Biostatistics Department, ICON Clinical Research UK Ltd, Marlow, Buckinghamshire, UK.
ICON Drug Development Services, ICON Clinical Research GmbH, München, Germany.
Pharm Stat. 2021 Jan;20(1):68-76. doi: 10.1002/pst.2056. Epub 2020 Sep 7.
The International Council for Harmonisation (ICH) guideline E9 Statistical Principles for Clinical Trials (1) was issued in 1998. In October 2014, an addendum to ICH E9 was proposed on statistical principles relating to estimands and sensitivity analyses. The final version of the addendum to ICH E9 (R1) (2) was issued in November 2019. This virtual edition of Pharmaceutical Statistics takes a closer look at some of the progress that has been made since 2018 when implementing the estimand framework within clinical research. The articles discussed in this virtual issue are not new, but a compilation from previous issues. This specific article will act as a refresher for those not familiar with the topic and discuss the ABCs of estimands and their proposed deployment for improving the quality of clinical research. An overview of the more recent Pharmaceutical Statistics articles on estimands will be provided, signifying areas where progress have been made. The articles should be considered as contributions to the ongoing discussions rather than the final word. Finally, a personal perspective on the estimand success story and remaining challenges with proposed solutions will be discussed.
ICH 指导原则 E9《临床试验的统计学原则》（1）于 1998 年发布。2014 年 10 月，ICH E9 就与目标值和敏感性分析相关的统计学原则提出了增编。ICH E9（R1）增编的最终版本（2）于 2019 年 11 月发布。本期《药物统计学》虚拟版更详细地探讨了自 2018 年在临床研究中实施目标值框架以来所取得的一些进展。本期虚拟版中讨论的文章并非新内容，而是之前各期文章的汇编。这篇特定的文章将为不熟悉该主题的人提供一个复习，讨论目标值的基础知识及其为提高临床研究质量而提出的应用。将提供最近关于药物统计学中目标值的文章的概述，表明取得进展的领域。这些文章应被视为对正在进行的讨论的贡献，而不是最终定论。最后，将讨论对目标值成功案例的个人看法以及拟议解决方案所面临的挑战。
