arXiv subjects

Nandita Mitra

Publications and source records attributed to Nandita Mitra.

At least 19 recordsLinked to original sources

arXiv2026

Small Samples and Short Panels: Evaluating Policy Evaluation Methods with Realistic Data

Methods for estimating causal effects in longitudinal, quasi-experimental settings are widely used in economics, public health, political science, and other fields. However, studies evaluating effects of health policies often rely on limited sample sizes, both in terms of study units and time periods analyzed. Synthetic difference-in-differences (SDiD) and the augmented synthetic control method (ASCM) are recently developed methods for evaluating the effects of policy interventions. Although SDiD and ASCM generally rely on weaker assumptions than both DiD and SCM, they lack theoretical performance guarantees with respect to bias or coverage in relevant settings with small sample sizes or short panel lengths. To evaluate the performance of SDiD and ASCM in realistic, small-sample settings, we employ a calibrated simulation strategy that allows the injection of a known treatment effect into existing data in a setting of interest. Drawing on findings from these empirical investigations, we offer practical guidance for researchers and policymakers on when SDiD and ASCM are likely to yield reliable estimates and inferences under realistic scenarios.

stat.ME↗

arXiv2026

Nonparametric Estimation of Optimal Stochastic Just-In-Time Adaptive Interventions for Distal Outcomes

Mobile and wearable technologies enable the delivery of just-in-time adaptive interventions (JITAIs) -- interventions that adapt treatment delivery to an individual's rapidly changing internal state and context in real-time, real-world settings. Estimating optimal JITAIs, however, remains challenging because these studies often involve dozens of decision points per individual, and existing methods can produce unstable and irregular estimators with substantial bias and slow convergence rates. Advanced reinforcement learning approaches may be difficult to interpret and often target proximal, discounted outcomes rather than the distal end-of-study outcomes that define long-term success in many behavioral and clinical studies. To address these challenges, we develop a nonparametrically efficient estimator of the regimen-response curve for distal outcomes under a class of stochastic policies and introduce a data-adaptive tilting procedure to stabilize estimation in settings with many decision points. We show that the estimated regimen-response curve converges weakly to a Gaussian process, enabling simultaneous confidence bands, and we derive asymptotic theory for the optimizer of the curve, thereby enabling inference for the learned optimal stochastic policy. These developments provide a unified framework for estimation, inference, and optimization of stochastic JITAIs for distal outcomes.

stat.ME↗

arXiv2026

A Bayesian approach to the survivor average causal effect in cluster-randomized crossover trials

In cluster-randomized crossover (CRXO) trials, groups of individuals are randomly assigned to two or more sequences of alternating treatments. Since clusters serve as their own control, the CRXO design is typically more statistically efficient than the usual parallel-arm design. CRXO trials are increasingly popular in many areas of health research where the number of available clusters is limited. Further, in trials among severely ill patients, researchers often want to assess the effect of treatments on secondary non-terminal outcomes, but frequently in these studies, there are patients who do not survive to have these measurements fully recorded. In this paper, we provide a causal inference framework and treatment effect estimation methods for addressing truncation by death in the setting of CRXO trials. We target the survivor average causal effect (SACE) estimand, a well-defined subgroup treatment effect obtained via principal stratification. We propose novel structural and standard modeling assumptions that enable estimating the SACE within a Bayesian paradigm. We evaluate the small-sample performance of our proposed Bayesian approach for estimation of the SACE in CRXO trial settings via simulation studies. We apply our methods to a previously conducted two-period cross-sectional CRXO study examining the impact of proton pump inhibitors compared to histamine-2 receptor blockers on length of hospitalization among adults requiring invasive mechanical ventilation.

stat.ME↗

arXiv2026

A Bayesian framework for cost-effectiveness analysis with time-varying treatment decisions

