FDA bolstering rare disease oversight

FDA continues to rebuff calls from patient advocates to create a center of excellence and a standing advisory committee dedicated to rare diseases. The agency’s Center for Drug Evaluation and Research, however, is putting more horsepower into the oversight of therapies for rare diseases.

CDER has established the Accelerating Rare disease Cures (ARC) Program, an initiative that will serve as the “connective tissue” linking the center’s staff, patient communities, drug developers and the medical community, CDER Director Patrizia Cavazzoni said at a press briefing. It will coordinate CDER’s implementation of commitments it has made for the upcoming PDUFA reauthorization, she said. This includes initiatives to advance the use of Bayesian statistics, and to conduct workshops and release guidance documents on topics that will facilitate development of therapies for rare diseases...