The vast majority of literature on evaluating the significance of a treatment effect based on observational data has been confined to discrete treatments. These methods are not applicable to drawing inference for a continuous treatment, which arises in many important applications. To adjust for confounders when evaluating a continuous treatment, existing inference methods often rely on discretizing the treatment or using (possibly misspecified) parametric models for the effect curve. Recently, Kennedy et al. (2017) proposed nonparametric doubly robust estimation for a continuous treatment effect in observational studies. However, inference for the continuous treatment effect is a harder problem. To the best of our knowledge, a completely nonparametric doubly robust approach for inference in this setting is not yet available. We develop such a nonparametric doubly robust procedure in this paper for making inference on the continuous treatment effect curve. Using empirical process techniques for local U- and V-processes, we establish the test statistic's asymptotic distribution. Furthermore, we propose a wild bootstrap procedure for implementing the test in practice. We illustrate the new method via simulations and a study of a constructed dataset relating the effect of nurse staffing hours on hospital performance. We implement our doubly robust dose response test in the R package DRDRtest on CRAN.
Continuous treatments (e.g., doses) arise often in practice, but many available causal effect estimators are limited by either requiring parametric models for the effect curve, or by not allowing doubly robust covariate adjustment. We develop a novel kernel smoothing approach that requires only mild smoothness assumptions on the effect curve, and still allows for misspecification of either the treatment density or outcome regression. We derive asymptotic properties and give a procedure for data-driven bandwidth selection. The methods are illustrated via simulation and in a study of the effect …
Instrumental variable (IV) methods allow us the opportunity to address unmeasured confounding in causal inference. However, most IV methods are only applicable to discrete or continuous outcomes with very few IV methods for censored survival outcomes. In this work we propose nonparametric estimators for the local average treatment effect on survival probabilities under both nonignorable and ignorable censoring. We provide an efficient influence function-based estimator and a simple estimation procedure when the IV is either binary or continuous. The proposed estimators possess double-robustnes…
When studying treatment effects in multilevel studies, investigators commonly use (semi-)parametric estimators, which make strong parametric assumptions about the outcome, the treatment, and/or the correlation structure between study units in a cluster. We propose a novel estimator of treatment effects that does not make such assumptions. Specifically, the new estimator is shown to be doubly robust, asymptotically Normal, and often more efficient than existing estimators, all without having to make any parametric modeling assumptions about the outcome, the treatment, and the correlation struct…
Causal mediation analysis has historically been limited in two important ways: (i) a focus has traditionally been placed on binary treatments and static interventions, and (ii) direct and indirect effect decompositions have been pursued that are only identifiable in the absence of intermediate confounders affected by treatment. We present a theoretical study of an (in)direct effect decomposition of the population intervention effect, defined by stochastic interventions jointly applied to the treatment and mediators. In contrast to existing proposals, our causal effects can be evaluated regardl…
We propose a doubly robust approach to characterizing treatment effect heterogeneity in observational studies. We develop a frequentist inferential procedure that utilizes posterior distributions for both the propensity score and outcome regression models to provide valid inference on the conditional average treatment effect even when high-dimensional or nonparametric models are used. We show that our approach leads to conservative inference in finite samples or under model misspecification, and provides a consistent variance estimator when both models are correctly specified. In simulations, …
Recently, conditional average treatment effect (CATE) estimation has been attracting much attention due to its importance in various fields such as statistics, social and biomedical sciences. This study proposes a partially linear nonparametric Bayes model for the heterogeneous treatment effect estimation. A partially linear model is a semiparametric model that consists of linear and nonparametric components in an additive form. A nonparametric Bayes model that uses a Gaussian process to model the nonparametric component has already been studied. However, this model cannot handle the heterogen…