Cost-effectiveness analyses (CEAs) compare the costs and health outcomes of treatment regimes to inform medical decisions. With observational claims data, CEAs must address nonrandom treatment assignment, administrative censoring, and irregularly spaced medical visits that reflect the continuous timing of care and treatment initiation. In high-risk, early-stage endometrial cancer (HR-EC), adjuvant radiation is initiated at patient-specific times following hysterectomy, causing confounding between treatment and outcomes that can evolve with post-surgical recovery and clinical course. Most existing CEA methods use point-treatment or discrete-time models. However, point-treatment approaches break down with time-varying confounding, while discrete-time models bin continuous time, expand the data into a person-period format, and can induce zero-inflation by creating many intervals with no cost-accruing events. We propose a Bayesian framework for CEAs with sequential decision-making that jointly models costs and event times in continuous time, accounts for administrative censoring, and supports dynamic treatment regimes with minimal parametric assumptions. We use Bayesian g-computation to estimate causally interpretable cost-effectiveness measures, including net monetary benefit, and to compare regimes through posterior contrasts. We evaluate the finite-sample performance of the proposed method in simulations across censoring levels and compare it against discrete-time and fully parametric alternatives. We then use SEER-Medicare data to assess the cost-effectiveness of initiating adjuvant radiation therapy within six months following hysterectomy among HR-EC patients.

stat.ME↗

arXiv2025

Estimating the average treatment effect in cluster-randomized trials with misclassified outcomes and non-random validation subsets

Randomized trials are viewed as the benchmark for assessing causal effects of treatments on outcomes of interest. Nonetheless, challenges such as measurement error can undermine the standard causal assumptions for randomized trials. In ASPIRE, a cluster-randomized trial, pediatric primary care clinics were assigned to one of two treatments aimed at promoting clinician delivery of a secure firearm program to parents during well-child visits. A key outcome of interest is thus parent receipt of the program at each visit. Clinicians documented program delivery in patients' electronic health records for all visits, but their reporting is a proxy measure for the parent receipt outcome. Parents were also surveyed to report directly on program receipt after their child's visit; however, only a small subset of them completed the survey. Here, we develop a causal inference framework for a binary outcome that is subject to misclassification through silver-standard measures (clinician reports), but gold-standard measures (parent reports) are only available for a non-random internal validation subset. We propose a method for identifying the average treatment effect (ATE) that addresses the risk of bias due to misclassification and non-random validation selection, even when the outcome (parent receipt) may directly impact selection propensity (survey responsiveness). We show that ATE estimation relies on specifying the relationship between the gold- and silver-standard outcome measures in the validation subset, which may depend on treatment and covariates. Additionally, the clustered design is reflected in our causal assumptions and in our cluster-robust approach to estimation of the ATE. Simulation studies demonstrate acceptable finite-sample operating characteristics of our ATE estimator, supporting its application to ASPIRE.

stat.ME↗

arXiv2025

Bayesian Sensitivity Analyses for Policy Evaluation with Difference-in-Differences under Violations of Parallel Trends

Violations of the parallel trends assumption pose significant challenges for causal inference in difference-in-differences (DiD) studies, especially in policy evaluations where pre-treatment dynamics and external shocks may bias estimates. In this work, we propose a Bayesian DiD framework to allow us to estimate the effect of policies when parallel trends is violated. To address potential deviations from the parallel trends assumption, we introduce a formal sensitivity parameter representing the extent of the violation, specify an autoregressive AR(1) prior on this term to robustly model temporal correlation, and explore a range of prior specifications - including fixed, fully Bayesian, and empirical Bayes (EB) approaches calibrated from pre-treatment data. By systematically comparing posterior treatment effect estimates across prior configurations when evaluating Philadelphia's sweetened beverage tax using Baltimore as a control, we show how Bayesian sensitivity analyses support robust and interpretable policy conclusions under violations of parallel trends.

stat.ME↗

arXiv2025

A Causal Framework for Evaluating Drivers of Policy Effect Heterogeneity Using Difference-in-Differences

