When units in observational studies are clustered in groups, such as students in schools or patients in hospitals, researchers often address confounding by adjusting for cluster-level covariates or cluster membership. In this paper, we develop a unified weighting framework that clarifies how different estimation methods control two distinct sources of imbalance: global balance (differences between treated and control units across clusters) and local balance (differences within clusters). We show that inverse propensity score weighting (IPW) with a random effects propensity score model -- the current standard in the literature -- targets only global balance and constant level shifts across clusters, but imposes no constraints on local balance. We then present two approaches that target both forms of balance. First, hierarchical balancing weights directly control global and local balance through a constrained optimization problem. Second, building on the recently proposed Generalized Mundlak approach, we develop a novel Mundlak balancing weights estimator that adjusts for cluster-level sufficient statistics rather than cluster indicators; this approach can accommodate small clusters where all units are treated or untreated. Critically, these approaches rest on different assumptions: hierarchical balancing weights require only that treatment is ignorable given covariates and cluster membership, while Mundlak methods additionally require an exponential family structure. We then compare these methods in a simulation study and in two applications in education and health services research that exhibit very different cluster structures.
Propensity score weighting is a tool for causal inference to adjust for measured confounders. Survey data are often collected under complex sampling designs such as multistage cluster sampling, which presents challenges for propensity score modeling and estimation. In addition, for clustered data, there may also be unobserved cluster effects related to both the treatment and the outcome. When such unmeasured confounders exist and are omitted in the propensity score model, the subsequent propensity score adjustment will be biased. We propose a calibrated propensity score weighting adjustment fo…
Estimating the individual treatment effect (ITE) from observational data is meaningful and practical in healthcare. Existing work mainly relies on the strong ignorability assumption that no hidden confounders exist, which may lead to bias in estimating causal effects. Some studies consider the hidden confounders are designed for static environment and not easily adaptable to a dynamic setting. In fact, most observational data (e.g., electronic medical records) is naturally dynamic and consists of sequential information. In this paper, we propose Deep Sequential Weighting (DSW) for estimating I…
Identifying causal treatment (or exposure) effects in observational studies requires the data to satisfy the unconfoundedness assumption which is not testable using the observed data. With sensitivity analysis, one can determine how the conclusions might change if assumptions are violated to a certain degree. In this paper, we propose a new technique for sensitivity analysis applicable to clusters observational data with a normally distributed or binary outcome. The proposed methods aim to assess the robustness of estimated treatment effects in a single study as well as in multiple studies, i.…
Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups with respect to possible confounders, which makes the analysis straightforward. However, when analysing observational data, the potential for unmeasured confounding makes comparing treatment effects much more challenging. Causal inference methods such as Instrumental Variable and Prior Even Rate Ratio approaches make it possible to circumvent the need to ad…
The era of big data has witnessed an increasing availability of multiple data sources for statistical analyses. We consider estimation of causal effects combining big main data with unmeasured confounders and smaller validation data with supplementary information on these confounders. Under the unconfoundedness assumption with completely observed confounders, the smaller validation data allow for constructing consistent estimators for causal effects, but the big main data can only give error-prone estimators in general. However, by leveraging the information in the big main data in a principle…