This paper constructs a doubly robust estimator for continuous dose-response estimation. An outcome regression model is augmented with a set of inverse generalized propensity score covariates to correct for potential misspecification bias. From the augmented model we can obtain consistent estimates of mean average potential outcomes for distinct strata of the treatment. A polynomial regression is then fitted to these point estimates to derive a Taylor approximation to the continuous dose-response function. The bootstrap is used for variance estimation. Analytical results and simulations show t…
In this paper, we apply doubly robust approach to estimate, when some covariates are given, the conditional average treatment effect under parametric, semiparametric and nonparametric structure of the nuisance propensity score and outcome regression models. We then conduct a systematic study on the asymptotic distributions of nine estimators with different combinations of estimated propensity score and outcome regressions. The study covers the asymptotic properties with all models correctly specified; with either propensity score or outcome regressions locally / globally misspecified; and with…
The method of instrumental variables provides a fundamental and practical tool for causal inference in many empirical studies where unmeasured confounding between the treatments and the outcome is present. Modern data such as the genetical genomics data from these studies are often high-dimensional. The high-dimensional linear instrumental-variables regression has been considered in the literature due to its simplicity albeit a true nonlinear relationship may exist. We propose a more data-driven approach by considering the nonparametric additive models between the instruments and the treatment…
We develop a Bayesian nonparametric (BNP) approach to evaluate the causal effect of treatment in a randomized trial where a nonterminal event may be censored by a terminal event, but not vice versa (i.e., semi-competing risks). Based on the idea of principal stratification, we define a novel estimand for the causal effect of treatment on the nonterminal event. We introduce identification assumptions, indexed by a sensitivity parameter, and show how to draw inference using our BNP approach. We conduct simulation studies and illustrate our methodology using data from a brain cancer trial.
Existing tests for factorial designs in the nonparametric case are based on hypotheses formulated in terms of distribution functions. Typical null hypotheses, however, are formulated in terms of some parameters or effect measures, particularly in heteroscedastic settings. Here this idea is extended to nonparametric models by introducing a novel nonparametric ANOVA-type-statistic based on ranks which is suitable for testing hypotheses formulated in meaningful nonparametric treatment effects in general factorial designs. This is achieved by a careful in-depth study of the common distribution of …
We consider the problem of testing for treatment effect heterogeneity in observational studies, and propose a nonparametric test based on multisample U-statistics. To account for potential confounders, we use reweighted data where the weights are determined by estimated propensity scores. The proposed method does not require any parametric assumptions on the outcomes and bypasses the need for modeling the treatment effect for each study subgroup. We establish the asymptotic normality for the test statistic, and demonstrate its superior numerical performance over several competing approaches vi…
Missing attributes are ubiquitous in causal inference, as they are in most applied statistical work. In this paper, we consider various sets of assumptions under which causal inference is possible despite missing attributes and discuss corresponding approaches to average treatment effect estimation, including generalized propensity score methods and multiple imputation. Across an extensive simulation study, we show that no single method systematically out-performs others. We find, however, that doubly robust modifications of standard methods for average treatment effect estimation with missing…
In this paper, we propose a doubly robust method to present the heterogeneity of the average treatment effect with respect to observed covariates of interest. We consider a situation where a large number of covariates are needed for identifying the average treatment effect but the covariates of interest for analyzing heterogeneity are of much lower dimension. Our proposed estimator is doubly robust and avoids the curse of dimensionality. We propose a uniform confidence band that is easy to compute, and we illustrate its usefulness via Monte Carlo experiments and an application to the effects o…
Conditional Distributional Treatment Effects: Doubly Robust Estimation and Testing
Department of Statistics, University of Washington, Seattle, USA（华盛顿大学统计系，美国西雅图）；Departments of Health Care Policy and Statistics, Harvard University, USA（哈佛大学健康政策与统计系，美国）
Identifying heterogeneity in a population's response to a health or policy intervention is crucial for evaluating and informing policy decisions. We propose a novel heterogeneous treatment effect estimator in the difference-in-differences design with repeated cross sectional data, where we observe different samples of a population at two time periods separated by the onset of a policy intervention, as well as samples of a population that serves as the control. Our estimator has orthogonality properties that enable fast rates on learning the treatment effect while allowing slower rates for esti…
