingly as possible going forward because it's most labor intensive. I think in the middle of the kind of patient-mediated and the patient-directed and then the traditional site is also going to be where the decentralized trial capabilities come in. That's where you're going to see things like someone going to a local lab for diagnostics, or going to a local imaging center, or going to a general practitioner, rather than going to a clinical site in a major city for their clinical trial. That's where you're going to see some of that middle ground of how we can leverage things that are closer to a patient's home. There's still medical clinics, so there's still burden there. Closer to a patient's home, more convenient to make this more approachable for everyone. It's really just a continuum and you really just try to maximize getting the most robust data you can with the least burden you can possibly do it.
Evelyn Pyper:
I like that. Andrew, would you agree with what Gaelan said? Would you add anything? What are your thoughts?
Andrew Larsen:
Yeah. Absolutely. I think Sneha brought it up even earlier where I don't think the site-based component is going to go away anytime soon. To Gaelan's point, it's very laborious, but it is so critical for a lot of having those controlled settings, being able to administer whatever assessment you want, whatever sort of imaging study, having that controlled environment. It's more about what are the ways that you can create the greatest wealth of data, like balancing that with other modalities in addition to that. I think tokenization is a great way to get a lot of low hanging fruit information, and if that is what you need to answer your research questions, fantastic. It's really about stepping back and being like, what do you need this evidence to show? If there is a tertiary data set that you can link to that has that outcome, just making sure you are taking the consideration: When you think about the waterfall of how many patients will actually be able to match? How many patients will have the temporality coverage that you need? Additionally, if there's a secondary or third data set that you need to link as well, applying that subsequent downstream waterfall from those criteria as well.
Evelyn Pyper:
Right. Thanks so much. Sneha, where do you see the most challenges with traditional site-based approaches? Alternatively, when do other approaches make the most sense?
Sneha Kishnani:
Yeah. It's a good question. I mean, thinking very specifically, we can think about rare disease studies–rare disease, ultra rare disease studies–where it's difficult to bring up a site across a geography, a specific country or region, and each site can only enroll 1-2 patients a year because that's all they have. The industry addresses this by targeting specialty centers where there's a larger population of these types of patients, but it's not a perfect solution. I mean, this is where I think the decentralized approaches can really help allow patients to, like Gaelan was saying, get their labs done through the local lab, imaging done through their own imaging centers near their houses.
I think the other thing that we also need to take into consideration is that the geographical landscape of all of the solutions that we're talking about, sometimes the regulatory landscape of that is the regulators are a little bit slower to adapt to some of these solutions that we're talking through, so that also needs to be considered. And in that decision tree that Gaelan was talking about, consider what is the relative risk if we are to take an innovative approach or an innovative data collection acquisition solution here, and is it something that the regulators could maybe be open to? So having those connections and having those pre-discussions with the regulators is also critical in this case.
Evelyn Pyper:
I'm imagining that…. there's a lot of external factors as you mentioned, so even with a very well-thought-out, comprehensive real-world data strategy from a researcher, from industry researchers, the factor of timing is also always going to come into play. So Company X has an urgent need for a data set, they need evidence in six months from now. Probably not an uncommon request for some of you to get. Sneha, how does the timing of evidence generation planning come into play in the work that you do around decentralized trials and what, if anything, needs to change in order for folks to see greater success with these approaches?
Sneha Kishnani:
We've all been there, right, where like you mentioned, there's an urgent need to fill a data gap and we need the data yesterday. So the pre-planning, the early planning, the sooner you can get the right players in the room to start talking through that evidence generation and evidence even dissemination, the better. Before your Phase III study begins probably is the right time. Even earlier is better. But I think that's really it, I don't know that I need to be verbose about this, but early is good, earlier is better. I think that there are all kinds of issues with the lack of data being available. There's cost concerns to the sponsor. There are concerns around product uptake during launch. It creates all kinds of knock-on effects, so if we can be proactive about those conversations, we can avoid some of that and we can make the whole research gathering process a bit smoother.
Evelyn Pyper:
You made a really good point. It's not just about ‘early’, but it's about getting the right people in the room early, which can also be part of the challenge. So like within a biopharma organization like yourself, Gaelan, I know we're seeing this cross-functional decision-making happening and thinking about how data can be used to serve multiple groups. From your perspective, Gaelan, does having related questions coming from across multiple parts of the organization impact what data sources or approaches you use, and if so, can you share any examples of some common cross-functional real-world data needs?
Gaelan Ritter:
Yeah. Absolutely. It certainly impacts it. I mean, the reality is that high quality, large real-world data sources are still rare in a lot of instances, so there's a huge amount of cross-functional need for these data sources, and some obvious examples are going to be ones that no one's surprised by. Long-term patient safety and efficacy is an easy example. That's an important endpoint for clinical trials. It's an important factor for the kind of progression of trials from Phase I to II to III. It's also hugely important for the patient safety monitoring teams and for the health economics and outcomes research teams and for your field medical colleagues. You see everyone asking the same question, and in the past, one of the things we found with normal traditional clinical trials is that the trials weren't designed to answer the questions of some of the teams that came downstream. It was designed to answer the questions of the specific person designing that specific trial.
To the points that everyone's made, that world is a little bit gone in the sense that everyone now recognizes that there's other users for these data sets inside pharmaceutical companies, and the need is to really expand the capabilities by acquiring the data that you need in the different locations and then leveraging that across everyone involved in the pipeline of that assets journey. So, patient safety is an easy one. Patient journeys. I mean, Andrew mentioned tokenization and the value of linking the claims data sets and other things. When you look at patient journeys for years, those have been used in outcomes research and in those types of capabilities, and we're seeing more and more of patient journeys creeping into the kind of earlier development cycles of assets. You're thinking about: How do patients experience the drug that I'm creating? How's it going to change the treatment pathway? How's it going to impact the way medical practice is handled in some of these settings, especially in rare diseases?
One of the keys is better understanding that patient journey. Understanding the journey that exists today. Understanding the journey that's going to be created from the asset that you're hoping will be successful, and if you're seeing a lot of cross-functional use. It's great you're seeing teams communicate with teams that they never did before because they're realizing that they share common interests. And that also means that then on the real-world data side, you're seeing demand for data sets that can accomplish, that can serve multiple people's goals. And that might mean incorporating data into a clinical trial data set that wouldn't have been there before, or incorporating some of those patient-reported outcomes into a later-phase study that wouldn't have classically done them because it's not the primary efficacy endpoint for the trial. But being able to get those learnings means that everyone can cross-use some of those data sets.
Evelyn Pyper:
Right. Thanks. I mean, curious, do you think that we'll get towards the kind of ‘unicorn’ data sets, where they are serving all stakeholders? Or do you feel like this is aspirational at the moment?
Gaelan Ritter:
We're getting close. I think it's going to be a while until you really get there. I don't think your phase four safety studies are going away tomorrow. I think we're getting closer to being able to answer the questions of multiple teams, but there are still pieces missing at different journeys. And I think a lot of that goes to Sneha's point about, unfortunately, as you're building out the depth and completeness of real-world data sets, you're also realizing where your gaps still are and you're realizing how often you rely on some of those traditional clinical techniques in your trials and in your data acquisition, and so we're getting closer and then also realizing just how big the gap still is. I think there's a lot of room left to make improvement there, but absolutely it's moving in that right direction.
Evelyn Pyper:
Yeah. I guess the more we learn, the more we know what we don't know and still need to fill. As we are moving towards more of this kind of personalized healthcare vision, precision medicine, we know that the reality is that decisions–whether it's regulatory, payer, HTA decisions–might not always be made with the same types of coded data that we've seen in the past, so across all types of studies, clinical studies, post-launch studies, there's a growing need we know to go deeper with the data. Andrew, I'm wondering if you can share some examples of how these emerging–and maybe not so novel anymore, but in some ways novel–patient reported, caregiver reported, or clinician documented data are being used in clinical research today?
Andrew Larsen:
Yeah. Absolutely. Maybe starting with the clinician-documented. Coded information is usually the backbone of a lot of go-to sources, but I think there's a growing appreciation for the sort of wealth of information that is buried in the physician notes underneath the just general coded information, where it's really all about understanding what is either under coded, non-coded or inaccurately coded. And so that can be things about just understanding what the patient population is, subtypes, disease severity. It can be about understanding outcomes for that, so either physician assessments, major sort of signals of activity, disease activity, symptomology, as well as treatment use and effectiveness including mentions for treatment switch or discontinuation. Really I think this is a backbone of a lot of the different studies that PicnicHealth engages in, and I think it's….
There are a lot of places that we expected the value to emerge, but there's even some of the sort of less ‘cookie-cutter’ value adds have even been about understanding the sort of inefficiency of the coded information. So we published in lupus nephritis about the ability to actually more accurately diagnose around 100% more patients by not just relying on ICD-10 codes alone. But going by additional information across the medical record, including narrative text, as well as the shortcomings of relying on those patient populations that are coded today in hemophilia B, where at least 40% of them had an inaccurate hemophilia A diagnosis, a third of which didn't even have any mention of hemophilia B. I think it's actually truly understanding that the patient and their outcomes really relies on going deeper within that information, and then the ability where you're actually talking about a patient-centric platform is like, how can you bring the input of them and their care team into this equation? Which is nothing new for the field, as this has been growing steadily over the past decade where the majority of regulatory submissions now include some component of patient-reported outcomes data, whether it's about symptomology, functionality, quality of life, and those are increasingly making their ways into the actual labels of products.
The way that sort of we think about another unique application of it as it relates to trials is you'll have these assessments done that are patient-reported as part of the study, like for actually understanding bleeds at a very frequent rate is key for a lot of hemophilia studies. And what we've tried to recreate in our real-world data studies is biweekly bleed patient-reported outcome assessments to create the ability to track these outcomes that are never really assessed in the real world. Like you can go in the documented physician notes, but they'll never be as comparable to what you have going on in the trial, so I think that's another major advantage as you sort of look to more holistically understand how trial populations compare to the real world.
Then additionally I would say for the caretaker, this has been a growing focus of PicnicHealth over the last year. A lot of the major areas of therapeutic development are beginning to appreciate that the impact that the therapy brings is not just on the patient, it is on the entire care network of that patient. We are doing caretaker-reported outcomes in Alzheimer's, Huntington’s, a lot of pediatric conditions where it's about: What is the burden that this is bringing on as a societal for the entire care network? How do we actually characterize that? Because this is part of the holistic value that it's actually bringing to society, of here are all the other people afflicted that are absent in almost any data set you look at, that actually, there's a huge way for these advancements to bring relief to the sort of families in addition to the direct patient afflicted by these conditions.
Evelyn Pyper:
Thanks. We're getting a lot of great questions in. We want to make sure we have time for the audience Q&A, but I do want to wrap up with a prospective looking question. We've talked today about how changes and how drug therapies are developed and studied, continues to evolve, and to wrap up this main portion of our Q&A, can each of you share one example of something that you think will look very different in, let's say, five years when it comes to the use of real-world data or alternative approaches in clinical trials? Like what's your five year outlook? We'll start with Sneha.
Sneha Kishnani:
Five year outlook, I'm going to maybe answer this question with a little bit longer time horizon, but I think patients are becoming more knowledgeable and more empowered about their data and I think patients are really the linchpin here in terms of getting access to data. If we can continue to educate them on the value proposition of the data that we are using of theirs and the methods that we're using, and then return those results back to them in a way, I think that five years from now we're really going to see an unlocking of all of the types of data, and to Gaelan's point, “closing the canyon” of the gaps that we're seeing. Maybe in five years. Maybe the time scale is a little bit longer on that, but I am generally hopeful that knowledge is power and it is going to lead us to an overall healthcare system situation of patients being able to be managing their illnesses better, one, but then also leading to just a healthier population. Because I think if, I'll just tell you if it were me, if I am getting information back and insights back about my health and my health status, if I can make changes to improve my health and my life, I'm going to be making those changes, so I think that there's something there.
Evelyn Pyper:
I love that. Andrew, what does your crystal ball say for five years from now?
Andrew Larsen:
Yeah. Also love that. Clearly biased as our entire platform is built around empowering patients with more of their medical knowledge, so very excited to see that growing as a focus about how we make patients more front-and-center of their own care journey. Not to sort of echo what Sneha and Gaelan have already brought up, but I think integrated evidence planning, while not new right now, is really growing. And I think hopefully less and less there are going to be people showing up at mine or Sneha's doorstep asking for a data set that doesn't exist and needing it yesterday, as there's sort of this more forward thinking about cross-functionally of what we need. I think that's going to play out in more initiatives to future-proof the study by having part of the consent forms ease ability to collect longer term real-world data associated with these participants.
