A New FDA Initiative

The FDA announced work on a framework to rethink how rare disease drugs and biologics are developed and reviewed.

Why Change Is Being Considered

The agency says only about 5% of rare conditions have an FDA-approved treatment, despite existing support efforts.

One Size Won’t Fit All

The framework would consider disease characteristics, patient needs, product type, and evidence realistically gathered from small populations.

Earlier Patient Perspectives

The FDA intends to include patients’ perspectives early in development and at defined milestones during development and review.

More Expertise and Shared Resources

Plans include external expertise, shared resources, staff training, and institutional knowledge focused on rare disease drug development and review.

Risk-Based Review and New Science

The initiative prioritizes risk-based approaches and cutting-edge science, novel methods, and innovative statistical approaches; specific applications remain unspecified.

Ways to Share Feedback

An October 28 meeting will gather stakeholder feedback, followed by planned roundtables, Requests for Information, and internal FDA sessions.