arXiv subjects

Luke Keele

Publications and source records attributed to Luke Keele.

At least 19 recordsLinked to original sources

arXiv2026

A Weighting Framework for Clusters as Confounders in Observational Studies

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.

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arXiv2025

Forest Kernel Balancing Weights: Outcome-Guided Features for Causal Inference

While balancing covariates between groups is central for observational causal inference, selecting which features to balance remains a challenging problem. Kernel balancing is a promising approach that first estimates a kernel that captures similarity across units and then balances a (possibly low-dimensional) summary of that kernel, indirectly learning important features to balance. In this paper, we propose forest kernel balancing, which leverages the underappreciated fact that tree-based machine learning models, namely random forests and Bayesian additive regression trees (BART), implicitly estimate a kernel based on the co-occurrence of observations in the same terminal leaf node. Thus, even though the resulting kernel is solely a function of baseline features, the selected nonlinearities and other interactions are important for predicting the outcome -- and therefore are important for addressing confounding. Through simulations and applied illustrations, we show that forest kernel balancing leads to meaningful computational and statistical improvement relative to standard kernel methods, which do not incorporate outcome information when learning features.

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arXiv2025

Sensitivity Analysis for Clustered Observational Studies with an Application to the Effectiveness of Magnet Nursing Hospitals

In a clustered observational study, treatment is assigned to groups and all units within the group are exposed to the treatment. Here, we use a clustered observational study (COS) design to estimate the effectiveness of Magnet Nursing certificates for emergency surgery patients. Recent research has introduced specialized weighting estimators for the COS design that balance baseline covariates at the unit and cluster level. These methods allow researchers to adjust for observed confounders, but are sensitive to unobserved confounding. In this paper, we develop new sensitivity analysis methods tailored to weighting estimators for COS designs. We provide several key contributions. First, we introduce a key bias decomposition, tailored to the specific confounding structure that arises in a COS. Second, we develop a sensitivity framework for weighted COS designs that constrain the error in the underlying weights. We introduce both a marginal sensitivity model and a variance-based sensitivity model, and extend both to accommodate multiple estimands. Finally, we propose amplification and benchmarking methods to better interpret the results. Throughout, we illustrate our proposed methods by analyzing the effectiveness of Magnet nursing hospitals.

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arXiv2025

Nonparametric Estimation of Local Treatment Effects with Continuous Instruments

Instrumental variable methods are widely used to address unmeasured confounding, yet much of the existing literature has focused on the binary instrument setting. Extensions to continuous instruments often impose strong parametric assumptions for identification and estimation, which can be difficult to justify and may limit their applicability in complex real-world settings. In this work, we develop theory and methods for nonparametric estimation of treatment effects with a continuous instrumental variable. We introduce an estimand that, under a monotonicity assumption, quantifies the treatment effect among the maximal complier class, generalizing the local average treatment effect framework to continuous instruments. Considering this estimand and the local instrumental variable curve, we draw connections to the dose-response function and its derivative, and propose doubly robust estimation methods. We establish convergence rates and conditions for asymptotic normality, providing valuable insights into the role of nuisance function estimation when the instrument is continuous. Additionally, we present practical procedures for bandwidth selection and variance estimation. Through extensive simulations, we demonstrate the advantages of the proposed nonparametric estimators. Finally, we apply our methods to data where excess travel time is an instrument for patients' likelihood of receiving care at specialized health care facilities. We use this instrument to estimate the effect of delivering at low-quality neonatal intensive care units (NICUs) on infant mortality.

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arXiv2024

Local Effects of Continuous Instruments without Positivity

Instrumental variables are a popular study design for the estimation of treatment effects in the presence of unobserved confounders. In the canonical instrumental variables design, the instrument is a binary variable. In many settings, however, the instrument is continuous. Standard estimation methods can be applied with continuous instruments, but they require strong assumptions. While recent work has introduced more flexible estimation approaches, these methods require a positivity assumption that is implausible in many applications. We derive a novel family of causal estimands using stochastic dynamic interventions that allows a range of intervention distributions that are continuous with respect to the observed distribution of the instrument. These estimands focus on a specific local effect but do not require a positivity assumption. Next, we develop doubly robust estimators for these estimands that allow for estimation of the nuisance functions via nonparametric estimators. We use empirical process theory and sample splitting to derive asymptotic properties of the proposed estimators under weak conditions. In addition, we derive methods for profiling the principal strata as well as a method of sensitivity analysis. We evaluate our methods via simulation and demonstrate their feasibility using an application on the effectiveness of surgery for specific emergency conditions.

