Nature Examines Potential Accelerated Approval Reforms
A just-published article in the journal Nature examines the problems with FDA’s accelerated approval program and potential reforms to reduce any gaming by the pharmaceutical industry. “Part of the problem is that drug companies aren’t upholding their end of the bargain with timely post-market studies confirming the benefits of the drug,” Johns Hopkins Bloomberg School of Public Health epidemiologist Caleb Alexander is quoted in the article as saying. It notes that some researchers question whether companies are given too much time to produce such data.
A 2021 analysis found that 13% of drugs granted accelerated approval between 1992 and 2016 hadn’t been converted to full approval within five years — and remained on the market for a median of 9.5 years without the data needed for conversion. The agency gave Biogen nine years to complete a confirmatory trial for its controversial Alzheimer’s therapy aducanumab, an amount of time that Alexander calls “frankly offensive.” Biogen has since reduced its trial completion projection to four years.
Legislative fixes have been proposed in the House, but it is still too early to tell how this ends up and how effective the changes will be, the article says. Although they would make it easier for the agency to withdraw approval, they would also lengthen the bureaucratic process of rescinding approvals, National Center for Health Research president Diana Zuckerman told Nature. She would have preferred to stick with an earlier proposal, which would have automatically revoked approvals once confirmatory trials were one year overdue.
Zuckerman also recommends that the FDA commissioner’s office create a separate independent advisory panel to review agency approvals that go against advisory committee recommendations, which was the case in the Biogen accelerated approval. “The vast majority of advisory committee votes recommend approval, so when they don’t recommend approval, there’s usually a really good reason,” she says.
Additionally, Alexander suggests the Centers for Medicare & Medicaid Services should consider a lower reimbursement rate for accelerated-approval treatments that have not yet gained full approval. Such a move could “light a fire underneath manufacturers” to complete their trials, he says. “Why should taxpayers be on the hook for paying the full price of a drug when we don’t know the full scope of its safety and effectiveness?” he asks.