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Studies of HPV vaccine efficacy usually record infections with vaccine targeted and nontargeted strains. Contrary to blinded randomized controlled trials, confounding bias can be a threat and risk compensation may occur in observational…

统计方法学 · 统计学 2026-05-13 Lola Etievant

In order to meet regulatory approval, pharmaceutical companies often must demonstrate that new vaccines reduce the total risk of a post-infection outcome like transmission, symptomatic disease, severe illness, or death in randomized,…

统计方法学 · 统计学 2024-09-23 Rob Trangucci , Yang Chen , Jon Zelner

Pathogens usually exist in heterogeneous variants, like subtypes and strains. Quantifying treatment effects on the different variants is important for guiding prevention policies and treatment development. Here we ground analyses of…

应用统计 · 统计学 2024-08-15 Gellert Perenyi , Mats J. Stensrud

Researchers increasingly have access to two types of data: (i) large observational datasets where treatment (e.g., class size) is not randomized but several primary outcomes (e.g., graduation rates) and secondary outcomes (e.g., test…

统计方法学 · 统计学 2025-05-29 Susan Athey , Raj Chetty , Guido Imbens

The advent and subsequent widespread availability of preventive vaccines has altered the course of public health over the past century. Despite this success, effective vaccines to prevent many high-burden diseases, including HIV, have been…

统计方法学 · 统计学 2020-11-17 Nima S. Hejazi , Mark J. van der Laan , Holly E. Janes , Peter B. Gilbert , David C. Benkeser

Adjustment for prognostic baseline variables can reduce bias due to covariate imbalance and increase efficiency in randomized trials. While the use of covariate adjustment in late-phase trials is justified by favorable large-sample…

统计方法学 · 统计学 2025-04-08 Ethan Ashby , Bo Zhang , Genevieve G Fouda , Youyi Fong , Holly Janes

Safe and effective preventive vaccines have the potential to help stem the HIV epidemic. The efficacy of such vaccines is typically measured in randomized, double-blind phase IIb/III trials and described as a reduction in newly acquired HIV…

统计方法学 · 统计学 2024-10-10 Yutong Jin , Alex Luedtke , Zoe Moodie , Holly Janes , David Benkeser

In vaccine studies for infectious diseases such as human immunodeficiency virus (HIV), the frequency and type of contacts between study participants and infectious sources are among the most informative risk factors, but are often not…

应用统计 · 统计学 2012-08-27 Yang Yang , Peter Gilbert , Ira M. Longini, , M. Elizabeth Halloran

Understanding vaccine effects on post-infection outcomes is critical for evaluating the full value proposition of a vaccine. However, defining appropriate causal effects on such outcomes is challenging because infection is affected by…

While the HVTN 505 trial showed no overall efficacy of the tested vaccine to prevent HIV infection over placebo, previous studies, biological theories, and the finding that immune response markers strongly correlated with infection in…

应用统计 · 统计学 2018-11-12 Peter B. Gilbert , Bryan S. Blette , Bryan E. Shepherd , Michael G. Hudgens

Knowing the true effect size of clinical interventions in randomised clinical trials is key to informing the public health policies. Vaccine efficacy is defined in terms of the relative risk or the ratio of two disease risks. However, only…

统计方法学 · 统计学 2021-02-03 Yasin Memari

Most statistical tests for treatment effects used in randomized clinical trials with survival outcomes are based on the proportional hazards assumption, which often fails in practice. Data from early exploratory studies may provide evidence…

统计理论 · 数学 2020-05-28 Andrea Arfé , Brian Alexander , Lorenzo Trippa

Given a collection of features available for inclusion in a predictive model, it may be of interest to quantify the relative importance of a subset of features for the prediction task at hand. For example, in HIV vaccine trials, participant…

统计方法学 · 统计学 2025-03-27 Charles J. Wolock , Peter B. Gilbert , Noah Simon , Marco Carone

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

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

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

Optimization of vaccine allocations among different segments of a heterogeneous population is important for enhancing the effectiveness of vaccination campaigns in reducing the burden of epidemics. Intuitively, it would seem that…

定量方法 · 定量生物学 2022-12-01 Nir Gavish , Guy Katriel

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

In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…

This paper aims to evaluate the potential cost-effectiveness of healthcare interventions against human papillomavirus (HPV). For this, we consider a two-sex epidemic model for the transmission dynamics of HPV which includes screening,…

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