Efficacy and Comparative Effectiveness of Off-Label Use of Atypical Antipsychotics

Executive Summary

Published in: Comparative Effectiveness Review No. 6, (Rockville, MD: Agency for Healthcare Research and Quality, Jan. 2007), p. 1-8

Posted on RAND.org on January 01, 2007

by Paul G. Shekelle, Margaret A. Maglione, Steven Bagley, Marika Booth, Walter Mojica, Jason Carter, Cony Rolon, Lara Hilton, Annie Jie Zhou, Susan Chen, et al.

BACKGROUND: Aripiprazole, olanzapine, quetiapine, risperidone, and ziprasidone are atypical antipsychotics approved by the U.S. Food and Drug Administration (FDA) for treatment of schizophrenia and bipolar disorder. These drugs have been studied for off-label use in the following conditions: dementia and severe geriatric agitation, depression, obsessive-compulsive disorder, posttraumatic stress disorder, and personality disorders. The atypicals have also been studied for the management of Tourette's syndrome and autism in children. The purpose of this report is to review the scientific evidence on the safety and effectiveness of such off-label uses. THE KEY QUESTIONS WERE: Key Question 1. What are the leading off-label uses of atypical antipsychotics in the literature? Key Question 2. What does the evidence show regarding the effectiveness of atypical antipsychotics for off-label indications, such as depression? How do atypical antipsychotic medications compare with other drugs for treating off-label indications? Key Question 3. What subset of the population would potentially benefit from off-label uses? Key Question 4. What are the potential adverse effects and/or complications involved with off-label prescribing of atypical antipsychotics? Key Question 5. What are the appropriate dose and time limit for off-label indications?

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