FDA Reassesses Rare-Disease Trial Standards and Endpoint Selection on September 14, 2026 Clinical Benefit endpoint selection Endpoints Natural-history studies Pilot Rare Disease Endpoint Advancement rare-disease clinical trials Surrogate United States Food and Drug Administration +
FDA Announces New Rare Disease Approval Programme on September 04, 2025 Accelerated Approved Biological Factors conditional approval pathway drug review Endpoints Infrequent real-world data streamlined process Surrogate United States Food and Drug Administration +