ASMD Accelerate

It takes about 4 weeks to retrieve most historical medical records. After that, it can take another couple of months for us to track down the oldest and trickiest records to access. The timing depends on how quickly your child’s doctors respond to PicnicHealth's requests for records.

PicnicHealth will see your child’s medical records to process them and Wylder Nation Foundation will see the records to understand the complete care journey and what anonymous data elements are relevant to the broader ASMD research community. You can choose to share your child’s records with your healthcare providers or loved ones by clicking "Send Records" at the top of the PicnicHealth timeline. By signing up for this study, you're agreeing to share your child’s de-identified health information and data from your child’s medical records with ASMD researchers to better understand ASMD. For purposes of the study, they will receive health data that are stripped of your child’s name, address, and similar personal identifiers. In certain situations, to make sure that the study is being done properly or to check the quality of the data, regulatory authorities, institutional review board members, or study auditors associated with may be granted direct access to copies of your child’s medical records (i.e. they may see your child’s medical and personal information) without violating the confidentiality of your child’s data. 

You or your child can join this study if you have been diagnosed with an acid sphingomyelinase deficiency (ASMD), have received medical care in an English speaking country, and copies of the medical records still exist. People of all ages can join, with parents or legally authorized representatives able to enroll children, including those who have passed away. The study is currently focusing on the following types of ASMD:

• Infantile Neurovisceral ASMD (Niemann-Pick Type A)
• Chronic Neurovisceral ASMD (Niemann-Pick Type A/B)

PicnicHealth works directly with people like you or your child to help collect and manage medical records. That means going out and collecting all of your medical records from different doctors and keeping them up-to-date over time. PicnicHealth will then transform the records into a timeline that makes it easier to find what you’re looking for. When you see the doctor again, PicnicHealth will update the timeline with new records. You can access your PicnicHealth timeline from anywhere and even share it with your doctors with a few clicks.

Yes. You can view this sample timeline.

For this research study, we will collect records going back as far as possible. In most states, doctors are required to keep records for at least 7 years, but many will keep them for much longer. So how far back we can go really depends on the record keeping practices of your child’s doctors.

PicnicHealth and Wylder Nation Foundation will always protect you and your child’s rights and privacy. PicnicHealth will de-identify your child’s data before sharing it with researchers outside of Wylder Nation Foundation, stripping away personally identifiable information. This includes, but is not limited to, your child’s name, email, date of birth, phone number, address, and the names of your child’s doctors.  

You can enter all your child’s doctors, including pediatrician or primary care physician, metabolic specialist, geneticist, cardiologist, neurologist, pulmonologist, hepatologist, and more. In addition, you can enter other allied healthcare professionals that help manage ASMD, such as dieticians or nutritionists.

You may choose to delete your child’s medical records and data from the PicnicHealth service at any time and for any reason. Just email PicnicHealth at [email protected]. However, any de-identified data that has already been shared with a partner, up until the time you notify PicnicHealth to stop participating in the study, cannot be modified or deleted. This data will not include your name or personally identifying information.

Participants may be enrolled in the study through the following informed consent approaches:

• Participants over the age of 18 signing the informed consent form
• Participants aged 7 – 17 signing the assent form and their parents or legally authorized representatives (LAR) signing the informed consent form
• Parents or LAR signing the informed consent form for participants under the age of 7
• Parents or LAR signing the consent form for posthumous participants

When you first see the timeline, it's possible that some of your child’s medical records are not yet available. That's because PicnicHealth is still working hard to collect them for you and your child. You should be able to see the medical records within a month of signing up. After the first month, the timeline will be updated regularly with information from any new doctor visits, if applicable.

In the first months, some records might be missing because PicnicHealth is still collecting them for you and your child. After the first month, if you suspect that records are still missing, you can let us know about those records by clicking "Request Records" at the top of the timeline.

The goal of this study is to advance the understanding of ASMD and to support research to improve quality of care. Information from medical records is particularly useful for diseases, such as ASMD, because it gives insight into disease progression and how treatment options may work in the real world, while being able to participate from home. Some ASMD research objectives that your health information can help answer include understanding:

• Disease progression
• Differences in outcomes by ASMD subtype
• Symptoms, health complications, and other disease-related conditions
• Treatment patterns and their effectiveness 
• Unmet needs

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You can just enter as many doctors as you remember. The hospital or clinic name is usually enough and the PicnicHealth team will follow up with you if there’s any confusion. If PicnicHealth finds doctors mentioned in your child’s records who you did not include on the doctor list, you will get an email asking for your permission to request records from that new doctor.

