Decision-theoretic designs for small trials and pilot studies: A review.
Decision-theoretic designs for small trials and pilot studies: A review.
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DOI：
10.1177/0962280215588245
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发表时间：
2016-06
影响因子：
2.3
通讯作者：
Stallard N
中科院分区：
文献类型：
--
作者：
Hee SW;Hamborg T;Day S;Madan J;Miller F;Posch M;Zohar S;Stallard N
试点研究和其他小型临床试验经常进行，但服务于各种目的，并且对其设计几乎没有共识。设计这类研究的一个范例是贝叶斯决策理论。在这篇文章中，我们回顾了文献，目的是总结当前在这一领域的方法发展。我们发现决策理论方法已被应用于许多领域的小型临床试验设计。我们将对已发表方法的讨论分为单阶段试验、多阶段试验（通过若干中期分析在试验过程中做出决定）和试图设计一系列临床试验或药物开发计划的方法。在这三种情况下，已经提出了许多方法，这取决于正在考虑的决策者的观点和用于构建最佳设计的效用函数的细节。
Pilot studies and other small clinical trials are often conducted but serve a variety of purposes and there is little consensus on their design. One paradigm that has been suggested for the design of such studies is Bayesian decision theory. In this article, we review the literature with the aim of summarizing current methodological developments in this area. We find that decision-theoretic methods have been applied to the design of small clinical trials in a number of areas. We divide our discussion of published methods into those for trials conducted in a single stage, those for multi-stage trials in which decisions are made through the course of the trial at a number of interim analyses, and those that attempt to design a series of clinical trials or a drug development programme. In all three cases, a number of methods have been proposed, depending on the decision maker’s perspective being considered and the details of utility functions that are used to construct the optimal design.