Internists, Specialists Lack Knowledge About FDA Drug Approval Process

Tara Haelle

April 12, 2016

Most doctors do not fully understand the drug approval process for the US Food and Drug Administration (FDA) or the FDA's "breakthrough" drug classification, according to a survey published April 12 in JAMA.

A drug can receive breakthrough status when preliminary clinical evidence suggests the drug is superior to other existing therapies. The FDA has designated 76 drugs as "breakthrough therapies" since 2012, when the designation was created, through April 2015.

"Physicians tended to overestimate the minimum evidence of efficacy required of new drugs," write Aaron S. Kesselheim, MD, JD, MPH, from Brigham and Women's Hospital in Boston, Massachusetts, and his colleagues. "Similarly, many misinterpreted the term breakthrough—believing these drugs were supported by stronger evidence than required by the statute."

The researchers surveyed 692 internists and specialists in endocrinology, hematology, and infectious disease by email to learn what they knew about FDA procedures and practices. Among the 1148 physicians initially contacted, 60% responded, of whom 79% were specialists. The survey included five questions about breakthrough therapies, three about FDA approval, and two hypothetical scenarios.

In terms of overall understanding of FDA approval processes, 73% of the physicians incorrectly believed FDA approval meant the effectiveness of the drug was similar to the effectiveness of other approved drugs, and 70% incorrectly believed both statistically significant and clinically significant effects were required for FDA approval.

In addition, just over half (52%) of the respondents incorrectly believed that breakthrough status requires randomized trial data. However, 74% of doctors correctly answered that the breakthrough designation does not mean that high-quality evidence shows the drug to be safer than existing approved treatments.

For both question types, specialists were slightly more likely to answer correctly compared with internists, particularly with regard to the breakthrough therapy questions. Three of the five questions on breakthrough designation were factual; the other two were opinion questions. For the factual ones, just 17% of the respondents correctly answered all three. For the three FDA approval questions, only 1% of the respondents answered all three questions correctly, 26% answered two correctly, 63% answered one correctly, and 10% answered none correctly.

In the first hypothetical scenario, doctors had to choose between prescribing Axabex or Zykanta, both new, identical hypothetical drugs that met the criteria for breakthrough therapy, although only Axabex was labeled as such. Overall, 94% of the doctors chose to prescribe Axabex (the breakthrough drug) while just 6% preferred the identical, non–breakthrough-designated Zykanta.

In the second hypothetical scenario, the physicians were randomly assigned to read one of four mock FDA press releases for Procampa, a hypothetical new drug for lung cancer. One release included only the facts about FDA approval data, one additionally mentioned that the drug was designated as a breakthrough therapy, one included both breakthrough status and how quickly the drug was approved, and the last included breakthrough status as well as a warning about lack of data on the drug's effect on survival.

After reading the press releases, doctors indicated whether they agreed, strongly agreed, disagreed, or strongly disagreed with three statements: that Procampa improves patient survival, that strong evidence exists showing Procampa benefits patients, and that they would suggest trying Procampa to a patient with late-stage lung cancer.

Nearly two thirds of physicians (61%) reading the facts-only release believed there was strong evidence of benefit for the hypothetical Procampa, and 64% believed the drug improved survival. However, among those who read the press release with a warning regarding lack of data on survival, 48% believed strong evidence of benefit existed, and 50% believed the drug improved survival.

Across all four groups reading press releases, those who read the release with the warning were least likely to believe Procampa improves survival, that strong evidence of benefit exists, and that they would prescribe it to a patient. The second least likely group included those who read the release that mentioned that it was a breakthrough therapy. The physicians who read the release noting Procampa's breakthrough status and its expedited time to approval were most likely to agree with the three statements.

"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," the authors conclude. Study limitations include survey response biases, such as social desirability, and the inability to generalize outside of internists and specialists.

The research was funded by the Greenwall Foundation. Two authors, Dr Schwartz and Dr Woloshin, are cofounders of the company Informulary, which provides data regarding the benefits, harms, and uncertainties of prescription drugs.

JAMA. 2016;315:1516-1518. Abstract

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