FDA to establish Rare Disease Innovation Hub

Article summary

The FDA plans to establish a Rare Disease Innovation Hub to address the significant unmet needs for treatments for over 10,000 rare diseases affecting 30 million Americans. The Hub will leverage expertise across the agency and enhance collaboration to spur development of rare disease treatments. It will serve as a connection point with the rare disease community, enhance intercentre collaboration on scientific and policy issues, and advance regulatory science in areas like novel endpoints and trial designs. It will be co-led by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) directors and collaborate with other FDA centres to build on existing rare disease programs and facilitate engagement with external stakeholders through a new leadership position. While progress has been made, the Hub aims to improve outcomes for rare disease patients by addressing common challenges and facilitating safe and effective product development through enhanced cross-agency collaboration. The FDA anticipates holding...