Clinical trials involving novel immuno-oncology (IO) therapies frequently exhibit survival profiles which violate the proportional hazards assumption due to a delay in treatment effect, and in such settings, the survival curves in the two treatment arms may have a crossing before the two curves eventually separate. To flexibly model such scenarios, we describe a nonparametric approach for estimating the treatment arm-specific survival functions which constrains these two survival functions to cross at most once without making any additional assumptions about how the survival curves are related…
Missing exposure information is a very common feature of many observational studies. Here we study identifiability and efficient estimation of causal effects on vector outcomes, in such cases where treatment is unconfounded but partially missing. We consider a missing at random setting where missingness in treatment can depend not only on complex covariates, but also on post-treatment outcomes. We give a new identifying expression for average treatment effects in this setting, along with the efficient influence function for this parameter in a nonparametric model, which yields a nonparametric …
The research described herewith is to re-visit the classical doubly robust estimation of average treatment effect by conducting a systematic study on the comparisons, in the sense of asymptotic efficiency, among all possible combinations of the estimated propensity score and outcome regression. To this end, we consider all nine combinations under, respectively, parametric, nonparametric and semiparametric structures. The comparisons provide useful information on when and how to efficiently utilize the model structures in practice. Further, when there is model-misspecification, either propensit…
This paper analyzes the effect of a discrete treatment Z on a duration T. The treatment is not randomly assigned. The confounding issue is treated using a discrete instrumental variable explaining the treatment and independent of the error term of the model. Our framework is nonparametric and allows for random right censoring. This specification generates a nonlinear inverse problem and the average treatment effect is derived from its solution. We provide local and global identification properties that rely on a nonlinear system of equations. We propose an estimation procedure to solve this sy…
This paper develops a nonparametric model that represents how sequences of outcomes and treatment choices influence one another in a dynamic manner. In this setting, we are interested in identifying the average outcome for individuals in each period, had a particular treatment sequence been assigned. The identification of this quantity allows us to identify the average treatment effects (ATE's) and the ATE's on transitions, as well as the optimal treatment regimes, namely, the regimes that maximize the (weighted) sum of the average potential outcomes, possibly less the cost of the treatments. …
In causal inference confounding may be controlled either through regression adjustment in an outcome model, or through propensity score adjustment or inverse probability of treatment weighting, or both. The latter approaches, which are based on modelling of the treatment assignment mechanism and their doubly robust extensions have been difficult to motivate using formal Bayesian arguments, in principle, for likelihood-based inferences, the treatment assignment model can play no part in inferences concerning the expected outcomes if the models are assumed to be correctly specified. On the other…
We carry out ANOVA comparisons of multiple treatments for longitudinal studies with missing values. The treatment effects are modeled semiparametrically via a partially linear regression which is flexible in quantifying the time effects of treatments. The empirical likelihood is employed to formulate model-robust nonparametric ANOVA tests for treatment effects with respect to covariates, the nonparametric time-effect functions and interactions between covariates and time. The proposed tests can be readily modified for a variety of data and model combinations, that encompasses parametric, semip…
This article proposes different tests for treatment effect heterogeneity when the outcome of interest, typically a duration variable, may be right-censored. The proposed tests study whether a policy 1) has zero distributional (average) effect for all subpopulations defined by covariate values, and 2) has homogeneous average effect across different subpopulations. The proposed tests are based on two-step Kaplan-Meier integrals and do not rely on parametric distributional assumptions, shape restrictions, or on restricting the potential treatment effect heterogeneity across different subpopulatio…
