FDA establishes rare disease innovation hub to accelerate treatment development
The US Food and Drug Administration (FDA) has announced plans to establish a new innovative hub aimed at improving treatment options for rare disease patients. The Rare Disease Innovation Hub will leverage cross-agency expertise to expedite the development and approval of safe and effective drugs and biologics.
More than 30 million people in the US are affected by a rare disease, many of which are life-threatening and without approved treatments.
Co-led by Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), and Patrizia Cavazzoni, director of the agency’s Center for Drug Evaluation and Research (CDER), the hub will have a particular focus on products intended for smaller populations or for diseases where the natural history is “variable and not fully understood”.
Its three main functions will include serving as a single point of connection and engagement with the rare disease community for matters that intersect CDER and CBER, including medical devices and combination products; and enhancing inter-centre collaboration to address scientific, clinical and policy issues relating to rare disease product development.
It will also aim to advance regulatory science with “dedicated workstreams for consideration of novel endpoints, biomarker development and assays, innovative trial design, real-world evidence and statistical methods”, according to Marks and Cavazzoni.
The directors said they will work “in close collaboration” with colleagues in the FDA’s Center for Devices and Radiological Health, Oncology Center of Excellence, Office of Orphan Products Development, as well as the Office of Combination Products.
“Many rare diseases lack treatment options and therefore many patients have high unmet medical needs… We see huge potential in establishing a new model within the FDA to leverage cross-agency expertise and greatly enhance our inter-centre connectivity to spur the development of treatments for rare diseases,” they wrote in the announcement.
The plans come just three months after the CDER announced the launch of a new hub focused on promoting innovation in clinical trials.
The Center for Clinical Trial Innovation is focused on facilitating the sharing of lessons learned across the CDER’s existing clinical trial innovation initiatives and will communicate and collaborate with external parties.
July 22, 2024
