[5/10/2022] FDA’s Center for Drug Evaluation and Research (CDER) announced the launch of the new Accelerating Rare disease Cures (ARC) Program. The vision of CDER’s ARC Program is speeding and increasing the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases. This is a CDER-wide effort with leadership represented from several offices throughout the Center.

FDA defines rare disease as any disease that affects less than 200,000 people in the U.S. 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.
• Endpoint selection can be complex if there is a limited understanding of the natural history of the disease.
• Small patient populations can make it difficult to perform and interpret rare disease clinical trials.

For these and other reasons, many of these diseases have few or no available treatments. Through scientific and regulatory innovation and engagement, CDER’s ARC Program will help support the development and approval of safe and effective treatment options for the more than 30 million people living with a rare disease in the U.S.

In its first year, CDER’s ARC Program will focus on strengthening internal and external partnerships with stakeholders and will engage with external experts to help identify solutions for the challenges in rare disease drug development.

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, caregivers, advocacy groups, academics, industry, and other partners — to address the significant unmet medical needs of patients and families living with rare diseases.

For more information:

• CDER’s ARC Program
• FDA Voices: CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development
• CDER Rare Diseases Team