ICHE9(R1)指南概述了估计和框架,这与规划保持一致,设计,行为,分析,和临床试验的解释。在临床试验中使用这个框架的好处和价值已经在文献中概述,并就如何选择估计和定义估计和属性提供了指导。尽管在临床试验中实施Estmands方面取得了进展,据我们所知,对于临床试验中的估计应满足的基本原则,目前还没有发表过与ICHE9(R1)指南中提出的观点一致的讨论.因此,在这篇观点文章中,我们提出了定义估计和的四个关键原则。这些原则构成了明确定义的治疗效果的基础,反映了评估和思维过程。我们希望这个观点将补充ICHE9(R1),并激发关于估计和临床试验中应该具有哪些基本特性的讨论,并且这样的讨论最终将导致在临床试验中定义估计的清晰度和精确度的提高。
The ICH E9(R1) guideline outlines the estimand framework, which aligns planning, design, conduct, analysis, and interpretation of a clinical trial. The benefits and value of using this framework in clinical trials have been outlined in the literature, and guidance has been provided on how to choose the estimand and define the estimand attributes. Although progress has been made in the implementation of
estimands in clinical trials, to the best of our knowledge, there is no published discussion on the basic principles that
estimands in clinical trials should fulfill to be well defined and consistent with the ideas presented in the ICH E9(R1) guideline. Therefore, in this Viewpoint article, we propose four key principles for defining an estimand. These principles form a basis for well-defined treatment effects that reflect the estimand thinking process. We hope that this Viewpoint will complement ICH E9(R1) and stimulate a discussion on which fundamental properties an estimand in a clinical trial should have and that such discussions will eventually lead to an improved clarity and precision for defining
estimands in clinical trials.