Estimands in practice: Bridging the gap between study objectives and statistical analysis

摘要

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.