English

Identifying treatment response subgroups in observational time-to-event data

Methodology 2025-12-10 v6 Artificial Intelligence

Abstract

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily rely on Randomised Controlled Trials (RCTs), which tend to feature more homogeneous patient groups, making them less relevant for uncovering subgroups in the population encountered in real-world clinical practice. Subgroup analyses established for RCTs suffer from significant statistical biases when applied to observational studies, which benefit from larger and more representative populations. Our work introduces a novel, outcome-guided, subgroup analysis strategy for identifying subgroups of treatment response in both RCTs and observational studies alike. It hence positions itself in-between individualised and average treatment effect estimation to uncover patient subgroups with distinct treatment responses, critical for actionable insights that may influence treatment guidelines. In experiments, our approach significantly outperforms the current state-of-the-art method for subgroup analysis in both randomised and observational treatment regimes.

Keywords

Cite

@article{arxiv.2408.03463,
  title  = {Identifying treatment response subgroups in observational time-to-event data},
  author = {Vincent Jeanselme and Chang Ho Yoon and Fabian Falck and Brian Tom and Jessica Barrett},
  journal= {arXiv preprint arXiv:2408.03463},
  year   = {2025}
}

Comments

Presented at ML4H 2025

R2 v1 2026-06-28T18:05:53.677Z