Policymakers and researchers often seek to understand how a policy differentially affects a population and the pathways driving this heterogeneity. For example, when studying an excise tax on sweetened beverages, researchers might assess the roles of cross-border shopping, economic competition, and store-level price changes on beverage sales trends. However, traditional policy evaluation tools, like the difference-in-differences (DiD) approach, primarily target average effects of the observed intervention rather than the underlying drivers of effect heterogeneity. Common approaches to evaluate sources of heterogeneity often lack a causal framework, making it difficult to determine whether observed outcome differences are truly driven by the proposed source of heterogeneity or by other confounding factors. In this paper, we present a framework for evaluating such policy drivers by representing questions of effect heterogeneity under hypothetical interventions and use it to evaluate drivers of the Philadelphia sweetened beverage tax policy effects. Building on recent advancements in estimating causal effect curves under DiD designs, we provide tools to assess policy effect heterogeneity while addressing practical challenges including confounding and neighborhood dynamics.

stat.ME↗

arXiv2024

Weighting methods for truncation by death in cluster-randomized trials

Patient-centered outcomes, such as quality of life and length of hospital stay, are the focus in a wide array of clinical studies. However, participants in randomized trials for elderly or critically and severely ill patient populations may have truncated or undefined non-mortality outcomes if they do not survive through the measurement time point. To address truncation by death, the survivor average causal effect (SACE) has been proposed as a causally interpretable subgroup treatment effect defined under the principal stratification framework. However, the majority of methods for estimating SACE have been developed in the context of individually-randomized trials. Only limited discussions have been centered around cluster-randomized trials (CRTs), where methods typically involve strong distributional assumptions for outcome modeling. In this paper, we propose two weighting methods to estimate SACE in CRTs that obviate the need for potentially complicated outcome distribution modeling. We establish the requisite assumptions that address latent clustering effects to enable point identification of SACE, and we provide computationally-efficient asymptotic variance estimators for each weighting estimator. In simulations, we evaluate our weighting estimators, demonstrating their finite-sample operating characteristics and robustness to certain departures from the identification assumptions. We illustrate our methods using data from a CRT to assess the impact of a sedation protocol on mechanical ventilation among children with acute respiratory failure.

stat.ME↗

arXiv2024

Multiply Robust Difference-in-Differences Estimation of Causal Effect Curves for Continuous Exposures

Researchers commonly use difference-in-differences (DiD) designs to evaluate public policy interventions. While methods exist for estimating effects in the context of binary interventions, policies often result in varied exposures across regions implementing the policy. Yet, existing approaches for incorporating continuous exposures face substantial limitations in addressing confounding variables associated with intervention status, exposure levels, and outcome trends. These limitations significantly constrain policymakers' ability to fully comprehend policy impacts and design future interventions. In this work, we propose new estimators for causal effect curves within the DiD framework, accounting for multiple sources of confounding. Our approach accommodates misspecification of a subset of treatment, exposure, and outcome models while avoiding any parametric assumptions on the effect curve. We present the statistical properties of the proposed methods and illustrate their application through simulations and a study investigating the heterogeneous effects of a nutritional excise tax under different levels of accessibility to cross-border shopping.

stat.ME↗

arXiv2023

Policy effect evaluation under counterfactual neighborhood interventions in the presence of spillover

Policy interventions can spill over to units of a population that are not directly exposed to the policy but are geographically close to the units receiving the intervention. In recent work, investigations of spillover effects on neighboring regions have focused on estimating the average treatment effect of a particular policy in an observed setting. Our research question broadens this scope by asking what policy consequences would the treated units have experienced under hypothetical exposure settings. When we only observe treated unit(s) surrounded by controls -- as is common when a policy intervention is implemented in a single city or state -- this effect inquires about the policy effects under a counterfactual neighborhood policy status that we do not, in actuality, observe. In this work, we extend difference-in-differences (DiD) approaches to spillover settings and develop identification conditions required to evaluate policy effects in counterfactual treatment scenarios. These causal quantities are policy-relevant for designing effective policies for populations subject to various neighborhood statuses. We develop doubly robust estimators and use extensive numerical experiments to examine their performance under heterogeneous spillover effects. We apply our proposed method to investigate the effect of the Philadelphia beverage tax on unit sales.

stat.ME↗

arXiv2023

Estimation of Policy-Relevant Causal Effects in the Presence of Interference with an Application to the Philadelphia Beverage Tax

