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We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

Target trial emulation has improved comparative effectiveness research by making the causal question, assumptions, and analysis plan explicit. However, target trial protocols are usually developed iteratively. After examining the data,…

Methodology · Statistics 2026-04-30 Mats Julius Stensrud

Randomized controlled trials are considered the gold standard to evaluate the treatment effect (estimand) for efficacy and safety. According to the recent International Council on Harmonisation (ICH)-E9 addendum (R1), intercurrent events…

Methodology · Statistics 2021-11-24 Junxiang Luo , Stephen J. Ruberg , Yongming Qu

Methods to handle missing data have been extensively explored in the context of estimation and descriptive studies, with multiple imputation being the most widely used method in clinical research. However, in the context of clinical risk…

Methodology · Statistics 2024-11-25 Junhui Mi , Rahul D. Tendulkar , Sarah M. C. Sittenfeld , Sujata Patil , Emily C. Zabor

Binary endpoints are common in clinical trials and conditional odds ratios have traditionally been used to assess treatment effects. However, the interpretation of odds ratios is difficult, they are non-collapsible and rely on strong…

Methodology · Statistics 2026-05-20 Martin Schnuerch , Alex Ocampo , Klaus Kähler Holst , Christian Stock

In various biomedical studies, analysis often focuses on data magnitudes, particularly when algebraic signs are irrelevant or lost. For repeated measures studies involving magnitude outcomes, incorporating random effects is essential as…

Methodology · Statistics 2025-07-16 Wen Teng , Niall D. Ferguson , Ewan C. Goligher , Anna Heath

We introduce a new sufficient statistic for the population parameter vector by allowing for the sampling design to first be selected at random amongst a set of candidate sampling designs. In contrast to the traditional approach in survey…

Statistics Theory · Mathematics 2014-11-11 Kyle Vincent , Christopher S. Henry

This paper provides a review of model selection and model averaging methods for multinomial probit models estimated using the MACML approach. The proposed approaches are partitioned into test based methods (mostly derived from the…

Methodology · Statistics 2017-04-04 Manuel Batram , Dietmar Bauer

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Under current policy decision making paradigm, we make or evaluate a policy decision by intervening different socio-economic parameters and analyzing the impact of those interventions. This process involves identifying the causal relation…

Methodology · Statistics 2020-01-07 Md Saiful Islam , Md Sarowar Morshed , Gary J. Young , Md. Noor-E-Alam

Data-driven individualized decision making has recently received increasing research interests. Most existing methods rely on the assumption of no unmeasured confounding, which unfortunately cannot be ensured in practice especially in…

Methodology · Statistics 2022-12-26 Zhengling Qi , Rui Miao , Xiaoke Zhang

Fluid approximation is a widely used approach for solving two-stage stochastic optimization problems, with broad applications in service system design such as call centers and healthcare operations. However, replacing the underlying random…

Optimization and Control · Mathematics 2025-12-19 Can Er , Mo Liu

Joint multivariate longitudinal and time-to-event data are gaining increasing attention in the biomedical sciences where subjects are followed over time to monitor the progress of a disease or medical condition. In the insurance context,…

Methodology · Statistics 2019-02-12 Edward W. Frees , Catalina Bolancé , Montserrat Guillen , Emiliano Valdez

Propensity score matching (PSM) has been widely used to mitigate confounding in observational studies, although complications arise when the covariates used to estimate the PS are only partially observed. Multiple imputation (MI) is a…

Applications · Statistics 2021-07-22 Albee Y. Ling , Maria E. Montez-Rath , Maya B. Mathur , Kris Kapphahn , Manisha Desai

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

Recent success in Deep Reinforcement Learning (DRL) methods has shown that policy optimization with respect to an off-policy distribution via importance sampling is effective for sample reuse. In this paper, we show that the use of…

Machine Learning · Computer Science 2023-02-07 Zichuan Lin , Xiapeng Wu , Mingfei Sun , Deheng Ye , Qiang Fu , Wei Yang , Wei Liu

Model selection aims to identify a sufficiently well performing model that is possibly simpler than the most complex model among a pool of candidates. However, the decision-making process itself can inadvertently introduce non-negligible…

Methodology · Statistics 2024-08-08 Yann McLatchie , Aki Vehtari

Time-to-event endpoints are central to evaluate treatment efficacy across many disease areas. Many trial protocols include interim analyses within group-sequential designs that control type I error via spending functions or boundary…

Methodology · Statistics 2026-01-19 Edoardo Ratti , Federico L. Perlino , Stefania Galimberti , Maria G. Valsecchi

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

In this article, we develop nonparametric inference methods for comparing survival data across two samples, which are beneficial for clinical trials of novel cancer therapies where long-term survival is a critical outcome. These therapies,…

Methodology · Statistics 2024-09-05 Yi-Cheng Tai , Weijing Wang , Martin T. Wells
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