The estimands framework: a primer on the ICH E9(R1) addendum

2024/01/23 by Brennan C Kahan, Joanna Hindley, Mark Edwards +2 · 2 voices · 12 citations
Economics, Econometrics and Finance · Mathematics · #Advanced Causal Inference Techniques #Health Systems, Economic Evaluations, Quality of Life #Statistical Methods and Inference

paper · pdf · doi:10.1136/bmj-2023-076316

openalex publication_date 2024/01/23 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/02

Abstract

Estimands can be used in studies of healthcare interventions to clarify the interpretation of treatment effects. The addendum to the ICH E9 harmonised guideline on statistical principles for clinical trials (ICH E9(R1)) describes a framework for using estimands as part of a study. This paper provides an overview of the estimands framework, as outlined in the addendum, with the aim of explaining why estimands are beneficial; clarifying the terminology being used; and providing practical guidance on using estimands to decide the appropriate study design, data collection, and estimation methods. This article illustrates how to use the estimands framework by applying it to an ongoing trial in emergency bowel surgery. Estimands can be a useful way of clarifying the exact research question being evaluated in a study, both to avoid misinterpretation and to ensure that study methods are aligned to the overall study objectives.

Citations

• On the Application of Probability Theory to Agricultural Experiments. Essay on Principles. Section 9
• A Theorem on the Manipulability of Redundant Serial Kinematic Chains
• Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available: Table 1.
• Prediction meets causal inference: the role of treatment in clinical prediction models

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Discussions

• Check out the below for a nice description. Also can check out a similar article by the same lead author here: doi.org/10.1136/bmj-... [bsky, 2 points, 1 comments]
• Indeed. This paper is a good overview of the ICH E9 addendum on estimands on this topic: doi.org/10.1136/bmj-... [bsky, 0 points, 0 comments]

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