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Knowledge of the protection afforded by vaccines might, in some circumstances, modify a vaccinated individual's behaviour, potentially increasing exposure to pathogens and hindering effectiveness. Although vaccine studies typically do not…

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

In the presence of competing events, many investigators are interested in a direct treatment effect on the event of interest that does not capture treatment effects on competing events. Classical survival analysis methods that treat…

Methodology · Statistics 2025-10-29 Takuya Kawahara , Sean McGrath , Jessica G Young

It is often of interest to study the association between covariates and the cumulative incidence of a right-censored time-to-event outcome. When time-varying covariates are measured on a fixed discrete time scale, it is desirable to account…

Methodology · Statistics 2026-04-28 Hongxiang Qiu , Marco Carone , Alex Luedtke , Peter B. Gilbert

Estimating the causal effect of a time-varying public health intervention on the course of an infectious disease epidemic is an important methodological challenge. During the COVID-19 pandemic, researchers attempted to estimate the effects…

Applications · Statistics 2025-08-20 Yichi Zhang , Forrest W. Crawford

Causal inference is best understood using potential outcomes. This use is particularly important in more complex settings, that is, observational studies or randomized experiments with complications such as noncompliance. The topic of this…

Statistics Theory · Mathematics 2007-06-13 Donald B. Rubin

Competing risk is a common phenomenon when dealing with time-to-event outcomes in biostatistical applications. An attractive estimand in this setting is the "number of life-years lost due to a specific cause of death", Andersen et al.…

Methodology · Statistics 2025-02-04 Simon Christoffer Ziersen , Torben Martinussen

In situations with non-manipulable exposures, interventions can be targeted to shift the distribution of intermediate variables between exposure groups to define interventional disparity indirect effects. In this work, we present a…

Methodology · Statistics 2023-05-18 Helene C. W. Rytgaard , Amalie Lykkemark Møller , Thomas A. Gerds

This study aims to statistically assess the effectiveness of vaccination against SARS-CoV-2. It is indispensable to investigate the relationship between Covid-19 deadliness and vaccination in order to study the impact of vaccine in…

Quantitative Methods · Quantitative Biology 2022-02-04 MD. Rayhan , M. Abdullah-Al-Wadud , M. Helal Uddin Ahmed

Estimands can help clarify the interpretation of treatment effects and ensure that estimators are aligned to the study's objectives. Cluster randomised trials require additional attributes to be defined within the estimand compared to…

Methodology · Statistics 2024-02-23 Brennan C Kahan , Bryan Blette , Michael Harhay , Scott Halpern , Vipul Jairath , Andrew Copas , Fan Li

Inference for causal effects can benefit from the availability of an instrumental variable (IV) which, by definition, is associated with the given exposure, but not with the outcome of interest other than through a causal exposure effect.…

Methodology · Statistics 2012-01-13 Stijn Vansteelandt , Jack Bowden , Manoochehr Babanezhad , Els Goetghebeur

We discuss some causal estimands used to study racial discrimination in policing. A central challenge is that not all police-civilian encounters are recorded in administrative datasets and available to researchers. One possible solution is…

Applications · Statistics 2021-06-16 Qingyuan Zhao , Luke J Keele , Dylan S Small , Marshall M Joffe

Streaming data routinely generated by mobile phones, social networks, e-commerce, and electronic health records present new opportunities for near real-time surveillance of the impact of an intervention on an outcome of interest via causal…

Methodology · Statistics 2021-11-30 Xu Shi , Lan Luo

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

Machine Learning · Computer Science 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

Early on during a pandemic, vaccine availability is limited, requiring prioritisation of different population groups. Evaluating vaccine allocation is therefore a crucial element of pandemics response. In the present work, we develop a…

Knowing whether vaccine protection wanes over time is important for health policy and drug development. However, quantifying waning effects is difficult. A simple contrast of vaccine efficacy at two different times compares different…

Methodology · Statistics 2025-10-15 Matias Janvin , Mats J. Stensrud

Interference bias is a major impediment to identifying causal effects in real-world settings. For example, vaccination reduces the transmission of a virus in a population such that everyone benefits -- even those who are not treated. This…

Methodology · Statistics 2025-03-25 Michael O'Riordan , Ciarán M. Gilligan-Lee

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

Methodology · Statistics 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

Prediction models are used amongst others to inform medical decisions on interventions. Typically, individuals with high risks of adverse outcomes are advised to undergo an intervention while those at low risk are advised to refrain from…

In many randomized clinical trials of therapeutics for COVID-19, the primary outcome is an ordinal categorical variable, and interest focuses on the odds ratio (active agent vs. control) under the assumption of a proportional odds model.…

Methodology · Statistics 2021-08-25 Anastasios A. Tsiatis , Marie Davidian , Shannon T. Holloway