Many Hold Incorrect Beliefs About FDA Approval Standards and Breakthrough Therapies

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US Food and Drug Administration
US Food and Drug Administration

A survey of internists and specialists revealed the majority of respondents held incorrect beliefs about both U.S. Food and Drug Administration (FDA) approval standards and breakthrough therapy designations.1

The FDA designates a treatment as a breakthrough therapy if preliminary clinical evidence suggests an improvement over available options. Improvements can include improved pharmacodynamic biomarkers, for example. The FDA has had this designation since 2012.

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Prescribing resources and press releases frequently use the term breakthrough therapy, which can result in excessively optimistic beliefs about a drug's effectiveness. This study, published in JAMA, examined whether physicians understand breakthrough therapies specifically and FDA approval standards generally.

Researchers used a survey to ask participants 3 questions about FDA approval and 5 questions about breakthrough therapies. Of 1,148 physicians contacted, 692 (60%) completed the survey. Respondents included physicians who answered at least 1 survey question.

Mean age of responders was 46 years (standard deviation = 10). The respondents were 45% women and 79% specialists. These characteristics were no different than the non-responder group.

Respondents demonstrated limited knowledge of FDA approval standards and breakthrough therapies. Of respondents, 73% (95% CI, 69%-76%) incorrectly thought FDA approval means comparable effectiveness to other approved drugs, 70% (95% CI, 66%-73%) incorrectly thought approval necessitated a significant and clinically important effect, and more than half (52%) incorrectly thought that randomized trials are necessary for breakthrough designation.

“A national survey of board-certified internists and specialists revealed substantial deficits in knowledge of the meaning of FDA approval. Physicians tended to overestimate the minimum evidence of efficacy required of new drugs. Similarly, many misinterpreted the term breakthrough—believing these drugs were supported by stronger evidence than required by the statute,” wrote the authors.

Specialists were more likely to answer correctly than internists (1.5 vs 1.2, P = .01).

“Our results are limited by social desirability and other inherent survey response biases and may not be generalizable beyond internists and medical specialists,” concluded the authors.

“The misconceptions identified may lead physicians to overprescribe newly approved drugs, particularly breakthrough therapies, and inadequately communicate how well these drugs work to the patients who will use them.”


1. Kesselheim AS, Woloshin S, Eddings W, et al. Physicians' Knowledge About FDA Approval Standards and Perceptions of the “Breakthrough Therapy” Designation. JAMA. [Published April 12, 2016]. doi:10.1001/jama.2015.16984.

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