Marks: FDA open to using single-arm studies for accelerated approval of rare disease gene therapies
Peter Marks, the FDA official who oversees gene therapies, is again advocating for the use of accelerated approvals for gene therapies that treat rare diseases.
Marks, who is the director of the FDA’s Center for Biologics Evaluation and Research, said Monday during a webinar organized by the NYU Grossman School of Medicine that the FDA is “trying to be patient-focused.” Under Marks, the FDA has appeared more flexible with accelerated approvals and the kinds of trials and results needed to bring rare disease therapies to market.
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