Date: May 18, 2026 Time: 9:00 a.m. – 4:30 p.m. ET

To facilitate rare disease drug development, and as part of a performance goal and requirement related to the FDA User Fee Reauthorization Act of 2022 and the Food and Drug Omnibus Reform Act of 2022 (FDORA), respectively, the U.S. Food and Drug Administration (FDA) has established a pilot program for supporting the development of efficacy endpoints for rare disease treatments. The Rare Disease Endpoint Advancement (RDEA) Pilot Program offers additional engagement opportunities with FDA to sponsors of rare disease development programs that meet specific criteria. The PDUFA [Prescription Drug User Fee Act] Reauthorization Performance Goals and Procedures Fiscal Years 2023-2027, known as the PDUFA VII Commitment Letter, contains detailed requirements for participating in the program and outlines FDA’s commitment to conduct up to three public workshops by the end of fiscal year 2027 to discuss topics relevant to endpoint development for rare diseases. In addition, FDORA requires FDA to conduct up to three public workshops to discuss topics relevant to the development of endpoints for rare diseases by September 30, 2026. This public workshop is intended to meet a performance goal under PDUFA VII and a requirement under FDORA. Continuing Medical Education (CME) credit will be available.

FDA is convening a one-day virtual public workshop on May 18, 2026, focused on the development of surrogate endpoints for drugs and biological products intended to treat rare diseases. 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 stakeholders 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.

Learn more about the program and register to attend here.