I think sort of the aspects of the planning around it may shift as well. If you think of just more of the broader landscape across drug development, there's obviously some legislation changes that are going to shift a little bit about the focus and urgency that's around the timeline that you need evidence–to not only get approval, but once that approval is there, how do you maximize what you can communicate to all of the stakeholders involved in the care journey? What is the evidence you can show up to payers with day one vs. one year vs. two years–that we used to have a lot more flexibility for that timeline for. Which shows the tangible benefits of this therapy in ways that they can understand. And also bring that same pace to patients and their providers to make sure that they can make the right decision of whether this therapy is a choice worth considering. And so I think there's going to be a lot of evidence planning that is really about how do we ensure that all the stakeholders in the continuum here are going to have the evidence that they need much faster than we've historically considered what is permissible?
Evelyn Pyper:
Yeah. I like that too. Gaelan, final word, what does that five-year future look like from your eyes?
Gaelan Ritter:
I love both of the ones that came before. I think those are exactly on point. I guess for me, the other thing that I might add to it would be I think our kind of patient recruitment tactics and patient enrollment tactics are going to look totally different in five years. I think the advent of using patient journeys and claims data and new world EHR sources to find patients in their medical records and be able to identify best-fit patients for trials is emerging so quickly that I think in five years it's going to become the dominant way that patients are identified for clinical trials.
I think the days of, well, when I was a clinical research coordinator at Georgetown, the days of sitting there on Friday afternoons going through all the charts of the patients coming in, going through many charts of patients coming in next week as if I could get through all of them to find patients for trials is going to be over. And it's going to be kind of targeted lists using real-world data and other data sources and algorithm development to be able to create target lists for sites to be able to really understand what the flow-through of patients is, who's best fit for the trials, and really being able to triage that out is going to drastically change the landscape of how sponsors function and also how sites function in that kind of enrollment space.
Evelyn Pyper:
Thank you. Yeah, certainly the possibilities have me excited. I think it has people in the chat excited. We have some great questions coming from the audience, so I will pass things back to Sydney to facilitate this next, final part of the session.
Sydney Perelmutter:
Thank you all for that very insightful presentation and I would like to continue to ask our audience to continue sending in their questions for the Q&A portion of the webinar. But I've already received many questions, so I'll start with our first one. Our first question is, what are the current challenges in reconciling different RWD collected using a variety of recording techniques and technologies within RWE studies?
Gaelan Ritter:
Who wants to go first? I can jump in with some of the challenges that we see, I guess.
I think one of the things we see is that disparate data acquisition sources can have different….it's not that the data's conflicting, it's that the data's collected with different intent, so if you had a patient-reported symptom versus a clinician reported symptom, those are going to look different. Even though they might be getting to the same underlying part of the disease, they're going to be represented differently and we see that across different types of real-world data. So you'll see a very different kind of catalog of information from a patient-entered source versus a site-entered source. We see the same thing when you start looking at the kind of linkages between datasets that have different intended end uses. So a claims data set is going to represent a disease state very differently than an EHR dataset because they're intended for different purposes.
We actually see a lot of data that needs more, rather than cleaning, it needs concordance. And so we see that a lot more often now as you're starting to, like Andrew mentioned, starting to tokenize and link different data sets that had different reasons for existing. You're starting to kind of see the emergence of a lot of need for harmonization across those, and that takes a little bit more sophisticated effort than just a mapping table in controlled terminologies. So I think that's something that's emerging more for us as we start to get into these more complex, compiled real-world data sets.
Andrew Larsen:
Yeah. I think that's all right. And what Gaelan teed up earlier where it's like data acquisition is one of the challenges. I think the first step for PicnicHealth at least, a lot of because we collect medical records across any care facility, rural PCP versus like high-end academic institution, and part of what we have really put a lot of thought and energy to is like, how do we actually harmonize these data sets, these different disparate data sources into a single usable data set? I think as we've overcome that challenge, the exact next one is sort of concordance and it's like how do you reconcile all of these different pieces of information with different intent? I think our sort of fallback is there is no silver bullet. It's very much like, what is the question you're asking and what are the pieces of information that should be indexed on to best actually reach a defendable conclusion? I'll stop. I'll end it there and I'm sure there's probably a lot left unsaid there, but happy to discuss further if interested.
Sydney Perelmutter:
Thank you for those answers. Another audience member would like to know, what are your thoughts and what are you seeing with sponsors using registry or open access patient journey data as quote unquote "placebo group" for open-label studies?
Gaelan Ritter:
I'll start this one too, I guess. One of the things…so we are seeing more and more of that. You're seeing a lot of [this] especially in rare diseases. If it's hard enough to find patients to participate in the trial and treatment arms of some of these diseases, being able to find patients to participate in control arms is next to impossible. And especially we're seeing more and more that obviously clinical trials are becoming a therapeutic alternative, especially in rare disease and in late stage disease, and because of that, in those assets you literally don't have many patients that aren't part of clinical trials. We're seeing a lot more use of those data sets both in the classic kind of external control space, but then also seeing them being used as–whether it's digital twins or other types of capabilities that are emerging–to be able to provide insight about the patients on the treatment arm of the trial without having to classically enroll like you would for other trials.
You're seeing that emerge in a lot of ways, whether that's in hybrid control models, synthetic models, and like the digital twin model. It's actually been very beneficial though, to be totally honest, because getting drugs to patients that are in rare diseases is critically important, and the timing is so frustrating because delays in those–there are no other alternatives for those patients–so delays in those asset journeys literally impacts people's lives, and so being able to kind of build bodies of evidence through new statistical techniques has been a real benefit to a lot of those patient groups.
Sneha Kishnani:
Yeah. I mean, I can add to this one. We do see this all the time with respect to disease registries as well as natural history of disease studies that are also being run in addition to any kind of broader registry. I think that there is an acceptability around it and it's happening more and more.
Andrew Larsen:
Yeah. I would just say I think at least for the last three years, every year there's been at least one drug approved with this sort of approach, so I think while regulatory guidance still always indexes on preferential sort of the ability to have a classically randomized controlled trial with a placebo or standard of care arm, I think this is something where when you actually…you have to consider on a case-by-case basis is like, is this truly a landscape where the sort of unmet need and the actual feasibility considerations for a trial justify this? I think we've consistently seen an openness to that, which is great for patients. I think then there's getting that initial access to patients, whether it's an accelerated approval potentially, and then there's the follow-on confirmatory studies, which can take a very long time, right? But I think usually there's that consideration where it's like, let's do the right thing now based on this body of evidence that we feel comfortable doing it, and then confirmatory studies as appropriate for any outstanding questions.
Sydney Perelmutter:
Thank you. Again, we have a question for Gaelan specifically and then we'll do one more question. That question for Gaelan is, can you talk about innovative ways that can help to identify the best participant for a research study?
Gaelan Ritter:
Sure. Absolutely. We're doing a lot more with using access to EHR records and to claims records to be able to map where patients are. Some of that is kind of just looking at population dynamics and heat mapping and then taking the next level and deploying algorithms that actually search medical records for patients that meet not only the I/E criteria of the study but look like patient groups that we've seen in previous trials that would benefit from those studies. So it's really kind of about leveraging that EHR data access and the real-world data sources that we have and being able to create algorithms that look for those best fits. It's a lot of the work that the clinical research coordinators and investigators do manually today, which takes an enormous amount of their time, and so being able to automate some of that and present them with those best fits has been a real advantage and a real opportunity for all of us.
Sydney Perelmutter:
All right. We have time for one more question. We've talked comparisons and differences across patient mediated vs. tokenized vs. traditional site-based approaches, so can you all share some examples of if or how these approaches can be used together?
Evelyn Pyper:
I guess maybe a question to that question that I would put out there is, if the spectrum between site-based and decentralized has infinite points in between it, what does that kind of ideal hybrid model look like to folks on the call? It feels like it could be so many things. The way… patient mediated data is not the antithesis of collecting site-based data, so what does that ideal hybrid look like?
Sneha Kishnani:
I think there is no ideal hybrid. Right? I think that's the beauty of ‘hybrid’ is it can be what it needs to be for whatever research question we're trying to answer. It's having the awareness around, what are the different data types and data sources out there. And having the deep insight and the deep knowledge into: does that data source have the information that is going to help us to answer the question that we want, and where do we need to get the data from? So in essence, looking at a protocol, looking at the study objectives, the endpoints you're after and understanding who is the right source of all of this data? Does it need to come from a site? Can it come from the EMR? You know, et cetera. So really understanding deeply where that data lives and who can best provide it in an accurate and high quality way.
Andrew Larsen:
Yeah. I echo that same thought. I mean, it has to be fit-for-purpose. What are the questions? What are the data sources that support it? And really think critically about the feasibility of what you're considering, right? Just because a data source has ‘data element X’, are you actually going to get the coverage you need or the temporality you need for the patient populations that match? I think being diligent pays off and makes this all a much easier process downstream.
Sydney Perelmutter:
All right. Well, thank you very much for those answers. This concludes the Q&A portion of this webinar, and if we couldn't attend to your questions, the team at PicnicHealth may follow up with you, or if you have further questions, you can direct them to the email address on your screen. Thank you everyone for participating in today's webinar. You'll be receiving a follow-up email from Xtalks with access to the recorded archive for this event and the survey window will be popping up on your screen and your participation is appreciated as it will help us to improve our webinars. Now, please join us in thanking our speakers, Gaelan Ritter, Sneha Kishnani, Andrew Larsen and Evelyn Pyper. We hope you found this webinar informative. Have a great day everyone.
Sydney Perlmutter:
Good day to everyone joining us and welcome to today's Xtalks webinar. Today's talk is entitled The Reimagined Registry: Revolutionizing Patient Care and Research through Real-World Data. My name is Sydney Perlmutter and I'll be your Xtalks host for today. Today's webinar will run for approximately 60 minutes. This presentation includes a Q&A session with our speakers. This webinar is designed to be interactive and webinars work best when you're involved. So please feel free to submit questions and comments for our speakers throughout the presentation using the questions chatbox, and we'll try to attend to your questions during the Q&A session. This chat box is located in the control panel on the right-hand side of your screen. If you require any assistance, please contact me at any time by sending a message using this chat panel. At this time, all participants are in listen only mode.
Sydney Perlmutter:
Please note this event will be recorded and made available for streaming on xtalks.com. At this point, I'd like to thank PicnicHealth who developed the content for this presentation. PicnicHealth is a healthcare technology company that partners directly with patients to build deep real-world data sets. The company leverages state-of-the-art machine learning combined with human curation to put complete medical records into an easy to use online application. The platform gives patients unprecedented access to and control over their medical records, and with their consent, the opportunity to contribute this valuable data to further scientific research.
Sydney Perlmutter:
Now I'd like to introduce our speakers for today's event. Heather Fitzpatrick Medlin is CureDuchenne's Senior Director of Clinical Development. In this role, Heather leads the CD Link program, a decentralized data integrated biobank platform to connect researchers to coded data and biosamples from individuals with Duchenne and Becker muscular dystrophy, as well as the carriers of these mutations. Heather has over 25 years experience in the clinical research industry across all phases and therapeutic areas with a concentration in the late phase medical device and diagnostics projects focused on rare disease. Her prior experience working in the public health arena and hospital settings has enabled her to understand the perspective of patients, healthcare professionals and industry professionals. She completed her master's degree at the University of South Carolina and her undergraduate degree at Davidson College.
Sydney Perlmutter:
Clara Lam received her PhD and MHP from The George Washington University Milken Institute School of Public Health. She continued her studies at the university and pursued her pre-doctoral and post-doctoral fellowships in collaboration with the National Cancer Institute in the Radiation Epidemiology Branch. Clara went on to be a mathematical statistician in the Surveillance Research Program in the Division of Cancer Control and Population Sciences. During the last three years, Clara has taken an opportunity to join AstraZeneca in their US Health Economics and Outcomes Research group. and then became a strategy director. Then finally as an asset lead directing a comprehensive portfolio of epidemiologic and HEOR studies supporting AZ breast assets in the US and globally.
Sydney Perlmutter:
Daniel R. Drozd is the Chief Medical Officer at PicnicHealth. Prior to joining PicnicHealth he was on faculty at the University of Washington in the Department of Allergy & Infectious Diseases where he led research into the use of electronic health record data to power observational research and enhance the understanding of the chronic burden of HIV infection. At PicnicHealth, he oversees scientific collaborations with PicnicHealth's industry and academic partners and works extensively with both the product and commercial teams. Prior to attending medical school, he worked for numerous technology startups as an engineer and at the University of Washington in the Clinical Informatics Research group where he led the development of a large EHR data integration platform used to power HIV real-world research.
Sydney Perlmutter:
And lastly, Evelyn Pyper is a Senior Evidence Strategy Lead at PicnicHealth, a patient-centric real-world data company. Her career in real-world evidence (RWE) spans the public and private sectors as well as regional and global markets. Prior to PicnicHealth, she worked as Associate Director of Market Access at J&J Global Public Health, focused on securing access to HIV treatments in Sub-Saharan Africa and as RWE Manager of a diverse portfolio of partnerships and research projects at Janssen Canada. Evelyn has a bachelor's degree in Health Sciences with a minor in Psychology from McMaster University and a Master of Public Health degree from Queen's University. Evelyn is currently completing her doctorate in evidence-based healthcare at the University of Oxford, with a focus on digital health.
