The Case for RWE in Neurodegenerative Diseases
This article was inspired by content discussed in our webinar, Cutting Edge Conversations: Fighting Neurodegenerative Diseases.
Imagine treating a Parkinson’s Disease (PD) patient long before the event that puts them in the hospital. Or identifying an Alzheimer's Disease (AD) patient using caregiver feedback on behavioral symptoms, often forgotten and unreported by those who suffer from dementia. Real-world evidence (RWE) can make the hypothetical a reality.
RWE offers insights generated from real-world data (RWD) and analytics, which can be used to accelerate medical research and improve patient care. Once considered a bold new approach, RWE is growing recognition and acceptance by decision-makers across the life sciences ecosystem. The interest in RWE comes now not only from regulators like the U.S. Food and Drug Administration (FDA), payers, and health technology assessment (HTA) bodies. Increasingly, biopharma companies seek to demonstrate their products' effectiveness in the real world, outside a randomized controlled trial (RCT) setting.
Currently, most life science (LS) companies rely on repositories of routinely collected RWD from claims and electronic medical records (EMR) to identify patients and the providers who treat them.
The information generated from claims data typically captures many patients but may be limited to short time frames and a shallow, incomplete view of patient care in the real world.
Structured EMR datasets have begun to provide some clinical value but lack information on patient characteristics and treatment decisions derived from provider-patient conversations (which can often only be found within the narrative text).
Seeing a comprehensive and continuous picture from blurry, truncated, and siloed data is nearly impossible for any disease. Building a complete picture is especially difficult for patients visiting multiple healthcare facilities – primary care doctors, specialists, supportive care, diagnostic laboratories, urgent care, and so on – an everyday reality for patients suffering from neurodegenerative diseases (ND). Moreover, the routinely-collected data may miss vital information about what happens between visits, an essential part of the story for the neurodegenerative population. We may consider such information anecdotal and supportive in other diseases, however, this data missing in the context of ND can prevent researchers from:
- Understanding the natural history of the disease, from the onset of the first symptoms to the diagnosis
- Tracking disease progression and long-term outcomes, including changes in cognition, disability, or quality of life
- Capturing symptom information in-between-visits, which can inform when and why a patient should switch therapies
- Leveraging patient- and care partner-reported outcomes (PRO/CRO) to understand the total burden of the disease
How can we overcome traditional data limitations to improve neurodegenerative research, treatment, and outcomes?
The therapeutic field of neurodegenerative diseases has been plagued with multiple failures across the industry. FDA Commissioner Robert M. Califf recently called for “innovative approaches to better understand [neurodegenerative] diseases while also building on current scientific and research capabilities.” (FDA)
By working directly with consenting patients who contribute their medical data to research, LS companies could build the complete picture of a patient’s health across all providers - not just one care site or a specialist. The result? Relevant and reliable patient-centric data poised to support real-world evidence-generation programs.
Why neurodegeneration research needs real-world data
Patient-centric data is poised to support RWE generation programs through all development and launch stages. Today, while there are more RWD sources than ever, LS companies face the challenge of finding fit-for-use data that meet their specific needs.
The value of RWE—spanning clinical development, market access, reimbursement, surveillance, and sustained/expanded value demonstration—can be realized for all therapeutic areas; however, patient-centric RWD are especially conducive to accelerating neurodegeneration research.
First, there is more to the neurodegenerative story than what is captured by routinely collected data. Despite serving functional administrative purposes, these sources often miss critical information on what is covered in-clinic and practical details between clinic visits. Moreover, we know that neuropathological change (or what is happening at the cellular level) does not always equate to clinical significance (or a noticeable effect on a patient’s daily life).
Second, people experiencing neurodegenerative disease can experience various clinical symptoms and complications that require a range of care provided by different specialists. Accordingly, the RWD characterizing these complex care pathways spans many treatment sites and specialists. Bringing together data from separate, siloed sources begins with the patient—both in terms of the initial consent and the critical information that can only come from cognitive, functional, and patient-reported measures.
Third, for these neurodegenerative disorders, which are characterized by progressive degeneration, the role of the care partner is paramount. While most traditional RWD sources cannot integrate data beyond the individual patient, direct-to-patient channels are a novel exception that can extend to care partners. Not only does care partner data provide valuable information on patient outcomes, but it also helps to minimize the burden placed on patients to recall/document important events, experiences, or outcomes.
PicnicHealth's patient-centric, patient-first approach results in an expansive capture of historical longitudinal data on the neurodegenerative patient’s journey, even years before diagnosis. By starting with patient consent, we’re empowering patients and care partners to own their data and contribute to research, while enabling long-term engagement prospectively through their treatment. We also know that not all patients with neurodegenerative diseases experience the same care journey. By capturing hundreds to thousands of unique patient journeys, researchers can derive valuable insights from analyzing the constellation of their clinical features. Patient-centric RWD collection means better data, and it’s changing the game for neurodegeneration evidence.
Find out how PicnicHealth’s patient cohorts can help power your research. Contact us
Webinar: Cutting Edge Conversations: Fighting Neurodegenerative Diseases
Infographic: Resolving challenges in data completeness with machines and humans
Article: Comparing PicnicHealth data with existing research databases
As Senior Evidence Strategy Manager at PicnicHealth, Evelyn Pyper is passionate about empowering patients to “make their data work for them” and informing research partners on how patient-centered data can advance their evidence generation goals. Her career in real world evidence (RWE) spans the public and private sector, and regional and global markets - most recently as Associate Director of Market Access at J&J Global Public Health. Evelyn is completing her DPhil in Evidence-Based Health Care at the University of Oxford, with a focus on digital health. Connect with Evelyn on LinkedIn.