Doctors to FDA: Don't Call Them 'Breakthrough' Drugs

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The U.S. Food and Drug Administration should avoid using words like "breakthrough" and "promising" to describe new drugs when making announcements aimed at the public, some researchers argue.

These researchers contend that the general public may not understand the FDA's usage of these words. In everyday language, the word "breakthrough" tends to denote an important advance; however, the FDA uses the word "breakthrough" as part of its drug review process, but it does not necessarily mean that a drug is effective or that it will help patients live longer, according to a new study by researchers at Dartmouth College's Geisel School of Medicine and Carnegie Mellon University.

"Unless patients understand the FDA's usage of 'breakthrough,' they may have unwarranted confidence in the evidence supporting drug claims," researchers wrote in the Sept. 21 issue of the journal JAMA Internal Medicine. [Wishful Thinking: 6 'Magic Bullet' Cures That Don't Exist]

The FDA officially designates certain drugs as "breakthrough therapies" if they meet specific criteria, including that the drug treats people with a "serious or life-threatening condition" and "may demonstrate substantial improvement over existing therapies." Drugs that are designated "breakthrough therapies" undergo a faster review process and are often approved more quickly, with the idea that more evidence on the drug's effectiveness will come later.

Previous studies have found that, when drugs with the "breakthrough" designation are approved, the FDA's news releases announcing the approvals have often used the word "breakthrough," and about half of these releases also used the word "promising."

In a new study, the researchers surveyed nearly 600 American adults, asking them to read short descriptions of a hypothetical drug to treat lung cancer. These descriptions were based on wording used in real FDA press releases. One description presented just the facts, such as stating that half of participants in a study of the drug had their tumors shrink, and that this effect lasted for about 7 months. A second description said the FDA called the medication a "breakthrough" drug, and a third description said the FDA called the drug "promising."

When presented with only the facts, 10 percent of participants in the survey said they thought the drug was "very effective" at treating lung cancer, but this increased to 22 to 25 percent when the words "promising" or "breakthrough" were added, the study found.

In addition, the percentage of participants who said the evidence supporting the drug was "strong" increased from 43 percent with just the facts, to 57 to 60 percent when the words "promising" or "breakthrough" were added.

"The terms 'breakthrough' and 'promising' increased people's beliefs in a drug's effectiveness and strength of supporting evidence," the researchers said.

The researchers noted that the FDA is not required to use the terms "breakthrough" or "promising" in its news releases.

"Press releases with neutral terms … might help consumers make more accurate judgments about these drugs," the researchers said.

In an editorial accompanying the study, Dr. Rita F. Redberg and Dr. Joseph S. Ross, both editors of JAMA Internal Medicine, agreed. "To protect patients from spurious hopes for miracle cures, Congress and the FDA should abandon the adoption of terminology like 'breakthrough' and focus on strengthening the evidentiary requirements for meaningful clinical data to ensure the promise of new drugs and devices," they wrote.

Two of the researchers on the new study have started a company that provides consumers with information about the benefits, harms and uncertainties of prescription drugs.

Live Science reached out to the FDA for comment, and the agency said that press releases "are written to be factual and non-promotional, with the intention of conveying as much information as possible to all of the audiences." The FDA added that "language in press releases on therapies receiving breakthrough designation or accelerated approval, match the descriptions for both programs" as required by law, and "are intended to inform of the designation only."

Editor's note: This article was updated to include comments from the FDA.

Follow Rachael Rettner @RachaelRettner. Follow Live Science @livescience, Facebook & Google+. Original article on Live Science.

Rachael Rettner

Rachael is a Live Science contributor, and was a former channel editor and senior writer for Live Science between 2010 and 2022. She has a master's degree in journalism from New York University's Science, Health and Environmental Reporting Program. She also holds a B.S. in molecular biology and an M.S. in biology from the University of California, San Diego. Her work has appeared in Scienceline, The Washington Post and Scientific American.