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

arXiv 2408.03463 ↗First posted 6 Aug 2024stat.ME cs.AIcited by 0

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Abstract (arXiv)

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.

Key facts. No citing papers in the map yet (data through 7 Oct 2026). Builds on 23 of its 69 references that are in the map. First posted on arXiv 6 Aug 2024.

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23 of 69 references.

References · 23

  1. 2015Estimation and Inference of Heterogeneous Treatment Effects using Random Forestscited by 597
  2. 2017Causal Effect Inference with Deep Latent-Variable Modelscited by 408
  3. 2016Learning Representations for Counterfactual Inferencecited by 327
  4. 2019Adapting Neural Networks for the Estimation of Treatment Effectscited by 285
  5. 2015Recursive Partitioning for Heterogeneous Causal Effectscited by 254
  6. 2019Intensity-Free Learning of Temporal Point Processescited by 138
  7. 2020Estimating the Effects of Continuous-valued Interventions using Generative Adversarial Networkscited by 78
  8. 2020Deep Survival Machines: Fully Parametric Survival Regression and Representation Learning for Censored Data with Competing Riskscited by 70
  9. 2022Causal Machine Learning for Healthcare and Precision Medicinecited by 68
  10. 2021Deep Cox Mixtures for Survival Regressioncited by 43
  11. 2021Survival Regression with Proper Scoring Rules and Monotonic Neural Networkscited by 42
  12. 2021SurvITE: Learning Heterogeneous Treatment Effects from Time-to-Event Datacited by 25
11 more
  1. 2023Neural Fine-Gray: Monotonic neural networks for competing riskscited by 20
  2. 2022auton-survival: an Open-Source Package for Regression, Counterfactual Estimation, Evaluation and Phenotyping with Censored Time-to-Event Datacited by 17
  3. 2017Causal Rule Sets for Identifying Subgroups with Enhanced Treatment Effectcited by 15
  4. 2019Interpretable Subgroup Discovery in Treatment Effect Estimation with Application to Opioid Prescribing Guidelinescited by 13
  5. 2022Counterfactual Phenotyping with Censored Time-to-Eventscited by 13
  6. 2020Estimating heterogeneous survival treatment effect in observational data using machine learningcited by 12
  7. 2019Targeted Estimation of Heterogeneous Treatment Effect in Observational Survival Analysiscited by 9
  8. 2014A regression tree approach to identifying subgroups with differential treatment effectscited by 6
  9. 2018Efficient Discovery of Heterogeneous Quantile Treatment Effects in Randomized Experiments via Anomalous Pattern Detectioncited by 2
  10. 2023Recovering Sparse and Interpretable Subgroups with Heterogeneous Treatment Effects with Censored Time-to-Event Outcomescited by 1
  11. 2024Subgroup analysis methods for time-to-event outcomes in heterogeneous randomized controlled trialscited by 1

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