To comprehensively evaluate a public policy intervention, researchers must consider the effects of the policy not just on the implementing region, but also nearby, indirectly-affected regions. For example, an excise tax on sweetened beverages in Philadelphia was shown to not only be associated with a decrease in volume sales of taxed beverages in Philadelphia, but also an increase in sales in bordering counties not subject to the tax. The latter association may be explained by cross-border shopping behaviors of Philadelphia residents and indicate a causal effect of the tax on nearby regions, which may offset the total effect of the intervention. To estimate causal effects in this setting, we extend difference-in-differences methodology to account for such interference between regions and adjust for potential confounding present in quasi-experimental evaluations. Our doubly robust estimators for the average treatment effect on the treated and neighboring control relax standard assumptions on interference and model specification. We apply these methods to evaluate the change in volume sales of taxed beverages in 231 Philadelphia and bordering county stores due to the Philadelphia beverage tax. We also use our methods to explore the heterogeneity of effects across geographic features.

stat.ME↗

arXiv2023

Hierarchical Bayesian Bootstrap for Heterogeneous Treatment Effect Estimation

A major focus of causal inference is the estimation of heterogeneous average treatment effects (HTE) - average treatment effects within strata of another variable of interest such as levels of a biomarker, education, or age strata. Inference involves estimating a stratum-specific regression and integrating it over the distribution of confounders in that stratum - which itself must be estimated. Standard practice involves estimating these stratum-specific confounder distributions independently (e.g. via the empirical distribution or Rubin's Bayesian bootstrap), which becomes problematic for sparsely populated strata with few observed confounder vectors. In this paper, we develop a nonparametric hierarchical Bayesian bootstrap (HBB) prior over the stratum-specific confounder distributions for HTE estimation. The HBB partially pools the stratum-specific distributions, thereby allowing principled borrowing of confounder information across strata when sparsity is a concern. We show that posterior inference under the HBB can yield efficiency gains over standard marginalization approaches while avoiding strong parametric assumptions about the confounder distribution. We use our approach to estimate the adverse event risk of proton versus photon chemoradiotherapy across various cancer types.

stat.ME↗

arXiv2022

Leveraging baseline covariates to analyze small cluster-randomized trials with a rare binary outcome

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 weighting and multivariable regression as two individual-level covariate adjustment strategies for estimating the participant-average causal effect in small CRTs with a rare binary outcome and identify scenarios where each adjustment strategy has a relative efficiency advantage over the other to make practical recommendations. We also examine the finite-sample performance of the bias-corrected sandwich variance estimators associated with propensity score weighting and multivariable regression for quantifying the uncertainty in estimating the participant-average treatment effect. To illustrate the methods for individual-level covariate adjustment, we reanalyze a recent CRT testing a sedation protocol in 31 pediatric intensive care units.

stat.ME↗

arXiv2022

Addressing Positivity Violations in Causal Effect Estimation using Gaussian Process Priors

In observational studies, causal inference relies on several key identifying assumptions. One identifiability condition is the positivity assumption, which requires the probability of treatment be bounded away from 0 and 1. That is, for every covariate combination, it should be possible to observe both treated and control subjects, i.e., the covariate distributions should overlap between treatment arms. If the positivity assumption is violated, population-level causal inference necessarily involves some extrapolation. Ideally, a greater amount of uncertainty about the causal effect estimate should be reflected in such situations. With that goal in mind, we construct a Gaussian process model for estimating treatment effects in the presence of practical violations of positivity. Advantages of our method include minimal distributional assumptions, a cohesive model for estimating treatment effects, and more uncertainty associated with areas in the covariate space where there is less overlap. We assess the performance of our approach with respect to bias and efficiency using simulation studies. The method is then applied to a study of critically ill female patients to examine the effect of undergoing right heart catheterization.

stat.ME↗

arXiv2021

Identifying optimally cost-effective dynamic treatment regimes with a Q-learning approach

