两阶段设计在医疗器械非随机临床试验中的应用

摘要

目的 介绍两阶段设计的概念、使用流程和注意事项,并以某外周血管支架临床试验为例介绍其在医疗器械非随机临床试验中的应用.方法 基于倾向性评分的两阶段设计能使医疗器械非随机临床试验满足随机化和前瞻性的原则.我们以某外周血管支架的非劣效试验为例,介绍两阶段设计的流程和细节.结果 两阶段设计解决了非随机临床试验由于非随机所带来的可能的研究偏倚及倾向性评分方法使用过程中的主观不确定性,实现了对随机临床试验的模拟,从而保障了非随机临床试验研究设计和统计分析的客观性和前瞻性.结论 两阶段设计能够增加临床试验的可行性,整合高质量的外部数据,缩短试验周期,提高试验效率,最终得到可靠的结论,具有较大的推广价值.%Objective This article introduces the concept,process as well as considerations of two-stage design.The application to non-randomized medical device clinical trials is further illustrated by example of aperipheral vascular stent study.Methods Two-stage design based on propensity score serves to make non-randomized clinical trials randomized and prospectively.The details of design process are presented through a non-inferiority trial evaluating the efficacy of a peripheral vascular stent.Results Two-stage design handles the issue of potential biases due to non-randomization and subjective uncertainty when applying propensity score to non-randomized clinical trials,aiming to mimic randomized clinical trials and ensure the objectivity and prospectiveness of both trial design and statistical analysis.Conclusion With great potential,two-stage design enhances the feasibility of clinical trial and makes it possible to get full use of existing data of high quality,shorten the duration of a trial,improve trial efficiency and draw a reliable conclusion ultimately.