The growing demand for personalized decision-making has led to a surge of interest in estimating the Conditional Average Treatment Effect (CATE). Various types of CATE estimators have been developed with advancements in machine learning and causal inference. However, selecting the desirable CATE estimator through a conventional model validation procedure remains impractical due to the absence of counterfactual outcomes in observational data.

Numerous algorithms have been developed for Conditional Average Treatment Effect (CATE) estimation. In this paper, we first highlight a common issue where many algorithms exhibit inconsistent learning behavior for the same instance across different group assignments. We introduce a metric to quantify and visualize this inconsistency. Next, we present a theoretical analysis showing that this inconsistency indeed contributes to higher test errors and cannot be resolved through conventional machine learning techniques. To address this problem, we propose a general method called \textbf{Consistent Labeling Across Group Assignments} (CLAGA), which eliminates the inconsistency and is applicable to any existing CATE estimation algorithm. Experiments on both synthetic and real-world datasets demonstrate significant performance improvements with CLAGA.

The growing demand for personalized decision-making has led to a surge of interest in estimating the Conditional Average Treatment Effect (CATE). Various types of CATE estimators have been developed with advancements in machine learning and causal inference. However, selecting the desirable CATE estimator through a conventional model validation procedure remains impractical due to the absence of counterfactual outcomes in observational data. First, they must determine the metric form and the underlying machine learning models for fitting nuisance parameters (e.g., outcome function, propensity function, and plug-in learner). Second, they lack a specific focus on selecting a robust CATE estimator.

To test scientific theories and develop individualized treatment rules, researchers often wish to learn heterogeneous treatment effects that can be consistently found across diverse populations and contexts. We consider the problem of generalizing heterogeneous treatment effects (HTE) based on data from multiple sites. A key challenge is that a target population may differ from the source sites in unknown and unobservable ways. This means that the estimates from site-specific models lack external validity, and a simple pooled analysis risks bias. We develop a robust CATE (conditional average treatment effect) estimation methodology with multisite data from heterogeneous populations. We propose a minimax-regret framework that learns a generalizable CATE model by minimizing the worst-case regret over a class of target populations whose CATE can be represented as convex combinations of site-specific CATEs. Using robust optimization, the proposed methodology accounts for distribution shifts in both individual covariates and treatment effect heterogeneity across sites. We show that the resulting CATE model has an interpretable closed-form solution, expressed as a weighted average of site-specific CATE models. Thus, researchers can utilize a flexible CATE estimation method within each site and aggregate site-specific estimates to produce the final model. Through simulations and a real-world application, we show that the proposed methodology improves the robustness and generalizability of existing approaches.

Digital health interventions (DHIs) and remote patient monitoring (RPM) have shown great potential in improving chronic disease management through personalized care. However, barriers like limited efficacy and workload concerns hinder adoption of existing DHIs; while limited sample sizes and lack of interpretability limit the effectiveness and adoption of purely black-box algorithmic DHIs. In this paper, we address these challenges by developing a pipeline for learning explainable treatment policies for RPM-enabled DHIs. We apply our approach in the real-world setting of RPM using a DHI to improve glycemic control of youth with type 1 diabetes. Our main contribution is to reveal the importance of clinical domain knowledge in developing state and action representations for effective, efficient, and interpretable targeting policies. We observe that policies learned from clinician-informed representations are significantly more efficacious and efficient than policies learned from black-box representations. This work emphasizes the importance of collaboration between ML researchers and clinicians for developing effective DHIs in the real world.

We present unexpected findings from a large-scale benchmark study evaluating Conditional Average Treatment Effect (CATE) estimation algorithms. By running 16 modern CATE models across 43,200 datasets, we find that: (a) 62\% of CATE estimates have a higher Mean Squared Error (MSE) than a trivial zero-effect predictor, rendering them ineffective; (b) in datasets with at least one useful CATE estimate, 80\% still have higher MSE than a constant-effect model; and (c) Orthogonality-based models outperform other models only 30\% of the time, despite widespread optimism about their performance. These findings expose significant limitations in current CATE models and suggest ample opportunities for further research. Our findings stem from a novel application of \textit{observational sampling}, originally developed to evaluate Average Treatment Effect (ATE) estimates from observational methods with experiment data. To adapt observational sampling for CATE evaluation, we introduce a statistical parameter, $Q$, equal to MSE minus a constant and preserves the ranking of models by their MSE. We then derive a family of sample statistics, collectively called $\hat{Q}$, that can be computed from real-world data. We prove that $\hat{Q}$ is a consistent estimator of $Q$ under mild technical conditions. When used in observational sampling, $\hat{Q}$ is unbiased and asymptotically selects the model with the smallest MSE. To ensure the benchmark reflects real-world heterogeneity, we handpick datasets where outcomes come from field rather than simulation. By combining the new observational sampling method, new statistics, and real-world datasets, the benchmark provides a unique perspective on CATE estimator performance and uncover gaps in capturing real-world heterogeneity.

The estimation of Conditional Average Treatment Effects (CATE) is crucial for understanding the heterogeneity of treatment effects in clinical trials. We evaluate the performance of common methods, including causal forests and various meta-learners, across a diverse set of scenarios, revealing that each of the methods struggles in one or more of the tested scenarios. Given the inherent uncertainty of the data-generating process in real-life scenarios, the robustness of a CATE estimator to various scenarios is critical for its reliability. To address this limitation of existing methods, we propose two new ensemble methods that integrate multiple estimators to enhance prediction stability and performance - Stacked X-Learner which uses the X-Learner with model stacking for estimating the nuisance functions, and Consensus Based Averaging (CBA), which averages only the models with highest internal agreement. We show that these models achieve good performance across a wide range of scenarios varying in complexity, sample size and structure of the underlying-mechanism, including a biologically driven model for PD-L1 inhibition pathway for cancer treatment. Furthermore, we demonstrate improved performance by the Stacked X-Learner also when comparing to other ensemble methods, including R-Stacking, Causal-Stacking and others.