Skip to Main Content

Eight years ago, the US Food and Drug Administration coined the term “breakthrough designation” for faster reviews of drugs that may be substantial improvements over existing treatments. But a new analysis suggests that doctors regularly misinterpret the meaning and, as a result, the term may have become an effective marketing tool for the pharmaceutical industry.

More than half — 52 percent of nearly 700 physicians — incorrectly believed that strong evidence is required for a breakthrough designation when, in fact, the FDA requires preliminary evidence. And 64 percent wrongly answered that a drug anointed as a breakthrough represents a major advance, according to a research letter published in the Journal of the American Medical Association.

The researchers also showed physicians four different mock press releases about a fictitious lung cancer drug called Procampa. The doctors took a more taciturn view of the drug only after reading one release that made a point of including a caveat that the medicine’s effect on patient survival was unknown.


The other releases, however, included FDA approval facts with or without breakthrough designation language, but not the lack of available information about patient survival. And without that sort of language, physicians may be inclined to consider prescribing the medicine, one researcher told us.

“There’s already a tendency to believe that ‘new’ is better and breakthrough can amplify that impression,” said Dr. Steven Woloshin, a professor of medicine at Dartmouth College, who was one of the coauthors. “This may lead doctors and patients to think these drugs are better than they’re known to be and make decisions based on that language.”


To underscore the point, the researchers asked the physicians to imagine a patient with a serious medical condition for which there has been no effective treatment. The FDA recently approved two drugs, both of which were once-daily tablets with similar side effects and insurance coverage. But only one was designated as a breakthrough. Ninety-four percent preferred this drug as their first choice.

The perceptions surrounding breakthrough designation may reflect what the researchers called a “limited knowledge” that physicians hold of FDA approval language, in general.

For instance, 73 percent of the doctors incorrectly believed that regulatory approval meant that a drug displayed comparable effectiveness with other approved treatments. And 70 percent wrongly said that FDA approval required both a statistically significant and clinically important effect on patients.

The analysis involved providing questionnaires to 692 internists and specialists — endocrinologists, hematologists, and infectious disease physicians. Through April 2015, by the way, the FDA had given 76 drugs a breakthrough designation.

Of course, every study has its limitations. In this case, consider that there are likely to be instances where physicians are compelled to consider a drug that’s designated as a breakthrough simply because there are no alternative treatments. The researchers, however, did not have data on the number of breakthrough drugs for conditions for which no other treatment had been approved by the FDA.