Cost-optimal Sequential Testing via Doubly Robust Q-learning

Precision medicine aims to tailor therapeutic decisions to individual patient characteristics. This objective is commonly formalized through dynamic treatment regimes, which use statistical and machine learning methods to derive sequential decision rules adapted to evolving clinical information. In most existing formulations, these approaches produce a single optimal treatment at each stage, leading to a unique decision sequence. However, in many clinical settings, several treatment options may yield similar expected outcomes, and focusing on a single optimal policy may conceal meaningful alternatives. We extend the Q-learning framework for retrospective data by introducing a worst-value tolerance criterion controlled by a hyperparameter $\varepsilon$, which specifies the maximum acceptable deviation from the optimal expected value. Rather than identifying a single optimal policy, the proposed approach constructs sets of $\varepsilon$-optimal policies whose performance remains within a controlled neighborhood of the optimum. This formulation shifts Q-learning from a vector-valued representation to a matrix-valued one, allowing multiple admissible value functions to coexist during backward recursion. The approach yields families of near-equivalent treatment strategies and explicitly identifies regions of treatment indifference where several decisions achieve comparable outcomes. We illustrate the framework in two settings: a single-stage problem highlighting indifference regions around the decision boundary, and a multi-stage decision process based on a simulated oncology model describing tumor size and treatment toxicity dynamics.

When treatment effects are naturally expressed as ratios -- as in medicine, pricing, and marketing -- the ratio-based CATE $τ(x) = E[Y|W=1,X=x] / E[Y|W=0,X=x]$ is the appropriate estimand. Yet existing estimators either impose a log-linear parametric structure or apply generic regression without robustness guarantees for this functional. We introduce the Q-Learner, which decomposes $τ(x)$ into a product of two odds ratios, reducing ratio-CATE estimation for binary outcomes to two propensity classification tasks. We further derive doubly robust augmentations for both S/T- and Q-style ratio learners and characterize their distinct robustness properties. In benchmarks on seven RCT datasets, the Q-Learner is the most consistently competitive method in low-conversion regimes, where its propensity-only construction sidesteps the imbalanced regression that hurts outcome-based estimators. On four observational datasets, where propensity must be estimated and confounding cannot be ruled out, the DR learners introduced here decisively come out on top, making them practitioners' natural default for confounded observational data.

Policy learning methods are increasingly used to inform treatment allocation under budget constraints. Most proposed methods assume complete treatment data, yet applications frequently suffer from missingness that can bias estimates and lead to suboptimal policies. We address this gap by extending efficient estimators for average treatment effect (ATE) estimation to policy value and conditional average treatment effect (CATE) estimation under missing at random (MAR) and missing completely conditionally at random (MCCAR) treatment data. Through asymptotic efficiency analysis, we prove that the MAR estimator, which leverages partially-observed units, is both valid and more efficient than the MCCAR estimator when MCCAR assumptions hold. This result provides formal justification for preferring MAR-based estimation in policy learning under both missing data settings. Our comprehensive experiments using synthetic and semi-synthetic datasets confirm that correctly specifying the missingness mechanism is crucial: misspecified estimators remain biased regardless of sample size, while our estimators achieve near-oracle performance when assumptions are satisfied. Our work provides practitioners with theoretically grounded, empirically validated tools for robust policy learning in the presence of missing treatment data.