New Study Finds Public May Misunderstand FDA “Breakthrough” Designation for New Drugs

In a newly published article in JAMA Internal Medicine, researchers say the U.S. Food and Drug Administration (FDA) should be cautious in using words like “breakthrough” and “promising” when making announcements about new drugs because the public may not understand the agency’s usage.

“Unless patients understand the FDA’s usage of ‘breakthrough,’ they may have unwarranted confidence in the evidence supporting drug claims,” the researchers wrote. The public may understand a “breakthrough” to be an important advance, but to the FDA, the designation does not necessarily mean that a drug is highly effective or that it will help patients live longer, according to researchers at Dartmouth College’s Geisel School of Medicine and Carnegie Mellon University, Live Science reports.

To the FDA, a “breakthrough therapy” is a drug that meets specific criteria, including that it treats a “serious or life-threatening condition” and “may demonstrate substantial improvement over existing therapies.” Breakthrough therapies undergo a faster FDA review process and are often approved more quickly, with additional evidence on the drug’s effectiveness to come later. When a breakthrough therapy is approved, FDA news releases often use the word “breakthrough,” and about half of the news releases also use the word “promising.”

For the JAMA Internal Medicine study, the researchers asked about 600 American adults, to read and give judgments of short descriptions of a hypothetical drug to treat lung cancer, Live Science reports. The descriptions were based on actual FDA press releases. The first description presented just the facts, stating, for example, that half of participants in a trial of the drug had their tumors shrink, and that this effect lasted about seven months. The second description called the medication a “breakthrough” drug, and the third described the drug as “promising.”

About 10 percent of the study participants who read only the facts thought the drug was “very effective” at treating lung cancer. Their opinion of the drug’s effectiveness increased to 22 to 25 percent when the words “promising” or “breakthrough” were used in the description, according to Live Science. Forty-three percent of participants who read just the facts judged the evidence supporting the drug as “strong,” but the percentage increased to 57 to 60 percent when “promising” or “breakthrough” were in the description.

The words “breakthrough” and “promising” increased belief in a drug’s effectiveness and in the strength of the supporting evidence, the researchers say. The FDA is not required to use those words in news releases, and the researchers suggest that neutral terms in press releases “might help consumers make more accurate judgments about these drugs,” according to Live Science.

Dr. Rita F. Redberg and Dr. Joseph S. Ross, editors of JAMA Internal Medicine, wrote in an editorial accompanying the study that these terms might give patients “spurious hope” for a miracle cure. They recommend that Congress and the FDA abandon “terminology like ‘breakthrough’ and focus on strengthening the evidentiary requirements for meaningful clinical data to ensure the promise of new drugs and devices.”



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