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

Fuente: arXiv
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Hauptverfasser: Jeanselme, Vincent, Yoon, Chang Ho, Falck, Fabian, Tom, Brian, Barrett, Jessica
Format: Preprint
Veröffentlicht: 2024
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author Jeanselme, Vincent
Yoon, Chang Ho
Falck, Fabian
Tom, Brian
Barrett, Jessica
author_facet Jeanselme, Vincent
Yoon, Chang Ho
Falck, Fabian
Tom, Brian
Barrett, Jessica
contents 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.
format Preprint
id arxiv_https___arxiv_org_abs_2408_03463
institution arXiv
publishDate 2024
record_format arxiv
spellingShingle Identifying treatment response subgroups in observational time-to-event data
Jeanselme, Vincent
Yoon, Chang Ho
Falck, Fabian
Tom, Brian
Barrett, Jessica
Methodology
Artificial Intelligence
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.
title Identifying treatment response subgroups in observational time-to-event data
topic Methodology
Artificial Intelligence
url https://arxiv.org/abs/2408.03463