P313: Policy Opportunities to Advance Rare Disease Innovation
Poster Presenter
Mia Williams
Policy Analyst
Duke-Margolis Institute For Health Policy (DMI) United States
Objectives
Through public, expert workshops, this work aimed to identify opportunities to support and advance rare disease therapy development.
Method
Beginning in May 2025 through March 2026, the Duke-Margolis Institute for Health Policy co-convened three public workshops with the FDA’s Rare Disease Innovation Hub to examine emerging issues in rare disease innovation. These workshops were informed by submissions to a Federal Register Notice.
Results
These efforts led to the identification of cross-cutting themes and priority policy recommendations, informed by expert insights, to outline proposed next steps for the rare disease community. Overarching themes that emerged include the need to aggregate data across similar diseases to address limitations in highly heterogeneous populations; greater use of real-world data from registries, natural history studies, and patient-generated data to support research in small populations; and the demand to clarify the Investigational New Drug (IND) process and provide clearer regulatory guidance for obtaining approval in these unique populations.
Conclusion
The identified recommendations highlight areas for action across developers, regulators, academic researchers, clinicians, and patient communities. This work illustrates one approach to achieving multi-stakeholder input and engagement to advance rare disease innovation through practical policy reforms.
