As part of its Prescription Drug User Fee commitments, the FDA has commenced the Rare Disease Endpoint Advancement (RDEA) pilot to offer agency support to sponsors developing novel efficacy endpoints for rare disease trials.

Sponsors with an active investigational new drug (IND)/pre-IND as well as sponsors who don’t have an active development program but are doing a natural history trial studying the proposed endpoint, are invited to apply. Those selected for participation will be afforded greater engagement opportunities with experts from the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research to discuss their novel efficacy endpoints in depth.

RDEA proposals — which the FDA will begin accepting on a quarterly basis starting July 1, 2023, and ending June 30, 2027 — should be submitted as early as possible in the design of efficacy trials with active INDs/pre-INDs and natural history studies. The agency said it will accept just one proposal among those submitted by Sept. 30, 2023, and up to a maximum of three proposals per fiscal year between FY 2024 and 2027.

Preference will be given to proposals “that have the potential to impact drug development in rare disease more broadly, such as one that uses a novel approach to develop an efficacy endpoint or an endpoint that could potentially be relevant to other rare diseases,” the agency said. In addition, it will favor proposals that involve a range of different types of endpoints.

For surrogate endpoint proposals, the agency will give preference to those using novel approaches to gather additional premarket clinical data to advance endpoint validation.

The FDA said it may also accept a proposal for a development program for a common disease if it includes innovative or novel endpoint components that could apply to a rare disease.

Access the pilot program page here: https://bit.ly/3e9SoeC.