Mehrotra Devan V, Hemmings Robert J, Russek-Cohen Estelle
Merck Research Laboratories, North Wales, PA, USA
Medicines and Healthcare Products Regulatory Agency, London, UK.
Clin Trials. 2016 Aug;13(4):456-8. doi: 10.1177/1740774516633115. Epub 2016 Feb 17.
In October 2014, the Steering Committee of the International Conference on Harmonization endorsed the formation of an expert working group to develop an addendum to the International Conference on Harmonization E9 guideline ("Statistical Principles for Clinical Trials"). The addendum will focus on two topics involving randomized confirmatory clinical trials: estimands and sensitivity analyses. Both topics are motivated, in part, by the need to improve the precision with which scientific questions of interest are formulated and addressed by clinical trialists and regulators, specifically in the context of post-randomization events such as use of rescue medication or missing data resulting from dropouts. Given the importance of these topics for the statistical and medical community, we articulate the reasons for the planned addendum. The resulting "ICH E9/R1" guideline will include a framework for improved trial planning, conduct, analysis, and interpretation; a draft is expected to be ready for public comment in the second half of 2016.
2014年10月，国际协调会议指导委员会批准成立一个专家工作组，以制定《国际协调会议E9指南》（“临床试验的统计原则”）的增编。该增编将聚焦于涉及随机确证性临床试验的两个主题：估计量和敏感性分析。这两个主题部分是受临床试验人员和监管机构提高对感兴趣的科学问题进行表述和处理的精准度需求的推动，特别是在随机分组后出现的事件（如使用抢救药物或因受试者退出导致数据缺失）的背景下。鉴于这些主题对统计学和医学界的重要性，我们阐述了制定该计划增编的理由。最终的“ICH E9/R1”指南将包括一个改进试验设计、实施、分析和解读的框架；预计草案将于2016年下半年准备好供公众评论。
