Existing methods typically rely on either interpretable but inflexible models or highly flexible black-box approaches that sacrifice interpretability; moreover, most impose a single global decision rule across patients. We introduce the Locally Interpretable Individualized Treatment Rule (LI-ITR) method, which combines flexible machine learning models to accurately learn complex treatment outcomes with locally interpretable approximations to construct subject-specific treatment rules. LI-ITR employs variational autoencoders to generate realistic local synthetic samples and learns individualized decision rules through a mixture of interpretable experts. Simulation studies show that LI-ITR accurately recovers true subject-specific local coefficients and optimal treatment strategies. An application to precision side-effect management in breast cancer illustrates the necessity of flexible predictive modeling and highlights the practical utility of LI-ITR in estimating optimal treatment rules while providing transparent, clinically interpretable explanations.

Treatment individualization and adaptation over time are crucial for managing chronic diseases.

The latent constructs are the lower-dimensional representations of these instruments.

We introduce an algorithm for identifying interpretable subgroups with elevated treatment effects, given an estimate of individual or conditional average treatment effects (CATE). Subgroups are characterized by "rule sets"--easy-to-understand statements of the form (Condition A AND Condition B) OR (Condition C) --which can capture high-order interactions while retaining interpretability. Our method complements existing approaches for estimating the CATE, which often produce high dimensional and uninterpretable results, by summarizing and extracting critical information from fitted models to aid decision making, policy implementation, and scientific understanding. We propose an objective function that trades-off subgroup size and effect size, and varying the hyperparameter that controls this trade-off results in a "frontier" of Pareto optimal rule sets, none of which dominates the others across all criteria. Valid inference is achievable through sample splitting. We demonstrate the utility and limitations of our method using simulated and empirical examples. In causal inference, average treatment effects (ATE) and average treatment effects on the treated (ATT) are the estimands that garner the most interest. Even if the effect of a treatment is known to be positive on average, it can vary greatly across individuals; some individuals will benefit, but some may experience no effect, and others may even be hurt.

Dynamic Treatment Regimes (DTRs) provide a systematic approach for making sequential treatment decisions that adapt to individual patient characteristics, particularly in clinical contexts where survival outcomes are of interest. Censoring-Aware Tree-Based Reinforcement Learning (CA-TRL) is a novel framework to address the complexities associated with censored data when estimating optimal DTRs. We explore ways to learn effective DTRs, from observational data. By enhancing traditional tree-based reinforcement learning methods with augmented inverse probability weighting (AIPW) and censoring-aware modifications, CA-TRL delivers robust and interpretable treatment strategies. We demonstrate its effectiveness through extensive simulations and real-world applications using the SANAD epilepsy dataset, where it outperformed the recently proposed ASCL method in key metrics such as restricted mean survival time (RMST) and decision-making accuracy. This work represents a step forward in advancing personalized and data-driven treatment strategies across diverse healthcare settings.

There is a fast-growing literature on estimating optimal treatment rules directly by maximizing the expected outcome. In biomedical studies and operations applications, censored survival outcome is frequently observed, in which case the restricted mean survival time and survival probability are of great interest. In this paper, we propose two robust criteria for learning optimal treatment rules with censored survival outcomes; the former one targets at an optimal treatment rule maximizing the restricted mean survival time, where the restriction is specified by a given quantile such as median; the latter one targets at an optimal treatment rule maximizing buffered survival probabilities, where the predetermined threshold is adjusted to account the restricted mean survival time. We provide theoretical justifications for the proposed optimal treatment rules and develop a sampling-based difference-of-convex algorithm for learning them. In simulation studies, our estimators show improved performance compared to existing methods. We also demonstrate the proposed method using AIDS clinical trial data.

The most common approach to causal modelling is the potential outcomes framework due to Neyman and Rubin. In this framework, outcomes of counterfactual treatments are assumed to be well-defined. This metaphysical assumption is often thought to be problematic yet indispensable. The conventional approach relies not only on counterfactuals but also on abstract notions of distributions and assumptions of independence that are not directly testable. In this paper, we construe causal inference as treatment-wise predictions for finite populations where all assumptions are testable; this means that one can not only test predictions themselves (without any fundamental problem) but also investigate sources of error when they fail. The new framework highlights the model-dependence of causal claims as well as the difference between statistical and scientific inference.

A century ago, Neyman showed how to evaluate the efficacy of treatment using a randomized experiment under a minimal set of assumptions. This classical repeated sampling framework serves as a basis of routine experimental analyses conducted by today's scientists across disciplines. In this paper, we demonstrate that Neyman's methodology can also be used to experimentally evaluate the efficacy of individualized treatment rules (ITRs), which are derived by modern causal machine learning algorithms. In particular, we show how to account for additional uncertainty resulting from a training process based on cross-fitting. The primary advantage of Neyman's approach is that it can be applied to any ITR regardless of the properties of machine learning algorithms that are used to derive the ITR. We also show, somewhat surprisingly, that for certain metrics, it is more efficient to conduct this ex-post experimental evaluation of an ITR than to conduct an ex-ante experimental evaluation that randomly assigns some units to the ITR. Our analysis demonstrates that Neyman's repeated sampling framework is as relevant for causal inference today as it has been since its inception.