Words That Inappropriately Enhance Perception of New Drug’s Effectiveness

The findings are especially relevant for drugs designated by the US Food and Drug Administration (FDA) as meeting certain criteria.

In everyday usage, the term “breakthrough” represents a highly significant or definitive advance. However, since the FDA Safety and Innovation Act became law in 2012, 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. Such drugs often receive “accelerated approval.” Since the Safety and Innovation Act, all FDA press releases (PRs) announcing approval of a breakthrough-designated drug have used the term “breakthrough” and about half use “promising.”

Researchers from Carnegie Mellon University (Pittsburgh, PA, USA) and The Dartmouth Institute for Health Policy and Clinical Practice (Hanover, NH, USA) studied how catchphrase terms affect public perception of a new drug. “Today, patients and their families can easily find FDA PRs on the internet, or they often hear about them in the news,” said Prof. Woloshin, Dartmouth Institute, “But the reality is that unless patients fully understand how the FDA is using the term ‘breakthrough,’ they may have unwarranted confidence in the evidence supporting drug claims. So, we thought it was important to test how these terms affect the judgment of people without medical training.”

Participants in the online study were randomly given one of various short descriptions of a recently approved drug. The descriptions were based on an FDA PR for a metastatic lung cancer breakthrough-designated drug, conditionally approved based on the surrogate outcome tumor shrinkage. The facts-only description described the drug as meeting the breakthrough-criteria, but did not use the term “breakthrough.” A second and a third description added the terms “breakthrough” and “promising” respectively. A fourth, classified as “tentative,” used FDA-required language for professional labeling. Another, classified as “definitive,” changed the phrasing from “may be contingent” to “is contingent.” Participants were then asked to judge the drug’s benefit, harm, and strength of evidence.

Adding either “breakthrough” or “promising” in the description very significantly increased the percentage of participants who rated the drug as “very” or “completely” effective compared with the facts-only description (23% and 25% vs. 11%); it also significantly increased the number of people who reported believing that evidence supporting the drug is “strong” or “extremely strong” (59% and 63% vs. 43%). The tentative and definitive descriptions significantly reduced the percentage who believed (incorrectly) that the drug was “proven to save lives” (16%-tentative and 10%-definitive vs. 31%-breakthrough).

When participants were asked which of two drugs—one described as “breakthrough,” the other as meeting the breakthrough criteria—they would take for a potentially deadly condition, 92% chose the “breakthrough” drug.

“Our findings clearly indicate that words like ‘breakthrough’ and ‘promising’ increase people’s belief in a drug’s effectiveness (sometimes incorrectly),” said Prof. Schwartz, Dartmouth Institute, “PRs with neutral terms and that clearly explain the limited evidence supporting what breakthrough-designation and accelerated-approval mean might help consumers make more accurate judgments.”

The study, by Krishnamurti T et al., was published online September 21, 2015, in the journal JAMA Internal Medicine.

Related Links:
Carnegie Mellon University
The Dartmouth Institute