FDA Rare Disease Innovation Hub: Enhancing Collaboration and Speed?

In 2023, over half of all the novel drugs and biologics approved by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) were to prevent, diagnose or treat a rare disease or condition. The recently-formed FDA Rare Disease Innovation Hub is a new model of collaboration between CDER and CBER – and a key indicator of their commitment to explore new approaches for the development and approval of safe and effective drugs and biologics for rare diseases. 

This webinar discussion focuses on the desired priorities and potential outcomes of this initiative from the perspectives of the rare disease clinical research community – including those of a rare disease clinician, former FDA regulator and rare disease clinical research scientists. Key questions raised in this discussion include:

• What outcomes are most important for the research community? For patients and their families? What are the top priorities for improvement from the drug development ecosystem in support of rare disease?
• Which longstanding challenges are most critical for the FDA to address and why? What resources are needed to help solve these challenges?
• Are there  similar regulatory initiatives that are helping the FDA keep pace with drug development complexity and the recent breakthroughs in genetic science, personalized medicine and novel therapies? What learnings and advancements are currently being applied to the rare disease space or can be? 
• What emerging practices and development approaches are likely here to stay? What expanded role can the research community take in partnering with the FDA and how?

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