RISE Together: Data Sharing across the Rare Disease Ecosystem
The RISE Workshop 3 convened on March 30, 2026, to discuss optimizing data sharing for therapy development and the collaboration, infrastructure, and education opportunities to promote data sharing as a consistent reality in the rare disease space.
The EveryLife Foundation was delighted to support Duke Margolis Institute for Health Policy and the FDA Rare Disease Innovation Hub during the planning of this workshop, as the meeting topic was based on submissions from the community, including our July submission to the Federal Register. Annie Kennedy, Chief Mission Officer, was invited to provide opening remarks to help set the tone for the day’s discussions.
The day kicked off with reflections across the patient, regulator, and industry communities about the impact of data sharing and the successes in research. It also focused on care coordination, which has led to successful approvals across disease states. Panels discussed the ethical considerations of informed consent and data privacy, including examples of data sharing in practice from partners at CPATH, the Cystic Fibrosis Foundation, and the Foundation for Angelman Syndrome Therapeutics (FAST).
These case studies highlighted the impact of robust data collection and sharing, the impact of patient-centered outcome measures, and the potential impacts on clinical trial design. Conversations also included stakeholder attitudes towards data sharing, the need to plan data collection with specific outcomes in mind, and infrastructure capabilities that work across research and clinical systems. The day ended with a conversation around scalable solutions that support education and communication across stakeholders to ensure functional and feasible data sharing.
Across all discussions of patient registries, data repositories, therapy approvals, and infrastructure development, panelists returned to the central idea of “Fit for Purpose” data collection. This “Fit for Purpose” data collection is the idea that we should collect data to:
- Maximize the function of the information to limit the burden on families,
- Inform better research,
- Allow other disease states to learn,
- Ensure robust outcomes that align with regulatory requirements.
This framework, if made scalable, was discussed as a way to make data sharing the norm and enhance systematic rare disease therapy innovation.
These hybrid RISE (Rare disease Innovation, Science, and Exploration) workshops are co-convened by the Duke-Margolis Institute for Health Policy and the U.S. Food and Drug Administration (FDA) Rare Disease Innovation Hub under a cooperative agreement with the FDA. RISE workshops bring together innovators in drug development, rare disease research, patient advocacy, and regulatory science to discuss challenges in the development of medical products for rare diseases that are common to multiple rare diseases or a class of diseases and for which evolving science offers innovative solutions.
Thank you to our partners at the Rare Disease Innovation Hub and at Duke-Margolis for convening this workshop and facilitating important conversations around this topic.
The meeting materials – including the agenda, speaker bios, slides, and the event recording – will be posted to the Duke-Margolis website.
