JD Supra

FDA Announces Rare Disease Evidence Principles to Accelerate Treatments for Ultra-Rare Genetic Diseases

The U.S. Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) to allow for faster approval of treatments for ultra-rare genetic diseases. A product is eligible if it ...
JD Supra

FDA’s Rare Disease Evidence Principles Process Provides Guidance for Applications Supported by a Single Pivotal Trial

On September 3, 2025, the US Food and Drug Administration (FDA) announced a new process to support the development of drugs intended to treat rare genetic diseases. The Rare Disease Evidence ...