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The preclinical stage of many neurodegenerative diseases can span decades before symptoms become apparent. Understanding the sequence of preclinical biomarker changes provides a critical opportunity for early diagnosis and effective…

Methodology · Statistics 2024-06-11 Yizhen Xu , Scott Zeger , Zheyu Wang

Historical data about disease outcomes can be integrated into the analysis of clinical trials in many ways. We build on existing literature that uses prognostic scores from a predictive model to increase the efficiency of treatment effect…

Methodology · Statistics 2020-12-25 David Walsh , Alejandro Schuler , Diana Hall , Jon Walsh , Charles Fisher

In this paper we develop a general framework for quantifying how binary risk factors jointly influence a binary outcome. Our key result is an additive expansion of odds ratios as a sum of marginal effects and interaction terms of varying…

Survival analysis is a statistical technique used to estimate the time until an event occurs. Although it is applied across a wide range of fields, adjusting for reporting delays under practical constraints remains a significant challenge…

Machine Learning · Statistics 2025-10-27 Yuta Shikuri , Hironori Fujisawa

In cancer epidemiology using population-based data, regression models for the excess mortality hazard is a useful method to estimate cancer survival and to describe the association between prognosis factors and excess mortality. This method…

Methodology · Statistics 2019-04-19 Francisco J. Rubio , Bernard Rachet , Roch Giorgi , Camille Maringe , Aurelien Belot

Accurate cancer risk estimation is crucial to clinical decision-making, such as identifying high-risk people for screening. However, most existing cancer risk models incorporate data from epidemiologic studies, which usually cannot…

Methodology · Statistics 2023-10-26 Lingxiao Wang , Yan Li , Barry Graubard , Hormuzd Katki

Exposure measurement error is a ubiquitous but often overlooked challenge in causal inference with observational data. Existing methods accounting for exposure measurement error largely rely on restrictive parametric assumptions, while…

The progression-free survival ratio (PFSr) is a widely used measure in personalized oncology trials. It evaluates the effectiveness of treatment by comparing two consecutive event times - one under standard therapy and one under an…

Methodology · Statistics 2025-12-23 Merle Munko , Simon Mack , Marc Ditzhaus , Stefan Fröhling , Dennis Dobler , Dominic Edelmann

A case-control study is designed to help determine if an exposure is associated with an outcome. However, since case-control studies are retrospective, they are often subject to recall bias. Recall bias can occur when study subjects do not…

Methodology · Statistics 2021-02-23 Kwonsang Lee , Francesca Dominici

Safety analyses in terms of adverse events (AEs) are an important aspect of benefit-risk assessments of therapies. Compared to efficacy analyses AE analyses are often rather simplistic. The probability of an AE of a specific type is…

Applications · Statistics 2021-05-20 Regina Stegherr , Claudia Schmoor , Michael Lübbert , Tim Friede , Jan Beyersmann

When an infection spreads in a community, an individual's probability of becoming infected depends on both her susceptibility and exposure to the contagion through contact with others. While one often has knowledge regarding an individual's…

Social and Information Networks · Computer Science 2017-12-05 Maggie Makar , John Guttag , Jenna Wiens

We discuss a shift in perspective from traditional approaches to breast cancer risk prediction: modelling families rather than individuals as unit of analysis. By investigating the latent familial risk underlying breast cancer diagnoses, we…

Applications · Statistics 2025-08-25 Maria Veronica Vinattieri , Marco Bonetti , Kamila Czene

This paper develops an empirical balancing approach for the estimation of treatment effects under two-sided noncompliance using a binary conditionally independent instrumental variable. The method weighs both treatment and outcome…

Econometrics · Economics 2020-07-10 Phillip Heiler

Recently, we have proposed a new illness-death model that comprises a state of undiagnosed chronic disease preceding the diagnosed disease. Based on this model, the question arises how case-finding can be assessed in the presence of…

Populations and Evolution · Quantitative Biology 2016-03-22 Ralph Brinks

With the advent of effective pre-exposure prophylaxis agents, active-controlled HIV prevention trials have become a common study design. Nevertheless, estimating absolute efficacy relative to a placebo remains important. In this paper, we…

Genomic imprinting and maternal effects are two epigenetic factors that have been increasingly explored for their roles in the etiology of complex diseases. This is part of a concerted effort to find the "missing heritability." Accordingly,…

Applications · Statistics 2013-04-18 Jingyuan Yang , Shili Lin

Two simple agent based models are often employed in epidemic studies: the susceptible-infected (SI) and the susceptible-infected-susceptible (SIS). Both models describe the time evolution of infectious diseases in networks in which vertices…

In causal inference, the correct formulation of the scientific question of interest is a crucial step. Here we apply the estimand framework to a comparison of the outcomes of patient-level clinical trials and observational data to help…

In this paper, we develop a semiparametric sensitivity analysis approach designed to address unmeasured confounding in observational studies with time-to-event outcomes. We target estimation of the marginal distributions of potential…

Methodology · Statistics 2025-11-21 Linda Amoafo , Shiyao Xu , Elizabeth Platz , Daniel Scharfstein

Causal inference methods can be applied to estimate the effect of a point exposure or treatment on an outcome of interest using data from observational studies. For example, in the Women's Interagency HIV Study, it is of interest to…

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