The integrity of the FDA’s accelerated approval process should be called into question if the agency grants full approval to treatments that have failed their required confirmatory trials, the chief medical officer at the drug pricing watchdog the Institute for Clinical and Economic Review wrote in a new viewpoint.
David RindPublished in the Journal of the American Medical Association on Wednesday, ICER’s David Rind put the FDA’s accelerated approval of Sarepta’s Elevidys, a gene therapy for Duchenne muscular dystrophy, under the microscope, taking issue with the surrogate endpoint used in the approval, and the potential for it to be granted full approval next month. He also argues in the piece that the therapy’s high cost — $3.2 million — is hard to justify, contrasting it with other expensive treatments that have shown curative benefit.
“This is an enormous price tag for a therapy that has failed to meet its primary end point in the 2 randomized trials in which it has been studied and that is clearly not curative,” Rind wrote in the piece.
Still, Rind said in an interview with Endpoints News that the FDA shouldn’t consider price when making approval decisions, adding that it should be easier for payers to consider price when determining benefit.
Elevidys won accelerated approval last June to treat 4- and 5-year-old boys with DMD, following a rare move in which the FDA’s biologics center director Peter Marks overrode the application’s reviewers. The therapy then failed to meet the primary endpoint in its confirmatory trial this past October, though Sarepta pointed to secondary measures that suggested benefits.
In January, Marks hinted that he might expand the gene therapy’s label, and the agency has until next month to decide.
In the paper, Rind takes issue with the surrogate endpoint used in the accelerated approval decision, which hinged on levels of microdystrophin, a shortened version of the gene dystrophin that is affected by DMD. Sarepta’s thinking was that increased levels of microdystrophin could reasonably predict clinical benefit, but Rind asserts that there’s no evidence to support that idea.
Rind said that in his view, it could make sense to allow younger boys to access the therapy, granted that a clinical signal suggests benefit while awaiting confirmatory data in older boys. But, he added, the FDA doesn’t have a pathway to allow that.
“It’s not unreasonable if you’re FDA or anyone else to say, we have a signal, we have a clinical signal — not a surrogate signal — we have a clinical signal that suggests that maybe younger boys got a benefit here, and we’d like them to get this therapy sooner,” he said.
The FDA did not immediately return Endpoints’ request for comment.