US spent $3B on DMD treat­ments as con­fir­ma­to­ry stud­ies still un­der­way, JA­MA pa­per says

The US has so far doled out about $3 bil­lion on three of Sarep­ta Ther­a­peu­tics’ ther­a­pies for Duchenne mus­cu­lar dy­s­tro­phy, even as all three of the con­fir­ma­to­ry tri­als have yet to read out, Har­vard re­searchers found.

In the pa­per pub­lished Mon­day, the au­thors called on pol­i­cy­mak­ers to en­sure con­fir­ma­to­ry tri­als are com­plet­ed in a time­ly man­ner, and they sug­gest lim­it­ing Medicare and Med­ic­aid re­im­burse­ment for ac­cel­er­at­ed ap­proval drugs un­til clin­i­cal ben­e­fits are ver­i­fied.

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