FDA’s Center for Drug Evaluation and Research (CDER) has announced the launch of the new Accelerating Rare disease Cures (ARC) Program. The goal of the CDER ARC Program is to speed and increase the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases.

Launched in May 2022, CDER’s Accelerating Rare disease Cures (ARC) Program harnesses CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. CDER’s ARC Program is governed by leadership from across CDER’s Office of the Center Director, Office of New Drugs, and the Office of Translational Sciences. The program is managed by CDER’s Rare Diseases Team.

Drug development for the approximately 7,000 rare diseases and conditions can be complex for many reasons. There can be challenges with using well-established trial designs, and endpoint selection can be complex if there is a limited understanding of the natural history of the disease. Small patient populations can also make it difficult to perform and interpret rare disease clinical trials. Despite an increase in approvals for novel drugs to treat rare diseases and conditions, there is still a tremendous unmet need for FDA-approved treatments. The new CDER ARC Program will help support the development and approval of safe and effective treatment options for patients through scientific and regulatory innovation and engagement. 

CDER is optimistic about the future of rare disease drug development and is looking forward to continuing this important work under the new CDER ARC Program — together with patients, advocacy groups, academics, industry, and other partners — to address the significant unmet needs of patients and families living with rare diseases.