Management guideline for the off-label use of medicine in China (2021)

Off-label drug use embodies a thorough clinical diagnosis and evaluation of treatment needs and should not be confused with unreasonable drug use, but it also faces potential risks with drug safety and legal issues. We first established a guideline working group. Following the guideline development process recommended by the World Health Organization Handbook and the Chinese Medical Association, the key questions were determined through literature searches of PubMed, CNKI (Chinese National Knowledge Infrastructure) and other databases. Both the evidence and the clinicians’ diagnosis and treatment workload were considered to formulate the initial recommendations. Finally, two rounds of Delphi surveys and one expert seminar were organized to determine the final recommendations of this guideline. Meanwhile, we graded the recommendations based on the body of evidence. We determined nine questions and proposed a total of 23 recommendations regarding the definition of off-label use of drugs, applicable circumstances, classification of evidence, informed consent, legal basis, adverse drug reaction monitoring and evaluation, management procedure, responsibilities and obligations of different stakeholders, medical insurance reimbursement, and the national approval system. This guideline standardized clinical off-label drug use and provided suggestions and references for the management of off-label drug use.

药品超说明书用药（off-label drug use）体现了对治疗需求的全面临床诊断与评估，不应与不合理用药相混淆，但同时也面临药品安全与法律合规等潜在风险。本研究首先组建了指南制定工作组，遵循《世界卫生组织指南制定手册》（World Health Organization Handbook）及中华医学会推荐的指南制定流程，通过检索PubMed、中国知网（Chinese National Knowledge Infrastructure，CNKI）等数据库确定核心研究问题，综合考量循证证据与临床医师诊疗工作量，制定初步指南推荐意见。最终通过两轮德尔菲（Delphi）专家咨询及1次专家研讨会，确定本指南的最终推荐意见。同时，本研究基于证据体对推荐意见进行了分级。本研究明确了9个核心议题，共提出23条推荐意见，涉及药品超说明书用药的定义、适用情形、证据分级、知情同意、法律依据、药品不良反应监测与评价、管理流程、不同利益相关方的权责、医保报销及国家审批制度等内容。本指南实现了临床药品超说明书用药的规范化管理，为超说明书用药的管理工作提供了参考依据与实践建议。