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arXiv2024

Nonparametric identification and efficient estimation of causal effects with instrumental variables

Instrumental variables are widely used in econometrics and epidemiology for identifying and estimating causal effects when an exposure of interest is confounded by unmeasured factors. Despite this popularity, the assumptions invoked to justify the use of instruments differ substantially across the literature. Similarly, statistical approaches for estimating the resulting causal quantities vary considerably, and often rely on strong parametric assumptions. In this work, we compile and organize structural conditions that nonparametrically identify conditional average treatment effects, average treatment effects among the treated, and local average treatment effects, with a focus on identification formulae invoking the conditional Wald estimand. Moreover, we build upon existing work and propose nonparametric efficient estimators of functionals corresponding to marginal and conditional causal contrasts resulting from the various identification paradigms. We illustrate the proposed methods on an observational study examining the effects of operative care on adverse events for cholecystitis patients, and a randomized trial assessing the effects of market participation on political views.

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arXiv2023

Flexibly Estimating and Interpreting Heterogeneous Treatment Effects of Laparoscopic Surgery for Cholecystitis Patients

Laparoscopic surgery has been shown through a number of randomized trials to be an effective form of treatment for cholecystitis. Given this evidence, one natural question for clinical practice is: does the effectiveness of laparoscopic surgery vary among patients? It might be the case that, while the overall effect is positive, some patients treated with laparoscopic surgery may respond positively to the intervention while others do not or may be harmed. In our study, we focus on conditional average treatment effects to understand whether treatment effects vary systematically with patient characteristics. Recent methodological work has developed a meta-learner framework for flexible estimation of conditional causal effects. In this framework, nonparametric estimation methods can be used to avoid bias from model misspecification while preserving statistical efficiency. In addition, researchers can flexibly and effectively explore whether treatment effects vary with a large number of possible effect modifiers. However, these methods have certain limitations. For example, conducting inference can be challenging if black-box models are used. Further, interpreting and visualizing the effect estimates can be difficult when there are multi-valued effect modifiers. In this paper, we develop new methods that allow for interpretable results and inference from the meta-learner framework for heterogeneous treatment effects estimation. We also demonstrate methods that allow for an exploratory analysis to identify possible effect modifiers. We apply our methods to a large database for the use of laparoscopic surgery in treating cholecystitis. We also conduct a series of simulation studies to understand the relative performance of the methods we develop. Our study provides key guidelines for the interpretation of conditional causal effects from the meta-learner framework.

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arXiv2023

Doubly robust machine learning for an instrumental variable study of surgical care for cholecystitis

Comparative effectiveness research frequently employs the instrumental variable design since randomized trials can be infeasible for many reasons. In this study, we investigate and compare treatments for emergency cholecystitis -- inflammation of the gallbladder. A standard treatment for cholecystitis is surgical removal of the gallbladder, while alternative non-surgical treatments include managed care and pharmaceutical options. As randomized trials are judged to violate the principle of equipoise, we consider an instrument for operative care: the surgeon's tendency to operate. Standard instrumental variable estimation methods, however, often rely on parametric models that are prone to bias from model misspecification. We outline instrumental variable estimation methods based on the doubly robust machine learning framework. These methods enable us to employ various machine learning techniques for nuisance parameter estimation and deliver consistent estimates and fast rates of convergence for valid inference. We use these methods to estimate the primary target causal estimand in an IV design. Additionally, we expand these methods to develop estimators for heterogeneous causal effects, profiling principal strata, and a sensitivity analyses for a key instrumental variable assumption. We conduct a simulation study to demonstrate scenarios where more flexible estimation methods outperform standard methods. Our findings indicate that operative care is generally more effective for cholecystitis patients, although the benefits of surgery can be less pronounced for key patient subgroups.

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arXiv2023

Improving instrumental variable estimators with post-stratification

Experiments studying get-out-the-vote (GOTV) efforts estimate the causal effect of various mobilization efforts on voter turnout. However, there is often substantial noncompliance in these studies. A usual approach is to use an instrumental variable (IV) analysis to estimate impacts for compliers, here being those actually contacted by the investigators. Unfortunately, popular IV estimators can be unstable in studies with a small fraction of compliers. We explore post-stratifying the data (e.g., taking a weighted average of IV estimates within each stratum) using variables that predict complier status (and, potentially, the outcome) to mitigate this. We present the benefits of post-stratification in terms of bias, variance, and improved standard error estimates, and provide a finite-sample asymptotic variance formula. We also compare the performance of different IV approaches and discuss the advantages of our design-based post-stratification approach over incorporating compliance-predictive covariates into the two-stage least squares estimator. In the end, we show that covariates predictive of compliance can increase precision, but only if one is willing to make a bias-variance trade-off by down-weighting or dropping strata with few compliers. By contrast, standard approaches such as two-stage least squares fail to use such information. We finally examine the benefits of our approach in two GOTV applications.

