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Firms increasingly trying to sway FDA with families' stories

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An FDA advisory committee voted 7-6 last year to oppose approval for eteplirsen (Exondys—Sarepta Therapeutics), a position later overruled. Sarepta leveraged the emotional appeal of parents' stories about their children who had Duchenne muscular dystrophy to obtain that approval.

An FDA advisory committee voted 7-6 last year to oppose approval for eteplirsen (Exondys—Sarepta Therapeutics), a position later overruled. Sarepta leveraged the emotional appeal of parents' stories about their children who had Duchenne muscular dystrophy to obtain that approval. The Wall Street Journal found that a majority did not know how Sarepta's consultant had guided the patients' families. "More might have voted no if they were aware," said Bruce Ovbiagele, MD, a committee member, when he learned the role the company consultant played in helping parents prepare for FDA hearings. Relationships between companies and advocacy groups are important to know about, said G. Caleb Alexander, MD, codirector of the Johns Hopkins Center for Drug Safety and Effectiveness, who served as chairman of FDA's advisory committee. "The advisory committee received little information about how that played out in the development of eteplirsen." Companies have turned to such efforts as hidden involvement with grass-roots organizations to lobby decision makers and help sway public opinion. Many of these emerging tactics fall outside the government's longstanding definition of lobbying and remain hidden to both officials and the public, making it hard to know how companies are interacting with the people charged with running the country. Eteplirsen's drug application coincided with a shift at FDA to give a larger role to patient families and advocacy groups.

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https://www.wsj.com/articles/how-the-fda-approved-a-300-000-a-year-drug-its-own-experts-didnt-believe-worked-1495116544

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