在肿瘤试验中使用真实世界数据作为外部控制手段的可行性。

IF 6.5 2区医学 Q1 PHARMACOLOGY & PHARMACY

Drug Discovery Today Pub Date : 2025-03-01 DOI:10.1016/j.drudis.2025.104324

Lishi Lin , Merel J.J. Lucassen , Vincent van der Noort , Toine C.G. Egberts , Jos H. Beijnen , Alwin D.R. Huitema

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引用次数: 0

摘要

在真实世界数据（RWD）衍生的外部控制臂（ECAs）可以应用于肿瘤领域的临床试验之前，必须确定这些ECAs是否可以作为合适和可靠的控制臂。本综述概述了构建rwd来源ECAs的研究，并与随机临床试验（RCT）组进行了比较。在纳入的8项研究中，有6项rwd衍生的eca与RCT组的生存率相似。因此，当进行随机对照试验不可行时，RWD衍生的eca可以作为单臂临床试验的对照组，前提是使用合适的RWD来源并做出合适的方法决定。

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The feasibility of using real world data as external control arms in oncology trials

Before real world data (RWD)-derived external control arms (ECAs) can be applied in clinical trials within the field of oncology, it must be determined whether these ECAs can function as appropriate and reliable control arms. This review provides an overview of studies in which RWD-derived ECAs were constructed and compared to a randomized clinical trial (RCT) arm. RWD-derived ECAs had similar survival outcomes as the RCT arm of comparison in six of the eight included studies. Therefore, when performing RCTs is not feasible, RWD-derived ECAs can act as control arms in single-arm clinical trials, provided that suitable RWD sources are used and suitable methodological decisions are made.

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来源期刊

Drug Discovery Today 医学-药学

CiteScore

14.80

自引率

2.70%

发文量

293

审稿时长

6 months

期刊介绍： Drug Discovery Today delivers informed and highly current reviews for the discovery community. The magazine addresses not only the rapid scientific developments in drug discovery associated technologies but also the management, commercial and regulatory issues that increasingly play a part in how R&D is planned, structured and executed. Features include comment by international experts, news and analysis of important developments, reviews of key scientific and strategic issues, overviews of recent progress in specific therapeutic areas and conference reports.