US FDA Plans Rare Disease Center Of Excellence, But Not "That" Kind Of COE
A COE modeled after the Oncology Center of Excellence, which stakeholders had proposed, is not in the works, but rather a more virtual structure.
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Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.
‘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.
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.