Biogen, Sarepta Quick Drug Approvals Show FDA Gaps, Report Says-OxBig News Network

(Bloomberg) — Approvals for drugs from Biogen Inc. and Sarepta Therapeutics Inc. show flaws in the process US drug regulators use to speed approval for drugs to fill unmet needs, federal watchdogs said in a report. 

Biogen’s Aduhelm, Sarepta’s Exondys 51, along with Covis Pharma’s Makena were all cleared quickly by the US Food and Drug Administration despite weak evidence and disagreements among reviewers, according to the report Tuesday from Department of Health and Human Services’ Office of Inspector General. In one case, documentation about key decisions was missing, the report found. 

While Aduhelm, an Alzheimer’s treatment, and Makena, developed to ward off preterm births, were pulled from the market, Sarepta’s drug for Duchenne muscular dystrophy is still sold. The report echoes and extends findings from a 2023 Bloomberg News investigation that found companies made billions of dollars from medicines that received accelerated approvals without conclusive evidence of patient benefit. 

In the case of Aduhelm, the OIG report also noted serious issues with missing documentation, raising concerns around transparency. While FDA records showed 13 meetings between company and agency officials, a 2022 investigation by the House Oversight and Reform and Energy and Commerce committees revealed that there were at least 40. Some recorded meetings lacked detailed minutes or summaries, the report said.

The report focused on the three drugs after analyzing 24 accelerated approvals. It recommended the creation of a special advisory group involved in approvals and better documentation of meetings. 

“FDA must balance the goal of expediting the development and review of new drugs when there are no alternative treatments with the risk of approving drugs without confirmed clinical benefit,” it said.

Requiring a special advisory council for elevated approvals would be inefficient, according to an agency response in the report. The FDA agreed that documentation of meetings with drugmakers is important. 

The agency’s accelerated approval pathway has been used for more than three decades to give patients access to experimental drugs based on promising preliminary data. While it has been hailed for quickly addressing unmet medical needs with novel treatments, it has faced intense scrutiny for allowing drugs to linger on the market without conclusive evidence that they improve patient survival or quality of life. 

Bloomberg’s investigation also found that confirmatory trials were delayed for numerous drugs approved via the accelerated pathway, sometimes for years.

The FDA needs to “strengthen guardrails in certain circumstances to ensure appropriate and consistent use of the accelerated approval pathway,” the report concluded. 

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