Rare Disease Framework Public Meeting

Rare Disease Framework
Hybrid Public Meeting

October 28, 2026 | 1pm - 5pm (Eastern)

About the meeting

On October 28, 2026, the U.S. Food and Drug Administration, in partnership with the Reagan-Udall Foundation for the FDA, will convene a hybrid public meeting to seek input related to the FDA’s Rare Disease Framework Initiative to reimagine the agency’s approach to rare disease drug development. The intent of this new initiative is to develop a fit-for-purpose Framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type, and the evidence that realistically can be generated in small patient populations.

Opportunity for public comment

The public meeting will be an opportunity for members of the rare disease community – including patients, disease advocates, drug developers, and others – to provide input on the direction and focus of the Framework. Insights from the public comment will inform the agency’s upcoming work.

Both in-person and virtual attendees may provide comments. To request a comment slot, use the registration buttons on this page and make your request when you register.

Topics for input

Feedback is welcome related to the Framework Initiative, and in particular, the following topic areas:

Fit-for-purpose review of rare disease therapies based on attainable evidence, particular disease state, and population.
FDA consistency and predictability of drug development and review regulatory requirements.
FDA engagements with external parties – including patients, disease experts, and researchers – to inform review decision-making.
Considerations for chemistry, manufacturing, and controls (CMC) specific to rare disease therapies.

The deadline to request to present public comment is 5pm ET Tuesday, October 20, 2026.

The Foundation will work to ensure a variety of representative voices can be heard.