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Principles for Defining Estimands in Clinical Trials-A Proposal.

Tobias Mütze1, James Bell2, Stefan Englert3

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|August 14, 2024
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Summary
This summary is machine-generated.

This viewpoint proposes four key principles for defining estimands in clinical trials, enhancing the ICH E9(R1) guideline. These principles aim to improve clarity and precision in trial design and analysis.

Keywords:
ICH E9 (R1) addendumcausalityclinical question of interestestimands

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Area of Science:

  • Clinical Trials
  • Biostatistics
  • Regulatory Science

Background:

  • The ICH E9(R1) guideline introduced the estimand framework to align clinical trial processes.
  • Existing literature details the benefits of estimands and guidance on their selection and attribute definition.
  • A gap exists in published discussions on fundamental principles for well-defined estimands consistent with ICH E9(R1).

Purpose of the Study:

  • To propose four key principles for defining estimands in clinical trials.
  • To provide a basis for well-defined treatment effects aligned with the estimand thinking process.
  • To complement the ICH E9(R1) guideline and stimulate discussion on estimand properties.

Main Methods:

  • This is a viewpoint article presenting proposed principles.
  • The principles are derived from the ICH E9(R1) guideline and existing literature on estimands.
  • The approach involves outlining fundamental properties for estimand definition.

Main Results:

  • Four key principles for defining estimands are proposed.
  • These principles aim to ensure estimands are well-defined and consistent with ICH E9(R1).
  • The proposed principles facilitate a clearer and more precise estimand definition process.

Conclusions:

  • The proposed principles offer a foundation for robust estimand definition in clinical trials.
  • Implementing these principles can enhance the clarity and precision of treatment effect estimation.
  • Further discussion on these fundamental properties is encouraged to improve clinical trial practices.