Rare Disease Framework

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 FDA’s Rare Disease Framework Initiative to reimagine the agency’s regulatory approach to rare disease development. The intent of this new initiative is to replace the FDA’s approach designed for larger diseases with a customized framework for rare disease drug development that considers the nuances of a specific disease, patient needs, product type, and attainable evidence.

Opportunity for public comment

The public meeting will be an opportunity for members of the rare disease community – including patients, advocates, drug developers, and others – to provide input on the direction and focus of the Framework by requesting to provide public comment. Insights from the public comment will inform the Agency’s upcoming work to develop a tailored approach to the regulatory review of rare disease therapies.

Topics for input

All feedback related to the Framework Initiative is welcome. FDA seeks input, in particular, on the following topic areas:

Customized review of rare disease therapies based on attainable evidence, particular disease state, and population
Consistency and predictability for rare disease drug sponsors
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

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.

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

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