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

Med Decis Making. 2022 Oct;42(7):923-936. doi: 10.1177/0272989X221100717. Epub 2022 May 24.

Abstract

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.

Keywords: causal forests; heterogeneous treatment effects; machine learning; personalized medicine.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Machine Learning*