ICER push­es back on FDA's po­ten­tial con­ver­sion of Sarep­ta's gene ther­a­py to full ap­proval

The in­tegri­ty of the FDA’s ac­cel­er­at­ed ap­proval process should be called in­to ques­tion if the agency grants full ap­proval to treat­ments that have failed their re­quired con­fir­ma­to­ry tri­als, the chief med­ical of­fi­cer at the drug pric­ing watch­dog ICER wrote in a new view­point.

Pub­lished in the Jour­nal of the Amer­i­can Med­ical As­so­ci­a­tion on Wednes­day, ICER’s David Rind put the FDA’s ac­cel­er­at­ed ap­proval of Sarep­ta’s Ele­v­idys, a gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy, un­der the mi­cro­scope, tak­ing is­sue with the sur­ro­gate end­point used in the ap­proval, and the po­ten­tial for it to be grant­ed full ap­proval next month. He al­so ar­gues in the piece that the ther­a­py’s high cost — $3.2 mil­lion — is hard to jus­ti­fy, con­trast­ing it with oth­er ex­pen­sive treat­ments that have shown cu­ra­tive ben­e­fit.

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