New FDA Program to Focus on Driving Rare Disease Drug Development

The FDA’s Center for Drug Evaluation and Research (CDER) will launch a new Accelerating Rare disease Cures (ARC) program to help speed and increase development of treatment options to address unmet needs in these patients, according to an agency statement.

The National Institute of Health estimates that 7000 rare diseases affect between 25 million and 30 million Americans. Any disease that affects fewer than 200,000 individuals is considered rare.

Treatment for these conditions can be complex for a variety of reasons, including hurdles in carrying out well-established trial designs due to the low number of affected individuals, and challenges in selecting end points as researchers may not fully understand the natural history of the disease.

Many of these conditions thus have few or no available treatments, so the new ARC aims to address this shortage through “scientific and regulatory innovation and engagement.”

According to the statement, within the first year of the program, priorities will include strengthening partnerships with internal and external stakeholders and engaging with external experts to better identify solutions to challenges in drug development for rare diseases.

“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,” the statement reads.

The ACR program will be overseen by leaders from CDER’s Office of the Center Director, Office of New Drugs, and Office of Translational Sciences, with the program being managed by CDER’s Rare Diseases Team.

Upcoming events this month include a regulatory fitness in rare disease clinical trial workshop and a workshop on translational science in drug development focused on surrogate endpoints and biomarkers.

“Over the past decade or so, we have seen an increase in the percentage of novel drugs approved to treat rare or orphan diseases,” said Kerry Jo Lee, MD, associate director for rare diseases at the FDA Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in a promotional video for the program.

She continued to explain that in 2021 more than half of CDER’s novel drug approvals were for orphan diseases.

“However, 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,” Lee said, adding ARC will help address that need.