Sarepta influences FDA testimony through patients’ parents

Industry influence in patient advocacy groups became a hot topic when the US Food and Drug Administration approved eteplirsen, a drug to treat Duchenne Muscular Dystrophy, based heavily on parents’ accounts of their children’s clinical improvement. A report from the Wall Street Journal in May 2017 found that a consultant from Sarepta Therapeutics, the maker of epitlersen, had guided patients through the approval process and helped them create “slickly packaged testimony” for the FDA. For taking advantage of parents desperate to find a cure for their children, in order to get their drug approved without sufficient evidence, Sarepta deserves a spot in the Shkreli Awards.

SOURCE: Susan Pulliam and Brody Mullins, The Wall Street Journal

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