Drug Evaluation Committee Implementation of Estimand in Clinical Trial Protocols
Data Science Subcommittee
March 2023
ICH E9(R1) "Addendum on Estimands and Sensitivity Analysis in Clinical Trials to The Guideline on Statistical Principles for Clinical Trials" was finalized as an ICH Harmonized Guideline. It has been finalized as an ICH Harmonized Guideline and is expected to be issued as Step 5 in Japan in the future. The "estimand" defined and introduced in ICH E9(R1) is presented as a framework that links study objectives with corresponding study design and analysis methods. Using this framework, it will be important to agree in advance with each stakeholder on the treatment effect to be estimated according to the clinical question of interest at the trial planning stage, and to conduct clinical trials with a document that precisely defines it.
In the first part of this report, we have explained the outline of the estimand for all those who are involved in clinical trials, and summarized the estimand settings and considerations in accordance with the thinking process in the Training Material. It also proposes a policy for the description of the estimand in the clinical trial protocol and presents specific contents of the estimand in the clinical trial protocol using a hypothetical example. In the latter part of the book, we introduce relevant literature for those who wish to deepen their understanding of estimand, and summarize each author's insights on estimand-related considerations. We hope that this publication will be of assistance in setting up an estimand and implementing it into a clinical trial protocol.
Published March 2023
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