RARE Daily

FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

October 2, 2023

Rare Daily Staff

The U.S. Food and Drug Administration announced a pilot program aimed at accelerating the development of rare disease therapies by allowing for more frequent communication with FDA staff to provide a mechanism for addressing clinical development issues.

“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,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “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.”

Selected participants of the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including clinical study design, choice of control group, and fine-tuning the choice of patient population.

The program will be open to sponsors of products currently in clinical trials under an active Investigational New Drug application, regulated by either the Center for Biologics Evaluation and Research or the Center for Drug Evaluation and Research.

Eligibility criteria for the pilot differs between CBER and CDER-regulated products.

In addition to having an active IND, eligible CBER-regulated products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life.

Under CDER’s eligibility criteria, the product must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic type. More information on the program’s eligibility requirements can be found in the Federal Register Notice.

The FDA will be accepting applications to the START program from January 2, 2024 to March 1, 2024. Pilot participants will be selected based on application readiness. The agency will select up to three participants for each center. Following an evaluation of this pilot and feedback from selected sponsors, the agency said it may consider a second iteration, which would be announced in the Federal Register at a later date.

Photo: Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research

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