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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