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 weighting (OW) for covariate adjustment. The OW method has a unique advantage of completely removing chance imbalance when the propensity score is estimated by logistic regression. We show that the OW estimator attains the same semiparametric variance lower bound as the most efficient ANCOVA estimator and the IPW estimator for a continuous outcome, and derive closed-form variance estimators for OW when estimating additive and ratio estimands. Through extensive simulations, we demonstrate OW consistently outperforms IPW in finite samples and improves the efficiency over ANCOVA and augmented IPW when the degree of treatment effect heterogeneity is moderate or when the outcome model is incorrectly specified. We apply the proposed OW estimator to the Best Apnea Interventions for Research (BestAIR) randomized trial to evaluate the effect of continuous positive airway pressure on patient health outcomes. All the discussed propensity score weighting methods are implemented in the R package PSweight.
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…
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…
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…
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?
We study Bayesian approaches to causal inference via propensity score regression. Much of the Bayesian literature on propensity score methods have relied on approaches that cannot be viewed as fully Bayesian in the context of conventional `likelihood times prior' posterior inference; in addition, most methods rely on parametric and distributional assumptions, and presumed correct specification. We emphasize that causal inference is typically carried out in settings of mis-specification, and develop strategies for fully Bayesian inference that reflect this. We focus on methods based on decision…
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…
Covariate balance is crucial for unconfounded descriptive or causal comparisons. However, lack of balance is common in observational studies. This article considers weighting strategies for balancing covariates. We define a general class of weights---the balancing weights---that balance the weighted distributions of the covariates between treatment groups. These weights incorporate the propensity score to weight each group to an analyst-selected target population. This class unifies existing weighting methods, including commonly used weights such as inverse-probability weights as special cases…
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…
Covariate balance is crucial for unconfounded descriptive or causal comparisons. However, lack of balance is common in observational studies. This article considers weighting strategies for balancing covariates. We define a general class of weights---the balancing weights---that balance the weighted distributions of the covariates between treatment groups. These weights incorporate the propensity score to weight each group to an analyst-selected target population. This class unifies existing weighting methods, including commonly used weights such as inverse-probability weights as special cases…
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…
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…
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…
Propensity score matching is a common tool for adjusting for observed confounding in observational studies, but is known to have limitations in the presence of unmeasured confounding. In many settings, researchers are confronted with spatially-indexed data where the relative locations of the observational units may serve as a useful proxy for unmeasured confounding that varies according to a spatial pattern. We develop a new method, termed Distance Adjusted Propensity Score Matching (DAPSm) that incorporates information on units' spatial proximity into a propensity score matching procedure. We…
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 betw…
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…
The propensity score analysis is one of the most widely used methods for studying the causal treatment effect in observational studies. This paper studies treatment effect estimation with the method of matching weights. This method resembles propensity score matching but offers a number of new features including efficient estimation, rigorous variance calculation, simple asymptotics, statistical tests of balance, clearly identified target population with optimal sampling property, and no need for choosing matching algorithm and caliper size. In addition, we propose the mirror histogram as a us…
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 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…
Propensity score methods are common for estimating a binary treatment effect when treatment assignment is not randomized. When exposure is measured on an ordinal scale (i.e., low - medium - high), however, propensity score inference requires extensions which have received limited attention. Estimands of possible interest with an ordinal exposure are the average treatment effects between each pair of exposure levels. Using these estimands, it is possible to determine an optimal exposure level. Traditional methods, including dichotomization of the exposure or a series of binary propensity score …
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.…
The Consent-to-Contact (C2C) registry at the University of California, Irvine collects data from community participants to aid in the recruitment to clinical research studies. Self-selection into the C2C likely leads to bias due in part to enrollees having more years of education relative to the US general population. Salazar et al. (2020) recently used the C2C to examine associations of race/ethnicity with participant willingness to be contacted about research studies. To address questions about generalizability of estimated associations we estimate propensity for self-selection into the conv…
Use of historical control data to augment a small internal control arm in a randomized control trial (RCT) can lead to significant improvement of the efficiency of the trial. It introduces the risk of potential bias, since the historical control population is often rather different from the RCT. Power prior approaches have been introduced to discount the historical data to mitigate the impact of the population difference. However, even with a Bayesian dynamic borrowing which can discount the historical data based on the outcome similarity of the two populations, a considerable population diffe…
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 …
Adjustment for Unmeasured Spatial Confounding in Settings of Continuous Exposure Conditional on the Binary Exposure Status: Conditional Generalized Propensity Score-Based Spatial Matching
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…
The pervasive use of prevalent cohort studies on disease duration, increasingly calls for appropriate methodologies to account for the biases that invariably accompany samples formed by such data. It is well-known, for example, that subjects with shorter lifetime are less likely to be present in such studies. Moreover, certain covariate values could be preferentially selected into the sample, being linked to the long-term survivors. The existing methodology for estimation of the propensity score using data collected on prevalent cases requires the correct conditional survival/hazard function g…
We investigate the estimation of subgroup treatment effects with observational data. Existing propensity score matching and weighting methods are mostly developed for estimating overall treatment effect. Although the true propensity score should balance covariates for the subgroup populations, the estimated propensity score may not balance covariates for the subgroup samples. We propose the subgroup balancing propensity score (SBPS) method, which selects, for each subgroup, to use either the overall sample or the subgroup sample to estimate propensity scores for units within that subgroup, in …
Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account for chance imbalances between treatment arms and increase statistical efficiency in individually-randomized trials, analytical methods for individual-level covariate adjustment in small CRTs have received little attention to date. In this paper, we systematically investigate, through extensive simulations, the operating characteristics of propensity score weig…
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…
In most real-world systems units are interconnected and can be represented as networks consisting of nodes and edges. For instance, in social systems individuals can have social ties, family or financial relationships. In settings where some units are exposed to a treatment and its effect spills over connected units, estimating both the direct effect of the treatment and spillover effects presents several challenges. First, assumptions on the way and the extent to which spillover effects occur along the observed network are required. Second, in observational studies, where the treatment assign…
