FDA’s ‘Conundrum’: What To Do With Approved Drugs That Fail Pivotal or Confirmatory Trials
FDA’s ‘Conundrum’: What To Do With Approved Drugs That Fail Pivotal or Confirmatory Trials
FDA’s ‘Conundrum’: What To Do With Approved Drugs That Fail Pivotal or Confirmatory Trials
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former reg...
Joe Ross, professor of medicine and public health at Yale School of Medicine and co-author of the study, told BioSpace he was “surprised to find that about 10% of all approvals are made despite the primary efficacy endpoint being null.” But, he added, “perhaps it isn’t surprising because the FDA has such flexibility in reviewing a marketing application.”
The most notable case—highlighted by the authors—is Biogen’s Aduhelm (aducanumab), greenlit under the FDA’s accelerated approval pathway in June 2021 as the first Alzheimer’s drug to treat the underlying cause of the disease. It was also among the most controversial approvals of the past decade, leading to the resignation of three prominent FDA reviewers.