No Arabic abstract
Randomized controlled trials typically analyze the effectiveness of treatments with the goal of making treatment recommendations for patient subgroups. With the advance of electronic health records, a great variety of data has been collected in clinical practice, enabling the evaluation of treatments and treatment policies based on observational data. In this paper, we focus on learning individualized treatment rules (ITRs) to derive a treatment policy that is expected to generate a better outcome for an individual patient. In our framework, we cast ITRs learning as a contextual bandit problem and minimize the expected risk of the treatment policy. We conduct experiments with the proposed framework both in a simulation study and based on a real-world dataset. In the latter case, we apply our proposed method to learn the optimal ITRs for the administration of intravenous (IV) fluids and vasopressors (VP). Based on various offline evaluation methods, we could show that the policy derived in our framework demonstrates better performance compared to both the physicians and other baselines, including a simple treatment prediction approach. As a long-term goal, our derived policy might eventually lead to better clinical guidelines for the administration of IV and VP.
Recent development in the data-driven decision science has seen great advances in individualized decision making. Given data with individual covariates, treatment assignments and outcomes, policy makers best individualized treatment rule (ITR) that maximizes the expected outcome, known as the value function. Many existing methods assume that the training and testing distributions are the same. However, the estimated optimal ITR may have poor generalizability when the training and testing distributions are not identical. In this paper, we consider the problem of finding an optimal ITR from a restricted ITR class where there is some unknown covariate changes between the training and testing distributions. We propose a novel distributionally robust ITR (DR-ITR) framework that maximizes the worst-case value function across the values under a set of underlying distributions that are close to the training distribution. The resulting DR-ITR can guarantee the performance among all such distributions reasonably well. We further propose a calibrating procedure that tunes the DR-ITR adaptively to a small amount of calibration data from a target population. In this way, the calibrated DR-ITR can be shown to enjoy better generalizability than the standard ITR based on our numerical studies.
Recent development in data-driven decision science has seen great advances in individualized decision making. Given data with individual covariates, treatment assignments and outcomes, researchers can search for the optimal individualized treatment rule (ITR) that maximizes the expected outcome. Existing methods typically require initial estimation of some nuisance models. The double robustness property that can protect from misspecification of either the treatment-free effect or the propensity score has been widely advocated. However, when model misspecification exists, a doubly robust estimate can be consistent but may suffer from downgraded efficiency. Other than potential misspecified nuisance models, most existing methods do not account for the potential problem when the variance of outcome is heterogeneous among covariates and treatment. We observe that such heteroscedasticity can greatly affect the estimation efficiency of the optimal ITR. In this paper, we demonstrate that the consequences of misspecified treatment-free effect and heteroscedasticity can be unified as a covariate-treatment dependent variance of residuals. To improve efficiency of the estimated ITR, we propose an Efficient Learning (E-Learning) framework for finding an optimal ITR in the multi-armed treatment setting. We show that the proposed E-Learning is optimal among a regular class of semiparametric estimates that can allow treatment-free effect misspecification. In our simulation study, E-Learning demonstrates its effectiveness if one of or both misspecified treatment-free effect and heteroscedasticity exist. Our analysis of a Type 2 Diabetes Mellitus (T2DM) observational study also suggests the improved efficiency of E-Learning.
Understanding how treatment effects vary on individual characteristics is critical in the contexts of personalized medicine, personalized advertising and policy design. When the characteristics are of practical interest are only a subset of full covariate, non-parametric estimation is often desirable; but few methods are available due to the computational difficult. Existing non-parametric methods such as the inverse probability weighting methods have limitations that hinder their use in many practical settings where the values of propensity scores are close to 0 or 1. We propose the propensity score regression (PSR) that allows the non-parametric estimation of the heterogeneous treatment effects in a wide context. PSR includes two non-parametric regressions in turn, where it first regresses on the propensity scores together with the characteristics of interest, to obtain an intermediate estimate; and then, regress the intermediate estimates on the characteristics of interest only. By including propensity scores as regressors in the non-parametric manner, PSR is capable of substantially easing the computational difficulty while remain (locally) insensitive to any value of propensity scores. We present several appealing properties of PSR, including the consistency and asymptotical normality, and in particular the existence of an explicit variance estimator, from which the analytical behaviour of PSR and its precision can be assessed. Simulation studies indicate that PSR outperform existing methods in varying settings with extreme values of propensity scores. We apply our method to the national 2009 flu survey (NHFS) data to investigate the effects of seasonal influenza vaccination and having paid sick leave across different age groups.
Precision medicine is an emerging scientific topic for disease treatment and prevention that takes into account individual patient characteristics. It is an important direction for clinical research, and many statistical methods have been recently proposed. One of the primary goals of precision medicine is to obtain an optimal individual treatment rule (ITR), which can help make decisions on treatment selection according to each patients specific characteristics. Recently, outcome weighted learning (OWL) has been proposed to estimate such an optimal ITR in a binary treatment setting by maximizing the expected clinical outcome. However, for ordinal treatment settings, such as individualized dose finding, it is unclear how to use OWL. In this paper, we propose a new technique for estimating ITR with ordinal treatments. In particular, we propose a data duplication technique with a piecewise convex loss function. We establish Fisher consistency for the resulting estimated ITR under certain conditions, and obtain the convergence and risk bound properties. Simulated examples and two applications to datasets from an irritable bowel problem and a type 2 diabetes mellitus observational study demonstrate the highly competitive performance of the proposed method compared to existing alternatives.
We study the problem of estimating the continuous response over time to interventions using observational time series---a retrospective dataset where the policy by which the data are generated is unknown to the learner. We are motivated by applications where response varies by individuals and therefore, estimating responses at the individual-level is valuable for personalizing decision-making. We refer to this as the problem of estimating individualized treatment response (ITR) curves. In statistics, G-computation formula (Robins, 1986) has been commonly used for estimating treatment responses from observational data containing sequential treatment assignments. However, past studies have focused predominantly on obtaining point-in-time estimates at the population level. We leverage the G-computation formula and develop a novel Bayesian nonparametric (BNP) method that can flexibly model functional data and provide posterior inference over the treatment response curves at both the individual and population level. On a challenging dataset containing time series from patients admitted to a hospital, we estimate responses to treatments used in managing kidney function and show that the resulting fits are more accurate than alternative approaches. Accurate methods for obtaining ITRs from observational data can dramatically accelerate the pace at which personalized treatment plans become possible.