FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes

The U.S. Food and Drug Administration has announced the establishment of a Rare Disease Innovation Hub. The Hub will connect and engage the rare disease community; help the community navigate FDA projects/programs that impact people with rare diseases; enhance collaboration to address scientific, clinical, and policy issues related to rare disease product development; and advance regulatory science with dedicated workstreams.

“The ALS Network applauds the FDA for creation of its Rare Disease Innovation Hub. This represents acceleration in discovering better treatments and cures for ALS while improving outcomes for the entire rare disease community.” –  Sheri Strahl, MPH, MBA, President & CEO, ALS Network

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 conditions are life threatening, and most do not have approved treatments. 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.