Approvals for medications of doubtful efficacy

Writing today on the Harvard Law Bill of Health blog, Associate Professor Rachel Sachs reports on the accelerated approval (by the US Food and Drug Administration (FDA)) of a medication for the treatment of Duchenne Muscular Dystrophy. She writes:

As regular FDA observers already know, Exondys’ path to approval has been highly contentious.  The key clinical trial used to support approval contained just twelve patients and no placebo, and the FDA Advisory Committee voted against both full approval of the drug and accelerated approval.  …  FDA staff scientists charged with reviewing the drug recommended against approval but were overruled by Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research.  FDA Commissioner Califf deferred to her decision.

Associate Professor Sachs expects that other companies developing rare disease drugs to submit clinical trial data for approval by the FDA that, until today, had seemed insufficient in terms of demonstrated efficacy.

Sarepta is reported to have announced that Exondys would cost on average $US300,000 per patient per year, in line with other rare disease drugs but lower than what some Wall Street analysts had estimated.

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