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 Index Drugs Whole-Life Cycle Management Policies and Guidances from the Perspective of Medication Safety

关键词:  窄治疗指数药物  生物等效性评价  用药替换  上市后监测

Key words:  narrow therapeutic index drugs  bioequivalence evaluation  drug substitution  post-market surveillance

摘要:

目的 梳理我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，为我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 我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，能够为我国优化相关政策提供借鉴。结论 建议通过推动我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经构建全周期安全用药体系。

Abstract:

OBJECTIVE This study aims to identify the deficiencies in China's regulation policies and guidances for Narrow Therapeutic Index Drugs (NTIDs) and provide practical recommendations for better ensuring the safe practices medication of NTIDs in China, with policies of developed countries acting as references. METHODS This study employs the perspective of pharmaceutical lifecycle management, with a primary focus on ensuring medication safety. It will conduct a comprehensive comparative analysis of policies and guidances in four crucial areas of NTIDs regulation: definition and identification, development and marketing, clinical use, post-market surveillance. Results Regulations and guidances for quality control and bioequivalence evaluation of NTIDs have been in place in China. However, there are still certain gaps in terms of defining NTIDs, regulating clinical substitution and establishing post-market surveillance mechanism. However, several countries (regions) such as the United States and the European Union have preliminarily established a comprehensive regulatory framework covering the whole-life cycle, which can provide valuable insights and serve as a reference for optimizing Chinese regulations. Conclusions This study suggests promoting the coordination of governance among stakeholders in lifecycle management of NTIDs, with regulatory authority continuously updating product-specific bioequivalence guidelines. In the context of conducting volume-base procurement of NTIDs, priority should be given to the surveillance of clinical substitution. Additionally, it is crucial to standardize post-market surveillance mechanisms for NTIDs.