Sydney Perlmutter:
And now without further ado, I'd like to hand the presentation over to our speakers so you all may begin when ready.
Evelyn Pyper:
Great, thank you so much Sydney and welcome everyone. Welcome to our speakers. We are really excited to have you all here live for the third webinar in PicnicHealth RWE ROI series. Some of you may have actually joined us earlier in the year as we discussed the value of real-world data for drug development and regulatory decision making in our first webinar or perhaps in incorporating different data types into clinical trials for our second webinar. But for those who are joining us for the first time today, the goal of the RWE ROI series is really twofold. It's to dive deeper into today's uses or untapped opportunities when it comes to real-world evidence and to convene a diverse group of experts with different perspectives from across the life sciences ecosystem.
Evelyn Pyper:
And today we are very thrilled to have Clara, Heather, and Dan with us to discuss the innovative ways that stakeholders are optimizing registries to generate more robust research insights. So given the topic of this session is the Reimagined Registry, it's probably worth us starting off our conversation with a perspective around what constitutes a traditional registry. While no single definition exists, a patient registry which might also be referred to as a clinical registry, disease registry or outcomes registry, typically refers to an organized system that uses observational study methods in order to collect standardized information about a group of patients in this case. We know that registries are a really key source of real-world data that can meet a variety of needs and answer a wide range of research questions.
Evelyn Pyper:
At the highest level, registries serve an important purpose, which is evaluating and improving outcomes for a population, often that population being defined by a condition, a disease or some exposure. So with that foundational level setting, it's clear that patient registries have an important role to play in healthcare research. However, we know, and this group here knows all too well that there's many challenges with traditional registries that can have broad implications for researchers and participants alike. So starting with you, Clara, what are some of the methodological challenges that you've seen or experienced yourself with traditional registries?
Clara Lam:
Thanks for the question, Evelyn. Thanks so much for the opportunity to be part of this really nice distinguished panel. Thank you all as well for joining. I think registries can be extremely important. They can provide population-based data, longitudinal information. It can be really important for looking at trends over time amongst just understanding the population that we're trying to help and improve their outcomes. But there are issues with that. A lot of it is site-based location. And so there's a lot of restrictions in terms of where the representation might be coming from. And a lot of the time it's actually restricted to academic centers, so we might not get great representation coming from a community setting. They're often specific to different specific sites. So there might be a limit on the scope of data collection that one might actually be able to get.
It's a static data model in many ways as well. It's not necessarily changing with emerging technologies and information that might be coming down in the future. And a lot of the time it's also restricted based on resources, time and budget of the sites themselves. And so I think there are wonderful abstracters out there who have just done so much in collecting this information, but it can be very limited in terms of the resources. And so I think while it's a phenomenal way to collect information, we might want to think about more innovative ways going into the future because I think some of these limitations just cannot be overcome.
Evelyn Pyper:
Thanks so much. That very much resonates. And Dan, would you agree with what Clara said? Would you add anything? And maybe taking a step further beyond methods, do you see any other challenges with traditional registries when it comes to the data that's then available from those sites?
Dr. Dan Drozd:
Absolutely. Thanks for the question, Evelyn. I completely agree with all the things that Clara outlined. I think just to call out very explicitly, registries are incredibly expensive to run, right? They have very high burden both on sites and on patients. And I know we'll dive into both of those topics in a little bit more detail later. But I think it's increasingly difficult to get sites with limited time and budget to run registries compared potentially to observational studies that may be better compensated for sites. I think as Clara mentioned, data elements need to be pre-specified in terms of registries, and there is the potential for a lot of expense and additional time requirements if you need to add an additional data element. Something as relatively simple as adding an additional data element can add months of delay as well as significant cost burden to sponsors.
I think it's just increasingly difficult to get patients to contribute data to registries in an increasingly crowded space and field. So specifically though about data, I think piggybacking off of those, loss to follow-up can be a huge issue for registries. And so that definitely limits the usability and generalizability of the data that's being collected. I think as Clara mentioned, questions of representativeness are key in terms of the data as well. So are the patients that we're capturing data from in the registry, do they look like patients broadly within the larger population? And can we generalize results that are derived from a particular registry to another population of patients if we're only collecting information from large tertiary academic care centers, etc.
And so I think that representativeness becomes a major question, a major challenge in terms of the underlying data as well. I think representativeness, loss to follow-up and long-term follow-up in terms of the data. And then that, as Clara mentioned, the static nature of the data and registries present real challenges to the way that we have done things historically.
Evelyn Pyper:
Thanks, Dan. Taking what you've said in total, it sounds like it's not just an issue of where the data is collected, but also how it's collected and from whom. There's layers to that challenge, which means complex challenges needs complex solutions, I suppose. And before we start talking more solutions oriented, we know that beyond the data is of course patients themselves. And Heather, from your perspective, what are the shortcomings you've seen with traditional registries when it comes to the patient experience? So traditionally, what might some of those barriers be to either initial or ongoing participation in registries?
Heather Fitzpatrick Medlin:
Thank you so much for the question, and thank you for the great conversation so far. I think what we know is that especially in the rare disease space, everyone is looking for an answer. They're spending a lot of time trying to go to different places, collect information from lots of different sources. So there's a burden, especially when you're looking at a pediatric population, we put this on the parent or the caregiver to go to these different sources. One of the biggest challenges we've seen, and I think we see now, is understanding that oftentimes we present the request for this information in English and not everybody speaks English. So making sure we're connecting with the larger community, especially in diseases where we see them more broadly, both geographically, demographically, et cetera.
I think the return on investment, what's in it for me, and making sure we push the message about the fact that they're providing this information, whether it's patient reported data or site or research center provided data, that it may not lead to a treatment right away, but there is that downstream potential for an impact to maybe the next generation or a later change in treatment that could impact the individual participating. I think the ROI, and then what we see is specifically working now in the Duchenne space is that we have a lot of individuals who can't participate in this type of research due to the structure of clinical trials and the exclusion criteria from participating. So talking about loss to follow-up, we lose people sometimes because they have to decline participation or stop participation while proceeding forward with intervention.
Evelyn Pyper:
Thanks Heather. And I'm really glad you mentioned the ROI from a patient perspective, because I do feel like all too often and throughout this series we've considered multiple different perspectives, but we're often thinking about what's the ROI to invest in the infrastructure to do something like this for the people developing it. And often it's an afterthought to think about the actual ROI for the patients themselves and the families of those patients. I'm really glad you mentioned that. And so at this point now, we've discussed the variety of ways that traditional registries really need to evolve and have needed to for some time. And fortunately for those listening, we don't just have to imagine what that evolved registry might look like.
Evelyn Pyper:
Our panelists here have actual hands-on experience with moving us to that next phase of what registries could be. So as a follow-up question for you, Heather, can you tell us a little bit about CureDuchenne link and what makes that an innovative model?
Heather Fitzpatrick Medlin:
Sure. Coming from experience running, a lot of what we would say are traditional registries, I was excited when I joined CureDuchenne because we've taken not only the component where we collect information directly from what we call our participants, so survey-based information directly from a parent caregiver. But we're also asking for donation of bio samples that we're able to collect aliquot into smaller samples and provide back to researchers. But one thing we were missing, and I think we all talk about this, is the larger set of data that we no longer want to send somebody to abstract or have just limited information coming over, but really understanding that especially in a rare space there's multiple providers, there's often lots of geographies that are covered as parents go to seek care.
So what we've done is partnered to be able to pull that data in to make a large and more robust data source, collecting both retrospectively looking to go back maybe towards the age of diagnosis and then moving forward with no endpoint or upper limit of participation at this point.
Evelyn Pyper:
Thanks so much. And maybe just a side question that comes to mind and might be a question that our audience would have, is typically when folks think of registers, they think of the prospective collection. Do any of the panelists want to speak to what it means to have that retrospective data with a real example or a hypothetical? I think often people think of observational prospective cohorts and not that retrospective lens.
Heather Fitzpatrick Medlin:
I can jump in because we are collecting this retrospective understanding specifically in Duchenne, that the diagnostic journey can be five to seven years. There's a lot of information on that journey that's not presented initially. I did want to speak to the fact that the way we're collecting our data now using PicnicHealth, we're actually able for that ROI question to provide it back to participants so they have a one-stop place where they can access their information, take it to a healthcare provider or just review it themselves. And that's been a really great system, because again, that's not something that has traditionally existed in the registry or even in the medical record space.
Clara Lam:
I'm going to chime in a little bit as well. I think the collection of that retrospective data and in the situation that we're working with for PicnicHealth is we're looking at early breast cancer patients. And breast cancer patients if they receive a diagnosis, it could be a very long time for their treatment journey. And survival in early breast cancer is quite high in the United States, and obviously that's where we want to keep it, but there's a lot of information that we can glean from that retrospective data collection upon their first diagnosis and following them through their patient journey. It's invaluable that we're able to get access to all of that. And then importantly, as Heather was mentioning, being able to provide that to the patient themselves.
I think as we all know, it's just so difficult to keep track of all your medical appointments and all the information that comes along, every scan, every blood test, and having all that in one convenient location that is easily accessible and easily understandable, that's just so invaluable. And I think that's something that is a huge benefit to the patients who are involved in the early breast cancer registry.
Evelyn Pyper:
Thanks so much for sharing, really appreciate that. It seems like it really underscores the point that for these patients, the clock is not starting at the time of diagnosis, at the time of entry into the study, there's a whole wealth of information that is all too often forgotten about or just inaccessible, that can tell a totally different story. And so this starts to introduce some really exciting ideas, I think, and paints a clear picture of the value that innovative registries have for rare disease research. And Clara, building on what you alluded to already, you're someone working in a very different therapeutic area than Heather is. You're working in oncology. And so are these innovative registry approaches relevant to early breast cancer with a variety of other cancers you've worked with, and are there any other types of novel designs or types of data that have you most excited in working in this space?
Clara Lam:
Absolutely. I think you touched upon it already, Evelyn, that everybody wants to do an observational prospective study, but the reality is that those are very difficult to get off the ground. They're expensive, they're time-consuming. They require a lot of personnel and a lot of procedures and logistics to be in place and to be able to have retrospective medical records from patients and following them forward, it really provides a very unique environment that we can look at emerging biomarkers. Every day I feel as though there's a new biomarker that we've discovered, and it might have a key role in whatever cancer we might be looking at. And breast cancer in particular, we know the ones like BRCA for example, and that's something that we all can test for, but there might be more.
Clara Lam:
And so it'd be really fantastic to be able to not only capture the retrospective information for the patient journey, but to follow forward and see what happens with emerging biomarkers. I think another huge innovation that's really helpful is treatment and oncology is just exploding. It changes so rapidly. Every other day in the news you're going to hear about new treatments that are available for a different breast cancer, lung cancer, pancreatic. And so being able to track this forward in time also is really, really important so that we can see, what do the patients get in early breast cancer for their first sign of treatment? When did they have their surgery? What are they getting after? Did they have neoadjuvant treatment?
I think a lot of just being able to have that level of detail and granularity for the treatment journey for these patients, it's so important. I think the other huge component, and this is something that Heather touched upon earlier, but getting those patient reported outcomes, that is something that has become that much more important and become really central to the studies that we do. We want to know, how are the patients doing? What goes into their decision making? How are they discussing this with their caregivers and their patient care team? There's a lot of different important factors that may have prognostic value that we need to include as part of these registries. So to be able to capture the retrospective data but then follow patients forward, it's just invaluable. And that's just something that we really can't do in a traditional setting.
Evelyn Pyper:
Thanks so much. I'm glad you mentioned the patient reported outcome piece at the end. It seems like the traditional registry world, things really fall neatly into categories of researcher driven or patient driven in terms of who birthed it, who's really owning the collection of that data. And it's really exciting to see those boxes be broken down as we think about things that can be equal parts, information coming from patients, they're involved in the process and getting something out of it. Researchers are also really critical in defining what are those key data elements and continuously improving the data model if necessary as new treatments come on the market. I think we'll probably get into this as well a bit later, but just especially the treatment angle, really thinking beyond disease registries to product and drug specific registries where a data model from five years ago might not be relevant for the types of endpoints that are critical.
Evelyn Pyper:
I'm excited to talk more about that a bit later in the session. But for right now, Dan, PicnicHealth has partnerships with both CureDuchenne in DMD as well as AstraZeneca in early breast cancer. These are unique collaborations and different disease areas with very distinct research needs. And so what is it, if you could describe it about PicnicHealth's approach that drives value for researchers and patients across these varying diverse contexts?
Dr. Dan Drozd:
I think it's a really good question. I think the one thing that both of these conditions have in common is they certainly can have complex diagnostic journeys. And really as a patient in the United States in particular, you really are the only through line yourself in your journey through the US healthcare system. And so having data that is siloed in one organization or one institution, really inhibits the ability to understand that comprehensive care journey. I think if you think about what we actually mean by patient-centric research, I think this is a term that obviously gets used really often. I think ISPOR has a nice definition of patient-centric research. And they say that it is the active, meaningful and collaborative interaction between patients and researchers across all stages of research process where research decision-making is guided by patients' contributions as partners recognizing their specific experiences, values and expertise.