Propensity score weighting is a tool for causal inference to adjust for measured confounders in observational studies. In practice, data often present complex structures, such as clustering, which make propensity score modeling and estimation challenging. In addition, for clustered data, there may be unmeasured cluster-specific variables that are related to both the treatment assignment and the outcome. When such unmeasured cluster-specific confounders exist and are omitted in the propensity score model, the subsequent propensity score adjustment may be biased. In this article, we propose a ca…
It is important to draw causal inference from observational studies, which, however, becomes challenging if the confounders have missing values. Generally, causal effects are not identifiable if the confounders are missing not at random. We propose a novel framework to nonparametrically identify causal effects with confounders subject to an outcome-independent missingness, that is, the missing data mechanism is independent of the outcome, given the treatment and possibly missing confounders. We then propose a nonparametric two-stage least squares estimator and a parametric estimator for causal…
Many scientific questions require estimating the effects of continuous treatments. Outcome modeling and weighted regression based on the generalized propensity score are the most commonly used methods to evaluate continuous effects. However, these techniques may be sensitive to model misspecification, extreme weights or both. In this paper, we propose Kernel Optimal Orthogonality Weighting (KOOW), a convex optimization-based method, for estimating the effects of continuous treatments. KOOW finds weights that minimize the worst-case penalized functional covariance between the continuous treatme…
Causal inference in observational studies can be challenging when confounders are subject to missingness. Generally, the identification of causal effects is not guaranteed even under restrictive parametric model assumptions when confounders are missing not at random. To address this, We propose a general framework to establish the identification of causal effects when confounders are subject to treatment-independent missingness, which means that the missing data mechanism is independent of the treatment, given the outcome and possibly missing confounders. We give special consideration to commo…
Estimating average causal effect (ACE) is useful whenever we want to know the effect of an intervention on a given outcome. In the absence of a randomized experiment, many methods such as stratification and inverse propensity weighting have been proposed to estimate ACE. However, it is hard to know which method is optimal for a given dataset or which hyperparameters to use for a chosen method. To this end, we provide a framework to characterize the loss of a causal inference method against the true ACE, by framing causal inference as a representation learning problem. We show that many popular…
Marginal structural models (MSMs) estimate the causal effect of a time-varying treatment in the presence of time-dependent confounding via weighted regression. The standard approach of using inverse probability of treatment weighting (IPTW) can lead to high-variance estimates due to extreme weights and be sensitive to model misspecification. Various methods have been proposed to partially address this, including truncation and stabilized-IPTW to temper extreme weights and covariate balancing propensity score (CBPS) to address treatment model misspecification. In this paper, we present Kernel O…
Assessing treatment effects in observational data with missing confounders: A comparative study of practical doubly-robust and traditional missing data methods
Kaiser Permanente Washington Health Research Institute（凯撒永久华盛顿健康研究所）；University of Washington（华盛顿大学）；TL Revolution, LLC（TL革命有限责任公司）；Vanderbilt University（范德堡大学）；University of California at Berkeley（加州大学伯克利分校）；University of Auckland（奥克兰大学）；US Food and Drug Administration（美国食品药品监督管理局）
Confounding seriously impairs our ability to learn about causal relations from observational data. Confounding can be defined as a statistical association between two variables due to inputs from a common source (the confounder). For example, if $Z\rightarrow Y$ and $Z\rightarrow X$, then $X$ and $Y$ will be statistically dependent, even if there are no causal connections between the two. There are several approaches available to adjust for confounding, i.e. to remove, or reduce, the association between two variables due to the confounder. Common adjustment techniques include stratifying the a…
Estimating marginal treatment effects from observational studies and indirect treatment comparisons: When are standardization-based methods preferable to those based on propensity score weighting?
