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Public health researchers often estimate health effects of exposures (e.g., pollution, diet, lifestyle) that cannot be directly measured for study subjects. A common strategy in environmental epidemiology is to use a first-stage (exposure)…

Methodology · Statistics 2014-06-03 Adam A. Szpiro , Christopher J. Paciorek

Statistical models often require inputs that are not completely known. This can occur when inputs are measured with error, indirectly, or when they are predicted using another model. In environmental epidemiology, air pollution exposure is…

Methodology · Statistics 2025-12-23 Konstantin Larin , Daniel R. Kowal

This paper studies inference in two-stage randomized experiments under covariate-adaptive randomization. In the initial stage of this experimental design, clusters (e.g., households, schools, or graph partitions) are stratified and randomly…

Econometrics · Economics 2026-01-16 Jizhou Liu

The health effects of environmental exposures have been studied for decades, typically using standard regression models to assess exposure-outcome associations found in observational non-experimental data. We propose and illustrate a…

Applications · Statistics 2017-09-20 Marie-Abele C. Bind , Donald B. Rubin

Under two-phase designs, the outcome and several covariates and confounders are measured in the first phase, and a new predictor of interest, which may be costly to collect, can be measured on a subsample in the second phase, without…

Methodology · Statistics 2023-05-12 Jingjing Zou , Lori B. Daniels , Karen Messer , Daniel Rabinowitz

Increasingly, medical research is dependent on data collected for non-research purposes, such as electronic health records data (EHR). EHR data and other large databases can be prone to measurement error in key exposures, and unadjusted…

Methodology · Statistics 2020-05-13 Kyunghee Han , Thomas Lumley , Bryan E. Shepherd , Pamela A. Shaw

We present a simulation-based inference approach for two-stage estimators, focusing on extremum estimators in the second stage. We accommodate a broad range of first-stage estimators, including extremum estimators, high-dimensional…

Econometrics · Economics 2024-11-08 Aristide Houndetoungan , Abdoul Haki Maoude

Adaptive designs are commonly used in clinical and drug development studies for optimum utilization of available resources. In this article, we consider the problem of estimating the effect of the selected (better) treatment using a…

Statistics Theory · Mathematics 2023-01-24 Masihuddin , Neeraj Misra

Methods for causal inference are well developed for binary and continuous exposures, but in many settings, the exposure has a substantial mass at zero-such exposures are called semi-continuous. We propose a general causal framework for such…

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

Estimating causal effects under interference is pertinent to many real-world settings. Recent work with low-order potential outcomes models uses a rollout design to obtain unbiased estimators that require no interference network…

Methodology · Statistics 2025-02-12 Mayleen Cortez-Rodriguez , Matthew Eichhorn , Christina Lee Yu

Combination of several anti-cancer treatments has typically been presumed to have enhanced drug activity. Motivated by a real clinical trial, this paper considers phase I-II dose finding designs for dual-agent combinations, where one main…

Methodology · Statistics 2023-05-09 José L. Jiménez , Haiyan Zheng

We consider an experiment with at least two stages or batches and $O(N)$ subjects per batch. First, we propose a semiparametric treatment effect estimator that efficiently pools information across the batches, and show it asymptotically…

Methodology · Statistics 2023-09-28 Harrison H. Li , Art B. Owen

Given n experiment subjects with potentially heterogeneous covariates and two possible treatments, namely active treatment and control, this paper addresses the fundamental question of determining the optimal accuracy in estimating the…

Machine Learning · Statistics 2024-11-13 Jiachun Li , David Simchi-Levi , Yunxiao Zhao

Two-phase designs involve measuring extra variables on a subset of the cohort where some variables are already measured. The goal of two-phase designs is to choose a subsample of individuals from the cohort and analyse that subsample…

Applications · Statistics 2020-10-12 Tong Chen , Thomas Lumley

The growing availability of observational databases like electronic health records (EHR) provides unprecedented opportunities for secondary use of such data in biomedical research. However, these data can be error-prone and need to be…

Methodology · Statistics 2024-05-28 Sarah C. Lotspeich , Gustavo G. C. Amorim , Pamela A. Shaw , Ran Tao , Bryan E. Shepherd

In observational studies of treatment effects, estimates may be biased by unmeasured confounders, which can potentially affect the validity of the results. Understanding sensitivity to such biases helps assess how unmeasured confounding…

Methodology · Statistics 2026-02-02 Qishuo Yin , Dylan S. Small

Two-phase sampling designs are frequently employed in epidemiological studies and large-scale health surveys. In such designs, certain variables are exclusively collected within a second-phase random subsample of the initial first-phase…

Methodology · Statistics 2024-03-25 Lingxiao Wang

This paper studies a two-stage model of experimentation, where the researcher first samples representative units from an eligible pool, then assigns each sampled unit to treatment or control. To implement balanced sampling and assignment,…

Econometrics · Economics 2023-08-22 Max Cytrynbaum

For biological experiments aiming at calibrating models with unknown parameters, a good experimental design is crucial, especially for those subject to various constraints, such as financial limitations, time consumption and physical…

Applications · Statistics 2014-07-22 Xiao Lin , Gabriel Terejanu
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