A review of statistical designs for improving the efficiency of phase II studies in oncology
A review of statistical designs for improving the efficiency of phase II studies in oncology
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DOI：
10.1177/0962280215588247
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发表时间：
2016-06-01
影响因子：
2.3
通讯作者：
Jaki, Thomas
中科院分区：
文献类型：
--
作者：
Wason, James M. S.;Jaki, Thomas
进行II期肿瘤学试验是为了评估实验性抗癌治疗是否显示出足够的有效性迹象，以证明在III期试验中进行测试是合理的。传统上，此类试验是作为单组研究进行的，使用二元缓解率作为主要终点。在这篇文章中，我们回顾和对比这些研究的替代方法。每种方法只使用传统分析所需的数据。我们考虑了两大类方法：一种是旨在提高效率，使用新的设计思想，如多阶段和多臂多阶段设计;和那些旨在改善分析，更好地利用丰富的数据，在传统的分析中被忽略。前一类方法大大提高了效率，但也增加了审判中的行政和后勤问题。第二类包括标准分析的可行替代方案，这些替代方案几乎没有额外要求，并在效率方面提供了相当大的收益。
Phase II oncology trials are carried out to assess whether an experimental anti-cancer treatment shows sufficient signs of effectiveness to justify being tested in a phase III trial. Traditionally such trials are conducted as single-arm studies using a binary response rate as the primary endpoint. In this article, we review and contrast alternative approaches for such studies. Each approach uses only data that are necessary for the traditional analysis. We consider two broad classes of methods: ones that aim to improve the efficiency using novel design ideas, such as multi-stage and multi-arm multi-stage designs; and ones that aim to improve the analysis, by making better use of the richness of the data that is ignored in the traditional analysis. The former class of methods provides considerable gains in efficiency but also increases the administrative and logistical issues in running the trial. The second class consists of viable alternatives to the standard analysis that come with little additional requirements and provide considerable gains in efficiency.