I think if we think about that definition, that definition is so different from the way that we would've conceptualized what research historically has been, right? Historically participants have been subjects, they've been things we study, not active participants in the research process. And so I think one of the things that both of these collaborations have in common is a real desire and motivation to put patients at the center of that process. One, because it's the right thing to do, but also it is the only way that we can actually get at the data that we need in order to move forward treatments and decision-making in both of these conditions. I think that starts obviously with having fully consented patients, it means having a low lift for patients from an onboarding and ongoing participation perspective.
It means meeting patients where they're at. Some combination at times of data that's collective passively and data that we are actively collecting from patients, from a data perspective it really leverages patients individual experiences to compliment the data coming from electronic health records, whether that's codified data coming through in ICD 10 codes or information coming out of narrative text sections of patients records, which are important in different ways in both of these conditions. And then combined, that really gives us a much better 360 degree view of the patient journey. And then just from a technology perspective, I think in order for us to be able to do this at scale, it really does require a sophisticated underlying technology platform that's tuneable and responsive to the specific research needs within a particular condition.
And that it allows those things to change over time, that it becomes relatively straightforward when there's a new biomarker of interest or a new gene mutation of interest, to be able to interrogate the data and get additional information about that without having to go back and do a protocol revision and manually reconstruct a bunch of data, etc. And so I think that's the way that I view our overarching challenge here. And the reason I think that both of these collaborations, while the conditions are obviously in many ways very different, have an underlying through line that has made both these collaborations important and successful.
Evelyn Pyper:
Thanks, Dan. And maybe as a follow-up question for you, for some of our audience members that maybe haven't been to one of these sessions before, can you give even a brief flavor of what those core technology capabilities or platform as PicnicHealth drives this? It can be a fairly non-technical explanation of what that technical component is.
Dr. Dan Drozd:
I think when I think about it, it's having the infrastructure to be able to collect records from anywhere a patient has been seen, that is a foundational part of the electronic health record component. And that is both leveraging electronic means as well as other means of collecting data when necessary. From a data structuring standpoint, it really is the use of advanced machine learning technology to be able to have a scalable process that we then can tune to how much human review of a particular concept or idea we want to put into place. And so there are concepts that are relatively straightforward and we may need less human review. So an individual lab result for example.
And things that definitely need a lot more review, is a patient's cancer progressing? That's something that definitely has a lot more nuance around it. And so I think at its core, it's a technology platform that leverages state-of-the-art machine learning technology to enable us to tune the output of the process to whatever the particular research needs happen to be. And is cognizant of the fact that the needs for data that is being used for a regulatory submission are very different in many ways than data that might be used for an internal study or other purposes.
Evelyn Pyper:
Thanks, Dan. With that in mind, we know that there is value that these registries can bring to life sciences researchers, but what that particular value is is going to probably depend on a variety of factors, including whether or not there's a need to study a particular treatment or disease, what we've spoken to before, the stage of clinical research, what functional groups are involved. And Clara, you have extensive experience working in medical affairs. So in that case, and actually building on what Dan just mentioned in terms of it's really going to depend on what it's being used for. What might success look like for a medical affairs function when investing in a patient registry? What needs to be the output? What do you need to see to call it a success?
Clara Lam:
No, absolutely, and I think I alluded to it earlier, but the ever fast pace of oncology, we need to have the most current data available. Part of the problem with more traditional registries is that there's usually a time lag, there's a data lag, because it just takes time to collect this information. And so unfortunately with some of the registries that are available currently in a traditional setting, there might be a time lag of two to three years. And while it's still extremely irrelevant and very helpful, we are worried about the current treatments today, what is the current standard of care that the patients are dealing with? What are the new options that are being made available to them and what goes into that decision making? We need to have very current up-to-the-minute information about the patients who might be receiving the products that we might be supporting.
And then going into the future, I think the other thing is that we need to have patients who are really representative. Some of the restrictions when it comes to other traditional registries is that side based, it might be a state registry, it might be a local registry, but we really want to be able to capture patients from all over the United States and not just in one or two areas. And I think the other, there's a time element to that too, is that we want to have representative patients that can provide their information quickly and to be able to upload that information in a really timely manner. And so not just the medical records that we're going to be capturing, but also patient reported outcomes or potentially if we want to evolve caregivers somehow and building that into the platform, that's something that we can explore and that might be an option in the future.
But I think just having that more complete picture and we really need a patient population that's representative of folks who are outside of clinical trials. That's a really big component for us, is that clinical trials are only representative of certain subsection of population, and we want to have underrepresented groups, whether it be racial and ethnic minorities, it might be subgroups of different biomarker patients who are not well-represented and registries currently. We want to go after the patients that we don't see anywhere else. And so the novel approach that we have with the platform we're building for the early breast cancer registry with PicnicHealth, that's the patients that we're getting. So that's really phenomenal that we can do that in a really current fashion.
That's exciting for us and that's a sign of success that if this registry can really represent those patients, then we're doing something right.
Evelyn Pyper:
Definitely. Exciting on the PicnicHealth end too, all around huge in terms of what we're able to achieve together in this partnership. And as a follow-up question, some of the attributes you mentioned, you're able to, as a decision maker, you're deciding where to invest in a registry. You can ask the right questions and glean that information up front, but there's probably other attributes that you can only determine what's going to be successful once you're in the data, once you're there. And for the subset of folks that are listening in live who might be also deciding where to invest in a registry like, beyond the let's say representativeness, which you spoke to, are there any other specific registry attributes or signals that you would suggest to folks like yourself who are deciding what's the best approach or where best to invest their time and resources?
Clara Lam:
I have to say with the PicnicHealth team, we've asked a lot of questions repeatedly. I feel very confident in the PicnicHealth team and their level of expertise across all different levels to be able to answer a lot of the questions that we had about different data elements. We would want to capture the cadence of that information, how often we would have the data being delivered, the refresh of that data, how often the army of abstracters are sent out to collect additional information going forward. And two things that are really important is that just because we have a patient enrolled and we start collecting their medical information, that's not the stopping point. We actually keep following them and we keep on getting information at regular intervals. And so not only do we continue adding data to them, we just continue following them forward and have opportunities to interact.
I think the other huge thing, and I can't stress this enough, I'm really excited about this, is the source documentation. Being able to actually go back and look at this information in the event that there's another biomarker that comes up that actually has been collected and we just didn't know how to collect it and we didn't realize its importance. But that information is already in the medical record on some level for some data elements. And so being able to go back and get this information without having to go through the procedures of writing a new protocol or making an amendment or having to fix the static data model to add this in as well, and all the logistics for that, I think the fact that it's very flexible and that it's adaptable, I think that's a huge set of attributes that this registry really provide. It's a lot more straightforward.
And I think in terms of the patient perspective as well, and I say this as a patient myself and just for other things, but I think everyone is really appreciative of the fact that it's simple for us to get this information and we have it in a format that makes sense and is easily used and analyzed, but it makes sense for the patient too. It's a win-win on many different fronts. And so I think this very novel approach of capturing this information, it's really hard to not say yes to and be a part of.
Evelyn Pyper:
Absolutely. And it's wild to think that a three to five years, which might be like an average, let's say study time, to be able to avoid having to spin off a separate study, three to five years in the life of a patient is huge, is massive if that new biomarker becomes known and that information needs to be gleaned for a study. If you just think about timelines, it's massive for researchers, but also even more massive in terms of the life of a patient and saving that time. So Heather, question for you. We know that the integration of different real-world data sources seems to be at the core of your registry's resolution. Can you provide some insight examples about how integrated data translates into better research and ultimately better outcomes for patients in the context that you're working in?
Heather Fitzpatrick Medlin:
For us I think it was really important to understand that for these kids with Duchenne, CureDuchenne's focus is getting that research and getting to the cure. And oftentimes we have information, it's in different places, it's not systematically available, and that's time. We're finding out there's so much active research in the space right now that we're able to provide not only the data that we're collecting from the participants directly, we're able to supplement that with de-identified data from PicnicHealth. And then we have, as I mentioned, the bio samples. We're able to provide researchers, whether they're biotech, pharma or academia with that information on the ready.
It's another disease space where we're seeing so many new and innovative treatments come, and we really want to focus on being able to keep up with that and recognize that over time that the different elements of research are going to change, but we always are going to have a baseline in that data and the samples moving forward.
Evelyn Pyper:
That's great. And for those who are less familiar with the disease space, as a follow-up, are you able to speak any more to the significance of those biosamples? In your average DMD study, are bio-samples a core part of it? Is it pretty unusual to have it collected as part of a study or registry like this?
Heather Fitzpatrick Medlin:
Certainly not unique in the study space, but definitely in a data integrated biobank as we're running, it's uncommon. I wouldn't say we're the only one, but we're one of few. And for us, it's really important that we're able to offer that in a collective place to researchers quickly, because for these boys, time is of the essence.
Evelyn Pyper:
It's clear from our talk so far that registries are not what they used to be. We've talked about the way different types of data being collected, different ways of collecting that data, integration of data types, but also across time retrospective and prospective, and then the entire gamut of patient-centric elements that we can pull into things from patient-centric approaches to getting patients onboarded and aware of the registry in the first place all the way through to giving back data to patients themselves. It really feels like there's this suite of possibilities in this new era of registries. And this is undoubtedly enabled by advancements in data and tech, innovative partnership models like the ones that folks on this call are part of, as well as bringing together the strengths of stakeholders from different aspects of the ecosystem.
Evelyn Pyper:
I'm fairly certain that not one of us on our own could really have the impact that we could have together, particularly when it comes to registries and how many folks are involved. But with that, we've never quite solved the problem. Our work is never quite done. I'm sure all of our panelists have ideas or dreams, top of mind, that continue to move the needle for patient registries. And so as a larger final question before we head into more of the audience Q&A, I'll ask each of our panelists to imagine a world where resources, people, resources, financial resources are unlimited. What would you do with these infinite resources to further optimize the design or deployment of your registry or a registry? Dan, I will start with you.
Dr. Dan Drozd:
All right, I have lots of thoughts and ideas here, but I think maybe what would be more, what I'll do is start by just putting out a couple of north stars that reiterate a few of the things that we've talked about already. I think increasing focus on minimizing burden on patients while at the same time increasing their ability to contribute. We talked a little bit about how there's increasing choice and competition for patients about different sources of data that they can contribute to. I think the extent to which we live in a world where there are silos, no one is going to actively go out and contribute to 10 different disease registries within their condition. And so thinking about ways that we can simplify that process from a patient perspective is top of mind for me. I think it is also key to us recruiting diverse populations of patients and patient retention within registries.
And then in situations where the registry isn't entirely virtual, really a similar frame of mind thinking about sites and interaction at a site level. So what are the things we can do to make the process easier for sites getting from, here's another study that I'm being asked to do, to this is a study that I can show value to patients that can be relatively low impact to my clinic or my setting from a staffing perspective, from a cost perspective. And then also really from a technology burden perspective, I think sites are increasingly being asked to adopt many, many different flavors of e-consent systems or various sorts of things. And so these to me are things that are like north star, top of mind as we think about ways to expand the reach of registries in the ecosystem.
Evelyn Pyper:
I like it. I like it. Thanks Dan. And Heather, how about you, world where there's no constraints to what you can do with your registry?
Heather Fitzpatrick Medlin:
I think the ability to give back to patients or participants in a way that they can see the information more readily, perhaps more graphically, have timelines and being able to really empower them to make the best healthcare decisions with their providers, gives them a bit more understanding. And as I said, we can't dispute the fact that we do a lot of things in English. We need to broaden that. We can't assume people can read and write, so we have to make sure that we're able to provide this information perhaps in an audible way so that information can be shared for those that may not have access to information on the ready. I think that's our goal. And again, looking at how far we've come, it's pretty impressive. And I'm sure if we regroup in two years, we'll be talking about today being the good old days.
Evelyn Pyper:
We should indeed do that, regroup in two years and see if we've achieved any of these goals. That's great.
Heather Fitzpatrick Medlin:
Exactly.
Evelyn Pyper:
And Clara, how about you?
Clara Lam:
I think just to build on everything that Dan and Heather had said, I think if I had a wishlist aside from just capturing all this really wonderful information on the treatment, the journey, the patient perspectives, caregiver perspective, physician perspective, all of that, as a health economics person a little bit on the background, I'd also like to collect information to be able to address the financial toxicity that our patients are dealing with. There is no simple condition in the United States and everything seems to be expensive. So what can we do to help our patients address some of those concerns as well?
So if there were ways for us to be able to capture information on costs, disease progression, just the quality of life, just capturing a lot of this information just on all fronts for economic outcomes research to be able to quantify that and showcase if we can provide better treatment options for our patients earlier, can we delay disease progression? Can we delay the costs and the financial toxicity that comes along with that? I think that's another huge component of information that we don't necessarily have set up in this moment, but I think that could be coupled with the information that we are capturing in the registry would be incredibly valuable and just not available anywhere else. I think if I had my wishlist, I would add economic outcomes as well.
