Drug Evaluation Committee Literature Review Related to Estimand and Sensitivity Analysis in Clinical Trials
June 2019
The Data Science Subcommittee of the Drug Evaluation Committee’s FY2018 Task Force 4 examined the following issues, which are closely related to estimands and sensitivity analyses—topics under consideration in ICH E9 (R1): “1. Efficacy of Treatment Strategies Based on ITT Principles and Other Treatment Effects,” “2. Issues Related to Data Handling and Missing Data,” “3. Definition and Role of the Analysis Population,” and “4. Stability and Sensitivity Analyses”—all of which are closely related to estimands and sensitivity analyses under consideration in ICH E9 (R1)—and compiled the findings into a report.
This literature review primarily involved a comprehensive survey of literature published or made publicly available from 1998 to 2018, with the total number of documents examined amounting to approximately 400.Therefore, we believe this report not only helps organize the various discussions that have taken place to date but also deepens understanding of estimands and sensitivity analysis. As such, we recommend that not only statisticians but also all personnel involved in drug development read this document.
We hope this report will be of assistance in more appropriately planning and conducting future clinical trials.
Literature Review on Estimands and Sensitivity Analysis in Clinical Trials_Ver1 (1.2MB)