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Choosing Estimands in Clinical Trials: Putting the ICH E9(R1) Into Practice
Bohdana Ratitch1, James Bell2, Craig Mallinckrodt3
1Eli Lilly, Montreal, Quebec, Canada.
Therapeutic Innovation & Regulatory Science
|February 20, 2020
Summary
This paper clarifies estimands in clinical trials, focusing on intercurrent events (ICEs) and ICH E9(R1) guidelines. It offers practical insights for defining objectives and ensuring robust trial conclusions.
Area of Science:
- Clinical Trials Methodology
- Biostatistics
- Pharmaceutical Research
Background:
- The National Research Council (NRC) report emphasized defining objectives and estimands.
- Consideration has expanded beyond missing data to include intercurrent events (ICEs).
- The ICH E9(R1) draft addendum provides principles for choosing estimands in confirmatory trials.
Purpose of the Study:
- To provide practical insights and examples for implementing ICH E9(R1) estimand principles.
- To detail strategies for handling intercurrent events (ICEs) in clinical trials.
- To explore estimand considerations for various trial types and endpoints.
Main Methods:
- Discussion of stakeholder perspectives on estimand choice.
- Analysis of randomization and intention-to-treat principles.
- Detailed examination of strategies for managing intercurrent events (ICEs).
Main Results:
- Highlights the influence of stakeholder perspectives on estimand selection.
- Clarifies the definition of causal effects for treatment regimens.
- Presents strategies for handling ICEs with their assumptions and implications.
Conclusions:
- ICH E9(R1) provides a framework for robust estimand definition in clinical trials.
- Careful consideration of intercurrent events is crucial for valid trial conclusions.
- Practical examples aid in defining estimands across diverse clinical trial contexts.
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