When devising a course of treatment for a patient, doctors often have little quantitative evidence on which to base their decisions, beyond their medical education and published clinical trials. Stanford Health Care alone has millions of electronic medical records (EMRs) that are only just recently being leveraged to inform better treatment recommendations. These data present a unique challenge because they are high-dimensional and observational. Our goal is to make personalized treatment recommendations based on the outcomes for past patients similar to a new patient. We propose and analyze three methods for estimating heterogeneous treatment effects using observational data. Our methods perform well in simulations using a wide variety of treatment effect functions, and we present results of applying the two most promising methods to data from The SPRINT Data Analysis Challenge, from a large randomized trial of a treatment for high blood pressure.
Much of causal inference is based on the propensity score , which is the estimated probability that a patient would receive treatment, conditioned on the patient's covariates. If the estimate of the propensity function ) is @math , then the propensity score for a patient with covariate vector @math is @math . Throughout the present work, we estimate the propensity function using the probability forests of @cite_8 . We are able to do so quickly using the fast implementation in the R package ranger .
@cite_8
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We are interested in estimating the average effect of a binary treatment on a scalar outcome. If assignment to the treatment is independent of the potential outcomes given pretreatment variables, biases associated with simple treatment-control average comparisons can be removed by adjusting for differences in the pre-treatment variables. Rosenbaum and Rubin (1983, 1984) show that adjusting solely for differences between treated and control units in a scalar function of the pre-treatment, the propensity score, also removes the entire bias associated with differences in pre-treatment variables. Thus it is possible to obtain unbiased estimates of the treatment effect without conditioning on a possibly high-dimensional vector of pre-treatment variables. Although adjusting for the propensity score removes all the bias, this can come at the expense of efficiency. We show that weighting with the inverse of a nonparametric estimate of the propensity score, rather than the true propensity score, leads to efficient estimates of the various average treatment effects. This result holds whether the pre-treatment variables have discrete or continuous distributions. We provide intuition for this result in a number of ways. First we show that with discrete covariates, exact adjustment for the estimated propensity score is identical to adjustment for the pre-treatment variables. Second, we show that weighting by the inverse of the estimated propensity score can be interpreted as an empirical likelihood estimator that efficiently incorporates the information about the propensity score. Finally, we make a connection to results to other results on efficient estimation through weighting in the context of variable probability sampling. Abstract Causal effect modeling with naturalistic rather than experimental data is challenging. In observational studies participants in different treatment conditions may also differ on pretreatment characteristics that influence outcomes. Propensity score methods can theoretically eliminate these confounds for all observed covariates, but accurate estimation of propensity scores is impeded by large numbers of covariates, uncertain functional forms for their associations with treatment selection, and other problems. This article demonstrates that boosting, a modern statistical technique, can overcome many of these obstacles. The authors illustrate this approach with a study of adolescent probationers in substance abuse treatment programs. Propensity score weights estimated using boosting eliminate most pretreatment group differences and substantially alter the apparent relative effects of adolescent substance abuse treatment. Abstract : The results of observational studies are often disputed because of nonrandom treatment assignment. For example, patients at greater risk may be overrepresented in some treatment group. This paper discusses the central role of propensity scores and balancing scores in the analysis of observational studies. The propensity score is the (estimated) conditional probability of assignment to a particular treatment given a vector of observed covariates. Both large and small sample theory show that adjustment for the scalar propensity score is sufficient to remove bias due to all observed covariates. Applications include: matched sampling on the univariate propensity score which is equal percent bias reducing under more general conditions than required for discriminant matching, multivariate adjustment by subclassification on balancing scores where the same subclasses are used to estimate treatment effects for all outcome variables and in all subpopulations, and visual representation of multivariate adjustment by a two-dimensional plot. (Author) In observational studies, propensity scores are commonly estimated by maxi- mum likelihood but may fail to balance high-dimensional pre-treatment covariates even after specification search. We introduce a general framework that unifies and generalizes several recent proposals to improve covariate balance when designing an observational study. In- stead of the likelihood function, we propose to optimize special loss functions---covariate balancing scoring rules (CBSR)---to estimate the propensity score. A CBSR is uniquely determined by the link function in the GLM and the estimand (a weighted average treatment effect). We show CBSR does not lose asymptotic efficiency to the Bernoulli likelihood in estimating the weighted average treatment effect compared, but CBSR is much more robust in finite sample. Borrowing tools developed in statistical learning, we propose practical strategies to balance covariate functions in rich function classes. This is useful to estimate the maximum bias of the inverse probability weighting (IPW) estimators and construct honest confidence interval in finite sample. Lastly, we provide several numerical examples to demonstrate the trade-off of bias and variance in the IPW-type estimators and the trade-off in balancing different function classes of the covariates.