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  1. Medical products for rare diseases and conditions

FDA Rare Disease Innovation Hub

Fundamental to the mission of the U.S. Food and Drug Administration is to engage patients and caregivers – to understand their unique perspectives and experiences and keep these front of mind as we review medical products for rare disease patients.

An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare diseases and conditions are life threatening, and most do not have approved treatments. 

FDA created the Rare Disease Innovation Hub (the Hub) to serve as a point of collaboration and connectivity between Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) with the goal of ultimately improving outcomes for patients.  Although the Hub will work across rare diseases, it will particularly focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. 

The Hub is co-led by the directors of Center for Biologics Evaluation and Research (CBER) and the FDA’s Center for Drug Evaluation and Research (CDER), in close collaboration with the FDA’s Center for Devices and Radiological Health, Oncology Center of Excellence, Office of Orphan Products Development, and Office of Combination Products. 


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