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Topic Title

  • Developing Evidence-Based Research Priorities for Off-Label Drug Use

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Abstract - Final

Developing Evidence-Based Research Priorities for Off-Label Drug Use

Topic Abstract

Background. Drug use for indications not approved by the Food and Drug Administration exceeds 20% of outpatient prescriptions. Available compendia indicate that a minority of off-label uses are well supported by evidence. Policy makers, however, lack information to identify where systematic review of the evidence or other research would be most valuable.

Methods. Nationally representative prescribing data from the IMS Health (Plymouth Meeting PA) National Disease and Therapeutic Index were used to estimate the number of off-label drug uses by indication from 1/2005 through 6/2007. Off-label uses were categorized according to adequacy of scientific support based on the MICROMEDEX DRUGDEX compendium. Our model also incorporated FDA-identified safety concerns, mean prescription prices, marketing expenditures, and recent market entry.

Results. Top priority drugs were quetiapine, warfarin, escitalopram, risperidone, montelukast, bupropion, sertraline, venlafaxine, celecoxib, lisinopril, duloxetine, trazodone, olanzapine, and epoetin alfa.

Conclusion. Future research into off-label drug use should focus on drugs used frequently with inadequate supporting evidence, particularly if further concerns are raised by known safety issues, high drug cost, recent market entry, and extensive marketing. Our quantitative analysis identified particular concerns with the off-label use of antipsychotic and antidepressant medications.  Targeted research and policy activities on our list of prioritized drugs have high potential value.