PicnicHealth will collect medical records from all the U.S. doctors, hospitals, and clinics that you tell us about. PicnicHealth does not collect dental or optometry records.

PicnicHealth takes privacy very seriously. Your child’s personal data is never shared without your explicit permission and we abide by HIPAA (Health Insurance Portability and Accountability Act) compliant practices. To keep your child’s medical records safe PicnicHealth uses the highest encryption standard available: 256-bit SSL. This is the same technology that banks use to keep account information safe. 

Researchers interested in studying ASMD using the de-identified data collected can contact PicnicHealth at [email protected]. Researchers will be asked to submit an application which outlines their research study rationale, goals, and analysis approach. Applications require background information including personnel involved, relevant credentials, associated institutions, and any conflicts of interest. PicnicHealth then follows a process to evaluate potential partners based on the validity of the research questions and commitment to advance science for the public good with this information also being shared with Wylder Nation.

When you sign up, you’ll read and sign a form that authorizes PicnicHealth to request your child’s medical records on your behalf. You can e-sign this form online -- no printing or mailing involved. Your doctor will then know that it's okay to share your child’s medical records with PicnicHealth.

Children with ASMD may still be enrolled in this study posthumously. Your child’s records can still provide valuable information for researchers to use to advance ASMD research and hopefully improve future care.

Yes! You will be able to view imaging files right in the PicnicHealth timeline.

No problem. PicnicHealth can work with data in any format, whether your child’s doctor takes notes with a pen or on an iPad.

As part of this study, PicnicHealth will collect records on behalf of the participants when they physically exist from physician offices. In the event that it is unlikely PicnicHealth will be able to retrieve the records (e.g. the office no longer has the records, record office is located outside of the U.S.), your physical copies of the medical records will be requested. The optimal approach based on your unique situation will be determined through a series of questions when you sign-up for the study.  

PicnicHealth will update your child’s medical records regularly. To keep the records most up-to-date, you can always tell PicnicHealth when your child has seen the doctor. Just click the “Request Records” button at the top of the timeline, and PicnicHealth will retrieve the new records for you.

Your child’s PicnicHealth timeline provides a single source of information for you, your child, and their doctors. It is a tool to help you coordinate care and understand your child’s health. You can share the timeline with any of your child’s doctors with a few clicks. The doctor will receive a secure link and get access to your child’s medical history. This can make it easier when you meet a new member of your child’s care team - who likely doesn't know a lot about your child’s ASMD experience. You can provide them all of your child’s background more easily.

At the top of the timeline, click on "Doctors" to add new doctors or medical facilities. PicnicHealth will then collect records from your child’s new doctors.

At this time, the study is only enrolling patients with medical records in English. 

Yes. Your child’s participation in this study is completely voluntary and you can withdraw at any time. If you choose to stop participating, your child’s medical records will remain available for you through your PicnicHealth account. At that time we will stop collecting any new medical records. You can download copies of your child’s records at any time. You can always tell PicnicHealth to delete your child’s medical records, although the information that has already been shared with third party partners, such as other academic researchers, can’t be deleted.

We believe it’s important for you to see the results of research produced using your data and we’ll make every effort to share published results back with you (e.g., research publications, conference abstracts). Research takes time, so we appreciate your patience while scientists and clinicians are working on learning new things about ASMD.

You’ll receive PicnicHealth’s service free of charge for as long as your child is a participant in the study.

PicnicHealth and Wylder Nation Foundation are separate companies that are partnering for this study. PicnicHealth is a digital health company that helps patients collect & manage their medical records which can be used to support research. Wylder Nation Foundation is an organization committed to helping advance ASMD research by fostering collaboration between various research channels (e.g., academic partnerships).

Medical records tell a story of what’s actually happening when doctors see patients and can be really helpful for understanding how care is delivered in the real world. They also contain a lot of information about how you or your child experiences ASMD; their symptoms, health status, and outcomes. Putting all of this information together will allow the scientific community to see patterns that no one healthcare provider can see on his or her own. This is particularly insightful for rare diseases like ASMD, where there is typically little known about the disease.

This is an observational study, not a clinical trial. A clinical trial involves testing a specific treatment, like a drug or medical device. In this study, we are looking at data from medical records produced during your child’s visits to healthcare providers. The goal is to better understand how ASMD is diagnosed, managed, and experienced to advance researchers’ understanding of ASMD and hopefully improve future care.