Advancing Novel Surrogate Endpoints For Rare Disease Drug Development Workshop

Advancing treatments for rare diseases requires innovative ways to measure success. 

Join FDA, researchers, clinicians, and innovators for a free, one-day virtual workshop open to the public focused on emerging scientific and regulatory pathways for advancing surrogate endpoints in rare disease drug development.

This workshop is being convened in support of the Rare Disease Endpoint Advancement (RDEA) Pilot Program, established under the FDA User Fee Reauthorization Act of 2022 and the Food and Drug Omnibus Reform Act of 2022. The RDEA program is intended to support the development of efficacy endpoints for rare disease treatments by fostering dialogue among FDA, researchers, clinicians, industry, patient communities, and other stakeholders.

Topics to be Covered

1. Mechanistic and Translational Evidence Considerations for Supporting a Novel Surrogate Endpoint Biomarker (FDA Presentation)
2. Pathways for Engagement with FDA related to Biomarker Science (FDA Presentation)
3. Cases Studies of RLSEs in rare disease drug development (FDA Presentation)
4. Leveraging Disease Models from New Approach Methodologies and Biochemical Signatures as Endpoints (Invited Panelists)
5. Use of Digital Health Technologies as Biomarkers (Invited Panelists)
6. Real-World Whole-Person Data Management (Invited Panelists)

Please register for this event via the event website.