FDA’s CDER Introduces New ARC Program to Accelerate Rare Disease Cures

The mission of the ARC Program by the FDA’s Center for Drug Evaluation and Research (CDER) is to drive regulatory and scientific innovation and engagement to speed up the availability of treatments for patients with rare diseases.

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) announced the launch of the Accelerating Rare disease Cures (ARC) Program.

The ARC Program is managed by CDER’s Rare Diseases Team.

The vision of the ARC program is to accelerate and increase the development of safe and effective treatment options to address the unmet medical needs of patients with rare diseases.

The ARC program’s mission is to drive regulatory and scientific innovation and engagement to speed up the availability of treatments for patients with rare diseases.

In the US, a rare disease is defined by the Orphan Drug Act of 1983 as a disease or condition that affects less than 200,000 people in the country. Other nations may define rare disease in a slightly different manner. For instance, the European Union (EU) defines a rare disease as one that impacts no more than one person in 2,000 people.

Approximately one in 10 people in the US — which totals to around 30 million people — are living with a rare disease.

Rare disease clinical trials come with unique hurdles. These include small patient populations, the difficulty of using well-established trial designs and challenges in endpoint selection if the natural history of the disease isn’t well understood.

On the brighter side, there has been an upward trend since 2010 in CDER’s number of new drug approvals for orphan diseases.

“Despite this increase in approvals for novel drugs to treat rare diseases and conditions, there is still a tremendous unmet need for FDA-approved therapies. This is where CDER’s ARC Program comes in,” said Dr. Kerry Jo Lee, MD, associate director for rare diseases, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s YouTube video.

“I am optimistic about the future of rare disease drug development, and we are looking forward to continuing to work under the new CDER ARC Program together with you, patients, advocacy groups, academics, industry and all our other partners to address the significant unmet needs of patients and families living with rare diseases,” added Dr. Lee.

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