FDA 'Breakthrough' Drug Designation Can Mislead Doctors
Many believe that medications labelled "breakthrough" are supported by stronger evidence than the law requires for them to achieve that designation.
HealthDay News — Use of the word "breakthrough" in the US Food and Drug Administration's expedited approval process could mislead doctors about the new drugs' actual benefits, according to a research letter published in the April 12 issue of the Journal of the American Medical Association.
The US Congress in 2012 gave FDA the power to designate a drug as a "breakthrough therapy" if preliminary clinical evidence suggests an advantage over existing medications. But a survey of 692 doctors revealed that many tended to misinterpret "breakthrough." Doctors often believed the drugs were supported by stronger evidence than the law requires to achieve that designation, lead author Aaron Kesselheim, MD, JD, MPH., a faculty member at Brigham and Women's Hospital in Boston, told HealthDay.
In the survey, 9 out of 10 doctors chose one hypothetical drug over another identical one solely based on the "breakthrough" designation, even though both were taken on the same schedule, had similar side effects, and were equally covered by the patient's insurance. In addition, 52% incorrectly believed that strong evidence from randomized trials is needed to earn the breakthrough designation. The survey also revealed a widespread lack of understanding about the meaning of FDA approval for a new drug, the researchers reported.
The FDA released the following statement in response to the study: "In general, the FDA does not comment on specific studies, but evaluates them as part of the body of evidence to further our understanding about a particular issue and assist in our mission to protect public health."
Kesselheim AS, Woloshin S, Eddings W, et al. Physicians' Knowledge About FDA Approval Standards and Perceptions of the "Breakthrough Therapy" Designation. JAMA. 2016;315(14):1516-1518. doi:10.1001/jama.2015.16984.