15 years ago I wrote a piece, “The Seven Words You Shouldn’t Use in Medical News.” A revised version of that piece resides on this site. Two of those seven words were breakthrough and promising.
So when those same words were the focus of a research letter in JAMA Internal Medicine, it was worth a closer look.
“A randomized trial testing US Food and Drug Administration ‘breakthrough’ language,” was written by Tamar Krishnamurti, PhD and Baruch Fischhoff, PhD of Carnegie Mellon University, and Steven Woloshin, MD and Lisa Schwartz, MD of Dartmouth.
In colloquial terms, “breakthrough” connotes an important, definitive advance. Since the 2012 US Food and Drug Administration (FDA) Safety and Innovation Act became law, however, the FDA can assign the breakthrough designation to a drug that “treats a serious or life-threatening condition” and “may demonstrate a substantial improvement…over available therapies” based only on preliminary evidence (eg, uncontrolled studies, surrogate outcomes). Such drugs often receive “accelerated approval.”
All FDA press releases announcing approval of breakthrough-designated drugs use the term breakthrough; about half use promising. Patients can easily find these press releases searching the Internet or hearing about them in the news. Unless patients understand the FDA’s usage of breakthrough, they may have unwarranted confidence in the evidence supporting drug claims. In a randomized trial, we test how these terms affect lay judgments.
You can read the full results online (for now at least – it was published first online.)
Drs. Rita Redberg and Joseph Ross wrote an Editor’s Note in reaction to the study, “Would a breakthrough therapy by any other name be as promising?” Excerpts:
Not surprisingly, inclusion of the terms breakthrough or promising without any other change in vignettes led to higher ratings of the drug’s effectiveness and perceptions of the strength of supporting evidence. It is entirely reasonable to expect that patients, as well as health care professionals, will similarly perceive drugs as more promising based on this terminology. When patients learn about “breakthrough therapies” from FDA press releases, should we not expect them to request that their physicians prescribe these therapies instead of other available therapies, even if the evidence to support their use is less robust? Similarly, should we not expect physicians to preferentially adopt their use? …
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.
Reuters Health was one of the only news organizations to report on the study, under the headline, ” ‘Breakthrough’ drug may not mean what you think.” That story began:
What the Food and Drug Administration calls a ‘breakthrough’ drug is often not the same as what a layperson would call a breakthrough, a new study shows.
The FDA uses the term more often, and for smaller advances, than people use it colloquially, and this may lead patients to have unwarranted confidence in new drug claims.
Given how often “breakthroughs” and “promising” developments are trumpeted in news stories – even in this context of how the FDA uses the term – it’s probably not surprising that this study didn’t get much news attention. After all, news stories grab more eyeballs when they tout “breakthroughs,” so there’s no incentive to stop using those terms. Why bite the hand that feeds you?