Double machine learning methods for estimating average treatment effects: a comparative study

Observational cohort studies are increasingly being used for comparative effectiveness research to assess the safety of therapeutics. Recently, various doubly robust methods have been proposed for average treatment effect estimation by combining the treatment model and the outcome model via different vehicles, such as matching, weighting, and regression. The key advantage of doubly robust estimators is that they require either the treatment model or the outcome model to be correctly specified to obtain a consistent estimator of average treatment effects, and therefore lead to a more accurate and often more precise inference. However, little work has been done to understand how doubly robust estimators differ due to their unique strategies of using the treatment and outcome models and how machine learning techniques can be combined to boost their performance, which we call double machine learning estimators. Here, we examine multiple popular doubly robust methods and compare their performance using different treatment and outcome modeling via extensive simulations and a real-world application. We found that incorporating machine learning with doubly robust estimators such as the targeted maximum likelihood estimator gives the best overall performance. Practical guidance on how to apply doubly robust estimators is provided.

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Publication Details

Journal
Journal of Biopharmaceutical Statistics
Published
2025-04-21
DOI
https://doi.org/10.1080/10543406.2025.2489281
Citations
2
Primary Topic
Advanced Causal Inference Techniques
Type
article
Field-Weighted Citation Impact
5.26
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article

Double machine learning methods for estimating average treatment effects: a comparative study

Shu Yang, Ilya Lipkovich, Zbigniew Kadziola, Douglas E. Faries et al.
2 citations
Journal of Biopharmaceutical Statistics
Advanced Causal Inference Techniques
5.26
article

Double machine learning methods for estimating average treatment effects: a comparative study

Shu Yang, Ilya Lipkovich, Zbigniew Kadziola, Douglas E. Faries, Wenyu Ye, Xiaoqing Tan
article en
2 citations

Abstract

Observational cohort studies are increasingly being used for comparative effectiveness research to assess the safety of therapeutics. Recently, various doubly robust methods have been proposed for average treatment effect estimation by combining the treatment model and the outcome model via different vehicles, such as matching, weighting, and regression. The key advantage of doubly robust estimators is that they require either the treatment model or the outcome model to be correctly specified to obtain a consistent estimator of average treatment effects, and therefore lead to a more accurate and often more precise inference. However, little work has been done to understand how doubly robust estimators differ due to their unique strategies of using the treatment and outcome models and how machine learning techniques can be combined to boost their performance, which we call double machine learning estimators. Here, we examine multiple popular doubly robust methods and compare their performance using different treatment and outcome modeling via extensive simulations and a real-world application. We found that incorporating machine learning with doubly robust estimators such as the targeted maximum likelihood estimator gives the best overall performance. Practical guidance on how to apply doubly robust estimators is provided.

Journal of Biopharmaceutical StatisticsVol. 35(6)
North Carolina State University (US), Eli Lilly (United States) (US), University of Pittsburgh (US)
Openalex Percentile: Top 5%
Advanced Causal Inference Techniques
5.26
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