A Machine-Learning Approach for Estimating Subgroup- and Individual-Level Treatment Effects: An Illustration Using the 65 Trial.
Zia SadiqueRichard D GrieveKarla Diaz-OrdazPaul MounceyFrancois LamontagneStephen O'NeillPublished in: Medical decision making : an international journal of the Society for Medical Decision Making (2022)
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.
Keyphrases
- machine learning
- end stage renal disease
- randomized controlled trial
- clinical trial
- cystic fibrosis
- phase iii
- study protocol
- newly diagnosed
- ejection fraction
- chronic kidney disease
- type diabetes
- cardiovascular disease
- phase ii
- climate change
- prognostic factors
- combination therapy
- deep learning
- patient reported outcomes
- single cell
- patient reported
- replacement therapy
- double blind