Conflicts of interest come to light on controversial FDA approval

Back in September 2016, we reported on the controversial approval of a new drug to treat a rare form of muscular dystrophy. The drug, eteplirsen, was approved after a trial with a sample of 12 patients and no control group. Despite the meager evidence and recommendation of agency experts not to approve it, public pressure from patient advocacy groups swayed the Food and Drug Administration advisory committee.

Now, an in-depth report from the Wall Street Journal finds that ties between the Duchenne Muscular Dystrophy patient advocates and drugmaker Sarepta were closer than anyone thought. Sarepta partnered with parents of children with DMD, providing a consultant who guided them through the approval process and helped them create “slickly packaged testimony” for the FDA.

A majority of the FDA advisory committee that voted to approve eteplirsen did not know how the Sarepta consultant had helped the parents, according to the report. “More [committee members] might have voted no if they were aware” of these conflicts, said committee member Dr. Bruce Ovbiagele.

Industry-subsidized patient advocacy groups are common, providing an avenue for industry influence with the FDA. As the Trump administration moves to weaken the FDA approval process and replace federal funding for the agency with industry user fees, recognizing and publicizing conflicts of interest will become even more important.