Drugs

Ways to Fix Accelerated Approval: Professors

David McFarland

— 1 min read
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Two professors in the University of Pennsylvania Perelman School of Medicine and Boston University School of Law say the FDA accelerated approval process is “worth saving, but fixes are needed to make sure companies quickly follow through on rigorous confirmatory studies” of the drugs receiving accelerated approval. Writing in a Washington Post opinion column, the professors say that after three decades of using accelerated approval, problems are evident, most recently with the approval of Biogen’s Aduhelm (aducanumab) for Alzheimer’s disease in 6/2021.

The article says FDA “often sets unreasonably distant deadlines for companies to report confirmatory trial results and more than one in 10 accelerated approvals predating 2016 still haven’t produced evidence to support transition either to traditional approval or withdrawal.”

The authors say part of the problem is that once companies have received an accelerated approval, and thus can sell their drugs and generate revenue, they have little reason to pursue fast, high-quality confirmatory trials. Fixes recommended in the article are: 

  •          adding an expiration date to accelerated approvals, perhaps two to five years depending on how rare the disease is and how quickly it progresses;
  •          stating that withdrawal of a drug will be automatic when standards for approval aren’t met;
  •          making confirmatory trial participation more attractive by making it more convenient, by offering incentives, or by making clearer to patients the extent of uncertainty around accelerated approval drugs;
  •          considering moving confirmatory trials to foreign locales where the drug is not yet approved to attract more eager participants;
  •          insurers determining to pay less or nothing for accelerated approval drugs until they are proved to work; and
  •          Medicare choosing to pay only for an accelerated approval drug in the context of a clinical study.

“Accelerated approval has been a popular pathway for drugs for cancer and for some rare conditions, and after Aduhelm we’re likely to see an onslaught of drugmakers seeking similar approval for other Alzheimer’s drugs,” the authors conclude. “Some of these may prove to be boons. But others will be duds. The system needs to change so that patients not only have treatment options, but options they can be confident work.”

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