Propensity score (PS) weighting methods are often used in non-randomized studies to adjust for confounding and assess treatment effects. The most popular among them, the inverse probability weighting (IPW), assigns weights that are proportional to the inverse of the conditional probability of a specific treatment assignment, given observed covariates. A key requirement for IPW estimation is the positivity assumption, i.e., the PS must be bounded away from 0 and 1. In practice, violations of the positivity assumption often manifest by the presence of limited overlap in the PS distributions between treatment groups. When these practical violations occur, a small number of highly influential IPW weights may lead to unstable IPW estimators, with biased estimates and large variances. To mitigate these issues, a number of alternative methods have been proposed, including IPW trimming, overlap weights (OW), matching weights (MW), and entropy weights (EW). Because OW, MW, and EW target the population for whom there is equipoise (and with adequate overlap) and their estimands depend on the true PS, a common criticism is that these estimators may be more sensitive to misspecifications of the PS model. In this paper, we conduct extensive simulation studies to compare the performances of IPW and IPW trimming against those of OW, MW, and EW under limited overlap and misspecified propensity score models. Across the wide range of scenarios we considered, OW, MW, and EW consistently outperform IPW in terms of bias, root mean squared error, and coverage probability.
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…
Background: Subgroup analyses are frequently conducted in randomized clinical trials to assess evidence of heterogeneous treatment effect across patient subpopulations. Although randomization balances covariates within subgroups in expectation, chance imbalance may be amplified in small subgroups and harm the precision. Covariate adjustment in overall analysis of RCT is often conducted, via either ANCOVA or propensity score weighting, but for subgroup analysis has been rarely discussed. In this article, we develop propensity score weighting methodology for covariate adjustment to improve the p…
Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes constructs pseudo-observations and allows standard methods such as propensity score weighting to proceed as if the outcomes are completely observed. For a general class of model-free causal estimands with survival outcomes on user-specified target populations, we develop corresponding propensity score weighting estimators based on the pseudo-observations and es…
Estimating marginal treatment effects from observational studies and indirect treatment comparisons: When are standardization-based methods preferable to those based on propensity score weighting?
Chance imbalance in baseline characteristics is common in randomized clinical trials. Regression adjustment such as the analysis of covariance (ANCOVA) is often used to account for imbalance and increase precision of the treatment effect estimate. An objective alternative is through inverse probability weighting (IPW) of the propensity scores. Although IPW and ANCOVA are asymptotically equivalent, the former may demonstrate inferior performance in finite samples. In this article, we point out that IPW is a special case of the general class of balancing weights, and advocate to use overlap weig…
Causal or unconfounded descriptive comparisons between multiple groups are common in observational studies. Motivated from a racial disparity study in health services research, we propose a unified propensity score weighting framework, the balancing weights, for estimating causal effects with multiple treatments. These weights incorporate the generalized propensity scores to balance the weighted covariate distribution of each treatment group, all weighted toward a common pre-specified target population. The class of balancing weights include several existing approaches such as the inverse prob…
Propensity Score Weighting to Ensure Balance in Key Subgroups or Strata: A Practical Guide
IQVIA；Institute of Health Policy, Management and Evaluation, University of Toronto（多伦多大学健康政策、管理与评估研究所）；Li Ka Shing Knowledge Institute, St. Michael’s Hospital, Unity Health Toronto（多伦多联合医疗圣迈克尔医院李嘉诚知识研究院）；Department of Medicine, University of Toronto（多伦多大学医学系）
A common goal in comparative effectiveness research is to estimate treatment effects on pre-specified subpopulations of patients. Though widely used in medical research, causal inference methods for such subgroup analysis remain underdeveloped, particularly in observational studies. In this article, we develop a suite of analytical methods and visualization tools for causal subgroup analysis. First, we introduce the estimand of subgroup weighted average treatment effect and provide the corresponding propensity score weighting estimator. We show that balancing covariates within a subgroup bound…
The inverse probability weighting (IPW) is broadly utilized to address missing data problems including causal inference but may suffer from large variances and biases due to propensity score model misspecification. To solve these problems, I propose an estimation method called the navigated weighting (NAWT), which utilizes estimating equations suitable for a specific pre-specified parameter of interest (e.g., the average treatment effects on the treated). Since these pre-specified parameters determine the relative importance of each unit as a function of propensity scores, the NAWT prioritizes…
The inverse probability weighting approach is popular for evaluating treatment effects in observational studies, but extreme propensity scores could bias the estimator and induce excessive variance. Recently, the overlap weighting approach has been proposed to alleviate this problem, which smoothly down-weighs the subjects with extreme propensity scores. Although advantages of overlap weighting have been extensively demonstrated in literature with continuous and binary outcomes, research on its performance with time-to-event or survival outcomes is limited. In this article, we propose two weig…
Application of Propensity Score Models and Causal Estimators in Observational Studies under Model Misspecification
University of Nebraska Medical Center（内布拉斯加医学中心大学）；Medical College of Wisconsin（威斯康星医学院）；University of Kansas Medical Center（堪萨斯医学中心大学）；Daffodil International University（达芬奇国际大学）
Propensity score weighting is an important tool for comparative effectiveness research.Besides the inverse probability of treatment weights (IPW), recent development has introduced a general class of balancing weights, corresponding to alternative target populations and estimands. In particular, the overlap weights (OW) lead to optimal covariate balance and estimation efficiency, and a target population of scientific and policy interest. We develop the R package PSweight to provide a comprehensive design and analysis platform for causal inference based on propensity score weighting. PSweight s…
