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Electronic health records are being increasingly used in medical research to answer more relevant and detailed clinical questions; however, they pose new and significant methodological challenges. For instance, observation times are likely…

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…

Methodology · Statistics 2021-10-25 Daniel Daly-Grafstein , Paul Gustafson

When the outcome of interest is semicontinuous and collected longitudinally, efficient testing can be difficult. Daily rainfall data is an excellent example which we use to illustrate the various challenges. Even under the simplest…

Methodology · Statistics 2018-07-10 Harlan Campbell

Surrogate markers offer the potential to reduce the burden of data collection by replacing costly or invasive primary outcomes with more accessible measurements, provided that they can faithfully indicate the effectiveness of a treatment.…

Methodology · Statistics 2026-04-15 Silvaneo V. dos Santos , Layla Parast

Quality of Life (QOL) outcomes are important in the management of chronic illnesses. In studies of efficacies of treatments or intervention modalities, QOL scales multi-dimensional constructs are routinely used as primary endpoints. The…

Methodology · Statistics 2021-12-02 Yue Cui , Solomon W. Harrar

This paper considers the design of observational longitudinal studies with a continuous response and a binary time-invariant exposure, where, typically, the exposure is unbalanced, the mean response in the two groups differs at baseline and…

Methodology · Statistics 2022-09-27 Xavier Basagana , Donna Spiegelman

We study identification and estimation in the Regression Discontinuity Design (RDD) with a multivalued treatment variable. We also allow for the inclusion of covariates. We show that without additional information, treatment effects are not…

Econometrics · Economics 2020-07-02 Carolina Caetano , Gregorio Caetano , Juan Carlos Escanciano

Unobserved heterogeneous treatment effects have been emphasized in recent policy evaluation literature. In this paper, we extend Lu and White (2014)'s testing method for unobserved heterogeneous treatment effects by developing nonparametric…

Methodology · Statistics 2016-11-22 YuChin Hsu , TaCheng Huang , Haiqing Xu

Modeling and computation for multivariate longitudinal surveys have proven challenging, particularly when data are not all continuous and Gaussian but contain discrete measurements. In many social science surveys, study participants are…

Applications · Statistics 2016-06-09 Tsuyoshi Kunihama , Carolyn T. Halpern , Amy H. Herring

When the difference between treatments in a clinical trial is estimated by a difference in means, then it is well known that randomization ensures unbiassed estimation, even if no account is taken of important baseline covariates. However,…

Statistics Theory · Mathematics 2014-07-22 J. N. S. Matthews , Nuri H. Badi

Estimating the counterfactual outcome of treatment is essential for decision-making in public health and clinical science, among others. Often, treatments are administered in a sequential, time-varying manner, leading to an exponentially…

Machine Learning · Statistics 2024-07-16 Shenghao Wu , Wenbin Zhou , Minshuo Chen , Shixiang Zhu

Understanding treatment effect heterogeneity is vital to many scientific fields because the same treatment may affect different individuals differently. Quantile regression provides a natural framework for modeling such heterogeneity. We…

Methodology · Statistics 2023-07-12 Alexander Giessing , Jingshen Wang

We propose a procedure for imputing missing values of time-dependent covariates in a survival model using fully conditional specification. Specifically, we focus on imputing missing values of a longitudinal marker in joint modeling of the…

Methodology · Statistics 2024-03-29 Havi Murad , Nirit Agay , Rachel Dankner

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

We present a novel approach for extrapolating causal effects away from the margin between treatment and non-treatment in sharp regression discontinuity designs with multiple covariates. Our methods apply both to settings in which treatment…

Econometrics · Economics 2025-07-02 Ben Deaner , Soonwoo Kwon

Regression analyses based on transformations of cumulative incidence functions are often adopted when modeling and testing for treatment effects in clinical trial settings involving competing and semi-competing risks. Common frameworks…

Methodology · Statistics 2024-01-11 Alexandra Bühler , Richard J Cook , Jerald F Lawless

This paper addresses estimation in a longitudinal regression model for association between a scalar outcome and a set of longitudinally-collected functional covariates or predictor curves. The framework consists of estimating a time-varying…

Applications · Statistics 2020-06-30 Madan G. Kundu , Jaroslaw Harezlak , Timothy W. Randolph

Methods for addressing missing data have become much more accessible to applied researchers. However, little guidance exists to help researchers systematically identify plausible missing data mechanisms in order to ensure that these methods…

Applications · Statistics 2020-07-29 Adam Davey , Ting Dai

We study inference on the long-term causal effect of a continual exposure to a novel intervention, which we term a long-term treatment, based on an experiment involving only short-term observations. Key examples include the long-term health…

Applications · Statistics 2024-06-06 Allen Tran , Aurélien Bibaut , Nathan Kallus

Suppose we have a binary treatment used to influence an outcome. Given data from an observational or controlled study, we wish to determine whether or not there exists some subset of observed covariates in which the treatment is more…

Methodology · Statistics 2016-03-22 Alexander R. Luedtke , Mark J. van der Laan