Dive Brief:

• The Food and Drug Administration needs stricter standards for the accelerated approval process to ensure patients and the healthcare system don’t waste money on ineffective treatments, according to a report issued Tuesday from a federal watchdog
• Spurred by controversy over the 2021 approval of Biogen’s Alzheimer’s disease drug Aduhelm, the office of the inspector general for the Health and Human Services department examined 24 FDA reviews that led to accelerated approvals of new medicines. The office identified concerns with three, including Aduhelm.
• In those cases, the FDA failed to follow its own best practices in data analysis, held meetings with sponsors that weren’t properly documented and suffered from a lack of oversight that could have been triggered by conflicts within the agency, the report said. “These three cases underscore the need for additional guardrails,” the report said.

Dive Insight:

The inspector general’s office has two main recommendations to improve the accelerated approvals process. The FDA agreed with the watchdog that meetings with sponsors need to be better documented and said it’s already taken steps to improve its procedures. But the agency disagreed with the watchdog’s second recommendation, to outline certain factors that would trigger greater oversight.

Specifically, the inspector general’s office suggested the agency’s accelerated approvals council — itself a new creation — should be required to meet when FDA reviewers don’t reach consensus on an application, an advisory committee raises significant concerns or when a drugmaker relies on analyses not included in the original plan to win approval of a medicine. Other factors could be set up to trigger a meeting as well, it said.

The accelerated approval pathway exists to allow drugs to reach the market faster based on promising signs in research, such as a reduction in a biomarker linked to disease. Drugmakers are then required to keep testing the medicines to show a clinical benefit. The pathway is often used for diseases that don’t have adequate treatments and have patient advocates pushing the FDA to approve new medicines.

In the case of Aduhelm, messy study results led to unorthodox practices at the FDA that have already been well documented. Biogen ended up pulling the drug off the market in 2024. The inspector general’s office also found problems with the reviews of Makena, a drug to reduce the risk of preterm birth, and Exondys 51 to treat Duchenne muscular dystrophy.

The HHS inspector general’s office detailed some of the behind-the-scenes action on the applications. In the case of Exondys, the director of the FDA’s drug division overruled the review team, which had decided the research didn’t support approval. A dispute between high-ranking officials followed, and the FDA commissioner ultimately decided to approve the medicine.

The inspector general’s office chose 10 of the drugs for review after they were flagged by stakeholders as potentially problematic. It chose the other 14 at random. The office noted that both Aduhelm and Makena are no longer for sale. Exondys, sold by Sarepta Therapeutics, remains on the market, but its required trial to demonstrate clinical benefit has been delayed.