The FDA Accelerating Rare Disease Cures (ARC) Program: Opportunity for Innovation

Rare disease settings present unique challenges for drug development and regulatory decision-making. The challenges include, but are not limited to, small patient populations, phenotypic heterogeneity within diseases, lack of reliable and well-defined endpoints, and limited information on the natural history. These challenges represent significant opportunities for innovation in endpoint development, study designs, and statistical analyses. To drive innovation in rare diseases, the Center for Drug Evaluation and Research of the Food and Drug Administration launched the Accelerating Rare Disease Cures (ARC) Program in May 2022. This talk will provide an overview of the ARC program and some of the methodologic opportunities it affords.