Evelyn Pyper:
That's a great one. Especially it feels like both a probably common researcher wishlist item, but becoming more of a potential reality in the world of value-based healthcare where there'll be certain things that aren't just a wouldn't it be nice if we could capture, it'll be a thou capture this or we're not going to reimburse. And so that just feels like such, again, let's regroup in two years and see how much of this is in fact necessary and not just innovative. Thank you so, so much to our speakers. Oh yeah, Dan, go ahead.
Dr. Dan Drozd:
One other quick follow up to that, which is probably obvious, but I think it's not just patient economic burden, it's caregiver burden as well. And so in many, many conditions that we work in, there's a significant burden on caregivers that is historically under captured. And so I think really reflective of, if part of our aim is to really understand the true impact of disease and the true patient journey, the caregiver aspect is key as well.
Evelyn Pyper:
Definitely. Thank you for adding that. Any other thoughts from the group before we head to audience Q&A? Awesome. Well I think we have some great questions waiting for us, so thanks so much to our panelists and we'll head into the next phase with Sydney.
Sydney Perlmutter:
All right, thank you very much for that insightful presentation. Now I'd like to invite our audience to continue sending in their questions or comments right now using the questions window for the Q&A portion of the webinar. I've already received some great audience questions, so I'll start with those. So our first question is, one of the challenges of collecting RWD is patient and clinician participation. What are the common behavioral science approaches used today to increase participation in our RWD collection?
Heather Fitzpatrick Medlin:
I can start. I think for us it's really, again, not making it something manual when it can be so simple to be able to behind the scenes move this, not to ask the same questions time and time again, to make sure we recognize the value of individuals that are participating is time and being able to not consistently ask the same information or collect the same information and be willing to share. I think that's the other part about running a program like we do, is that we're independent and so we're able to offer this to all researchers and academic individuals to support us or for us to support them. So we're able to make sure everyone gets a chance or a piece of the data if they'd like it.
Clara Lam:
And maybe ust to build off of what Heather said, I think the other thing about patients is that they tend to be a very altruistic population. They understand the condition of their disease and how it impacts them and their family, but they also want to help make sure, at least in the early breast cancer setting, that this doesn't happen to their daughter, their aunts, their sisters, or their friends. And so it's a huge incentive for them to contribute to the research because on some level, even in an aggregate form, they're able to help find, an additional step to finding that cure, an additional step to finding a treatment that works. And so I think that incentive is there as well. I think it's hard to put a value on having all of your medical records in one place, and that's another huge incentive as part of this decentralized registry that this platform that we're building, it just saves so much time and energy on the patient.
They don't have that kind of time and energy. They have other things they need to do. And especially for early breast cancer patients, a lot of the patients that we have are younger women of reproductive age and they have families, they have other responsibilities. And so to take this burden off of them, I think it provides an incentive for them to really participate so then they can really help in terms of the research that we're contributing and that they're participating in, but also to make their lives easier on the day-to-day I think is a huge thing.
Dr. Dan Drozd:
I totally agree with all of that. I think the percentage of patients across conditions that we work in who are motivated really by the ability to give something back, whether it's something that they will directly benefit from or something that might be, as you said, Clara, hoping that their daughters are not diagnosed with this condition, I think does become a particularly important motivator for a large percentage of people. I think the other thing is just being very thoughtful when we are asking patients to do something and provide data. I think we've all taken surveys at some point in time in the past where you get asked 27 questions and you know that you could have gotten the information you actually needed in a couple of questions.
And so I think something that we spend a lot of time thinking about is patient or participant burden in the context of PRO instruments, right? Just very simple things. Let's try to give a questionnaire that requires only 30 seconds of someone's time instead of them needing to sit down and do something for 10 minutes. We obviously see very different responses in terms of follow through and completion of those surveys and those different situations.
Sydney Perlmutter:
Thank you all for those responses. Our next question is, what functionality, if any, does PicnicHealth provide for storing and working with images such as MRI scans, etc?
Dr. Dan Drozd:
I'm happy to take this one. We do as part of our studies, collect raw DICOM images. So these are the actual slices from various radiographic studies, whether those are plain film X-rays or CT scans, MRIs, et cetera. And do have the ability then to de-identify that data and make it the raw imaging data available to research partners when that's relevant for a particular condition. Obviously there are situations where it is more or less relevant. And then consistent with our commitment to share back information with patients, we then make the same imaging available to patients as part of their PicnicHealth timeline and then that data can then be shared with their providers.
So you can imagine how important this is in a patient who has an imaging study, and the provider or center where that study is being done doesn't have prior studies to compare it to. It can be very, very difficult to tell is this lesion in a patient's breast cancer a new lesion or is this an existing lesion? Is it bigger or smaller than was present before? So make that very simple for patients and then make it simple for them as well to share it with their care teams and providers.
Sydney Perlmutter:
Thank you. Another audience member would like to know, for what types of studies or applications are you seeing the greatest potential for impact of these innovative registry approaches?
Heather Fitzpatrick Medlin:
I think from my perspective in the rare space, it's become so critical because there's just so many different types of providers and so many different tests and encounters that they have with physical therapy, occupational therapy, even within reports for the school system. The fact that we're able to aggregate all of that is really critical because it paints that whole picture of what an individual's journey is not just in the healthcare setting, but in all healthcare interactions.
Dr. Dan Drozd:
We can just add settings where the management of the disease is complex and occurs over an extended period of time. So multiple providers, multiple centers, we've all certainly had the experience of changing physicians that we see because our insurance has changed. That's a reality of navigating the healthcare system in the US. And so I think really conditions that are chronic in some sense, that have a prolonged care journey, multiple providers involved either in the path up to diagnosis as we talked about the value of retrospective data earlier in the presentation, but also prospectively as patients are being followed over time across a variety of providers. Those end up being characteristics that I would say where we feel like this approach is particularly important.
Clara Lam:
And I think it also speaks in a way to some measure of precision medicine as well. At least I think in the early breast cancer space, for example, we're following patients for quite a long period of time. And their treatment journey is complicated and the continuity that we can capture by being able to find these patients is not based on a site or based on a particular physician or being able to trace back to their source. I think it's very helpful to be able to basically catalog all that information going forward. And as new treatments become available, new biomarker testing becomes available, to be able to incorporate that I think it really helps understand the ever-changing landscape that we're looking at in cancer care. I don't think this is specific to cancer, I think in general, I think just any condition out there, I think just being able to have the most current information and being able to make it specific to that patient. That's really important.
Sydney Perlmutter:
Thank you all. Our next question. In many cases the research questions that need to be answered using a patient registry are not just disease specific but also highly drug specific. Are there ways that these novel registries can be set up or enrolled to ensure sufficient sample size on the treatment of interest?
Evelyn Pyper:
Maybe I'll start out too just with thought out loud and partial answer to this question, is I often think that we think of disease registries and drug registries as two different things, but it is interesting when you start to dial into what folks mean by a drug specific registries, there's always still going to be that need to compare drug X with a comparator Y on the market, right? It's very rare to have the need to just study one treatment. And so I started to recognize that the real difference is less about are there more than one type of product being studied in patients with the same disease, but more about is there the granularity of data that's important to those treatments available?
And that seems to be, and Clara would love to know your thoughts on this too, the sense that we've gotten from life sciences partners where it's less about excluding patients on other drugs and more about is there at least a depth of data and enough patients on our treatment of interest to make this a worthwhile study type and an investment?
Clara Lam:
No, absolutely. And as a representative of AstraZeneca, obviously we are very keen on understanding what's happening to patients who are on AstraZeneca products. But as a step beyond that, our dedication to really understand breast cancer research is moving forward, and especially in the early breast cancer space, there's a lot of options available. And I think it's very important that we think bigger and not just in the sense of very statistical, you need to have proper sample size to do anything, because last time I checked you cannot do a robust analysis on five patients as much as we seem to think we can. The reality is that the more patients we have, the more represented they are, and the more we can understand with some confidence that the numbers that we're seeing, the results that we're seeing, outcome measures, that they're more reliable and that they really do speak to the population that we're trying to address.
So as much as it's important to understand a specific drug registry, I think the bigger research question that can involve that, is what's happening in our patient population that needs to be addressed on a bigger scale? And then from there you have specific research questions addressing some of those subgroups, and that could be an analysis that can be done.
Sydney Perlmutter:
All right, I think we have time for one last question and that is, what do the panelists think about the application of these approaches to pregnancy registries? There are many challenges with how these are done today, and it's an area of research that could benefit from innovative methods.
Heather Fitzpatrick Medlin:
I'm going to jump in first because that was my first registry I ever worked on, were pregnancy registries, and so they're still very near and dear to my heart. Obviously they've evolved a lot over time and we've watched the expansion by looking at a single product exposure, a class of products, following from a disease perspective. It gets a bit more complicated as we know, because if a baby's born, you have a separate set of medical records that are cleaved off and you want to be able to follow that information. You also have prenatal information that you want to follow with additional testing after birth. But I think, again, this is a place where as we know even women who are pregnant maybe seeing multiple practitioners. It's really important to have all of that. There could be a standing disease that they're seeking treatment for outside of their obstetric care during pregnancy.
I'm always excited to see the pregnancy registry space come to the forefront, but I think this is definitely well suited for the collection of data around that.
Sydney Perlmutter:
All right. Well, thank you very much for those answers. We've reached the end of the question and answer portion of this webinar. If we couldn't attend to your question, the team at PicnicHealth may follow up with you or if you have further questions, feel free to direct them to the email addresses on your screen. Thank you everyone for participating in today's webinar. You'll be receiving a follow-up email from Xtalks with access to the recorded archive for this event. A survey window will be popping up on your screen and your participation is appreciated as it will help us to improve our webinars. Now, please join us in thanking our speakers, Heather Fitzpatrick Medlin, Clara Lam, Dr. Dan Drozd, and Evelyn Pyper. We hope you found this webinar informative. Have a great day everyone.
Jane Myles:
You notice I didn't say decentralized trials space. That's a learning from a site listening session last week, which I had the privilege to lead. Where the sites really said, the more you talk about this as a different kind of trial, the more complicated you're making it. It's really a new set of tools. And it's like, I violently agree and so I'm changing my language. Anyway, welcome to the circle. Thrilled you're joining us. Matt is our circle steward. You have special guests today, and really delighted to welcome you here. Circles are new ish for DTRA, they got started earlier this year. But the benefit of the circle format is that people from any part of any member organization are welcome to join.
There's no title. There's no role. It's just about curiosity and passion. And there's no specific deliverables attached to being part of a circle, unless someone in the circle has this brilliant idea that we need to tackle and then we'll go charter an initiative that will be separate from the circle. So this is a spend your time, get curious and learn who else is thinking about these things forum? Yeah.
Matt Veatch:
Perfect. Thanks. Thanks, Jane. Yeah, super exciting space. And predicated on all the interest in real world data and evidence generation, it made sense to have the circle that would focus on the combination of decentralized research and real world data. Hence the topic today, it's the growing opportunity in the drug development lifecycle. Kind of a fireside chat, and I'm speaking from Colorado where it's getting a little bit colder. The fireside actually sounds pretty good. We'll be talking about where the industry is headed. We've got a distinguished panel. This is so exciting with our moderators Xerxes Sanii, who's the Managing Director of Corporate Strategy and Development at PicnicHealth. And panelists, Kim Barnholt, Executive Director of Evidence Generation at Genentech, Nancy Dreyer, President of Dreyer Strategies, and the former Chief Scientific Officer of IQVIA, as well as Dr. Dan Drozd, Chief Medical Officer for PicnicHealth. So welcome to our guests. Super excited for the discussion today. And I'll open it up initially, just for some very brief additional introductions if you would like. Xerxes, maybe I'll start with you.
Xerxes Sanii:
Sure. I'll start thanks for the intro, Matt. Yeah, Xerxes has been with PicnicHealth for a little over four years. Managing director of the company has spent the last decade or so in various evidence generation capacities in the life sciences space. So appreciate you all inviting us and I look forward to the conversation.
Matt Veatch:
Perfect. Thanks And Kim.
Kim Barnholt:
Great. Hi, everyone. Thank you so much for the opportunity to be here. Kim Barnholt. I am an evidence generation leader in our USMA, our US Medical Affairs group. I've been at Genentech for just over 10 years in various capacities from early stage to late stage and now in the evidence generation part in our medical affairs, which I love because it's actually closer to the patient. It's closer to the real world. And part of what I enjoy in my role now is really bridging across different parts of the drug development lifecycle, as well as bridging across the industry. So looking forward to the discussion today.
Matt Veatch:
Thanks so much. And Nancy, great again to see you and a former colleague and friend. Welcome.
Nancy Dreyer:
So thank you. Hi, everybody. I'm Nancy Dreyer, I'm an epidemiologist. As Matt introduced me I have formerly was the Chief Scientific Officer of Real World Solutions at IQVIA. And I take pride in having introduced Quintiles and IMS Health. But I've been in the business for over 30 years. And I think this is probably the most exciting development I've seen. And the idea of having the voice of the patient together with clinical information and wearables. It's there's not the only bright thing on the horizon at the moment.
