Survey Shows Physicians Don’t Understand FDA’s Approval Process

Sanket Dhruva, MD, MHS

Many physicians are unfamiliar with how the Food and Drug Administration (FDA) regulates new drugs and medical devices, and they may be under the impression that the data supporting these approvals are more rigorous than they are, according to a national survey of physicians conducted by researchers at UC San Francisco (UCSF).

Physicians said they wanted to see more rigorous pre-market evidence, as well as regulatory action against drugs and devices that do not demonstrate safety and effectiveness after they have been approved for use.

The study appears January 8, 2024, in Health Affairs.

“Many of the physicians we queried felt that the FDA has become more lenient over time,” said first author Sanket Dhruva, MD, MHS, a UCSF cardiologist, assistant professor of medicine, and member of the Philip R. Lee Institute for Health Policy Studies. “Physicians need – and want – to know if a drug or device was rigorously studied before they can make recommendations to their patients.”

Physicians’ knowledge of the FDA’s approval process is important in informing clinical decisions and patient discussions. In recent years, several aspects of the FDA review process have changed, including the nature of the evidence required for approval. One of the most prominent modifications is the increasing use of expedited pathways to make potentially promising drugs and devices available to patients based on earlier-stage data.

Among the 509 physicians participating in the survey, 41% reported a moderate or better understanding of the FDA’s drug approval process, and 17% reported a moderate or better understanding of the FDA’s medical device approval process. Fifty-nine percent of physicians thought that the FDA’s current bar for approval of new drugs was about right, with 66% feeling similarly for new devices. Yet, more than a quarter of the respondents thought that the FDA was too flexible in the data accepted for new approvals for both drugs and devices.

Nearly all physicians thought that randomized, blinded trials that met primary endpoints should be required to secure regulatory approval, with majorities saying that the FDA should require two or more randomized controlled trials of most new drugs (57%) and for most new devices (60%). Nearly all physicians thought the FDA should revoke approval for drugs in the accelerated approval program (95%) or breakthrough devices (89%) that did not show benefit in post-approval studies.

“Physicians overwhelmingly felt that randomization, blinding, and meeting primary endpoint thresholds were important characteristics of trials used to support regulatory approval,” Dhruva said. “Despite these preferences, the proportion of studies leading to FDA approval that feature these characteristics has fallen in recent years.”

The authors note that the prevalence of physicians reporting a lack of understanding of drug and medical device approval reflects the limited coverage of these topics during medical education. They also suggest that the FDA could explain clearly and concisely how and why a drug was authorized, including its risk-benefit assessment, and what knowledge gaps remain.

Co-authors: Rita F. Redberg, MD and Robin Z. Ji, of UCSF; Aaron S. Kesselheim, MD, Zhigang Lu, and Jonathan J. Darrow, SJD, of Harvard University, and Steven Woloshin, MD, of Dartmouth University.

Funding: This study was funded by the Greenwall Foundation and Arnold Ventures.