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

• URL: http://arxiv.org/abs/2408.03463v4
• Date: Mon, 24 Feb 2025 00:33:14 GMT
• Title: Identifying treatment response subgroups in observational time-to-event data
• Authors: Vincent Jeanselme, Chang Ho Yoon, Fabian Falck, Brian Tom, Jessica Barrett,
• Abstract summary: Existing approaches for treatment effect estimation rely on Randomised Controlled Trials (RCTs)<n>RCTs tend to feature more homogeneous patient groups, making them less relevant for uncovering subgroups in the population encountered in real-world clinical practice.<n>Our work introduces a novel, outcome-guided, subgroup analysis strategy for identifying subgroups of treatment response in both RCTs and observational studies alike.
• Score: 2.176207087460772
• License: http://creativecommons.org/licenses/by/4.0/
• 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 are often limited by insufficient power, multiple comparisons, and unbalanced covariates. In addition, RCTs 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.

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