Physicians need to be better educated about FDA-approved indications for drugs and evidence for off-label drug use

Research Activities, April 2010, No. 356

Physicians frequently prescribe FDA-approved drugs for nonapproved indications. This "off-label" use may be a source of innovation, and in some settings represent the standard of care. However, off-label use of drugs often lacks supporting evidence and may expose patients to unwarranted risk. The researchers used a survey to assess physicians' knowledge of the evidence supporting a set of 14 commonly used drug-indication pairs that varied in the FDA-approval status and level of supporting evidence. Physicians knew the FDA-approval status for only 55 percent of drug-indication pairs, according to Donna T. Chen, M.D., M.P.H., of the University of Virginia, and colleagues.

Physician accuracy increased only modestly (to 60 percent) when the drug-indication pairs were confined to those that the physician reportedly prescribed during the previous year. Also, 41 percent of physicians erroneously believed some off-label uses to be FDA-approved, despite uncertain or no evidence of efficacy for the off-label use. The survey results indicate an urgent need for effective methods of disseminating information to physicians about the level of evidence supporting off-label drug uses, with specific attention to common off-label uses known to be ineffective or to carry unacceptable risk of harm, suggest the researchers.

They surveyed 600 psychiatrists and 599 primary care physicians with a 62-item questionnaire. Psychiatrists were included in the survey since psychiatric medications are among those most often used off-label by psychiatrists and generalists alike. Drugdex, a commercial indexing service, was used to categorize labeling status and the level of evidence regarding efficacy. This study was supported in part by the Agency for Healthcare Research and Quality (HS15699).

See "U.S. physician knowledge of the FDA-approved indications and evidence base for commonly prescribed drugs: Results of a national survey," by Dr. Chen, Matthew K. Wynia, M.D., M.P.H., Rachael M. Moloney, B.A., and G. Caleb Alexander, M.D., M.S., in Pharmacoepidemiology and Drug Safety 18, pp. 1094-1100, 2009.

Current as of April 2010
Internet Citation: Physicians need to be better educated about FDA-approved indications for drugs and evidence for off-label drug use: Research Activities, April 2010, No. 356. April 2010. Agency for Healthcare Research and Quality, Rockville, MD.