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September 10, 2025Biometrics2 citationsOpen Access

Causal machine learning for heterogeneous treatment effects in the presence of missing outcome data

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MPMatthew PryceKDKarla Diaz‐OrdazRKRuth H. Keogh

Key Points

  • The proposed mDR-learner and mEP-learner effectively handle missing outcome data in causal machine learning.
  • Both estimators demonstrate oracle efficiency and outperform traditional methods in estimating conditional average treatment effect.
  • Simulation comparisons confirm the robust performance of these new estimators against existing CATE techniques.
  • Application to the GBSG2 trial illustrates significant treatment effect heterogeneity in breast cancer therapies using these novel methods.

Abstract

When estimating heterogeneous treatment effects, missing outcome data can complicate treatment effect estimation, causing certain subgroups of the population to be poorly represented. In this work, we discuss this commonly overlooked problem and consider the impact that missing at random outcome data has on causal machine learning estimators for the conditional average treatment effect (CATE). We propose 2 de-biased machine learning estimators for the CATE, the mDR-learner, and mEP-learner, which address the issue of under-representation by integrating inverse probability of censoring weights into the DR-learner and EP-learner, respectively. We show that under reasonable conditions, these estimators are oracle efficient and illustrate their favorable performance through simulated data settings, comparing them to existing CATE estimators, including comparison to estimators that use common missing data techniques. We present an example of their application using the GBSG2 trial, exploring treatment effect heterogeneity when comparing hormonal therapies to non-hormonal therapies among breast cancer patients post surgery, and offer guidance on the decisions a practitioner must make when implementing these estimators.

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Cite This Study

Pryce et al. (2025) studied this question.

synapsesocial.com/papers/68c1a77254b1d3bfb60e0961https://doi.org/10.1093/biomtc/ujaf098
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