FDA Launches Pilot Program to Develop Rare Disease Therapies

A limited number of sponsors will be allowed to participate.

The U.S. Food and Drug Administration (FDA) has launched the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Through this program, the agency intends to further accelerate the development of novel drug and biological products for rare disease. Participants will receive support from FDA staff regarding clinical study design, choice of control group and fine-tuning the choice of patient population.

“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 FDA’s Center for Biologics Evaluation and Research Director Peter Marks. “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.”

As the Lord Leads, Pray with Us…

  • For FDA Commissioner Robert Califf as he oversees the agency.
  • For FDA staff and scientists as they support the development of rare disease therapies.
  • For healing for Americans who live with rare diseases and conditions.

Sources: Food and Drug Administration


Back to top