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Statistics in Medicine
Article . 2025 . Peer-reviewed
License: Wiley Online Library User Agreement
Data sources: Crossref
https://dx.doi.org/10.48550/ar...
Article . 2025
License: arXiv Non-Exclusive Distribution
Data sources: Datacite
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Using Individualized Treatment Effects to Assess Treatment Effect Heterogeneity

Authors: Konstantinos Sechidis; Cong Zhang; Sophie Sun; Yao Chen; Asher Spector; Björn Bornkamp;

Using Individualized Treatment Effects to Assess Treatment Effect Heterogeneity

Abstract

ABSTRACT Assessing treatment effect heterogeneity (TEH) in clinical trials is crucial, as it provides insights into the variability of treatment responses among patients, influencing key decisions related to drug development. Furthermore, it can lead to personalized medicine by tailoring treatments to individual patient characteristics. This paper introduces novel methodologies for assessing treatment effects using the individualized treatment effect as a basis. To estimate this effect, we use a doubly robust (DR) learner to infer a pseudo‐outcome that reflects the causal contrast. This pseudo‐outcome is then used to perform three objectives: (1) a global test for heterogeneity, (2) ranking covariates based on their influence on effect modification, and (3) providing estimates of the individualized treatment effect. We compare the DR‐learner with various alternatives and competing methods in a simulation study, and also use it to assess heterogeneity in a pooled analysis of five Phase III trials in psoriatic arthritis (PsA). By integrating these methods with the recently proposed Workflow to Assess Treatment Effect Heterogeneity in Drug Development for Clinical Trial Sponsors (WATCH) workflow, we provide a robust framework for analyzing TEH, offering insights that enable more informed decision‐making in this challenging area.

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Keywords

Treatment Effect Heterogeneity, Methodology (stat.ME), FOS: Computer and information sciences, Treatment Outcome, Models, Statistical, Clinical Trials, Phase III as Topic, Applications, Arthritis, Psoriatic, Methodology, Humans, Computer Simulation, Applications (stat.AP), Precision Medicine

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    popularity
    This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
    Top 10%
    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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    impulse
    This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
3
Top 10%
Average
Average
Green