Rare Disease Challenges At US FDA: CDER Director Says More Staff, Not Reorganization, Will Fix Issues
Rather than changing the agency's structure to create more focus on rare diseases, Patrizia Cavazzoni wants funding for people to do the necessary work.
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‘Umbrella’ program is intended to ensure all rare disease and related programs at CDER are working together, which seems similar to the rare disease center of excellence concept that stakeholders want to coordinate activities across the FDA.
Measures aimed at spurring biomedical innovation, ensuring fewer roadblocks to development of biosimilars and generics, and strengthening the FDA’s interactions with rare disease patients could find their way into must-pass legislation reauthorizing the human drug and biologic user fee programs by 30 September.
Meetings are now primarily hosted by external patient advocacy groups, with accelerated approval and surrogate endpoints making their way into discussions. Stakeholders want more transparency on how FDA uses PFDD reports, and one agency staffer suggests adding other types of expertise to the meetings.