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Matching is a widely used causal inference design that aims to approximate a randomized experiment using observational data by forming matched sets of treated and control units based on similarities in their covariates. Ideally, treated…

Methodology · Statistics 2026-04-06 Jianan Zhu , Jeffrey Zhang , Zijian Guo , Siyu Heng

The phase III BNT162b2 mRNA COVID-19 vaccine trial is based on a Bayesian design and analysis, and the main evidence of vaccine efficacy is presented in Bayesian statistics. Confusion and mistakes are produced in the presentation of the…

Applications · Statistics 2021-03-10 Yuan Ji , Shijie Yuan

Results from randomized controlled trials (RCTs) help determine vaccination strategies and related public health policies. However, defining and identifying estimands that can guide policies in infectious disease settings is difficult, even…

Methodology · Statistics 2023-01-24 Mats J. Stensrud , Louisa H. Smith

This paper proposes a new statistical approach for assessing treatment effect using Bayesian Networks (BNs). The goal is to draw causal inferences from observational data with a binary outcome and discrete covariates. The BNs are here used…

Measuring treatment effects in observational studies is challenging because of confounding bias. Confounding occurs when a variable affects both the treatment and the outcome. Traditional methods such as propensity score matching estimate…

Methodology · Statistics 2021-12-23 Bevan I. Smith , Charles Chimedza

Measurement error is a common challenge for causal inference studies using electronic health record (EHR) data, where clinical outcomes and treatments are frequently mismeasured. Researchers often address measurement error by conducting…

Confounding and exposure measurement error can introduce bias when drawing inference about the marginal effect of an exposure on an outcome of interest. While there are broad methodologies for addressing each source of bias individually,…

Methodology · Statistics 2025-01-29 Brian D. Richardson , Bryan S. Blette , Peter B. Gilbert , Michael G. Hudgens

In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to…

Methodology · Statistics 2021-12-23 Janie Coulombe , Erica E M Moodie , Robert W Platt

Psychiatric and social epidemiology often involves assessing the effects of environmental exposure on outcomes that are difficult to measure directly. To address this problem, it is common to measure outcomes using a comprehensive battery…

Statistical hypothesis testing serves as statistical evidence for scientific innovation. However, if the reported results are intentionally biased, hypothesis testing no longer controls the rate of false discovery. In particular, we study…

Methodology · Statistics 2018-10-12 Junpei Komiyama , Takanori Maehara

Real-world vaccine effectiveness has increasingly been studied using matching-based approaches, particularly in observational cohort studies following the target trial emulation framework. Although matching is appealing in its simplicity,…

Methodology · Statistics 2026-04-07 Emily Wu , Elizabeth Rogawski McQuade , Mats Stensrud , Razieh Nabi , David Benkeser

Evidence-informed policy on infections requires estimates of their effects on health. However, pathogenic variation, whereby occurrence of adverse outcomes depends on the infecting strain, might complicate the study of many infectious…

Methodology · Statistics 2026-04-24 Bronner P. Gonçalves

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

Cross-sectional HIV incidence estimation leverages recency test results to determine the HIV incidence of a population of interest, where recency test uses biomarker profiles to infer whether an HIV-positive individual was "recently"…

Methodology · Statistics 2023-11-21 Qi Wang , Ann Duerr , Fei Gao

When there is interference, a subject's outcome depends on the treatment of others and treatment effects may take on several different forms. This situation arises often, particularly in vaccine evaluation. In settings where interference is…

Statistics Theory · Mathematics 2020-01-10 Erin E Gabriel

Evaluating causal effects in the presence of interference is challenging in network-based studies of hard-to-reach populations. Like many such populations, people who inject drugs (PWID) are embedded in social networks and often exert…

Precision medicine has led to a paradigm shift allowing the development of targeted drugs that are agnostic to the tumor location. In this context, basket trials aim to identify which tumor types - or baskets - would benefit from the…

Methodology · Statistics 2026-01-05 Marcio A. Diniz , Hulya Kocyigit , Erin Moshier , Madhu Mazumdar , Deukwoo Kwon

Predictive biomarkers are playing an essential role in precision medicine. Identifying an optimal cutoff to select patient subsets with greater benefit from treatment is critical and more challenging for predictive biomarkers measured with…

Applications · Statistics 2025-04-29 Chi Zhang , Wei Shi , Spencer Woody , Qing Liu

We study the performance of shape-constrained methods for evaluating immune response profiles from early-phase vaccine trials. The motivating problem for this work involves quantifying and comparing the IgG binding immune responses to the…

Methodology · Statistics 2022-11-01 Nilanjana Laha , Zoe Moodie , Ying Huang , Alex Luedtke

A treatment benefit predictor (TBP) is a function that maps patient characteristics to an estimate of the treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to…

Methodology · Statistics 2025-09-30 Yuan Xia , Mohsen Sadatsafavi , Paul Gustafson
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