Causal rule ensemble approach for multi-arm data

Abstract

Heterogeneous treatment effect (HTE) estimation 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 primarily designed for binary comparisons and often rely on black-box models, which limit 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 ensemble, 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 state-of-the-art multi-arm HTE estimation approaches. The results indicate that our approach achieved lower bias and higher estimation accuracy compared with those of 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.