医疗器械真实世界研究设计和统计分析指导原则解析和探讨

Guideline Analysis and Discussion on the Design and Statistical Methods for Real-World Study of Medical Devices

近年来,医疗器械真实世界研究(RWS)备受关注,全球许多监管机构制定发布了相关指南文件,尚未包含RWS设计、统计分析和报告相关具体内容。2024年1月,国家药品监督管理局医疗器械技术审评中心发布《医疗器械真实世界研究设计和统计分析注册审查指导原则》,这是全球首个针对医疗器械RWS设计和统计分析的指南文件。该指导原则围绕RWS的核心特征,对RWS关键内容进行了详细分析和论述,包括研究设计基本要素、数据质量控制、偏倚控制、统计分析方法和研究报告要求等,为医疗器械RWS提供了全面的设计和分析框架,有助于提升研究质量,规范和合理引导RWS在医疗器械临床评价中的应用。需要注意的是,当前RWS仍面临高质量真实世界数据缺乏、研究设计较复杂、控制偏倚较难等主要挑战。未来,随着相关经验的积累和制度框架的完善,RWS在医疗器械临床评价中可能发挥更加重要的作用。

In recent years,real-world study(RWS)of medical devices have attracted significant attention.Many regulatory agencies around the world have formulated and issued relevant guidelines,but none have included specific content related to RWS design,statistical analysis,and reporting.In January 2024,the Center for Medical Device Evaluation of the National Medical Products Administration released the Guidance on Real-World Study Design and Statistical Analysis for Medical Device Registration Review,which is the first guideline in the world specifically targeting the design and statistical analysis of RWS for medical devices.This guidance provides a comprehensive framework for RWS design and analysis,focusing on its core characteristics,with detailed analysis and discussion on key aspects such as study design essentials,data quality control,bias control,statistical analysis methods,and study reporting requirements It helps to improve research quality and standardize and reasonably guide the application of RWS in the clinical evaluation of medical devices.It is worth noting that RWS still faces significant challenges,such as the lack of high-quality real-world data(RWD),the complexity of study design,and difficulties in controlling bias.In the future,with the accumulation of relevant experience and the refinement of regulatory framework,RWS is likely to play a more significant role in the clinical evaluation of medical devices.