“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).

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Reliability of East German Pharmaceutical Investigations Questioned

MedicalResearch.com Interview with: Dr. med. Rainer Erices Institut für Geschichte und Ethik der MedizinMedicalResearch.com Interview with:
Dr. med. Rainer Erices
Institut für Geschichte und Ethik der Medizin

Medical Research: What are the main findings of the study?

Dr. Erices: The study presents the results of a first systematic investigation of clinical drug trials carried out by Western pharmaceutical firms in Eastern Germany in the 1980s. The scientific investigation of the East German Health system has only just started. The study shows that in that period of time, around 220 clinical trials were carried out. We now know how many patients took part and what remuneration the GDR received. It continues to be difficult to evaluate these tests. Despite intensive research efforts in different archives, we have been unable to find documentation on how detailed (and systematically) patients were informed about the trial they were taking part in. The responsible institutions repeatedly advised testers to stick to the law during the clinical trials. The law required that these trials should only be carried out on patients which had given their informed consent and had decided to participate voluntarily. However, so far there is no convincing proof that these legal requests were met.

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