Matt Veatch:
Thanks. Thanks, Nancy. Dan?
Dan Drozd:
All right. Thanks, Matt. So I'm really excited to be here. My name is Dan Drozd. As was mentioned, Chief Medical Officer of Picnic Health, had been at the company for about four years. I'm a technologist at heart, also trained as an epidemiologist. And really couldn't agree more with Nancy, that this is a particularly exciting time and I think really an opportunity for all of us to talk, and make real headway in terms of how do we empower patients to really be at the center of what we do clinically, as well as what we do from a research perspective? At the end of the day to improve the quality of life for patients, and to reduce some of that burden on patients for both contributing, engaging in research and also for managing their complex chronic condition. Looking forward to the conversation today.
Matt Veatch:
Great, thanks so much. Great. Again, wonderful panel, and really looking forward to kicking things off. So with that, and directed to you Xerxes, as a sort of moderator for the panel. RWD, what's the focus of PicnicHealth? How does it help advance RWD and RWE in the life sciences?
Xerxes Sanii:
Sorry, yeah. And I think just to keep brief, I think that the tagline that I think we've been using for a while, and nowadays, I think it's maybe becoming overused. But this kind of patient centric evidence generation is where we fall into this space. And I think what that means is, where there are deep data needs, and/or kind of the need to engage with patients, which I think each of the panelists also brought up. So just to kind of orient the group here. When a patient consents, and the infrastructure and technology we've built, enables us to get all of a patient's records. And so that's irrespective of a format where it's living, what EMR system, did they move, are they on the East Coast, the West Coast will ensure they have... And then and then on top of that, also a homegrown solution that's what we call machine guided and human curated, to generate high quality data out of the EMR. And then on top of that, engage with patients directly, allow them to participate and share their voice via surveys, clinically validated PRs or bespoke questionnaires.
And so you can think about, the output of that being almost if you had the time to kind of do a retrospective prospective multi-site chart review later on patient perspectives, and get that volume of data but do it in a very efficient way through the technology we've built. So that's what we've done. A lot of virtual registries that we've built in various diseases over the years, I think to use, Jane, I can't remember Jane's word it was the decentralized space where solutions are. But I think now that we're seeing these novel study designs, incorporating this technology into that direction where we see the industry moving, and they're very excited about the kind of new applications that that opens up for where we go.
Matt Veatch:
Perfect. Well, I think, just in terms of additional stage setting, maybe if you could focus just a little bit and directing this to the panel, some of your observations from the space. And there's obviously been a maturation and use of all types of real world data for evidence generation, and specific to clinical research. And again, broader definition than trials, and not trying to call out specifically decentralized it really is part of just the tools in the toolbox for conducting thorough clinical research of today. And again, spanning beyond trials and observational studies, the classic phase for research. Welcome, your thoughts on sort of where the industry has come from and where it's going.
Kim Barnholt:
Maybe I'll take a quick stab, I think where I'm really excited is that we talk a lot about patient centered trials. And I think decentralized trials or decentralized research is patient centric and that it's really trying to meet patients where there are, it's able to bring tools into their house into their community to allow them to sort of passively share evidence, gather evidence, as well as have more active elements that can be shared remotely. But we've always been, patients are still patients, they're still one element of who they are. And the term that I've heard evolve and start to be used more frequently is human centric trials. So we're taking into account not just the patient as somebody who may have disease, but the person who also has lived before their disease, who may have family and work obligations beyond their disease. And I really see the value of real world data being used in collaboration with clinical data as a way to add more humaneness to the research.
It allows us to have a wearable be out in the world where we're understanding how the person is going about their day to day life, without having it be an artificially contrived setting that we're trying to understand their disease burden or an impact of a drug response. It's allowing us to look at claims data or health record data that may even be before they were diagnosed with the disease and understand some of the progression and the trajectory to that point. So I really see the value and the impact and the opportunity with real world data as helping us be more human centric, and look beyond the patient and look at the more holistic aid end to end of their mind and sorry, and beginning and middle part of their lives and understanding how we can treat somebody to make their whole life better not just try to cure a disease or improve their current disease state.
Matt Veatch:
And Kim, speaking from the platform of a biopharmaceutical company, do you feel that there are specific areas of research that are particularly well benefited through real world data? Or is it across the board?
Kim Barnholt:
I mean, I think it's across the board. And I think we're really starting to understand the opportunities, where we've seen more immediate impact is around rare disease. I think there's an opportunity to start to connect in some of the dots and understand, link in some of the real world data with some of the clinical trial data and maybe fill in some missing evidence gaps. I think it's also in, in terms of some of the wearables and some of those devices, I think it's also where patients may not always be able to travel into the center, to a disease center. And I also think it's with improving access. So really more across the board from some representative and inclusive trials, it's a way we can start to better understand different populations, we can better understand how to provide access and really provide more equity in how we're conducting our research.
Matt Veatch:
Yeah, great thoughts, Kim. Thanks so much. And turning to either Nancy or Dan, we don't have to go in any particular order. But welcome your thoughts around, just observations that you've seen and the applicability of real world data to clinical research at large?
Nancy Dreyer:
Well, because Dan still on mute, I'll jump in. I think we're just scratching the the tip of the iceberg, whatever the right expression is about the missing information we've been blind to. And we always thought, "Well, it's not important." The idea of the decentralized trials, I mean, let's just follow up on Kim's suggestion or mention of the wearables. What you're finding year is not how I do in a six minute walk test after I've traveled hours to get to the clinic, and I'm exhausted and hungry and just... You get really a more accurate measurement of people's movement in natural settings. You also get information from patients that they may not have shared or may not be willing to share with their doc. Now I come out of drug safety, Matt, and that's where I had my roots. And it's really easy to take claims data and blame something on the drug. But when you start to talk to patients, you find out all that valuable information like, "Well, I was prescribed it, I filled it, but I didn't take it because of this or that."
Or, "I took it along with my meditation practice and I really think it's the meditation that made the difference." Or it's the, "I haven't told my doctor about my illicit recreational drugs, which maybe has an effect on my health." So I think you're referring you're giving the opportunity to patients to bring information in a confidential way that could be extremely important to your health, and really have a much more a fuller picture to allow us to evaluate both safety and effectiveness.
Matt Veatch:
Fantastic, thank. Yeah.
Dan Drozd:
I'll just build on that. I really, I think that we see in practice the difference at times between the efficacy of drugs in clinical trials and effectiveness of drugs in the real world. And I think that there are obviously many reasons for that. But the fact that patients at the end of the day are people, right? They're all of us, and you may go to visit your doctor once or twice a year, as Nancy mentioned maybe that's not the best time to do a six minute walk test for you are to check your blood pressure or various things like that. And that our ability to gather that information and really understand what the the overarching journey and experience of a patient as they move through their lives is, is I think incredibly powerful from a research perspective, also incredibly powerful obviously from a clinical and care delivery perspective, which is maybe a whole other topic.
But I think as a physician, anyone who has taken care of patients recognizes that that kind of day to day life challenges that patients have, ended up having significant impacts often on kind of their ability to comply with medications as they're prescribed, the potential effectiveness of those medications in their day to day life and ultimately the impact of those meta medications on improving their quality of life. So I think, lots of possibilities there, both from a research perspective in a care delivery and optimization perspective as well.
Matt Veatch:
Yeah, great thoughts as well. And so just kind of picking up on a thread and something that Jane mentioned in the opening comments is sort of the idea that decentralized is not a separate paradigm. And it is really part of mainstream political research today, and shouldn't be held out as distinct. Does the same hold true for real world data? I mean, should we call out real world data separately? Or is it really part of the ecosystem of how research is conducted today?
Dan Drozd:
Who can challenge that? I mean, it's clear it's become part of the landscape of research today. It's been an interesting journey watching how trust has been built in real world data. Now, anybody who's had access to their medical record might occasionally raise an eyebrow when they see what the doc wrote compared to what they told you. But overall, what you're seeing in medical records is useful. And that's been showing time and time again. So we started with health insurance claims. And we were all frustrated, because it's just a diagnosis code or a rollout code. Now we have the richness of the EMR. So the end, if you look at every real authority around the world, they're all interested in real world data now. I don't think there's anybody who's not open to it and starting to make decisions on it. So we've passed that hurdle.
The question now is, what can you trust that's happening outside the health insurer and outside the supervision of the doc? And my reaction to that is it's about time. Because what we're talking about is tremendously valuable information that we were blind to before because it was just too hard together.
Matt Veatch:
Yeah, great [inaudible 00:17:17]... Kim?
Kim Barnholt:
Oh, go ahead. No, I was going to build on that and say I absolutely think it is part of our ecosystem. And I think we would be stupid to ignore the valuable resource a wealth of information that it provides. I think Nancy said earlier, we're just scratching the tip of the surface of what's possible. And I think the more that we start to integrate it in, the more we'll learn with that. I would say we can't necessarily just consider it all folded in because it is a complex type of data, there's a lot of different considerations around how we work with it, how we structure it, how we are able to harmonize it and integrate it in. So I do think that we're still new in terms of learning how to manage it, and how to understand the quality of it that it takes a little bit of white glove to make sure that we're using it right and integrating it correctly.
So while I think yes, in terms of we shouldn't be shunning it, and we should absolutely be pulling it into the conversations, I think it's not quite at the level of some of the more traditional data sources that we understand really well, that we need to make sure we take some care to bring it in correctly so that we don't go too fast, and then lose the opportunity to actually kind of have it become more status quo, if that makes sense.
Matt Veatch:
True does. Thanks Kim. And Dan, I don't know if you would like to contribute to this particular thread. It seems that PicnicHealth's entire business model is kind of predicated around just the incorporation of real world data. It's central to the corporate strategy.
Dan Drozd:
Yeah, absolutely. I mean, I think it's a recognition that we are in this period of transition really between kind of these silos, that have existed in the past. Between what we called real world data, claims originally as Nancy mentioned kind of progression over time. And that what we're at is, I think, this exciting point where we are seeing more and more progress towards towards integration and use of this data across a variety of use cases, kind of throughout the development lifecycle for for pharmaceuticals as well as obviously post approval and kind of more, "traditional" use cases. I think that we're at a really interesting period of time, and as you said this really is core to the way at PicnicHealth that we think about real world data and about ensuring that the patient has a voice in that process.
And that by leveraging kind of the voice of the patient and the access that patients are entitled to to their own healthcare data, that there really is a unique unlock that's possible in terms of collecting more comprehensive and complete data coming out of EMRs. And then being able to link and leverage that data to other data sources as well.
Xerxes Sanii:
If I can add, I think like when Kim was, spilled on a dance that I think when Kim brought up the whole concept of like, human centric designs. As she was saying that in my head I was kind of, that's like practical problem solving for a question you're trying to answer. Right? And kind of to the question of is, real data part of that? I think it is. But I think what's really interesting now, and like what we're seeing a lot and what's I think very promising is, I think people are more willing to have like, are willing to engage in all the nuance that goes into having these decisions. I think a lot of the tension that you see from, I'll just say "traditionalists" and kind of people who are pushing more novel ways or, "This is not controlled," or, "I don't know how to interpret this." But I think as an industry, as a kind of community. I think having those nuanced discussions that again, Kim was alluding to, "Hey, this data is actually useful for this, or this wearable will solve this problem."
I think we're all kind of at a place where we're willing to really think about these things in more individualized ways. And I do feel like that has been a big driver of some of the progress that we've seen recently.
Matt Veatch:
Yeah, fantastic. And Xerxes, just out of curiosity, because of your role and really managing corporate strategy for the company. One of the things that I was kind of reflecting on is the some of the negativity in the press around accessing data. It's almost always, for the lay person, the lay consumer, the lay patient out there in public, it's always presented in kind of a negative light. There's data access breaches and things like that, how is it that... And what are some of the ways that that's mitigated? Where patients are made comfortable to share their data, to grant consent, to allow that access to their records?
Xerxes Sanii:
Yeah, it's a great question. And I do think that to some degree, there's no generalization, I think, still remains in. But I think there is sometimes a misconception that people don't want to share their data. I think, especially when you're going through something... We see people all the time, "I'm going to go do a walk for this disease." Or, "I want to do like..." I think wanting to make meaning I've been wanting to contribute in a certain way. And so I do think there is, in many individuals like an inherent nature to want to contribute. And I think those challenges you alluded to are certainly there of, "Hey, what about data breaches, or who will see my data?" And so I think our philosophy from the beginning has always been consent and transparency. Everyone on this call is in the aggregated claims data set, right? And it's going left and right. And so I think our model has always been own your data, be empowered with how it's used, who it's shared with.
And I think just a core principle of a company from our founder, that I believe will never change is just kind of giving patients that ability to turn that switch off if they want. And then just being able to explain... People don't want to hear about cybersecurity details. Right? But I think just being able to communicate in clear, layman's terms, how data is kept safe, and what certifications you may have things like that. So, I think trust within the patient populations that we serve is paramount, for sure in everything we do.
