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The test-negative design (TND) is widely used to evaluate vaccine effectiveness in real-world settings. In a TND study, individuals with similar symptoms who seek care are tested, and effectiveness is estimated by comparing vaccination…

统计方法学 · 统计学 2025-09-19 Christopher B. Boyer , Kendrick Qijun Li , Xu Shi , Eric J. Tchetgen Tchetgen

The test-negative design (TND) has become a standard approach to evaluate vaccine effectiveness against the risk of acquiring infectious diseases in real-world settings, such as Influenza, Rotavirus, Dengue fever, and more recently…

统计方法学 · 统计学 2023-03-09 Kendrick Qijun Li , Xu Shi , Wang Miao , Eric Tchetgen Tchetgen

Test-negative designs (TNDs), a form of case-cohort study, are widely used to evaluate infectious disease interventions, notably for influenza and, more recently, COVID-19 vaccines. TNDs rely on recruiting individuals who are tested for the…

统计方法学 · 统计学 2025-04-29 Mengxin Yu , Nicholas P. Jewell

We develop semiparametric methods for estimating subgroup-specific relative vaccine efficacy against multiple viral strains in a partially vaccinated population. Focusing on observational case-only studies, we address informative…

统计方法学 · 统计学 2025-03-12 Lars van der Laan , Peter B. Gilbert

The test-negative design has become popular for evaluating the effectiveness of post-licensure vaccines using observational data. In addition to its logistical convenience on data collection, the design is also believed to control for the…

统计方法学 · 统计学 2026-05-15 Soumyabrata Kundu , Peng Ding , Jingshu Wang , Xinran Li

The test-negative design (TND), which is routinely used for monitoring seasonal flu vaccine effectiveness (VE), has recently become integral to COVID-19 vaccine surveillance, notably in Qu\'ebec, Canada. Some studies have addressed the…

统计方法学 · 统计学 2024-12-13 Cong Jiang , Denis Talbot , Sara Carazo , Mireille E Schnitzer

Testing of symptomatic persons for infection with SARS-CoV-2 is occurring worldwide. We propose two types of case-control studies that can be carried out jointly in test-settings for symptomatic persons. The first, the test-negative…

Targeted Maximum Likelihood Estimation (TMLE) is increasingly used for doubly robust causal inference, but how missing data should be handled when using TMLE with data-adaptive approaches is unclear. Based on the Victorian Adolescent Health…

We evaluate the performance of targeted maximum likelihood estimation (TMLE) for estimating the average treatment effect in missing data scenarios under varying levels of positivity violations. We employ model- and design-based simulations,…

统计方法学 · 统计学 2026-05-12 Christoph Wiederkehr , Christian Heumann , Michael Schomaker

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,…

统计方法学 · 统计学 2026-04-07 Emily Wu , Elizabeth Rogawski McQuade , Mats Stensrud , Razieh Nabi , David Benkeser

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

统计方法学 · 统计学 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Revealing the underlying causal mechanisms in the real world is crucial for scientific and technological progress. Despite notable advances in recent decades, the lack of high-quality data and the reliance of traditional causal discovery…

机器学习 · 计算机科学 2026-02-17 Huaming Du , Tao Hu , Yijie Huang , Yu Zhao , Guisong Liu , Tao Gu , Gang Kou , Carl Yang

Observational data are often used to estimate real-world effectiveness and durability of vaccines. A sequence of trials can be emulated to draw inference from such data while minimizing selection bias, immortal time bias, and confounding.…

统计方法学 · 统计学 2025-09-03 Justin B. DeMonte , Bonnie E. Shook-Sa , Michael G. Hudgens

We propose a novel targeted maximum likelihood estimator (TMLE) for quantiles in semiparametric missing data models. Our proposed estimator is locally efficient, $\sqrt{n}$-consistent, asymptotically normal, and doubly robust, under…

统计方法学 · 统计学 2016-08-23 Iván Díaz

Missing data are ubiquitous in public health research. When estimating causal effects, there are well-established methods to address bias to due missing outcomes. Commonly, causal estimands are defined under hypothetical interventions to…

Tensor classification is gaining importance across fields, yet handling partially observed data remains challenging. In this paper, we introduce a novel approach to tensor classification with incomplete data, framed within high-dimensional…

机器学习 · 统计学 2024-11-01 Elynn Chen , Yuefeng Han , Jiayu Li

In a tie-breaker design (TBD), subjects with high values of a running variable are given some (usually desirable) treatment, subjects with low values are not, and subjects in the middle are randomized. TBDs are intermediate between…

统计方法学 · 统计学 2025-05-12 Tim P. Morrison , Art B. Owen

Vaccine randomized trials are typically designed to be blinded, ensuring that the estimated vaccine efficacy (VE) reflects the immunological effect of the vaccine. When blinding is broken, however, the estimated VE reflects not only the…

统计方法学 · 统计学 2026-03-12 Rachel Axelrod , Uri Obolski , Daniel Nevo

Identifying a biomarker or treatment-dose threshold that marks a specified level of risk is an important problem, especially in clinical trials. This risk, viewed as a function of thresholds and possibly adjusted for covariates, we call the…

统计方法学 · 统计学 2023-03-22 Lars van der Laan , Wenbo Zhang , Peter B. Gilbert

Unlike the commonly used parametric regression models such as mixed models, that can easily violate the required statistical assumptions and result in invalid statistical inference, target maximum likelihood estimation allows more realistic…

应用统计 · 统计学 2020-06-17 Chi Zhang , Jennifer Ahern , Mark J. van der Laan
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