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Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

 
• By Sarah Karlin-Smith

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads. 

FDA Commissioner Robert Califf speaks at a webinar sponsored by the Alliance for a Stronger FDA
FDA Commissioner Robert Califf discussed the FDA's new rare disease hub during a 27 September Alliance for a Stronger FDA event. • Source: Screenshot from Alliance for Stronger FDA webinar

The US Food and Drug Administration’s new Rare Disease Innovation Hub will have three main functions, including methods development work that should ultimately help better determine whether a drug meets the approval threshold, FDA Commissioner Robert Califf said.

Key Takeaways

  • A main function of the FDA’s new Rare Disease Innovation Hub will be developing methods that will help make risk-benefit determinations, Commissioner Robert Califf said.

  • The hub’s other primary objectives are ensuring consistent communication with outside groups, regardless of the center a rare disease application is assigned, and facilitating interagency communication on rare disease work

During a 27 September Alliance for a Stronger FDA event, Califf said he was “really excited about the joint effort to develop methods” between the FDA’s Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research

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