A rare disease is any disease that affects fewer than 200,000 people in the U.S. There are approximately 25 to 30 million Americans living with a rare disease (about 1 in 10 people), and many rare diseases have few or no available treatment options. FDA’s goal for this year’s Rare Disease Day (February 28) is to explore ways to engage and collaborate with patients, patient advocates, and other stakeholders to support the development of safe and effective therapies.

CDER is committed to accelerating the development of treatments for patients with rare diseases. In May 2022, CDER launched the Accelerating Rare disease Cures (ARC) Program. The ARC Program seeks to harness CDER’s collective expertise and activities to drive scientific and regulatory innovation for rare diseases. It also builds upon CDER’s existing capabilities to expand its interactions with the rare disease stakeholder community. The vision of the ARC Program is speeding and increasing the development of effective and safe treatment options to address the unmet needs of patients with rare diseases.

In its first year, the ARC Program has focused on strengthening partnerships with patients, advocacy groups, industry, and other stakeholders. Moving forward, the ARC Program has multiple efforts underway to support rare disease drug development. The program recently initiated Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) to better understand the challenges in bringing rare disease drug products to market. As part of LEADER 3D, CDER is seeking input to help identify knowledge gaps and produce educational materials on fundamental topics important to our stakeholders, such as:

  • Nonclinical and clinical pharmacology considerations
  • Clinical trial design and interpretation
  • Regulatory considerations for rare disease drug development1

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References

  1. ResearchCDER Continues to Advance Rare Disease Drug Development with New Efforts, Including the Accelerating Rare Disease Cures (ARC) Program. U.S. Food And Drug Administration. https://www.fda.gov/drugs/news-events-human-drugs/cder-continues-advance-rare-disease-drug-development-new-efforts-including-accelerating-rare-disease

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