``Statistical Performance Guarantee for Subgroup Identification with Generic Machine Learning.''

 

  Abstract

Across a wide array of disciplines, many researchers use machine learning (ML) algorithms to identify a subgroup of individuals who are likely to benefit from a treatment the most (``exceptional responders'') or those who are harmed by it. A common approach to this subgroup identification problem consists of two steps. First, researchers estimate the conditional average treatment effect (CATE) using an ML algorithm. Next, they use the estimated CATE to select those individuals who are predicted to be most affected by the treatment, either positively or negatively. Unfortunately, CATE estimates are often biased and noisy. In addition, utilizing the same data to both identify a subgroup and estimate its group average treatment effect results in a multiple testing problem. To address these challenges, we develop uniform confidence bands for estimation of the group average treatment effect sorted by generic ML algorithm (GATES). Using these uniform confidence bands, researchers can identify, with a statistical guarantee, a subgroup whose GATES exceeds a certain effect size, regardless of how this effect size is chosen. The validity of the proposed methodology depends solely on randomization of treatment and random sampling of units. Importantly, our method does not require modeling assumptions and avoids a computationally intensive resampling procedure. A simulation study shows that the proposed uniform confidence bands are reasonably informative and have an appropriate empirical coverage even when the sample size is as small as 100. We analyze a clinical trial of late-stage prostate cancer and find a relatively large proportion of exceptional responders. The open-source software package is available for implementing the proposed methodology.

  Software and Related Paper

Imai, Kosuke and Michael Lingzhi Li. (2023). ``Experimental Evaluation of Individualized Treatment Rules.'' Journal of the American Statistical Association, Vol. 118, No. 541, pp. 242-256.
Li, Michael Lingzhi and Kosuke Imai. (2024). ``Neyman Meets Causal Machine Learning: Experimental Evaluation of Individualized Treatment Rules.'' Journal of Causal Inference, Vol 12, No. 1, pp. 1-20. Special Issue on Neyman (1923) and its influences on causal inference.
Imai, Kosuke and Michael Lingzhi Li. (2025). ``Statistical Inference for Heterogeneous Treatment Effects Discovered by Generic Machine Learning in Randomized Experiments.'' Journal of Business & Economic Statistics, Vol. 43, No. 1, pp. 256-268.
Jia, Zeyang, Kosuke Imai, and Michael Lingzhi Li. ``Cramming Contextual Bandits for On-policy Statistical Evaluation.''
Li, Michael Lingzhi and Kosuke Imai. ``evalITR: Evaluating Individualized Treatment Rules.'' available through The Comprehensive R Archive Network and GitHub

© Kosuke Imai
 Last modified: Mon Apr 21 11:54:45 BST 2025