Optimizing therapeutic decision-making for off-label medicines use: A scoping review and consensus recommendations for improving practice and research .

Autor: Gazarian M; School of Biomedical Sciences, Faculty of Medicine and Health, The University of New South Wales, Sydney, New South Wales, Australia., Horton DB; Department of Pediatrics, Rutgers Robert Wood Johnson Medical School, New Brunswick, New Jersey, USA.; Rutgers Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Health Care Policy and Aging Research, New Brunswick, New Jersey, USA.; Department of Biostatistics and Epidemiology, Rutgers School of Public Health, Piscataway, New Jersey, USA., Carleton B; Division of Translational Therapeutics, Department of Paediatrics, Faculty of Medicine, University of British Columbia, Vancouver, Canada.; Pharmaceutical Outcomes Programme, BC Children's Hospital, Vancouver, Canada.; BC Children's Hospital Research Institute, Vancouver, Canada., Kinlaw AC; Division of Pharmaceutical Outcomes and Policy, University of North Carolina School of Pharmacy, Chapel Hill, North Carolina, USA.; Cecil G. Sheps Center for Health Services Research, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA., Bushnell GA; Rutgers Center for Pharmacoepidemiology and Treatment Science, Institute for Health, Health Care Policy and Aging Research, New Brunswick, New Jersey, USA.; Department of Biostatistics and Epidemiology, Rutgers School of Public Health, Piscataway, New Jersey, USA., Czaja AS; Department of Pediatrics, Critical Care section, University of Colorado School of Medicine, Aurora, Colorado, USA., Durrieu G; Department of Medical and Clinical Pharmacology, Centre of PharmacoVigilance and Pharmacoepidemiology, Faculty of Medicine, Toulouse University Hospital (CHU), Toulouse, France., Gorman EF; Health Sciences and Human Services Library, University of Maryland, Baltimore, Maryland, USA., Titievsky L; Real World Evidence, Vertex Pharmaceuticals, Boston, Massachusetts, USA., Zito J; Department of Pharmaceutical Health Services Research, University of Maryland School of Pharmacy, Baltimore, Maryland, USA., Slaughter JL; Center for Perinatal Research and Division of Neonatology, Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, College of Medicine and Division of Epidemiology, College of Public Health, The Ohio State University, Columbus, Ohio, USA., dosReis S; Department of Practice, Sciences, and Health Outcomes Research, University of Maryland School of Pharmacy, Baltimore, Maryland, USA.
Jazyk: angličtina
Zdroj: Pharmacoepidemiology and drug safety [Pharmacoepidemiol Drug Saf] 2023 Nov; Vol. 32 (11), pp. 1200-1222. Date of Electronic Publication: 2023 Jun 21.
DOI: 10.1002/pds.5640
Abstrakt: Purpose: Off-label medicines use is a common and sometimes necessary practice in many populations, with important clinical, ethical and financial consequences, including potential unintended harm or lack of effectiveness. No internationally recognized guidelines exist to aid decision-makers in applying research evidence to inform off-label medicines use. We aimed to critically evaluate current evidence informing decision-making for off-label use and to develop consensus recommendations to improve future practice and research.
Methods: We conducted a scoping review to summarize the literature on available off-label use guidance, including types, extent and scientific rigor of evidence incorporated. Findings informed the development of consensus recommendations by an international multidisciplinary Expert Panel using a modified Delphi process. Our target audience includes clinicians, patients and caregivers, researchers, regulators, sponsors, health technology assessment bodies, payers and policy makers.
Results: We found 31 published guidance documents on therapeutic decision-making for off-label use. Of 20 guidances with general recommendations, only 35% detailed the types and quality of evidence needed and the processes for its evaluation to reach sound, ethical decisions about appropriate use. There was no globally recognized guidance. To optimize future therapeutic decision-making, we recommend: (1) seeking rigorous scientific evidence; (2) utilizing diverse expertise in evidence evaluation and synthesis; (3) using rigorous processes to formulate recommendations for appropriate use; (4) linking off-label use with timely conduct of clinically meaningful research (including real-world evidence) to address knowledge gaps quickly; and (5) fostering partnerships between clinical decision-makers, researchers, regulators, policy makers, and sponsors to facilitate cohesive implementation and evaluation of these recommendations.
Conclusions: We provide comprehensive consensus recommendations to optimize therapeutic decision-making for off-label medicines use and concurrently drive clinically relevant research. Successful implementation requires appropriate funding and infrastructure support to engage necessary stakeholders and foster relevant partnerships, representing significant challenges that policy makers must urgently address.
(© 2023 The Authors. Pharmacoepidemiology and Drug Safety published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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