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FDA Announces Pilot Program For the Development of Rare Disease Therapies


The program will offer participants more opportunity to communicate with FDA staff.

FDA is launching a pilot program aimed at improving the development of rare disease therapies.

In a press release, the agency announced that the program will provide sponsors with frequent communication opportunities with FDA staff. This should allow for more assistance with addressing clinical development issues.

The program is named the Support for clinical Trials Advancing Rare Diseases, otherwise known as START.

Director of FDA’s Center for Biologics Evaluation and Research Peter Marks, MD, PhD, said in a press release, “We hope the insight gained from this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies with rare disease indications and help sponsors generate high-quality, compelling data to support a future marketing application. These are complex products and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs."

The press release also details which who the program will initially be open to. Sponsors must fit at least one of the requirements:

  • Have a product currently in clinical trials under an IND.
  • Be regulated by CBER
  • Be regulated by CDER

Patrizia Cavazzoni, MD, director of FDA’s Center for Drug Evaluation and Research, also said in a press release, “"We look forward to increased engagement with sponsors developing these important products for the rare disease community. We share the goal of delivering potentially life-saving products to patients, and are committed to helping sponsors achieve regulatory milestones, while ensuring the safety, effectiveness and quality of these products."


(Sept. 19, 2023); FDA; FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies; https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies

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