Matt Veatch:
Absolutely, yeah. And just the stark differences between the randomized control trial, which is informed consent is a critical component of that, versus real world research which is obviously can be consented, should be consented, depending on the the nature of the research. But those worlds in some ways have kind of collided. And it's interesting to look at the impact and how that affects patient engagement, specifically. Any other thoughts about, from the patient perspective, sort of signing up for something where their data is going to be accessed. Any sort of real world examples of where that's worked particularly well in a research project?
Nancy Dreyer:
I can offer pandemics. I mean the big push in the pandemic is when decisions can't wait, you have to do something. And we often saw patient centered, patient driven research come to the forefront is critically important. Kim?
Kim Barnholt:
Yeah, I was actually going to speak with a little bit of a previous role have that I used to work at 23andMe and with digital patient communities, and a lot of it was sharing genetic data in the spirit of research and moving the needle forward in terms of discovery. And we found or I guess, I personally found in working with patients, they were actually some of the most likely to share. I think they there was a real need to get answers and they were very driven to be part of that process, and it almost was empowering to be put in a position where they had some data to contribute towards something that was helping them, but also helping the community of people that that were having similar experiences. And I think there's almost, and again, I think you can't generalize and not everyone is the same.
But sometimes by making this part of something bigger, a part of a process, there is something very empowering about consenting to share and being part of research versus, as you said, kind of claims data on EHR data that may be passed around anonymously that you don't have any say in or control in. And so I do feel like when people feel like they're a partner, and they feel like they're empowered, and it's a decision, and they're doing something that is active and proactive, that people are more likely to share, and it almost builds more trust, because there's transparency to it. Versus sort of what you don't know, and you make assumptions and layer a narrative on around that.
Xerxes Sanii:
Kim, I think you just triggered one thought which when we worked... One thing I've come to realize more than I think I appreciate it before is oftentimes the way kind of patients see their disease, clinicians see it and researchers see it can can also be so different. And one, we've done some work in hemophilia recently. And as part of that did a lot of surveys that were geared around, do you feel anxious around when you have to infuse yourself? Or what are like, some of the things that people don't often talk about of like there's a whole treatment journey. And oftentimes researchers they see it as, how often are you bleeding in a six month period, a 12-month period? And the participation and just like the unsolicited messages we got from patients. "Oh, thank you for asking me about this." And they were really eager to share about their experiences with these things.
But it's something that they had just never been asked in either a research or clinical setting, but was very top of mind for them in terms of how their disease impacts them and how they feel day to day. So that was a pretty eye opening experience for me.
Dan Drozd:
This is building off that a little bit. I completely agree. I think that the kind of empowering patients is clearly a very different kind of model than many of the things that we read about in terms of data breaches. "My data was stolen from Home Depot, because I bought a hammer there." or something like that, right? This is very, very different. And so I think that giving people choice and giving people the power to proactively consent into a data set rather than being included without necessarily their knowledge, or in some little addendum at the bottom of a forum that they happen to have signed when they went see the doctor is very empowering to people. And as Xerxes said that kind of asking questions that aren't necessarily the sorts of questions that might have routinely been asked in a clinical trial, for example, and really understanding a much more complete and 360 degree view of a patient's experience and how their illness impacts their life.
Not necessary, which may be kind of a hard clinical outcome that we might look at in a trial, or it may be something completely different as Xerxes said, anxiety around time I need to infuse factor in hemophilia patients. And the other thing that I think we've danced around maybe a little bit is value to patients, right? And value can come in kind of many different ways. And certainly one value can be fulfillment of this. How do I make meaning out of my own journey with this condition or with this disease? And is there a way particularly for conditions that have some genetic predisposition? If you're diagnosed with something and you know that your child has the chance to develop whatever that might be, this becomes an incredibly powerful motivator for people to contribute, obviously. I think that our platform in particular really kind of places an emphasis on how do we ensure there's a voice for a patient? And how are we making sure that as researchers, we're thinking about the patient as more than a series of numbers or kind of traditional outcomes?
How do we provide value to patients in terms of in many cases, providing a platform or a vehicle for people to be developing some meaning from their own journey and what they've been through. And then lastly, very tactically, like just having your records makes your life easier, right? Like people change doctors and health care facilities all the time. All the time you changed insurance because you got a new job. And I think for patients with complex conditions, there's a project management kind of components of what they... We all have that in many different places within our lives, but the extent to which we can take some of that burden off the patients and really allow them to live more fulfilling and meaningful lives and have less of their time consumed with kind of managing the busy work of their condition, I think is a real value adds for patients and ends up being extremely powerful the motivator as well.
Matt Veatch:
I love that. It's the voice of patient. It's empowering the patient as part of the research process. It is part of decentralized. It's bringing the research, the modalities, the tools to them in a way that really changes the research paradigm overall. I think it's great. And that kind of leads to another question in my mind. It's one thing to have patients understand the value of their real world data and consenting for that. It's another to actually have them participate in a trial or a study. What's necessary to sort of bring the patient from that agreement around their real world data to actually participating in a study? Dan, you have any thoughts about that?
Dan Drozd:
Yeah, I think the answer is it depends, right? It depends on the objective of the study, it depends on what's being asked for a patient in a particular setting. But I think the more that we can move, reduce barriers towards patients participating in trials. So we've talked some about wearables and other kind of, we can talk about patient reported outcomes and other ways. In the Haemophilia cohort that Xerxes mentioned, while we do gather a lot of information around things like symptoms that patients may be having, or as he said, kind of anxiety in the context of infusing factor. We also ask patients about kind of much more traditional measures, how often are you having bleeding events, etc. And so I think the more that we can simplify and make kind of lower burden for patients, reduce the number of times that patients need to go in to travel long distances, to go into sites, if we can do phlebotomy at home, all of these kinds of things. There's collections of tools that we have in our toolkit, and the right combination of those tools obviously depends on a particular study.
But I think unquestionably the more that we can lower barriers to patients participating, the more, the easier it is to get patients to participate. And I think at the end of the day, the richer the data that we'll be able to collect.
Jane Myles:
And I'm going to double click there. I have two questions, but I'm going to stick to one for now. So when you give patients their medical record, are they also asked if they are willing to be contacted about clinical trial opportunities? How does that work? Because the last mile is just such a big hurdle right now?
Dan Drozd:
Yep. So we do have the ability to do that. And whether we do that depends entirely on specific situations. Right? So we do have the ability, we obviously... Part of what I think at the end of the day PicnicHealth's biggest value proposition is, is that contact and ability to work. That relationship that we have with patients directly. Right? And so there are a lot of things that that unlocks, potentially from a clinical care perspective, from a research perspective, etc. And I think that, yeah, so the simple answer to your question is that is the sort of thing that we have the ability to do, and whether we choose to do it in particular conditions, or that varies depending on the situations.
Jane Myles:
I would say don't undervalue that. There's tons of de-identified data sources which are helpful. But the direct contact to an identified patient with their consent is golden.
Dan Drozd:
I could not agree more that if you asked me to pick like what is our biggest differentiator it is as an organization amongst many other things that we've alluded to here, I think it is that relationship with patients, that direct relationship with patients.
Matt Veatch:
That's great. I think Kerri had a question. Oh, sorry, someone...
Nancy Dreyer:
That was me Matt, I was going to just amplify what Dan said about being human centric, to use Kim's word because it's great. Because that relationship, that connection and Jane your point about a faster way to recruit and get right to people is huge. But then that ability to... The thing I think that's cool about what Picnic is done is they're already doing something of value for the patient regardless of research. So we start out giving patients something they can benefit from day in day out, period. Now when you add things on that you've already got an enthusiastic user base. I think that's got to be important.
Kim Barnholt:
And building on that, Nancy, I think some of the hurdles to trial awareness, trust and access. And I think that the more that patients start engaging with their data in the real world, so a lot of people have an Apple watch or a Fitbit, they start to get more comfortable with data, they start to understand what they're seeing in their own life whether they're a patient or not a patient, you can start to see trends, you can start to, "I don't feel well today. Wow, I didn't sleep well." Also, we're starting to introduce... And then Picnic is a great company around introducing access to medical records, taking away that stigma that that's something a doctor take notes, and then you never see. So we're starting to onboard... Sorry, my phone is going. We're starting to familiarize people with data with kind of the what data means, how data translates into opportunities for improving your health or to be empowered around decision making as part of your health or part of research.
It removes raises that awareness, it removes some of the barriers that might start building the trust, because then you're shifting the conversation. And then I think with some of the decentralized approaches through real world data, but then also some of the decentralized trial elements, we're increasing that access. So you're starting to touch on each of those barriers that I do think real world data plays a critical role in helping us address and remove for broader participation.
Matt Veatch:
Great points. Thank you. Kerri, I know you had a question. And if not to put you on the spot. But we would welcome your question and maybe it's already been addressed.
Kerri Cali:
Hey, Matt, yes, of course, thanks so much. And I apologize, I was a little bit late, I'm just coming off of... I'm still on parental leave for two more weeks. So my baby's awake and alert right now. So I'm going to stay on mute as much as possible. But, two things. One, I wanted to build on something that Jane had said, and then something someone else said earlier. But what I find really interesting in using real world evidence is twofold. It's one using it to find patients for trials, and then to how we incorporate the data in the trials. And to build on what Jane said, it's like using it as clinical research as a care option. So having that as an option. So when a doctor tells the patient, "Hey, these are your options for treatment, including clinical trials." I worked a lot in multiple sclerosis, cancer, other neurology studies. And that's what the doctors, a lot of them who are trained in research, they just tend to do that. They say, "Here's what's available in terms of treatment in terms of clinical trials."
It would be better if more folks were educated about that so they could do that. And also, on the other end of finding patients, which leads into clinical research as a care option. One thing that we're looking at in some oncology trials is using some of these genetic testing vendors to find patients. However, for those mutations that are not treatable mutations, a doctor might not necessarily look for that. And it might be difficult to to find in an EMR record. So we are using some genetic testing vendors to find those patients and offer those trials to them as a care option. But a few and far between and it's still kind of newer, a newer thing that folks are doing. So we're testing it out seeing if it's going to work. And I didn't introduce myself from before. So I apologize. My name is Kerri Cali and I currently work on the sponsor side of things. I'm at recursion out in the Midwest. And we're a tech bio company working in rare disease and oncology right now. So I'll just pause there and see if anyone has any thoughts or comments on that.
Matt Veatch:
Great points, Kerri, and I think it is such a critical, I mean, taking your second points around the incorporation of genomic data and looking for various biomarkers. I think increasingly, that's becoming central to research, certainly talking to a lot of sponsors myself, understanding what they're looking for, and where their research is migrating towards, there are considerations. And anytime you're capturing genomic data and returning results to patients, there's an obligation to provide genetic counseling, for example, and how that's managed, those are really designed considerations, I think, for the nature of the research.
And Kim, turning to you not to put you on the spot, but given your background and former roles, is that something that you are actively thinking about in the new role. It's how genomic data can influence the design or conceptualization of a study, because fundamentally, we're talking about decentralized again, it's just another tool in the toolbox. So really the core issue, why is the research being done in the first place?
Kim Barnholt:
Yeah, I mean, and I'm a little bit far from the the genetic side of things now, but I do know there's a Genentech in many companies personalized healthcare is the Holy Grail. It's 10 week. Ideally best target the right drug, the right patient at the right time. And biomarkers are absolutely a key to that. So it is a core part of a lot of the design of our trials, particularly in the oncology space. I think where real world data can add value is thinking about patient burden, if they're already having lab tests, if they're already doing some biomarker testing, rather than making them go through biopsies again, and again, potentially, can we pipe those data and use that as part of their screening? Can we better understand, is this patient the right fit for this trial, rather than making them go through multiple test, drive hours to a new center. Even from a sponsor side screening can be very expensive or rolling up a site can be very expensive. So is there something we can know about the patient's fit for the trial, to optimize the patient's experience, to reduce screen fails, it's expensive to sponsors, it's frustrating to patients.
And I think leveraging the data, the genomic data that we already may have, through the course of a patient's treatment, would reduce frustrations, costs, and just barriers all around as well as ideally, setting the trial up for success, which then is more in the space of, more likely to get the drug out to the market for the right patients who it may work for. So I am a strong believer in that. I also believe, I think one of the things, Kerri, to your point about in the perfect world, we have physicians to understand about their patient, whether it be biomarker driven or certain disease characteristics, and recommend either the care option or a trial as a care option. I'm not sure we're quite there yet. I think that's something that we really should strive towards. And I think where we're seeing, maybe the greatest opportunity to continue through this education awareness is in some of the underserved populations where they aren't necessarily always given the trial option.
And so I think that's something also that we're working with, within our company is how can we better connect in with community sites? How can we provide them with data potentially, they may not already have about their patients that would help them better understand a fit for trial, as well as helping make sure we're aware of trials to be able to refer those options for their patients.
