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Causal inference on multiple non-independent outcomes raises serious challenges, because multivariate techniques that properly account for the outcome's dependence structure need to be considered. We focus on the case of binary outcomes…

Methodology · Statistics 2018-05-11 Monia Lupparelli , Alessandra Mattei

Pragmatic randomized trials are designed to provide evidence for clinical decision-making rather than regulatory approval. Common features of these trials include the inclusion of heterogeneous or diverse patient populations in a wide range…

Methodology · Statistics 2019-11-20 Eleanor J. Murray , Sonja A. Swanson , Miguel A. Hernán

Controlling and understanding epidemic outbreaks has recently drawn great interest in a large spectrum of research communities. Vaccination is one of the most well-established and effective strategies in order to contain an epidemic. In the…

Social and Information Networks · Computer Science 2023-05-29 Sourin Chatterjee , Ahad N. Zehmakan

Vaccination campaigns have saved thousands of lives, reaching the farthest places in the world. These campaigns have required substantial investments and accurate coordination between several actors within the vaccine supply chain. Despite…

The efficacy of an intervention can be assessed by randomizing patients to different diagnostic tests instead of directly to an intervention and control. This principle is applied by allocating individuals to intervention if the test result…

Applications · Statistics 2022-01-11 Huw Llewelyn

This study demonstrates the existence of a testable condition for the identification of the causal effect of a treatment on an outcome in observational data, which relies on two sets of variables: observed covariates to be controlled for…

Econometrics · Economics 2026-05-20 Martin Huber , Jannis Kueck

Three critical issues for causal inference that often occur in modern, complicated experiments are interference, treatment nonadherence, and missing outcomes. A great deal of research efforts has been dedicated to developing causal…

Methodology · Statistics 2023-04-06 Yuki Ohnishi , Arman Sabbaghi

Post-treatment variables often complicate causal inference. They appear in many scientific problems, including noncompliance, truncation by death, mediation, and surrogate endpoint evaluation. Principal stratification is a strategy to…

Methodology · Statistics 2024-04-04 Sizhu Lu , Zhichao Jiang , Peng Ding

Motivated by massive outbreaks of COVID-19 that occurred even in populations with high vaccine uptake, we propose a novel multi-population temporal network model for the spread of recurrent epidemic diseases. We study the effect of human…

Dynamical Systems · Mathematics 2022-09-19 Kathinka Frieswijk , Lorenzo Zino , Ming Cao

In clinical trials, it is often of interest to understand the principal causal effect (PCE), the average treatment effect for a principal stratum (a subset of patients defined by the potential outcomes of one or more post-baseline…

Methodology · Statistics 2022-10-06 Yongming Qu , Ilya Lipkovich , Stephen J. Ruberg

Real world data is an increasingly utilized resource for post-market monitoring of vaccines and provides insight into real world effectiveness. However, outside of the setting of a clinical trial, heterogeneous mechanisms may drive observed…

We introduce a novel compartmental model accounting for the effects of vaccine efficacy, deployment rates and timing of initiation of deployment. We simulate different scenarios and initial conditions, and we find that higher abundancy and…

Populations and Evolution · Quantitative Biology 2022-06-28 Omar El Deeb , Maya Jalloul

Meta-analysis, by synthesizing effect estimates from multiple studies conducted in diverse settings, stands at the top of the evidence hierarchy in clinical research. Yet, conventional approaches based on fixed- or random-effects models…

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

Here we treat the transmission of disease through a population as a standard Galton-Watson branching process, modified to take the presence of vaccination into account. Vaccination reduces the number of secondary infections produced per…

Probability · Mathematics 2021-06-15 Arni S. R. Srinivasa Rao , Chris T. Bauch

We address discrete-marks survival analysis, also known as categorical sieve analysis, for a setting of a randomized placebo-controlled treatment intervention to prevent infection by a pathogen to which multiple exposures are possible, with…

Methodology · Statistics 2014-01-06 Paul T. Edlefsen

Vaccination is essential for the management of infectious diseases, many of which continue to pose devastating public health and economic challenges across the world. However, many vaccines are imperfect having only a partial protective…

Populations and Evolution · Quantitative Biology 2022-08-04 Hetsron L. Nyandjo-Bamen , Jean Marie Ntaganda , Aurelien Tellier , Olivier Menoukeu-Pamen

In neoadjuvant trials on early-stage breast cancer, patients are usually randomized into a control group and a treatment group with an additional target therapy. Early efficacy of the new regimen is assessed via the binary pathological…

Methodology · Statistics 2022-04-04 Xiaoqing Tan , Judah Abberbock , Priya Rastogi , Gong Tang

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

Methodology · Statistics 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson
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