FDA seeks external input on rare disease trial criteria and endpoint selection

The FDA announced it will seek additional external feedback as it reviews how to evaluate efficacy and safety data for rare disease therapies.

The agency noted the difficulty of setting appropriate trial sizes, endpoints, and data thresholds given the small patient populations that characterize many rare conditions.

The initiative is intended to refine guidance to balance timely patient access with rigorous standards, and the FDA has opened a public comment period for stakeholders to submit their input.