“Breakthrough” FDA Labeling of New Drugs Can Lead To Unwarranted Expectations

Dr. Tamar Krishnamurti PhD Department of Engineering & Public Policy Carnegie Mellon University Pittsburgh, PA 15213MedicalResearch.com Interview with:
Dr. Tamar Krishnamurti PhD
Department of Engineering & Public Policy
Carnegie Mellon University
Pittsburgh, PA 15213 

Medical Research: What is the background for this study? What are the main findings?

Dr. Krishnamurti: In 2012, the Food and Drug Administration Safety and Innovation Act became law. As part of this law, FDA can assign drugs the “breakthrough” designation. Breakthrough drugs are drugs that are intended to treat a serious or life threatening condition and have shown preliminary evidence of a substantial improvement over existing therapies on at least one one clinically significant endpoint. These clinical endpoints can be surrogate outcomes and don’t have to be a direct outcome of the disease. All FDA press releases announcing approval of breakthrough-designated drugs use the term “breakthrough” and about half use the term “promising” when describing the drugs. Our study randomly assigned participants to read 1 of 5 short descriptions of a recently approved drug. These vignettes differed by the term assigned to the drug (e.g. “breakthrough” or “promising”) or by whether the basis for the designation was clearly and succinctly explained in the description. We found that using the terms “breakthrough” and “promising” to describe these drugs resulted in people having unwarranted confidence about the effectiveness of breakthrough drugs, which could prevent them from making a fully informed decision about whether to take the drug or not. The influence of these terms on peoples’ judgments was mitigated by explaining the regulatory meaning of the drug’s approval (which is required in the drug’s professional label, but not in public discourse about the drug).

Medical Research: What should clinicians and patients take away from your report?

Dr. Krishnamurti: In communicating about breakthrough drugs, clinicians should carefully examine and clearly communicate the basis for approval for these drugs, being aware that the designation “breakthrough” should not be interpreted in the colloquial sense.

Medical Research: What recommendations do you have for future research as a result of this study?

Dr. Krishnamurti: Future research should focus on determining the most effective ways to communicate with the public about how various drugs are approved, including the strength of the clinical evidence on which these approvals are made, so that both physicians and patients can confidently make informed decisions about treatment.


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Dr. Tamar Krishnamurti PhD (2015). “Breakthrough” FDA Labeling of New Drugs Can Lead To Unwarranted Expectations

Last Updated on September 21, 2015 by Marie Benz MD FAAD