林小莹,丹增赤列,王朵儿,朱映璇,鲁晔,高凡,李圆心,苏梦竹,张子龙,陈敏,李其泽,姜茹,赵延延,王杨

• 1:国家心血管病中心北京协和医学院中国医学科学院阜外医院医学统计部

摘要(Abstract)：

目的 探讨基于已完成的多项临床试验个体水平数据构建样本资源库，并利用其构建药物/器械临床试验外部对照的可行性及对应的实现方法。方法 选定经导管主动脉瓣置换术（TAVR）器械治疗主动脉瓣狭窄的上市前临床试验为例，将多项试验的个体水平数据库进行标准化处理后形成样本库。选定样本库中任意一项试验原始数据作为试验组，其余样本作为对照组，利用倾向性评分匹配及分层的方法处理潜在混杂因素，明确基于临床试验个体水平数据样本库构建外部对照的过程。结果 本研究共纳入4项TAVR器械的单组试验个体水平数据，共569例受试者（男59.2%）。试验1至试验4的受试者例数分别为120例、120例、163例、166例。基于倾向性评分匹配时分别可从其他试验个体水平数据中匹配具有可比性、相似特征的受试者例数分别为113例、117例、125例、147例，具有较高的匹配成功比例。倾向性评分分层后的倾向性评分分布图显示方案1中层1至层5试验组与对照组例数比例分别为4/103、11/103、22/92、32/87、51/64，所有构建的外部对照试验，倾向性评分各分层内均有一定数量与试验组基线特征相似的对照样本分布。模拟试验的结果在12个月全因死亡率上亦能体现出不同器械间的潜在差异。结论 以临床试验个体水平数据构建的样本库，作为一种高质量的数据来源，可成为同领域开展的单臂的外部对照来源，作为真实世界证据支持药物/器械研发外部对照场景的有益补充。同时也需要对相关领域的研究方法及偏倚控制措施开展针对性的研究。

关键词(KeyWords)： 临床试验;个体水平数据;样本库;外部对照

Abstract:

Keywords:

基金项目(Foundation): 国家重点研发计划资助项目(2024YFC2418905);; 真实世界数据支持药械监管决策的方法及应用研究项目(零余额2023-GSP-GG-1);; 国家科技创新2030“癌症、心脑血管、呼吸和代谢性疾病防治研究”重大专项项目(2023ZD0505606)

作者(Author): 林小莹,丹增赤列,王朵儿,朱映璇,鲁晔,高凡,李圆心,苏梦竹,张子龙,陈敏,李其泽,姜茹,赵延延,王杨

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