Hee Siew Wan, Hamborg Thomas, Day Simon, Madan Jason, Miller Frank, Posch Martin, Zohar Sarah, Stallard Nigel
Division of Health Sciences, Warwick Medical School, The University of Warwick, Coventry, UK
Division of Health Sciences, Warwick Medical School, The University of Warwick, Coventry, UK.
Stat Methods Med Res. 2016 Jun;25(3):1022-38. doi: 10.1177/0962280215588245. Epub 2015 Jun 5.
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.
试点研究和其他小型临床试验经常开展，但目的各异，且在其设计方面几乎没有共识。针对此类研究的设计提出的一种范式是贝叶斯决策理论。在本文中，我们回顾文献，旨在总结该领域当前的方法学进展。我们发现决策理论方法已应用于多个领域的小型临床试验设计。我们将已发表方法的讨论分为针对单阶段试验的方法、针对多阶段试验（在多个中期分析中在试验过程中做出决策）的方法，以及试图设计一系列临床试验或药物研发计划的方法。在所有这三种情况下，根据所考虑的决策者视角以及用于构建最优设计的效用函数细节，已经提出了多种方法。
