Overestimation of the Effect Size in Group Sequential Trials
Overestimation of the Effect Size in Group Sequential Trials
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DOI：
10.1158/1078-0432.ccr-11-3118
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发表时间：
2012-09-15
影响因子：
11.5
通讯作者：
Sridhara, Rajeshwari
中科院分区：
文献类型：
--
作者：
Zhang, Jenny J.;Blumenthal, Gideon M.;Sridhara, Rajeshwari
分组序贯设计 (GSD) 可以对疗效数据进行中期监测，并允许提前终止试验，同时保留 I 型错误率，这在肿瘤学临床试验中已变得司空见惯。尽管在伦理上很有吸引力，但 GSD 在早期中期分析中往往会高估真实的治疗效果大小。高估治疗效果可能会夸大药物的益处，并为医生及其患者提供有关药物真实效果的不准确信息。在临床试验实践中，许多人通常不太了解这种现象的原因和影响。在这篇文章中，我们用图解的方式解释了为什么会出现 GSD 的高估现象。对治疗效果程度的潜在高估在肿瘤学领域尤其值得关注，其中更主观的无进展生存终点已越来越多地被采用作为关键 III 期试验的主要终点。临床癌症研究； 18(18)； 4872-6。 (c) 2012 年 AACR。
Group sequential designs (GSD), which provide for interim monitoring of efficacy data and allow potential early trial termination while preserving the type I error rate, have become commonplace in oncology clinical trials. Although ethically appealing, GSDs tend to overestimate the true treatment effect size at early interim analyses. Overestimation of the treatment effect may exaggerate the benefit of a drug and provide imprecise information for physicians and their patients about a drug's true effect. The cause and effect of such a phenomenon are generally not well understood by many in clinical trial practice. In this article, we provide a graphical explanation for why the phenomenon of overestimation in GSDs occurs. The potential overestimation of the magnitude of the treatment effect is of particular concern in oncology, in which the more subjective endpoint of progression-free survival has increasingly been adopted as the primary endpoint in pivotal phase III trials. Clin Cancer Res; 18(18); 4872-6. (c) 2012 AACR.