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Reminder: FDA to Hold Workshop on Advancing Novel Surrogate Endpoints for Rare Disease Drug Development

The U.S. Food and Drug Administration is convening a one-day virtual public workshop to be held on May 18, 2026, focused on the development of surrogate endpoints for drugs and biological products intended to treat rare diseases. The agenda and new meeting materials are now publicly available. The workshop will discuss mechanistic and translational evidence considerations for supporting a novel surrogate endpoint in rare disease marketing applications, including a case study highlighting the evidentiary package to support the use of a novel surrogate endpoint. FDA, clinicians, researchers and other parties will also discuss various relevant and or emerging translational science topics to support the development of novel surrogate endpoints such as new approach methodologies, digital health technology, and harnessing varied sources of patient-level real world data.

For more information about the program and meeting registration, please visit this FDA webpage. Visit CDER’s ARC website for an overview and information about the ARC Program.

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