US FDA launches pilot programme to help further accelerate development of rare disease therapies

FDA launches pilot programme to help further accelerate development of rare disease therapies

The US Food and Drug Administration (FDA) is taking steps to help further accelerate the development of novel drug and biological products for rare diseases. The agency is announcing the opportunity for a limited number of sponsors to participate in a pilot programme 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, M.D., Ph.D, 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.”