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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

Popular guidance on observational data analysis states that outcomes should be blinded when determining matching criteria or propensity scores. Such a blinding is informally said to maintain the "objectivity" of the analysis, and to prevent…

Methodology · Statistics 2022-07-26 Aaron Fisher

Statistical methods are developed for analysis of clinical and virus genetics data from phase 3 randomized, placebo-controlled trials of vaccines against novel coronavirus COVID-19. Vaccine efficacy (VE) of a vaccine to prevent COVID-19…

Methodology · Statistics 2022-01-25 Fei Heng , Yanqing Sun , Peter B. Gilbert

Understanding how vaccine effectiveness (VE) changes over time can provide evidence-based guidance for public health decision making. While commonly reported by practitioners, time-varying VE estimates obtained using Cox regression are vul-…

Estimating population-level effects of a vaccine is challenging because there may be interference, i.e., the outcome of one individual may depend on the vaccination status of another individual. Partial interference occurs when individuals…

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

Applications · Statistics 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

Measurement error in observational datasets can lead to systematic bias in inferences based on these datasets. As studies based on observational data are increasingly used to inform decisions with real-world impact, it is critical that we…

Machine Learning · Statistics 2019-01-29 Roy Adams , Yuelong Ji , Xiaobin Wang , Suchi Saria

Clinical trials of a vaccine during an epidemic face particular challenges, such as the pressure to identify an effective vaccine quickly to control the epidemic, and the effect that time-space-varying infection incidence has on the power…

Populations and Evolution · Quantitative Biology 2021-05-21 Rob Johnson , Chris Jackson , Anne Presanis , Sofia S. Villar , Daniela De Angelis

Understanding how vaccines perform against different pathogen genotypes is crucial for developing effective prevention strategies, particularly for highly genetically diverse pathogens like HIV. Sieve analysis is a statistical framework…

Methodology · Statistics 2025-12-11 James Peng , Michal Juraska , Pamela A. Shaw , Peter B. Gilbert

While conducting probabilistic surveys is the gold standard for assessing vaccine coverage, implementing these surveys poses challenges for global health. There is a need for more convenient option that is more affordable and practical.…

Applications · Statistics 2026-03-30 Nathaniel Dyrkton , Shomoita Alam , Susan Shepherd , Ibrahim Sana , Kevin Phelan , Jay JH Park

We systematically investigate issues due to mis-specification that arise in estimating causal effects when (treatment) interference is informed by a network available pre-intervention, i.e., in situations where the outcome of a unit may…

Methodology · Statistics 2018-10-22 Vishesh Karwa , Edoardo M. Airoldi

Subgroup analyses are common in epidemiologic and clinical research. Unfortunately, restriction to subgroup members to test for heterogeneity can yield imprecise effect estimates. If the true effect differs between members and non-members…

In oncology, phase II studies are crucial for clinical development plans as such studies identify potent agents with sufficient activity to continue development in the subsequent phase III trials. Traditionally, phase II studies are…

Methodology · Statistics 2023-08-08 Takuya Yoshimoto , Satoru Shinoda , Kouji Yamamoto , Kouji Tahata

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

From scientific experiments to online A/B testing, the previously observed data often affects how future experiments are performed, which in turn affects which data will be collected. Such adaptivity introduces complex correlations between…

Machine Learning · Statistics 2018-01-03 Xinkun Nie , Xiaoying Tian , Jonathan Taylor , James Zou

When constructing a model to estimate the causal effect of a treatment, it is necessary to control for other factors which may have confounding effects. Because the ignorability assumption is not testable, however, it is usually unclear…

Methodology · Statistics 2022-09-07 Spencer Woody , Carlos M. Carvalho , Jared S. Murray

The development of high-throughput sequencing and targeted therapies has led to the emergence of personalized medicine: a patient's molecular profile or the presence of a specific biomarker of drug response will correspond to a treatment…

Applications · Statistics 2020-05-27 Jonas Béal , Aurélien Latouche

Motivation: In predicting HIV therapy outcomes, a critical clinical question is whether using historical information can enhance predictive capabilities compared with current or latest available data analysis. This study analyses whether…

Background: Pairwise and network meta-analyses using fixed effect and random effects models are commonly applied to synthesise evidence from randomised controlled trials. The models differ in their assumptions and the interpretation of the…

Methodology · Statistics 2017-08-04 Shijie Ren , Jeremy E. Oakley , John W. Stevens

We consider identification and inference for the average treatment effect and heterogeneous treatment effect conditional on observable covariates in the presence of unmeasured confounding. Since point identification of these treatment…

Methodology · Statistics 2025-03-04 Kan Chen , Jeffrey Zhang , Bingkai Wang , Dylan S. Small