Economic Evaluation of Off-Label Drug Use in Neurological Diseases: A Scoping Review Protocol of Global Evidence and Policy Implications
Résumé fourni par la source
Introduction Off-label use of drugs is common in clinical practice and may be clinically justified if supported by evidence or rationale; however, the market failure of the pharmaceutical industry and policy incentives for coverage and reimbursement vary widely with ambiguity. Understanding off-label drug policies will enhance the synthesis of economic evaluations, thereby aiding equitable and efficient decision-making. Objective The purpose of this scoping review is to map the published economic evaluations for the non-oncology chronic conditions to assess the policy implications for regulators, payers, and health-technology assessment bodies. Methods In concordance with the Preferred Reporting Items for Systematic Reviews and Meta-analysis Extension for Scoping Reviews (PRISMA-Scr). Pubmed, Scopus, and Web of Science databases will be searched from inception till May 16, 2026. All studies available in full-text and English will be considered. Economic evaluations including cost-minimizing, cost-effectiveness, cost-utility, and cost-benefit analyses of off-label use of pharmacological treatments for non-oncology chronic conditions (e.g., autoimmune, neurologic, cardiometabolic, psychiatric) will be included. Four reviewers will undergo the title and abstract screening then a full-text screening to extract data using a standardized sheet regarding demographics, disease area, evidence and rationale of the off-label use, perspective, modeling approach (decision tree, Markov, microsimulation), outcomes used (e.g., ICER, QUALY), uncertainty analysis tool (deterministic, probabilistic, Bayesian), value of information, and budget impact. Keywords Off-label drug use, cost-effectiveness analysis, cost-utility analysis, pharmacological treatments, chronic non-oncology disease, ICER, QALY.
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