Propensity score plays a central role in causal inference, but its use is not limited to causal comparisons. As a covariate balancing tool, propensity score can be used for controlled descriptive comparisons between groups whose memberships are not manipulable. A prominent example is racial disparities in health care. However, conceptual confusion and hesitation persists for using propensity score in racial disparities studies. In this commentary, we argue that propensity score, possibly combined with other methods, is an effective tool for racial disparities analysis. We describe relevant est…
Propensity score methods are increasingly being used to reduce estimation bias of treatment effects for observational studies. Previous research has shown that propensity score methods consistently estimate the marginal hazard ratio for time to event data. However, recurrent data frequently arise in the biomedical literature and there is a paucity of research into the use of propensity score methods when data are recurrent in nature. The objective of this paper is to extend the existing propensity score methods to recurrent data setting. We illustrate our methods through a series of Monte Carl…
Many epidemiologic studies forgo probability sampling and turn to nonprobability volunteer-based samples because of cost, response burden, and invasiveness of biological samples. However, finite population inference is difficult to make from the nonprobability samples due to the lack of population representativeness. Aiming for making inferences at the population level using nonprobability samples, various inverse propensity score weighting (IPSW) methods have been studied with the propensity defined by the participation rate of population units in the nonprobability sample. In this paper, we …
Propensity score weighting is widely used to improve the representativeness and correct the selection bias in the voluntary sample. The propensity score is often developed using a model for the sampling probability, which can be subject to model misspecification. In this paper, we consider an alternative approach of estimating the inverse of the propensity scores using the density ratio function satisfying the self-efficiency condition. The smoothed density ratio function is obtained by the solution to the information projection onto the space satisfying the moment conditions on the balancing …
In this paper, we propose a propensity score adapted variable selection procedure to select covariates for inclusion in propensity score models, in order to eliminate confounding bias and improve statistical efficiency in observational studies. Our variable selection approach is specially designed for causal inference, it only requires the propensity scores to be $\sqrt{n}$-consistently estimated through a parametric model and need not correct specification of potential outcome models. By using estimated propensity scores as inverse probability treatment weights in performing an adaptive lasso…
Most epidemiologic cohorts are composed of volunteers who do not represent the general population. To enable population inference from cohorts, we and others have proposed utilizing probability survey samples as external references to develop a propensity score (PS) for membership in the cohort versus survey. Herein we develop a unified framework for PS-based weighting (such as inverse PS weighting (IPSW)) and matching methods (such as kernel-weighting (KW) method). We identify a fundamental Strong Exchangeability Assumption (SEA) underlying existing PS-based matching methods whose failure inv…
The idea of covariate balance is at the core of causal inference. Inverse propensity weights play a central role because they are the unique set of weights that balance the covariate distributions of different treatment groups. We discuss two broad approaches to estimating these weights: the more traditional one, which fits a propensity score model and then uses the reciprocal of the estimated propensity score to construct weights, and the balancing approach, which estimates the inverse propensity weights essentially by the method of moments, finding weights that achieve balance in the sample.…
Propensity score matching has been a long-standing tradition for handling confounding in causal inference, however requiring stringent model assumptions. In this article, we propose double score matching(DSM) for general causal estimands utilizing two balancing scores including the propensity score and prognostic score. To gain the protection of possible model misspecification, we posit multiple candidate models for each score. We show that the de-biasing DSM estimator achieves the multiple robustness property in that it is consistent for the true causal estimand if any model of the propensity…
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…
Propensity score (PS) methods have been increasingly used in recent years when assessing treatment effects in nonrandomized studies. In terms of statistical methods, a number of new PS weighting methods were developed, and it was shown that they can outperform PS matching in efficiency of treatment effect estimation in different simulation settings. For assessing balance of covariates in treatment groups, PS weighting methods commonly use the weighted standardized difference, despite some deficiencies of this measure like, for example, the distribution of the weighted standardized difference d…
Nonresponse weighting adjustment using propensity score is a popular method for handling unit nonresponse. However, including all available auxiliary variables into the propensity model can lead to inefficient and inconsistent estimation, especially with high-dimensional covariates. In this paper, a new Bayesian method using the Spike-and-Slab prior is proposed for sparse propensity score estimation. The proposed method is not based on any model assumption on the outcome variable and is computationally efficient. Instead of doing model selection and parameter estimation separately as in many f…
Inverse probability of treatment weighting (IPTW) is a popular method for estimating the average treatment effect (ATE). However, empirical studies show that the IPTW estimators can be sensitive to the misspecification of the propensity score model. To address this problem, researchers have proposed to estimate propensity score by directly optimizing the balance of pre-treatment covariates. While these methods appear to empirically perform well, little is known about how the choice of balancing conditions affects their theoretical properties. To fill this gap, we first characterize the asympto…
In the causal inference literature an estimator belonging to a class of semi-parametric estimators is called robust if it has desirable properties under the assumption that at least one of the working models is correctly specified. In this paper we propose a crude analytical approach to study the large sample bias of semi-parameteric estimators of the average causal effect when all working models are misspecified. We apply our approach to three prototypical estimators, two inverse probability weighting (IPW) estimators, using a misspecified propensity score model, and a doubly robust (DR) esti…
