Outrage of the Month: FDA Increasingly Reluctant to Seek Input From External Experts Before Approving New Drugs

Health Letter, July 2022

By Michael Carome, M.D.

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Historically, the Food and Drug Administration (FDA) in a large proportion of cases has convened an advisory committee to obtain advice and recommendations from independent, external experts before deciding whether to approve new drugs that contain any active ingredient not previously marketed in the U.S. Such consultation with advisory committees — which are composed of physicians, epidemiologists, statisticians and consumer representatives, among other experts — fosters transparency in FDA decision-making and promotes public confidence in the agency’s review and approval process for new drugs.

Indeed, Congress many years ago passed a law requiring that the FDA either refer applications for new drugs containing any active ingredients not previously marketed to an advisory committee for review prior to approval or provide a written summary of the reasons why the drug was not referred to an advisory committee prior to approval.

However, a study published in the May 2022 issue of Health Affairs by researchers at Brigham and Women’s Hospital and Harvard University revealed a stunning drop over the past decade in the proportion of new drugs approved by the FDA each year that were referred to an advisory committee. Specifically, of all new drugs approved by the FDA in 2010 and 2011, the agency referred the majority — 55% and 59%, respectively — to an advisory committee prior to approval. In stark contrast, in 2020 and 2021, the FDA referred a small minority of the new drugs that it approved — only 8% and 6%, respectively — to an advisory committee before granting approval.

The Health Affairs study researchers also examined the specific voting questions that were posed by the FDA to the advisory committees that were convened prior to drug approval from 2010 to 2021. They found wide variation in the substance and wording of key voting questions posed to the committee that ultimately framed the committees’ advice to the agency. In some cases, the voting questions posed to the committee failed to directly address whether the drug under review should be approved.

Commenting on their findings, the researchers noted that the “decade-long decrease in referrals of approved drugs to advisory committees suggests that the FDA needs specific criteria for how it chooses to subject new drugs to external scrutiny.” They also concluded that inconsistency in the questions posed to the advisory committees raises concerns about the legitimacy of the FDA’s drug-approval decisions.

The FDA in recent years clearly has abused the discretion that was granted to it by Congress regarding whether the agency should seek recommendations from its advisory committees before deciding whether to approve new drugs. Congress must pass new legislation that would sharply limit such discretion and direct the FDA to convene its advisory committee far more often before new drugs are approved.