Health policy decisions regarding patient treatment strategies require consideration of both treatment effectiveness and cost. Optimizing treatment rules with respect to effectiveness may result in prohibitively expensive strategies; on the other hand, optimizing with respect to costs may result in poor patient outcomes. We propose a two-step approach for identifying an optimally cost-effective and interpretable dynamic treatment regime. First, we develop a combined Q-learning and policy-search approach to estimate an optimal list-based regime under a constraint on expected treatment costs. Second, we propose an iterative procedure to select an optimally cost-effective regime from a set of candidate regimes corresponding to different cost constraints. Our approach can estimate optimal regimes in the presence of time-varying confounding, censoring, and correlated outcomes. Through simulation studies, we illustrate the validity of estimated treatment regimes and examine operating characteristics under flexible modeling approaches. We also apply our methodology to evaluate optimally cost-effective treatment strategies for assigning adjuvant therapies to endometrial cancer patients.

stat.ME↗

arXiv2021

Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score

Clinical trials with a hybrid control arm (a control arm constructed from a combination of randomized patients and real-world data on patients receiving usual care in standard clinical practice) have the potential to decrease the cost of randomized trials while increasing the proportion of trial patients given access to novel therapeutics. However, due to stringent trial inclusion criteria and differences in care and data quality between trials and community practice, trial patients may have systematically different outcomes compared to their real-world counterparts. We propose a new method for analyses of trials with a hybrid control arm that efficiently controls bias and type I error. Under our proposed approach, selected real-world patients are weighted by a function of the "on-trial score," which reflects their similarity to trial patients. In contrast to previously developed hybrid control designs that assign the same weight to all real-world patients, our approach upweights of real-world patients who more closely resemble randomized control patients while dissimilar patients are discounted. Estimates of the treatment effect are obtained via Cox proportional hazards models. We compare our approach to existing approaches via simulations and apply these methods to a study using electronic health record data. Our proposed method is able to control type I error, minimize bias, and decrease variance when compared to using only trial data in nearly all scenarios examined. Therefore, our new approach can be used when conducting clinical trials by augmenting the standard-of-care arm with weighted patients from the EHR to increase power without inducing bias.

stat.ME↗

arXiv2021

Analysis of survival data with non-proportional hazards: A comparison of propensity score weighted methods

One of the most common ways researchers compare survival outcomes across treatments when confounding is present is using Cox regression. This model is limited by its underlying assumption of proportional hazards; in some cases, substantial violations may occur. Here we present and compare approaches which attempt to address this issue, including Cox models with time-varying hazard ratios; parametric accelerated failure time models; Kaplan-Meier curves; and pseudo-observations. To adjust for differences between treatment groups, we use Inverse Probability of Treatment Weighting based on the propensity score. We examine clinically meaningful outcome measures that can be computed and directly compared across each method, namely, survival probability at time T, median survival, and restricted mean survival. We conduct simulation studies under a range of scenarios, and determine the biases, coverages, and standard errors of the Average Treatment Effects for each method. We then apply these approaches to two published observational studies of survival after cancer treatment. The first examines chemotherapy in sarcoma, where survival is very similar initially, but after two years the chemotherapy group shows a benefit. The other study is a comparison of surgical techniques for kidney cancer, where survival differences are attenuated over time.

stat.AP↗

arXiv2021

A regression framework for a probabilistic measure of cost-effectiveness

To make informed health policy decisions regarding a treatment, we must consider both its cost and its clinical effectiveness. In past work, we introduced the net benefit separation (NBS) as a novel measure of cost-effectiveness. The NBS is a probabilistic measure that characterizes the extent to which a treated patient will be more likely to experience benefit as compared to an untreated patient. Due to variation in treatment response across patients, uncovering factors that influence cost-effectiveness can assist policy makers in population-level decisions regarding resource allocation. In this paper, we introduce a regression framework for NBS in order to estimate covariate-specific NBS and find determinants of variation in NBS. Our approach is able to accommodate informative cost censoring through inverse probability weighting techniques, and addresses confounding through a semiparametric standardization procedure. Through simulations, we show that NBS regression performs well in a variety of common scenarios. We apply our proposed regression procedure to a realistic simulated data set as an illustration of how our approach could be used to investigate the association between cancer stage, comorbidities and cost-effectiveness when comparing adjuvant radiation therapy and chemotherapy in post-hysterectomy endometrial cancer patients.

stat.ME↗