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arXiv2023

A Guide to Regression Discontinuity Designs in Medical Applications

We present a practical guide for the analysis of regression discontinuity (RD) designs in biomedical contexts. We begin by introducing key concepts, assumptions, and estimands within both the continuity-based framework and the local randomization framework. We then discuss modern estimation and inference methods within both frameworks, including approaches for bandwidth or local neighborhood selection, optimal treatment effect point estimation, and robust bias-corrected inference methods for uncertainty quantification. We also overview empirical falsification tests that can be used to support key assumptions. Our discussion focuses on two particular features that are relevant in biomedical research: (i) fuzzy RD designs, which often arise when therapeutic treatments are based on clinical guidelines but patients with scores near the cutoff are treated contrary to the assignment rule; and (ii) RD designs with discrete scores, which are ubiquitous in biomedical applications. We illustrate our discussion with three empirical applications: the effect of CD4 guidelines for anti-retroviral therapy on retention of HIV patients in South Africa, the effect of genetic guidelines for chemotherapy on breast cancer recurrence in the United States, and the effects of age-based patient cost-sharing on healthcare utilization in Taiwan. We provide replication materials employing publicly available statistical software in Python, R and Stata, offering researchers all necessary tools to conduct an RD analysis.

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arXiv2023

Covariate-assisted bounds on causal effects with instrumental variables

When an exposure of interest is confounded by unmeasured factors, an instrumental variable (IV) can be used to identify and estimate certain causal contrasts. Identification of the marginal average treatment effect (ATE) from IVs relies on strong untestable structural assumptions. When one is unwilling to assert such structure, IVs can nonetheless be used to construct bounds on the ATE. Famously, Balke and Pearl (1997) proved tight bounds on the ATE for a binary outcome, in a randomized trial with noncompliance and no covariate information. We demonstrate how these bounds remain useful in observational settings with baseline confounders of the IV, as well as randomized trials with measured baseline covariates. The resulting bounds on the ATE are non-smooth functionals, and thus standard nonparametric efficiency theory is not immediately applicable. To remedy this, we propose (1) under a novel margin condition, influence function-based estimators of the bounds that can attain parametric convergence rates when the nuisance functions are modeled flexibly, and (2) estimators of smooth approximations of these bounds. We propose extensions to continuous outcomes, explore finite sample properties in simulations, and illustrate the proposed estimators in an observational study targeting the effect of higher education on wages.

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arXiv2023

Approximate Balancing Weights for Clustered Observational Study Designs

In a clustered observational study, a treatment is assigned to groups and all units within the group are exposed to the treatment. We develop a new method for statistical adjustment in clustered observational studies using approximate balancing weights, a generalization of inverse propensity score weights that solve a convex optimization problem to find a set of weights that directly minimize a measure of covariate imbalance, subject to an additional penalty on the variance of the weights. We tailor the approximate balancing weights optimization problem to both adjustment sets by deriving an upper bound on the mean square error for each case and finding weights that minimize this upper bound, linking the level of covariate balance to a bound on the bias. We implement the procedure by specializing the bound to a random cluster-level effects model, leading to a variance penalty that incorporates the signal signal-to-noise ratio and penalizes the weight on individuals and the total weight on groups differently according to the the intra-class correlation.

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arXiv2022

Using Balancing Weights to Target the Treatment Effect on the Treated when Overlap is Poor

Inverse probability weights are commonly used in epidemiology to estimate causal effects in observational studies. Researchers can typically focus on either the average treatment effect or the average treatment effect on the treated with inverse probability weighting estimators. However, when overlap between the treated and control groups is poor, this can produce extreme weights that can result in biased estimates and large variances. One alternative to inverse probability weights are overlap weights, which target the population with the most overlap on observed characteristics. While estimates based on overlap weights produce less bias in such contexts, the causal estimand can be difficult to interpret. One alternative to inverse probability weights are balancing weights, which directly target imbalances during the estimation process. Here, we explore whether balancing weights allow analysts to target the average treatment effect on the treated in cases where inverse probability weights are biased due to poor overlap. We conduct three simulation studies and an empirical application. We find that in many cases, balancing weights allow the analyst to still target the average treatment effect on the treated even when overlap is poor. We show that while overlap weights remain a key tool for estimating causal effects, more familiar estimands can be targeted by using balancing weights instead of inverse probability weights.

