The intent of this discussion is to highlight opportunities and limitations of utility-based and decision theoretic arguments in clinical trial design. The discussion is based on a specific case study, but the arguments and principles remain valid in general. The example concerns the design of a randomized clinical trial to compare a gel sealant versus standard care for resolving air leaks after pulmonary resection. The design follows a principled approach to optimal decision making, including a probability model for the unknown distributions of time to resolution of air leaks under the two treatment arms, and an explicit utility function that quantifies clinical preferences for alternative outcomes. As is typical for any real application, the final implementation includes some compromises from the initial principled setup. In particular, we use the formal decision problem only for the final decision, but use reasonable ad-hoc decision boundaries for making interim group sequential decisions that stop the trial early. Beyond the discussion of the particular study, we review more general considerations of using a decision theoretic approach for clinical trial design and summarize some of the reasons why such approaches are not commonly used.

中文翻译：

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作为决策问题的临床试验设计

本次讨论的目的是强调临床试验设计中基于效用和决策理论的论点的机会和局限性。讨论基于特定的案例研究，但论点和原则在一般情况下仍然有效。该示例涉及一项随机临床试验的设计，以比较凝胶密封剂与标准护理以解决肺切除术后漏气问题。该设计遵循最佳决策的原则方法，包括用于解决两个治疗臂下漏气的未知时间分布的概率模型，以及量化替代结果的临床偏好的明确效用函数。正如任何实际应用程序的典型情况一样，最终实现包括对初始原则设置的一些妥协。特别是，我们仅将正式决策问题用于最终决策，但使用合理的临时决策边界来做出提前停止试验的临时组顺序决策。除了对特定研究的讨论之外，我们还回顾了使用决策理论方法进行临床试验设计的更一般性考虑，并总结了此类方法不常用的一些原因。