A Machine-Learning Approach for Estimating Subgroup- and Individual-Level Treatment Effects: An Illustration Using the 65 Trial.

Zia Sadique ORCID logo; Richard Grieve ORCID logo; Karla Diaz-Ordaz ORCID logo; Paul Mouncey; Francois Lamontagne; Stephen O'Neill ORCID logo; (2022) A Machine-Learning Approach for Estimating Subgroup- and Individual-Level Treatment Effects: An Illustration Using the 65 Trial. Medical decision making : an international journal of the Society for Medical Decision Making, 42 (7). pp. 923-936. ISSN 0272-989X DOI: 10.1177/0272989X221100717
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This article examines a causal machine-learning approach, causal forests (CF), for exploring the heterogeneity of treatment effects, without prespecifying a specific functional form.The CF approach is considered in the reanalysis of the 65 Trial and was found to provide similar estimates of subgroup effects to using a fixed parametric model.The CF approach also provides estimates of individual-level treatment effects that suggest that for most patients in the 65 Trial, the intervention is expected to reduce 90-d mortality but with wide levels of statistical uncertainty.The study illustrates how individual-level treatment effect estimates can be analyzed to generate hypotheses for further research about those patients who are likely to benefit most from an intervention.


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