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Robust estimation of heterogeneous treatment effects in randomized trials leveraging external data

arXiv 2507.03681 ↗First posted 4 Jul 2025stat.ML cs.LG stat.MEcited by 1

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Abstract (arXiv)

Randomized trials are typically designed to detect average treatment effects but often lack the statistical power to uncover individual-level treatment effect heterogeneity, limiting their value for personalized decision-making. To address this, we propose the QR-learner, a model-agnostic learner that estimates conditional average treatment effects (CATE) within the trial population by leveraging external data from other trials or observational studies. The proposed method is robust: it can reduce the mean squared error relative to a trial-only CATE learner, and is guaranteed to recover the true CATE even when the external data are not aligned with the trial. Moreover, we introduce a procedure that combines the QR-learner with a trial-only CATE learner and show that it asymptotically matches or exceeds both component learners in terms of mean squared error. We examine the performance of our approach in simulation studies and apply the methods to a real-world dataset, demonstrating improvements in both CATE estimation and statistical power for detecting heterogeneous effects.

Key facts. Cited by 1 paper in the map (data through 14 Oct 2026). Builds on 25 of its 52 references that are in the map. First posted on arXiv 4 Jul 2025.

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25 of 52 references.

References · 25

  1. 2015Estimation and Inference of Heterogeneous Treatment Effects using Random Forestscited by 600
  2. 2017Meta-learners for Estimating Heterogeneous Treatment Effects using Machine Learningcited by 478
  3. 2017Quasi-Oracle Estimation of Heterogeneous Treatment Effectscited by 332
  4. 2015Recursive Partitioning for Heterogeneous Causal Effectscited by 255
  5. 2019Orthogonal Statistical Learningcited by 151
  6. 2021Nonparametric Estimation of Heterogeneous Treatment Effects: From Theory to Learning Algorithmscited by 144
  7. 2017Approximate Kernel-based Conditional Independence Tests for Fast Non-Parametric Causal Discoverycited by 76
  8. 2018Removing Hidden Confounding by Experimental Groundingcited by 64
  9. 2022Combining Observational and Randomized Data for Estimating Heterogeneous Treatment Effectscited by 32
  10. 2024Applied Causal Inference Powered by ML and AIcited by 30
  11. 2020Elastic Integrative Analysis of Randomized Trial and Real-World Data for Treatment Heterogeneity Estimationcited by 17
  12. 2020Increasing the efficiency of randomized trial estimates via linear adjustment for a prognostic scorecited by 15
13 more
  1. 2024Meta-Learners for Partially-Identified Treatment Effects Across Multiple Environmentscited by 11
  2. 2023Falsification of Internal and External Validity in Observational Studies via Conditional Moment Restrictionscited by 10
  3. 2023On Weighted Orthogonal Learners for Heterogeneous Treatment Effectscited by 10
  4. 2025Efficient Randomized Experiments Using Foundation Modelscited by 10
  5. 2024Detecting critical treatment effect bias in small subgroupscited by 7
  6. 2023Prognostic Adjustment with Efficient Estimators to Unbiasedly Leverage Historical Data in Randomized Trialscited by 6
  7. 2021Transfer learning of individualized treatment rules from experimental to real-world datacited by 4
  8. 2023Multi-Study R-Learner for Estimating Heterogeneous Treatment Effects Across Studies Using Statistical Machine Learningcited by 4
  9. 2023Improving Precision of RCT-Based CATE Estimation using Data Borrowing with Double Calibrationcited by 4
  10. 2025The Hardness of Validating Observational Studies with Experimental Datacited by 4
  11. 2019Study designs for extending causal inferences from a randomized trial to a target populationcited by 3
  12. 2024Robust integration of external control data in randomized trialscited by 3
  13. 2024Identification strategies for combining an experimental study with external datacited by 1

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