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Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy

Posted in Publications on 12.20.2016

JAMA. 2016;316(22)2257-2258

In September 2016, the head of the FDA’s Center for Drug Evaluation and Research approved a new drug (etiplersen) for the treatment of Duchenne muscular dystrophy, despite an advisory committee who voted 7 to 3 finding no evidence that the drug had clinical efficacy. Drs. Kesselheim and Avorn recently published a Viewpoint in JAMA where they describe the problematic case of etiplersen. They highlight the troubling fact that the pivotal trials which supported the drug’s approval were based on an outcome that was not likely to translate into meaningful clinical benefit: a change in protein dystrophin levels based upon muscle biopsy specimens. The authors also point out that approving drugs on the basis of such surrogate outcomes may translate into worse outcomes for patients, by exposing them to the risk of adverse events and high drug costs.

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