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Assessing immune responses to study vaccines as surrogates of protection plays a central role in vaccine clinical trials. Motivated by three ongoing or pending HIV vaccine efficacy trials, we consider such surrogate endpoint assessment in a…

Applications · Statistics 2008-12-18 Li Qin , Peter B. Gilbert , Dean Follmann , Dongfeng Li

Evaluating observational estimators of causal effects demands information that is rarely available: unconfounded interventions and outcomes from the population of interest, created either by randomization or adjustment. As a result, it is…

Machine Learning · Computer Science 2024-10-29 Fredrik D. Johansson

This paper develops an individual-based stochastic network SIR model for the empirical analysis of the Covid-19 pandemic. It derives moment conditions for the number of infected and active cases for single as well as multigroup epidemic…

Econometrics · Economics 2022-01-05 M. Hashem Pesaran , Cynthia Fan Yang

Treatment effects estimated from a randomized controlled trial are local not only to the study population but also to the time at which the trial was conducted. The literature on generalizing experimental findings to new populations is…

Cluster randomization trials commonly employ multiple endpoints. When a single summary of treatment effects across endpoints is of primary interest, global hypothesis testing/effect estimation methods represent a common analysis strategy.…

Methodology · Statistics 2025-05-19 E. Davies Smith , V. Jairath , G. Zou

The dynamics of strongly immunizing childhood infections is still not well understood. Although reports of successful modeling of several incidence data records can be found in the literature, the key determinants of the observed temporal…

Populations and Evolution · Quantitative Biology 2012-10-09 Ganna Rozhnova , Ana Nunes

If we can lower the number of people needed to vaccinate for a community to be immune against contagious diseases, we can save resources and life. A key to reach such a lower threshold of immunization is to find and vaccinate people who,…

Populations and Evolution · Quantitative Biology 2012-10-10 Sungmin Lee , Luis E. C. Rocha , Fredrik Liljeros , Petter Holme

Real world observational data, together with causal inference, allow the estimation of causal effects when randomized controlled trials are not available. To be accepted into practice, such predictive models must be validated for the…

Several recently developed methods have the potential to harness machine learning in the pursuit of target quantities inspired by causal inference, including inverse weighting, doubly robust estimating equations and substitution estimators…

Many research questions concern treatment effects on outcomes that can recur several times in the same individual. For example, medical researchers are interested in treatment effects on hospitalizations in heart failure patients and sports…

Methodology · Statistics 2024-12-31 Matias Janvin , Jessica G. Young , Pål C. Ryalen , Mats J. Stensrud

Causal inference analysis is the estimation of the effects of actions on outcomes. In the context of healthcare data this means estimating the outcome of counter-factual treatments (i.e. including treatments that were not observed) on a…

Methodology · Statistics 2018-03-21 Yishai Shimoni , Chen Yanover , Ehud Karavani , Yaara Goldschmnidt

Countries officially record the number of COVID-19 cases based on medical tests of a subset of the population with unknown participation bias. For prevalence estimation, the official information is typically discarded and, instead, small…

Methodology · Statistics 2020-12-25 Stéphane Guerrier , Christoph Kuzmics , Maria-Pia Victoria-Feser

Previously, it has been shown that maximum-entropy models of immune-repertoire sequence can be used to determine a person's vaccination status. However, this approach has the drawback of requiring a computationally intensive method to…

Quantitative Methods · Quantitative Biology 2023-12-21 Josiah Couch , Rohit Arora , Jasper Braun , Joesph Kaplinsky , Elliot Hill , Anthony Li , Brett Altschul , Ramy Arnaout

Intercurrent (post-treatment) events occur frequently in randomized trials, and investigators often express interest in treatment effects that suitably take account of these events. A naive conditioning on intercurrent events does not have…

Methodology · Statistics 2021-11-17 Mats J. Stensrud , Oliver Dukes

Causal inference from observational data can be viewed as a missing data problem arising from a hypothetical population-scale randomized trial matched to the observational study. This links a target trial protocol with a corresponding…

Methodology · Statistics 2022-07-27 Andrew Yiu , Edwin Fong , Stephen Walker , Chris Holmes

Estimating heterogeneous treatment effects with machine learning has attracted substantial attention in both academic research and industrial practice. However, the two communities often evaluate models under markedly different conditions.…

Machine Learning · Computer Science 2026-05-26 George Panagopoulos

Longitudinal cohort studies, which follow a group of individuals over time, provide the opportunity to examine causal effects of complex exposures on long-term health outcomes. Utilizing data from multiple cohorts has the potential to add…

PURPOSE Providing rapid answers and early acces to patients to innovative treatments without randomized clinical trial (RCT) is growing, with benefit estimated from single-arm trials. This has become common in oncology, impacting the…

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

There are limited options to estimate the treatment effects of variables which are continuous and measured at multiple time points, particularly if the true dose-response curve should be estimated as closely as possible. However, these…

Methodology · Statistics 2024-10-11 Michael Schomaker , Helen McIlleron , Paolo Denti , Iván Díaz
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