Doctors misunderstand what FDA drug approval really means

Shouldn’t physicians and consumers hear about the negative studies?

Most doctors do not fully understand the drug approval process for the US Food and Drug Administration (FDA) or the FDA’s “breakthrough” drug classification.

” Physicians tended to overestimate the minimum evidence of efficacy required of new drugs.

Similarly, many misinterpreted the term breakthrough—believing these drugs were supported by stronger evidence than required by the statute. “

write Aaron S. Kesselheim, MD, JD, MPH, from Brigham and Women’s Hospital in Boston, Massachusetts


This survey study examines how well physicians understand the US Food and Drug Administration’s (FDA’s) statutory definition of a “breakthrough” therapy, and whether the term breakthrough affects their perceptions of a drug’s efficacy.

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, April 12, 2016.

Before US patients can use new prescription drugs, the US Food and Drug Administration (FDA) reviews the clinical trial results to confirm that benefits outweigh harms for the indication. Approval may involve superiority to placebo, not to an active comparator or standard of care (although approval can be based on uncontrolled or historically controlled studies). Numerous pathways expedite drug development and approval for serious or life-threatening conditions. For example, since 2012, the FDA can designate a drug as a “breakthrough therapy” if preliminary clinical evidence—such as an improvement in a pharmacodynamic biomarker—suggests an advantage over existing options Through April 2015, the FDA designated 76 “breakthrough” drugs and the term is routinely used in press releases and prescribing resources.

Press releases
  • Inside the Sausage Factory of Drug Approval: Nuplazid (pimavanserin) coverage didn’t inspect closely enough, healthnewsreview, May 23, 2016.
  • Internists, Specialists Lack Knowledge About FDA Drug Approval Process, medscape, April 12, 2016.

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