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arXiv2022

Estimating Racial Disparities in Emergency General Surgery

Research documents that Black patients experience worse general surgery outcomes than white patients in the United States. In this paper, we focus on an important but less-examined category: the surgical treatment of emergency general surgery (EGS) conditions, which refers to medical emergencies where the injury is "endogenous," such as a burst appendix. Our goal is to assess racial disparities for common outcomes after EGS treatment using an administrative database of hospital claims in New York, Florida, and Pennsylvania, and to understand the extent to which differences are attributable to patient-level risk factors versus hospital-level factors. To do so, we use a class of linear weighting estimators that re-weight white patients to have a similar distribution of baseline characteristics as Black patients. This framework nests many common approaches, including matching and linear regression, but offers important advantages over these methods in terms of controlling imbalance between groups, minimizing extrapolation, and reducing computation time. Applying this approach to the claims data, we find that disparities estimates that adjust for the admitting hospital are substantially smaller than estimates that adjust for patient baseline characteristics only, suggesting that hospital-specific factors are important drivers of racial disparities in EGS outcomes.

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arXiv2022

Nonparametric identification of causal effects in clustered observational studies with differential selection

The clustered observational study (COS) design is the observational study counterpart to the clustered randomized trial. In a COS, a treatment is assigned to intact groups, and all units within the group are exposed to the treatment. However, the treatment is non-randomly assigned. COSs are common in both education and health services research. In education, treatments may be given to all students within some schools but withheld from all students in other schools. In health studies, treatments may be applied to clusters such as hospitals or groups of patients treated by the same physician. In this manuscript, we study the identification of causal effects in clustered observational study designs. We focus on the prospect of differential selection of units to clusters, which occurs when the units' cluster selections depend on the clusters' treatment assignments. Extant work on COSs has made an implicit assumption that rules out the presence of differential selection. We derive the identification results for designs with differential selection and that contexts with differential cluster selection require different adjustment sets than standard designs. We outline estimators for designs with and without differential selection. Using a series of simulations, we outline the magnitude of the bias that can occur with differential selection. We then present two empirical applications focusing on the likelihood of differential selection.

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arXiv2021

Covariate Adjustment in Regression Discontinuity Designs

The Regression Discontinuity (RD) design is a widely used non-experimental method for causal inference and program evaluation. While its canonical formulation only requires a score and an outcome variable, it is common in empirical work to encounter RD analyses where additional variables are used for adjustment. This practice has led to misconceptions about the role of covariate adjustment in RD analysis, from both methodological and empirical perspectives. In this chapter, we review the different roles of covariate adjustment in RD designs, and offer methodological guidance for its correct use.

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arXiv2021

Profiling Compliers in Instrumental Variables Designs

Instrumental variable (IV) analyses are becoming common in health services research and epidemiology. IV analyses can be used both to analyze randomized trials with noncompliance and as a form of natural experiment. In these analyses, investigators often adopt a monotonicity assumption, which implies that the relevant effect only applies to a subset of the study population known as compliers. Since the estimated effect is not the average treatment effect of the study population, it is important to compare the characteristics of compliers and non-compliers. Profiling compliers and non-compliers is necessary to understand what subpopulation the researcher is making inferences about, and an important first step in evaluating the external validity (or lack thereof) of the IV estimate for compliers. Here, we discuss the assumptions necessary for profiling, which are weaker than the assumptions necessary for identifying the local average treatment effect if the instrument is randomly assigned. We then outline a simple and general method to characterize compliers and noncompliers using baseline covariates. Next, we extend current methods by deriving standard errors for these estimates. We demonstrate these methods using an IV known as tendency to operate (TTO) from health services research.

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arXiv2020

Hospital Quality Risk Standardization via Approximate Balancing Weights

Comparing outcomes across hospitals, often to identify underperforming hospitals, is a critical task in health services research. However, naive comparisons of average outcomes, such as surgery complication rates, can be misleading because hospital case mixes differ -- a hospital's overall complication rate may be lower due to more effective treatments or simply because the hospital serves a healthier population overall. In this paper, we develop a method of ``direct standardization'' where we re-weight each hospital patient population to be representative of the overall population and then compare the weighted averages across hospitals. Adapting methods from survey sampling and causal inference, we find weights that directly control for imbalance between the hospital patient mix and the target population, even across many patient attributes. Critically, these balancing weights can also be tuned to preserve sample size for more precise estimates. We also derive principled measures of statistical precision, and use outcome modeling and Bayesian shrinkage to increase precision and account for variation in hospital size. We demonstrate these methods using claims data from Pennsylvania, Florida, and New York, estimating standardized hospital complication rates for general surgery patients. We conclude with a discussion of how to detect low performing hospitals.

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