Addressing innovative off-label medication use at an academic medical center.
OBJECTIVE
A large hospital's systematic and evidence-based approach to adjudicating, monitoring, and ensuring the safety of off-label medication use is described.


CONCLUSIONS
In 2003 the University of Pittsburgh Medical Center (UPMC)-Presbyterian implemented a policy that created a formal process for the systematic evaluation of formulary requests and drug-utilization patterns indicating or suggesting off-label use. Explicit criteria were developed for differentiating "innovative off-label use" (i.e., use based on a reasonable rationale yet lacking definitive scientific support in the form of fully published randomized controlled trials) from medication use more appropriately classified as clinical research. The UPMC-Presbyterian policy also outlined a process for the development, implementation, and evaluation of guidelines on innovative off-label use, including the collection of efficacy and safety outcomes. As of October 2012, 31 proposals for off-label medication use had been evaluated by the medical center's pharmacy and therapeutics committee and formulary subcommittee. Thirteen requests resulted in a determination of innovative off-label use and the development of prescribing guidelines, and 10 prompted the extension of an agent's current formulary status; in 6 cases, proposed off-label uses were determined to constitute clinical research. In some instances, innovative off-label medication use generated safety and outcomes data that led to changes in local standards of care. An algorithm to guide decision-making with regard to requests and proposals for off-label medication use is provided.


CONCLUSIONS
The UPMC-Presbyterian experience indicates that off-label medication use can be effectively managed using evidence-based principles and peer review mechanisms.
Susan J. Skledar, B.S.Pharm., M.P.H., is Associate Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Pharmacy Specialist, Health System Formulary Management and Drug Use Policy, University of Pittsburgh Medical Center (UPMC), Pittsburgh, PA; at the time of the project described herein, she was Director, Drug Use and Disease State Management Program, UPMC-Presbyterian, Pittsburgh. Shelby L. Corman, Pharm.D., M.S., BCPS, is Senior Clinical Outcomes Scientist, Pharmerit International, Bethesda, MD; at the time of the project, he was Assistant Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Specialist, Drug Information, UPMC. Thomas Smitherman, M.D., is Professor of Medicine, Division of Cardiology, University of Pittsburgh School of Medicine, and Cardiologist, UPMC-Presbyterian. skledarsj@upmc.edu.
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Skledar
Susan J. Skledar, B.S.Pharm., M.P.H., is Associate Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Pharmacy Specialist, Health System Formulary Management and Drug Use Policy, University of Pittsburgh Medical Center (UPMC), Pittsburgh, PA; at the time of the project described herein, she was Director, Drug Use and Disease State Management Program, UPMC-Presbyterian, Pittsburgh. Shelby L. Corman, Pharm.D., M.S., BCPS, is Senior Clinical Outcomes Scientist, Pharmerit International, Bethesda, MD; at the time of the project, he was Assistant Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Specialist, Drug Information, UPMC. Thomas Smitherman, M.D., is Professor of Medicine, Division of Cardiology, University of Pittsburgh School of Medicine, and Cardiologist, UPMC-Presbyterian.
Shelby L
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Corman
Susan J. Skledar, B.S.Pharm., M.P.H., is Associate Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Pharmacy Specialist, Health System Formulary Management and Drug Use Policy, University of Pittsburgh Medical Center (UPMC), Pittsburgh, PA; at the time of the project described herein, she was Director, Drug Use and Disease State Management Program, UPMC-Presbyterian, Pittsburgh. Shelby L. Corman, Pharm.D., M.S., BCPS, is Senior Clinical Outcomes Scientist, Pharmerit International, Bethesda, MD; at the time of the project, he was Assistant Professor, Department of Pharmacy & Therapeutics, University of Pittsburgh School of Pharmacy, and Clinical Specialist, Drug Information, UPMC. Thomas Smitherman, M.D., is Professor of Medicine, Division of Cardiology, University of Pittsburgh School of Medicine, and Cardiologist, UPMC-Presbyterian.
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D000046:Academic Medical Centers; D000465:Algorithms; D003657:Decision Making; D019317:Evidence-Based Medicine; D006801:Humans; D056687:Off-Label Use / Q000592:standards*; D016134:Organizational Policy; D010380:Peer Review; D010607:Pharmacy Service, Hospital / Q000458:organization & administration*; D010606:Pharmacy and Therapeutics Committee; D017410:Practice Guidelines as Topic; D010818:Practice Patterns, Physicians' / Q000592:standards