FDA Documents Reveal Depths of Internal Rancor Over Drug’s Approval Process

In some ways, it reads less like the product of a dusty bureaucracy and more like a Game of Thrones novel. Compelled by a lawsuit, the Food and Drug Administration is releasing thousands of never-before-seen documents regarding the most controversial drug approval in many years. And these documents are beginning to reveal the inner workings of a government agency that’s being torn apart by conflicting loyalties.

Late last year, the FDA approved eteplirsen, a potentially lifesaving drug for young boys (and in rare cases, girls) doomed to die from Duchenne muscular dystrophy, an incurable degenerative disease. That the approval was controversial is an understatement: In approving the drug, the agency overruled its own scientific advisers, rejected the recommendations of its review panel, triggered a formal internal dispute process, and apparently sparked the resignation of one senior official and the retirement of another.

The approval also failed to quash doubts about whether the drug, in fact, works — and it raised questions about how well the agency fulfilled its legal and scientific obligations to ensure that the drugs Americans use are both safe and effective. […]

Evidence of deep disagreement within the FDA over the decision to approve eteplirsen began to surface almost immediately after the FDA issued its go-ahead last September. Central to the tension was the decision by Janet Woodcock, the director of the agency’s Center for Drug Evaluation and Research, to issue the approval despite the contrary opinions of experts empaneled by the agency to review the drug. […]

Diana Zuckerman, the president of the nonprofit National Center for Health Research, expressed similar concerns. “I’ve seen high-level FDA officials undercut their scientists at meetings, or where they clearly try to take a side,” she said, “but I’ve never seen anything like this from a center director.”

Even more surprising than the alleged breaches of bureaucratic norms were Woodcock’s alleged breaches of scientific ones. The FDA is required by law to allow a drug to market only based upon scientific evidence that the drug is safe and effective; nothing other than science should be the basis of a decision. Jenkins’ memo backs up earlier allegations made by other FDA officials that Woodcock had decided to approve the drug not just before the review team had prepared its final report, but also well before a last round of crucial data were in.

“It’s not supposed to be the mission of the FDA to give treatments to desperate patients because the desperate patients want the treatments,” says Zuckerman, who worries that Woodcock is allowing patient desires to trump scientific evidence. “The role of the FDA is to ensure that the benefits outweigh the risks.” […]

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