I am thrilled to announce PPMD’s plan to launch a patient- and physician-reported outcomes data warehouse – the Duchenne Outcomes Research Interchange (the Interchange) – which will combine data from The Duchenne Registry with clinician reported, post marketing surveillance data provided by industry partners with approved therapies.
The Duchenne Outcomes Research Interchange, scheduled to launch in 2019, will pull together patient and clinician entered data obtained through various means, providing critical information to clinicians, sponsors, payers, regulators, and the patients/families who participate in The Duchenne Registry. We are delighted to partner with Sarepta Therapeutics, as our initial partner.
The Duchenne Registry has been an invaluable resource for this community since its inception over 10 years ago, thanks to the participation of thousands of families, like you, who keep their records up-to-date. The creation of the Interchange, and initial partnership with Sarepta, firmly places The Duchenne Registry at the center of the Duchenne data world. With a new therapeutic landscape, the goal is to combine the powerful patient reported outcomes data currently being collected through The Duchenne Registry with clinician reported data now being collected through post marketing surveillance conducted by industry partners with approved therapies. Electronic health record data (EHR) will also be pulled into this new tool, combining all of this data, together in one centralized location, and benefitting our entire community. The Duchenne Outcomes Research Interchange creates a central warehouse for all types of Duchenne data and ‘real world evidence’ to be collected, aggregated, and analyzed in one place.
Why is the Interchange necessary?
Knowing the importance of ‘real-world evidence’ to regulators and payers, PPMD has been collecting patient reported outcomes (PRO) data – with your help – for over a decade, informing the natural history of Duchenne. PPMD will now build upon this knowledge by combining it with clinician-reported data to better meet the needs of the patient, research, industry, and clinical communities, enhancing the value of The Duchenne Registry’s patient-reported data in a post-marketing surveillance space.
We believe that the Interchange will be a tool that multiple stakeholders can turn to in an effort to combine all forms of patient data, accelerate the clinical trial process, inform access decisions, and bring safe and effective therapies to families faster.
How does it work?
The Duchenne Outcomes Research Interchange is in essence a centralized warehouse that combines data from various sources. Data is entered into the Interchange in various ways, including:
- Data directly captured through a mobile phone based app
- Data you enter on The Duchenne Registry website
- Data shared by your physician during clinic visits
- Electronic health records shared by health institutions and physician’s offices across the country
Having all this data stored in one place paints a very clear picture of Duchenne and how different people live with the disease. Your data, coming from any source, will only be shared in the Interchange with your consent.
What does this mean for my Registry experience?
We are so excited for the updates to the Registry experience for our families coming in early 2019. In fact, you probably have already noticed several changes already – read last month’s blog to learn more. If you are currently active in The Duchenne Registry, the transition will be relatively seamless, providing enhanced access, as well as new and improved features and real life conveniences for a more engaging and valuable user experience. We will be reaching out to all registrants for consent before moving your data to any new platforms.
Updates to the Registry platform will make it even more responsive, easier to navigate, and more convenient to access. The interface will be primarily an App (useable via iOS and Android) – but a traditional website will also be in place. We hope the App will make day-to-day life with Duchenne a little easier, with features including:
- App notifications about trial/study recruitment and reminders to update
- Appointment reminders, medication reminders and other calendar features
- Easy access to medical records
- Ability to integrate data from wearable devices
- Data visualization in real-time
We will accomplish these enhancements to the Registry by bringing together the leading talent and technology, working collaboratively with industry partners, and building from our 10 years of patient-powered data. PPMD and Sarepta are working with preeminent companies involved in data acquisition and data aggregation to ensure that the Interchange partners chosen understand the needs of the Duchenne community and can offer the most advanced technology possible. PPMD will also be working with partners on The Duchenne Registry side, to bring these enhancements to you and ensure that the Registry and Interchange work in sync.
If I join the Interchange, does all of my data become available to all members?
All researchers, including our initial industry partner Sarepta Therapeutics, would be required to request authorization to access data in the Interchange. And then, only de-identified aggregate data (combined, anonymous data) will be shared with a research partner – your personal information is never shared. Your data would be associated to you by a unique code. No one but you, and a PPMD administrator can access your personally identifiable data. This is similar to how The Duchenne Registry currently operates, but with the Interchange up and running, a more robust data set will become available.
Why partner with industry? Why partner with Sarepta?
To advance research and therapies for Duchenne, we must combine efforts and combine data. As PPMD and Sarepta share a common goal to develop treatments for Duchenne patients, and each brings a valuable asset to the Interchange—10 years of PRO data from PPMD combined with the data set from the first approved therapy in Duchenne—our partnership is a natural one. And both teams are equally dedicated to adding electronic health records and other data as it becomes available to create the most comprehensive database possible.
Sarepta is the first of what we hope will be many industry partners. The Interchange is being developed for all companies with approved therapies to contribute data and use as a resource. Both PPMD and Sarepta are firmly committed to building a comprehensive resource for all to use as therapies are approved.
Over the coming months, PPMD will keep you updated on the progress of the Duchenne Outcomes Research Interchange. We know that questions will arise, and we are here to answer them! Please call 888-520-8675 (option 2) or email firstname.lastname@example.org and one of the Registry genetic counselors will answer your questions.