Causal Rule Ensemble Approach for Multi‐Arm Data

The estimation of the heterogeneous treatment effect (HTE) is critical in medical research. It provides insights into how treatment effects vary among individuals, which can provide statistical evidence for precision medicine. While most existing methods focus on binary treatment situations, real-world applications often involve multiple interventions. However, current HTE estimation methods are designed primarily for binary comparisons and often rely on black-box models, which limits their applicability and interpretability in multi-arm settings. To address these challenges, we propose an interpretable machine learning framework for HTE estimation in multi-arm trials. Our method employs a rule-based ensemble approach consisting of rule generation, rule ensembling, and HTE estimation, ensuring both predictive accuracy and interpretability. Through extensive simulation studies and real data applications, the performance of our method was evaluated against that of state-of-the-art multi-arm HTE estimation approaches. The results indicate that our approach achieves lower bias and higher estimation accuracy than existing methods. Furthermore, the interpretability of our framework allows clearer insights into how covariates influence treatment effects, facilitating clinical decision-making. By bridging the gap between accuracy and interpretability, our study contributes a valuable tool for multi-arm HTE estimation, supporting precision medicine.

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Publication Details

Journal
Statistics in Medicine
Published
2026-02-01
DOI
https://doi.org/10.1002/sim.70443
Primary Topic
Advanced Causal Inference Techniques
Type
article
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article

Causal Rule Ensemble Approach for Multi‐Arm Data

Kensuke Tanioka, Toshio Shimokawa, Ke Wan
Statistics in Medicine
Advanced Causal Inference Techniques
article

Causal Rule Ensemble Approach for Multi‐Arm Data

Kensuke Tanioka, Toshio Shimokawa, Ke Wan
article en

Abstract

The estimation of the heterogeneous treatment effect (HTE) is critical in medical research. It provides insights into how treatment effects vary among individuals, which can provide statistical evidence for precision medicine. While most existing methods focus on binary treatment situations, real-world applications often involve multiple interventions. However, current HTE estimation methods are designed primarily for binary comparisons and often rely on black-box models, which limits their applicability and interpretability in multi-arm settings. To address these challenges, we propose an interpretable machine learning framework for HTE estimation in multi-arm trials. Our method employs a rule-based ensemble approach consisting of rule generation, rule ensembling, and HTE estimation, ensuring both predictive accuracy and interpretability. Through extensive simulation studies and real data applications, the performance of our method was evaluated against that of state-of-the-art multi-arm HTE estimation approaches. The results indicate that our approach achieves lower bias and higher estimation accuracy than existing methods. Furthermore, the interpretability of our framework allows clearer insights into how covariates influence treatment effects, facilitating clinical decision-making. By bridging the gap between accuracy and interpretability, our study contributes a valuable tool for multi-arm HTE estimation, supporting precision medicine.

Statistics in MedicineVol. 45(3-5)
Doshisha University (JP), Wakayama Medical University (JP)
Peace, Justice and strong institutions
Openalex Percentile: Top 60%
Advanced Causal Inference Techniques
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