On September 3, 2025, the FDA announced the RDEP, a new framework intended to accelerate the approval of treatments for ultra-rare genetic diseases.1 The RDEP was jointly developed by the Center for Drug Evaluation and...more
On September 3, 2025, the Food and Drug Administration (FDA) announced Rare Disease Evidence Principles (RDEP) with processes aimed at providing greater predictability and facilitating the development and review of drugs...more
Clinical trial sponsors and principal investigators can consider themselves on notice that the Food & Drug Administration (FDA) is poised to ramp up enforcement activity relating to responsible parties’ obligations regarding...more