Drugs

Workshop on Novel Surrogate Endpoints for Rare Disease Drugs

FDA is convening a 5/18 virtual public workshop aimed at advancing the use of novel surrogate endpoints in developing treatments for rare diseases.

The workshop is part of a broader agency effort to accelerate rare disease drug development under mandates tied to the FDA User Fee Reauthorization Act of 2022 and the Food and Drug Omnibus Reform Act of 2022. Both laws require FDA to support endpoint innovation, including through a series of public workshops.

Central to this initiative is the agency’s Rare Disease Endpoint Advancement Pilot Program, which provides sponsors of qualifying rare disease programs with enhanced opportunities for regulatory engagement. The program is designed to help developers establish efficacy endpoints — particularly in cases where traditional clinical outcomes may be difficult to measure due to small patient populations or disease complexity.

During the May workshop, FDA officials and external experts will discuss the evidentiary framework needed to support novel surrogate endpoints in marketing applications, according to an agency notice. Topics will include mechanistic and translational data requirements, as well as a case study illustrating how such endpoints can be validated.

Participants will also explore emerging tools and methodologies that could strengthen endpoint development, including digital health technologies, new approach methodologies, and the use of patient-level real-world data.

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