Background/aims: While randomized controlled trials are the gold standard for measuring causal effects, robust conclusions about causal relationships can be obtained using data from observational studies if proper statistical techniques are used to account for the imbalance of pretreatment confounders across groups. Propensity score (PS) and balance weighting are useful techniques that aim to reduce the observed imbalances between treatment groups by weighting the groups to be as similar as possible with respect to observed confounders. Methods: We have created CoBWeb, a free and easy-to-use w…
The fundamental problem in treatment effect estimation from observational data is confounder identification and balancing. Most of the previous methods realized confounder balancing by treating all observed pre-treatment variables as confounders, ignoring further identifying confounders and non-confounders. In general, not all the observed pre-treatment variables are confounders that refer to the common causes of the treatment and the outcome, some variables only contribute to the treatment and some only contribute to the outcome. Balancing those non-confounders, including instrumental variabl…
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…
Matched observational studies are commonly used to study treatment effects in non-randomized data. After matching for observed confounders, there could remain bias from unobserved confounders. A standard way to address this problem is to do a sensitivity analysis. A sensitivity analysis asks how sensitive the result is to a hypothesized unmeasured confounder U. One method, known as simultaneous sensitivity analysis, has two sensitivity parameters: one relating U to treatment assignment and the other to response. This method assumes that in each matched set, U is distributed to make the bias wo…
Inferring the causal effect of a treatment on an outcome in an observational study requires adjusting for observed baseline confounders to avoid bias. However, adjusting for all observed baseline covariates, when only a subset are confounders of the effect of interest, is known to yield potentially inefficient and unstable estimators of the treatment effect. Furthermore, it raises the risk of finite-sample bias and bias due to model misspecification. For these stated reasons, confounder (or covariate) selection is commonly used to determine a subset of the available covariates that is sufficie…
Causal Effect Estimation using identifiable Variational AutoEncoder with Latent Confounders and Post-Treatment Variables
College of Informatics, Huazhong Agricultural University（华中农业大学信息学院）；Data 61, Commonwealth Scientific and Industrial Research Organisation (CSIRO)（联邦科学与工业研究组织数据61分部）；STEM, University of South Australia（南澳大学 STEM 学院）
Using observational data to estimate the effect of a treatment is a powerful tool for decision-making when randomized experiments are infeasible or costly. However, observational data often yields biased estimates of treatment effects, since treatment assignment can be confounded by unobserved variables. A remedy is offered by deconfounding methods that adjust for such unobserved confounders. In this paper, we develop the Sequential Deconfounder, a method that enables estimating individualized treatment effects over time in presence of unobserved confounders. This is the first deconfounding me…
Conditioning on some set of confounders that causally affect both treatment and outcome variables can be sufficient for eliminating bias introduced by all such confounders when estimating causal effect of the treatment on the outcome from observational data. It is done by including them in propensity score model in so-called potential outcome framework for causal inference whereas in causal graphical modeling framework usual conditioning on them is done. However in the former framework, it is confusing when modeler finds a variable that is non-causally associated with both the treatment and th…
Just what the doctor ordered: An evaluation of provider preference-based Instrumental Variable methods in observational studies, with application for comparative effectiveness of type 2 diabetes therapy
Observational studies are often used to understand relationships between exposures and outcomes. They do not, however, allow conclusions about causal relationships to be drawn unless statistical techniques are used to account for the imbalance of confounders across exposure groups. Propensity score and balance weighting (PSBW) are useful techniques that aim to reduce the imbalances between exposure groups by weighting the groups to look alike on the observed confounders. Despite the plethora of available methods to estimate PSBW, there is little guidance on what one defines as adequate balance…
The problem of missingness in observational data is ubiquitous. When the confounders are missing at random, multiple imputation is commonly used; however, the method requires congeniality conditions for valid inferences, which may not be satisfied when estimating average causal treatment effects. Alternatively, fractional imputation, proposed by Kim 2011, has been implemented to handling missing values in regression context. In this article, we develop fractional imputation methods for estimating the average treatment effects with confounders missing at random. We show that the fractional impu…
University of California, Los Angeles（加州大学洛杉矶分校）；University of Oxford（牛津大学）；University at Buffalo（布法罗大学）
