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In clinical trials, principal stratification analysis is commonly employed to address the issue of truncation by death, where a subject dies before the outcome can be measured. However, in practice, many survivor outcomes may remain…

Methodology · Statistics 2025-07-08 Wei Li , Yuan Liu , Shanshan Luo , Zhi Geng

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

COVID-19 vaccines are highly efficacious at preventing symptomatic infection, severe disease, and death. Most of the evidence that COVID-19 vaccines also reduce transmission of SARS-CoV-2 is based on retrospective, observational studies.…

Applications · Statistics 2022-06-16 Marlena S. Bannick , Fei Gao , Elizabeth R. Brown , Holly E. Janes

During the COVID-19 pandemic, estimating the total deaths averted by vaccination has been of great public health interest. Instead of estimating total deaths averted by vaccination among both vaccinated and unvaccinated individuals, some…

Populations and Evolution · Quantitative Biology 2025-10-09 Katherine M. Jia , Christopher B. Boyer , Alyssa Bilinski , Marc Lipsitch

When assessing the causal effect of a binary exposure using observational data, confounder imbalance across exposure arms must be addressed. Matching methods, including propensity score-based matching, can be used to deconfound the causal…

Methodology · Statistics 2024-10-01 Ernesto Ulloa-Pérez , Marco Carone , Alex Luedtke

Principal stratification (PS) is a commonly used approach for understanding the mechanisms through which a treatment affects an outcome. The goal of this work is to extend the PS framework to studies with continuous treatments, which…

Methodology · Statistics 2025-05-20 Joseph Antonelli , Minxuan Wu , Fabrizia Mealli , Brenden Beck , Alessandra Mattei

Understanding waning of vaccine-induced protection is important for both immunology and public health. Population heterogeneities in underlying (pre-vaccination) susceptibility and vaccine response can cause measured vaccine effectiveness…

Populations and Evolution · Quantitative Biology 2023-08-02 Ariel Nikas , Hasan Ahmed , Veronika I. Zarnitsyna

The health effects of environmental exposures have been studied for decades, typically using standard regression models to assess exposure-outcome associations found in observational non-experimental data. We propose and illustrate a…

Applications · Statistics 2017-09-20 Marie-Abele C. Bind , Donald B. Rubin

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable…

Methodology · Statistics 2025-09-01 Jiaqi Tong , Chao Cheng , Guangyu Tong , Michael O. Harhay , Fan Li

Practitioners are interested in not only the average causal effect of the treatment on the outcome but also the underlying causal mechanism in the presence of an intermediate variable between the treatment and outcome. However, in many…

Methodology · Statistics 2016-02-04 Peng Ding , Jiannan Lu

Accurate estimates of malaria vaccine efficacy require a reliable definition of a malaria case. However, the symptoms of clinical malaria are unspecific, overlapping with other childhood illnesses. Additionally, children in endemic areas…

Methodology · Statistics 2019-08-27 Raiden B. Hasegawa , Dylan S. Small

The analysis of causal effects when the outcome of interest is possibly truncated by death has a long history in statistics and causal inference. The survivor average causal effect is commonly identified with more assumptions than those…

Methodology · Statistics 2020-03-24 Jaffer M. Zaidi , Eric J. Tchetgen Tchetgen , Tyler J. VanderWeele

Network-based interventions against epidemic spread are most powerful when the full network structure is known. However, in practice, resource constraints require decisions to be made based on partial network information. We investigated…

Applications · Statistics 2019-03-19 Yingrui Yang , Ashley McKhann , Sixing Chen , Guy Harling , Jukka-Pekka Onnela

The mitigation of an infectious disease spreading has recently gained considerable attention from the research community. It may be obtained by adopting sanitary measurements social rules, together with an extensive vaccination campaign.…

Social and Information Networks · Computer Science 2022-02-17 Francesco Petrizzelli , Pietro Hiram Guzzi , Tommaso Mazza

The ICH E9(R1) addendum (2019) proposed principal stratification (PS) as one of five strategies for dealing with intercurrent events. Therefore, understanding the strengths, limitations, and assumptions of PS is important for the broad…

Face masks have been widely used as a protective measure against COVID-19. However, pre-pandemic empirical studies have produced mixed statistical results on the effectiveness of masks against respiratory viruses. The implications of the…

Quantitative Methods · Quantitative Biology 2022-11-24 Pratyush K. Kollepara , Alexander F. Siegenfeld , Nassim Nicholas Taleb , Yaneer Bar-Yam

Randomization inference is a powerful tool in early phase vaccine trials when estimating the causal effect of a regimen against a placebo or another regimen. Randomization-based inference often focuses on testing either Fisher's sharp null…

Methodology · Statistics 2024-02-27 Zhe Chen , Xinran Li , Bo Zhang

In causal inference, principal stratification is a framework for dealing with a posttreatment intermediate variable between a treatment and an outcome, in which the principal strata are defined by the joint potential values of the…

Methodology · Statistics 2021-04-20 Zhichao Jiang , Peng Ding

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…

Methodology · Statistics 2025-04-08 Ethan Ashby , Bo Zhang , Genevieve G Fouda , Youyi Fong , Holly Janes
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