Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation

Abstract

The ICH E9 (R1) Addendum on “Estimands and Sensitivity Analysis in Clinical Trials (Step 4)” was finalized in November 2019 and subsequently implemented by many regulatory agencies, including FDA (May 2021). This article is based on a session organized to cover experience implementing the estimand framework, including its use, impact on drug/biologic development, common challenges and ways to address them, as well as keys to productive interdisciplinary collaboration.

Keywords:

• Clinical trials
• Intercurrent event
• Missing data
• Sensitivity analysis
• Treatment policy

Acknowledgment

The authors would like to thank Gregory Levin, PhD for his valuable comments and suggestions.

Disclaimer

This article reflects the views of the authors and should not be construed to represent FDA’s views or policies.

Statement

Frank Bretz owns stocks of and is employed at Novartis Pharma AG. The rest of the authors report there are no competing interests to declare.

Funding

The authors reported there is no funding associated with the work featured in this article.