FDA’s CDER Launches Accelerating Rare Disease Cures Program

The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research announced the launch of the new Accelerating Rare disease Cures (ARC) Program, an effort to accelerate and increase development of treatments for the unmet needs of patients with rare diseases.

The ARC program extends across the Center for Drug Evaluation with leadership represented from several offices throughout the Center.

The FDA said there can be challenges with using well-established trial designs for therapies to treat rare diseases. Endpoint selection can be complex if there is a limited understanding of the natural history of the disease. And small patient populations can make it difficult to perform and interpret rare disease clinical trials. These challenges have added to the lack of therapeutic options for most rare diseases.

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 United States.

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.

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