Estimating the individual treatment effect (ITE) from observational data is essential in medicine. A central challenge in estimating the ITE is handling confounders, which are factors that affect both an intervention and its outcome. Most previous work relies on the unconfoundedness assumption, which posits that all the confounders are measured in the observational data. However, if there are unmeasurable (latent) confounders, then confounding bias is introduced. Fortunately, noisy proxies for the latent confounders are often available and can be used to make an unbiased estimate of the ITE. I…
The idea of "stratified medicine" is an important driver of methodological research on the identification of predictive biomarkers. Most methods proposed so far for this purpose have been developed for the use on randomized data only. However, especially for rare cancers, data from clinical registries or observational studies might be the only available data source. For such data, methods for an unbiased estimation of the average treatment effect are well established. Research on confounder adjustment when investigating the heterogeneity of treatment effects and the variables responsible for t…
No unmeasured confounding is often assumed in estimating treatment effects in observational data when using approaches such as propensity scores and inverse probability weighting. However, in many such studies due to the limitation of the databases, collected confounders are not exhaustive, and it is crucial to examine the extent to which the resulting estimate is sensitive to the unmeasured confounders. We consider this problem for survival and competing risks data. Due to the complexity of models for such data, we adapt the simulated potential confounders approach of Carnegie et al. (2016), …
Arizona State University（亚利桑那州立大学）；University of Virginia（弗吉尼亚大学）
Counterfactual evaluation of novel treatment assignment functions (e.g., advertising algorithms and recommender systems) is one of the most crucial causal inference problems for practitioners. Traditionally, randomized controlled trials (A/B tests) are performed to evaluate treatment assignment functions. However, such trials can be time-consuming, expensive, and even unethical in some cases. Therefore, offline counterfactual evaluation of treatment assignment functions becomes a pressing issue because a massive amount of observational data is available in today's big data era. Counterfactual …
Estimating causal effects of continuous-time dynamic treatments with unmeasured confounders
Key Laboratory of Advanced Theory and Application in Statistics and Data Science -MOE, School of Statistics, East China Normal University（华东师范大学统计学院统计学与数据科学前沿理论及应用教育部重点实验室）
Estimating treatment effects plays a crucial role in causal inference, having many real-world applications like policy analysis and decision making. Nevertheless, estimating treatment effects in the longitudinal setting in the presence of hidden confounders remains an extremely challenging problem. Recently, there is a growing body of work attempting to obtain unbiased ITE estimates from time-dynamic observational data by ignoring the possible existence of hidden confounders. Additionally, many existing works handling hidden confounders are not applicable for continuous-time settings. In this …
Reconciling Overt Bias and Hidden Bias in Sensitivity Analysis for Matched Observational Studies
Department of Biostatistics, New York University（纽约大学生物统计系）；School of Economics, Nankai University（南开大学经济学院）；Data Science Institute, The University of Chicago（芝加哥大学数据科学研究所）
Weighting estimators based on propensity scores are widely used for causal estimation in a variety of contexts, such as observational studies, marginal structural models and interference. They enjoy appealing theoretical properties such as consistency and possible efficiency under correct model specification. However, this theoretical appeal may be diminished in practice by sensitivity to misspecification of the propensity score model. To improve on this, we borrow an idea from an alternative approach to causal effect estimation in observational studies, namely subclassification estimators. It…
Inverse probability of treatment weighting (IPTW) is a popular propensity score (PS)-based approach to estimate causal effects in observational studies at risk of confounding bias. A major issue when estimating the PS is the presence of partially observed covariates. Multiple imputation (MI) is a natural approach to handle missing data on covariates, but its use in the PS context raises three important questions: (i) should we apply Rubin's rules to the IPTW treatment effect estimates or to the PS estimates themselves? (ii) does the outcome have to be included in the imputation model? (iii) ho…
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…
Unobserved confounding is one of the main challenges when estimating causal effects. We propose a causal reduction method that, given a causal model, replaces an arbitrary number of possibly high-dimensional latent confounders with a single latent confounder that takes values in the same space as the treatment variable, without changing the observational and interventional distributions the causal model entails. This allows us to estimate the causal effect in a principled way from combined data without relying on the common but often unrealistic assumption that all confounders have been observ…
This paper discusses an application of Shapley values in the causal inference field, specifically on how to select the top confounder variables for coarsened exact matching method in a scalable way. We use a dataset from an observational experiment involving LinkedIn members as a use case to test its applicability, and show that Shapley values are highly informational and can be leveraged for its robust importance-ranking capability.