Matt Veatch:
You can just build on that for a moment and finding those underrepresented patients. There's a lot of talk and discussion. And in fact, an entire forum discussions around social determinants of health, is that something that is often from the medical record, it may or may not be as comprehensive as needed to really make those determinations. Is that something that you think about in terms of how to sort of enrich the existing and accessible data with social determinants?
Kim Barnholt:
Absolutely, I think it's a really valuable data source. I think it's still finding the right quality of social determinants and kind of the right data source, having that information. But one thing that we're really excited about is data linkage. It's having trial data but then it's layering on real world data sources to help enrich the information that we're gathering through the trial process. And social determinants is absolutely a critical data source for being able to better understand the influences of different societal factors on to a patient's health and health outcomes.
Matt Veatch:
Yeah, fantastic. So we've got about 15 minutes left, and I just want to encourage additional audience questions and sort of take advantage of some live participation. And while we're waiting for hand raises, I will turn to another question. And that's the the conundrum of working with real world data. It's powerful in many ways. It's also notorious for its data missingness. And trying to overcome that missingness is something that is critical to its uses. It sounds like PicnicHealth would be in an ideal position to bridge that because you can just ask the patient questions or get additional information. And I'm curious to see to the extent that PicnicHealth actually tries to enrich the data that's coming in. Could you touch on that just a little bit?
Dan Drozd:
Yeah, happy to. So yeah, we do think that this is a really unique position, that we're in a really unique position to address this issue. And so we think of missingness in a couple of different ways. So if you look at a traditional EHR data vendor for example, they may have data only from a subset of sites that a patient may have been seen about, is seen at. So you may be missing clinical outcomes, key predictors, a biopsy report, a result of imaging etc. And so because at the end of the day, the patient really is the only through line and their journey through the US healthcare system. We do think that by putting the patient at the center of that process that unlocks the ability for us to go out and collect more comprehensive and complete records for patients, we have an entire both technology and kind of operational components of the way that we think about trying to meet that challenge. So that's kind of one component of completeness, there's both a lot, there's a longitudinal component to it. And there is also a data depth and missingness component to it.
And then the other part that you alluded to, or the kind of the other way that we think about it, as you said is how can we enrich that data, things that may not be present in any EHR record? So we've talked about, okay, if there's a biomarker that isn't routinely tested, because it's for an untreatable mutation, that that may not be something that a doctor or physician is going to order for a patient. So there, we do have, depending on what the kind of key missingness is different mechanisms where we can link to other data sources, that direct relationship with patients obviously enables us in situations where it's as "simple as asking," we can go and ask the patient about particular data, administer ePRO or something like that to the patient, if that's the kind of core data that's missing. But I think lots of ways that by putting the patient at the center of that process that you really kind of unlock the ability to help address that missingness challenge.
Matt Veatch:
Great. Thanks Dan. Turning to Jane.
Jane Myles:
Yeah, I warned you ahead two questions. I have an infinite list. But this one is about what you were just talking about, Dan. So real life. I've lived in the same house for 22 years. But it turns out, there's nothing in my EMR before 2012. Nothing. I've had the same practitioner. But if you were trying to screen me for a trial, you wouldn't know anything about my diagnosis. So how do you solve for that gap? Like the data is there, but it's not in an EMR?
Dan Drozd:
Yeah, so our... The answer to that question is, at the end of the day, we can only collect from a record perspective what's there. So as long as it's there, right, and that's a big caveat in some situations, right? If you're a practitioner, your practitioner isn't required to hold on to your records in perpetuity, right. So there's expiration of requirements for maintenance of records. Obviously, the those laws largely come from a period of time in which physicians had paper charts, and we're having to pay to store things in warehouses. But so for older records, we do have the ability to get them as long as they're there. And the way that we approach our kind of data ingestion pipeline is at the end of the day, will collect records in whatever format they happen to come in. Any clinicians on the call certainly know that the fax machine is still five bar, the technological implementer of choice for data exchange, from physician to physician.
When I talk about this to non physicians, that boggles the mind, but there's no question that if you're at a doctor's office, and they want to get records from another doctor's office, they are going to call up that office and give them their fax number, and those pieces of paper will come rolling off the printer. But our data ingestion system is really built to ingest data no matter how it comes in. So we have records. To be clear, I'm not saying this is the norm. But we have records for patients dating back to the 1950s handwritten records dating back to the 1950s. And so we're able to leverage technology in our in our core platform, to take those records, run them through a data processing pipeline, generate text out of those records, and then structure that data.
And so at the end of the day we're able to harmonize that data that's ingested. And we should be mindful of the challenges there and limitations of that, of course. But core pieces of data, we're certainly able to abstract from paper records, faxes that come in, we're obviously very happy and increasingly seeing electronic access to data. But I think most people on this call probably know we're not really there yet.
Matt Veatch:
Great. Yeah. Thanks, Dan. Now Kerri, I think you may have had another question, not to put you on the spot again, but welcome your question.
Kerri Cali:
Yeah, it was just one building on something and I apologize asked this question earlier, a few minutes that I wasn't on. But I was thinking more about how you incorporated that real world data and clinical trials. There's so much burden on the site and the patient in especially in oncology trials for duplicative pieces of data. So I'm curious about how you utilize those records. And I recognize that it's probably an early stages, and a lot of folks are working to do this. But just curious of what you've seen so far in your experience, and how you can be more efficient using real world data to minimize the burden on sites and patients and duplicative data and errors as well.
Dan Drozd:
Yeah, I'm happy to offer kind of the PicnicHealth version of that, but I think it'd be good to hear from Nancy or Kim as well. So we do a significant amount of work in this area and multiple mechanisms, we're kind of integrating that data into existing clinical trial infrastructure, depending on what the specific use case happens to be, is the data being used for screening, is the data being fed into an EDC directly or indirectly, in some way, etc. And so is a real challenge for sure. And we definitely appreciate the fact that if we look at like what the substantial challenges are, obviously a number of challenges today, but site burden is clearly a large challenge and making sure that we're minimizing burden on sites and ultimately, hopefully, allowing more and a broader kind of diversity of sites to be able to contribute to research is certainly top of mind for us as well.
Kim Barnholt:
Yeah, leveraging the EHR, maybe reducing double data entry from sites so they can use just directly, have the EHR be fed into the EDC. I think wearables is one way, maybe we can do some of the remote collection at home. So it's not necessarily having to go in and have an assessment being done out of sight but something that patient just directly is gathering. We're also looking at real world data in some of our long term follow up studies. So are there ways that we can and this is sort of where we're applying data linkage. Are there some things that we can do if a patient who is willing to consent to be tokenized, or some other way where we can anonymously connect their trial data to their real world data, do a the latter end of a trial, through real world data follow up versus having it be part of a traditional trial setup.
So these are the some of the areas where we're looking at real world data, and implementing and trials. Obviously, we're leveraging it for helping with trial design helping us better understand inclusion exclusion criteria, helping us find patients and sites. But within the trial itself, and from the evidence perspective, it's I think that the biggest potential we're exploring right now that we see the most obvious immediate value is some of the longer term follow up areas. And also talking about missingness trials that may have some optional assessments where patients aren't completing all of the assessments, can we fill in some of those gaps with real world data, and so that we are more powered to get the insights that we're trying to derive through the trial itself?
Kerri Cali:
Thank you Kim and Dan. And one more question on that. And if you want me to be a little bit more specific, as well, so I'm thinking about scans, right? Sometimes in a trial, they'll say, "Okay, well, if you've had of scan in the last six months, that can be included. And then you have to have at your baseline visitors sometime in screening two weeks prior to the baseline. So I'm just curious in terms of scans, given that you have all these other vendors that maybe are on a study to perform the assessment, so then you have to get the scan in some format that works for them. So then that's it, this becomes a burden for the sponsor, for the participant, for the site. So I'm curious, how will it work for scans? Or how are you guys seeing it work for scans? Is it easier? Is it getting better?
Dan Drozd:
Yeah, so we do actually collect raw DICOM images from CT scans, MRIs, plain film, X rays, etc, and have access to those, both give you access to those patients directly so that patients can look at that data through a viewer within our application, but also then have the ability to share that data in that kind of industry standard DICOM format, which can be loaded onto a fax machine etc, for radiologists to read. So can share both with patients and then with sites potentially, both ORION and Jiang and to the extent that it's relevant for a particular process, the report that was generated by the reading, the original leading radiologists as well.
Matt Veatch:
And then just to pick up on one thread there, and that could have triggered in my mind the thought that often in research there's Core Labs involved with imaging. I presume PicnicHealth works with a variety of partners. At least looking at domain experts, is that something that is possible for the company to do?
Dan Drozd:
Can you...? I'm not sure I totally understand the question.
Matt Veatch:
Yeah. Say at Core Lab was involved in looking at images in a trial, is that a partnership that you would engage in depending on what the sponsor wanted to do?
Dan Drozd:
Yeah, absolutely. We have kind of a broad suite of partners that we've used and are certainly open to exploring kind of relationships with other partners as well for specific use cases. So, absolutely.
Matt Veatch:
Great. It pains me that the hour just kind of whipped by it's been a great discussion, I'd like to turn to all of the panel and Xerxes as well, to just kind of give final thoughts. Xerxes, maybe I'll start with you. The question being given your role and rebidding strategy, what comes next? I'm presumptive, it's US focused for now. Is there some international expansion for the company? What are some of your thoughts around your corporate strategy?
Xerxes Sanii:
Yeah, I mean, I think a lot of this conversation kind of alluded to it. There's a lot of kind of small incremental changes we've been making and how kind of research is done. And I think just continue to keep our head down that path. I keep going back now to Kim's kind of human centric, and what's the practical way in which we can create trials, I think there's advancements in technology that we've contributed to, in some part. But there's just the human centricity that we are able to design trials with is increasing, thanks to those advancements. And so just continuing to kind of move along that spectrum and just making these designs more possible, by ensuring that the scientific rigor and all the other kind of components of the study, still meet the overall goal. So I think continuing down the path of long term follow up came up a lot in the end of this conversation.
And I think that is one area where we see a lot of opportunity where you can imagine the future of every clinical trial there's almost like, a checkbox or when you're designing it and when pharma companies are designing their trials, "Hey, do you want to link to these datasets? Do you want to generate patient mediated Picnic like data." Then it becomes kind of more than norm. I think that that is kind of a big part of the future that we're looking to shape.
Matt Veatch:
Great. Well, that's fantastic. And, Nancy, in sort of closing commentary, I'm sure you're asked this a lot, given your vast experience in designing research. When is it appropriate to focus on the real world data, you start with a research design? Is that something that you consider the real world data source upfront? Is it sort of as you're conceptualizing the protocol? When does it become the factor that you incorporate into the design itself?
Nancy Dreyer:
Well, studies, I found, are always better when you start with real world data. Because if anyone on this call, who does clinical trials knows how challenging it is to enroll, and part of the enrollment challenges are often due to having nice to have, Ron generously called them inclusion/exclusion criteria, there may be somebody suggested sitting around the table thought it was a brilliant idea, they didn't know would knock out 90% of their eligible population. So we use real world data a lot to see if the patients exist. So I think that's important. But Matt, I can't leave, one of your comments about missing data. I mean, I think the myth was that all data that's not there is missing, because you didn't report it. Not that the test wasn't done. And when we have the EMR and when you can do source data verification by checking back with the EMR, I think that will reshape a lot of people's ideas of what you should have done, but you didn't for whatever reason, we're not missing it, it wasn't done. Thank you.
Matt Veatch:
Yeah, great, great points. Thanks, Nancy. Kim, turning to you, from the sponsor perspective. Just thoughts on the future and sort of your role and use of real world data. You've touched on many topics today. Any kind of closing comments?
Kim Barnholt:
No, I mean, I think we're working internally, as well as externally to really help shape the ecosystem where real world data does become more part of practice. And I think, taking a very practical approach to finding the use cases where it makes sense to continue the conversation. We started where I think in the past, historically real world data, and then your randomized clinical trials were very far apart and we're moving towards where these are now part of the same conversation and really working as a cohesive set of evidence to be able to understand the patient experience, understand disease and really understand drug response. And I think we're on that journey. I'm excited to be part of that journey. And I think, as a data geek at heart, we have so much data in the world, I think we could answer so many questions if we just start connecting it, mining it, reusing it. And I think real world data is just a key part of that treasure trove that we should not ignore.
Matt Veatch:
Love it. Great, great comments, great thoughts. Dan, turning to you, Chief Medical Officer at PicnicHealth, what are you most excited about in terms of the incorporation of data into clinical research at large?
Dan Drozd:
I think it's really at the end of the day, just increasing access and the ability of patients to contribute and making sure that that voice of the patient is really at the center of the research process. That when we're generating evidence that it's being generated with the patient and the impact that that our treatments can have on how they live their regular lives, not how they live their lives when they walk into physician's offices intermittently.
Matt Veatch:
Fantastic . I know unfortunately, we're out of time, but this has been fantastic. Special thanks to everyone, participants as well. And we look forward to the next circle discussion coming up, to be determined. Maybe our date has been set, I think it has. But I'm really looking forward to that. Closing comments from Jane and Paige from an administrative perspective.
