用药安全视角下窄治疗指数药物全周期管理政策及指导原则国际对比分析与启示

International Comparative Analysis and Implications of Narrow Therapeutic Index Drugs Whole-life Cycle Management Policies and Guidances from the Perspective of Medication Safety

目的 梳理中国对窄治疗指数药物的政策法规及指导原则现状,通过借鉴国际成熟经验,为中国实现窄治疗指数药物用药安全提供政策建议。方法 基于全周期管理视角,以用药安全为目标,全面对比分析典型国家(地区)对窄治疗指数药物的定义与识别、研发注册、临床使用、上市后监测4个环节的政策及相关指导原则内容。结果 中国在质量控制与生物等效性评价方面对窄治疗指数药物均出台了相关政策规定与技术指导原则,但在明确窄治疗指数药物品种、规范临床合理替换、药品上市后监测机制建设上仍存在政策“盲区”。而当前美国、欧盟等国家(地区)已初步形成全周期的监管思路,能够为中国优化相关政策提供借鉴。结论 建议通过推动中国窄治疗指数药物全周期管理的多方主体协同共治、监管部门持续更新特定药物生物等效性指导原则、关注窄治疗指数药物纳入集采时仿制药替换风险管理、规范窄治疗指数药物上市后安全性监管机制等途径构建全周期安全用药体系。

OBJECTIVE To review the current policies,regulations,and guidances governing Narrow Therapeutic Index Drugs(NTIDs)in China,and by drawing on international best practices,to provide policy recommendations aimed at ensuring the safe use of NTIDs in China.METHODS Adopting a whole-life cycle management perspective with a focus on medication safety,the study carried out a comprehensive analysis of NTIDs-related policies and guidances from typical countries(regions).The analysis covered four critical areas of NTIDs regulation:definition and identification,development and registration,clinical use,and post-market surveillance.RESULTS While China had introduced relevant policies and technical guidelines for NTIDs quality control and bioequivalence evaluation,significant gaps remained in the clear identification of NTIDs categories,the regulation of clinical substitution,and the establishment of robust post-market surveillance mechanisms.In contrast,countries(regions)such as the United States and the European Union had established preliminary whole-life cycle regulatory frameworks,offering valuable insights for optimizing China’s NTIDs policies.CONCLUSION It is recommended to build a comprehensive NTIDs whole-life cycle management system in China.Key strategies include promoting multi-stakeholder collaboration in lifecycle governance,continuously updating bioequivalence guidelines by regulatory authorities,managing the risks associated with generic substitution in volume-based procurement,and standardizing post-market safety monitoring mechanisms for NTIDs to enhance medication safety.