In randomized clinical trials with survival outcome, there has been an increasing interest in subgroup identification based on baseline genomic, proteomic markers or clinical characteristics. Some of the existing methods identify subgroups that benefit substantially from the experimental treatment by directly modeling outcomes or treatment effect. When the goal is to find an optimal treatment for a given patient rather than finding the right patient for a given treatment, methods under the individualized treatment regime framework estimate an individualized treatment rule that would lead to th…
Estimation of causal parameters from observational data requires complete confounder adjustment, as well as positivity of the propensity score for each treatment arm. There is often a trade-off between these two assumptions: confounding bias may be reduced through adjustment for a large number of pre-treatment covariates, but positivity is less likely in analyses with irrelevant predictors of treatment such as instrumental variables. Under empirical positivity violations, propensity score weights are highly variable, and doubly robust estimators suffer from high variance and large finite sampl…
Plausible identification of conditional average treatment effects (CATEs) may rely on controlling for a large number of variables to account for confounding factors. In these high-dimensional settings, estimation of the CATE requires estimating first-stage models whose consistency relies on correctly specifying their parametric forms. While doubly-robust estimators of the CATE exist, inference procedures based on the second stage CATE estimator are not doubly-robust. Using the popular augmented inverse propensity weighting signal, we propose an estimator for the CATE whose resulting Wald-type …
Inferring the heterogeneous treatment effect is a fundamental problem in the sciences and commercial applications. In this paper, we focus on estimating Conditional Average Treatment Effect (CATE), that is, the difference in the conditional mean outcome between treatments given covariates. Traditionally, Q-Learning based approaches rely on the estimation of conditional mean outcome given treatment and covariates. However, they are subject to misspecification of the main effect model. Recently, simple and flexible one-step methods to directly learn (D-Learning) the CATE without model specificat…
The regression discontinuity (RD) design is a popular approach to causal inference in non-randomized studies. This is because it can be used to identify and estimate causal effects under mild conditions. Specifically, for each subject, the RD design assigns a treatment or non-treatment, depending on whether or not an observed value of an assignment variable exceeds a fixed and known cutoff value. In this paper, we propose a Bayesian nonparametric regression modeling approach to RD designs, which exploits a local randomization feature. In this approach, the assignment variable is treated as a c…
This paper considers conducting inference about the effect of a treatment (or exposure) on an outcome of interest. In the ideal setting where treatment is assigned randomly, under certain assumptions the treatment effect is identifiable from the observable data and inference is straightforward. However, in other settings such as observational studies or randomized trials with noncompliance, the treatment effect is no longer identifiable without relying on untestable assumptions. Nonetheless, the observable data often do provide some information about the effect of treatment, that is, the param…
We revisit the problem of estimating the local average treatment effect (LATE) and the local average treatment effect on the treated (LATT) when control variables are available, either to render the instrumental variable (IV) suitably exogenous or to improve precision. Unlike previous approaches, our doubly robust (DR) estimation procedures use quasi-likelihood methods weighted by the inverse of the IV propensity score - so-called inverse probability weighted regression adjustment (IPWRA) estimators. By properly choosing models for the propensity score and outcome models, fitted values are ens…
Performing causal inference in observational studies requires we assume confounding variables are correctly adjusted for. G-computation methods are often used in these scenarios, with several recent proposals using Bayesian versions of g-computation. In settings with few confounders, standard models can be employed, however as the number of confounders increase these models become less feasible as there are fewer observations available for each unique combination of confounding variables. In this paper we propose a new model for estimating treatment effects in observational studies that incorp…
The research in this paper gives a systematic investigation on the asymptotic behaviours of four inverse probability weighting (IPW)-based estimators for conditional average treatment effect, with nonparametrically, semiparametrically, parametrically estimated and true propensity score, respectively. To this end, we first pay a particular attention to semiparametric dimension reduction structure such that we can well study the semiparametric-based estimator that can well alleviate the curse of dimensionality and greatly avoid model misspecification. We also derive some further properties of ex…
