FDA Creates Rare Disease Innovation Hub

Rare Daily Staff

The U.S. Food and Drug Administration launched a Rare Disease Innovation Hub to serves as a single point of engagement with the rare disease community, a recognition by the agency that new approaches are needed to expedite the development and approval therapies for these conditions.

The hub seeks to leverage expertise across the agency and enhance connections between centers with the FDA to spur the development of treatments for rare diseases. The Center for Drug Evaluation and Research Director Patrizia Cavazzoni and Director of the Center for Biologics Evaluation and Research Peter Marks will co-lead the hub. They will be responsible for developing a cross-center strategic agenda.

The agency said it is expected to fuel collaborations among existing programs, such as CDER Accelerating Rare disease Cures Program and CBER Rare Disease Program. It will also provide a forum to understand and leverage what the agency learns from pilot programs currently being implemented across the agency, including the Rare Disease Endpoint Advancement Pilot Program and the Support for clinical Trials Advancing Rare Disease Therapeutics Pilot Program.

The center is also expected to advance regulatory science by considering such things as novel endpoints, biomarker development, innovative trial design, and the use of real-world evidence.

“Many rare diseases lack treatment options and therefore many patients have high unmet medical needs. This requires new approaches to expedite development and approval of safe and effective drugs and biologics,” Cavazzoni and Marks wrote in a blog on the FDA website. “We see huge potential in establishing a new model, within the FDA, to leverage cross-agency expertise and greatly enhance our intercenter connectivity to spur the development of treatments for rare diseases.”

Read the full blog here.