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Instrumental variables (IV) are a useful tool for estimating causal effects in the presence of unmeasured confounding. IV methods are well developed for uncensored outcomes, particularly for structural linear equation models, where simple…

Methodology · Statistics 2019-02-01 Behzad Kianian , Jung In Kim , Jason P. Fine , Limin Peng

In conventional randomized controlled trials, adjustment for baseline values of covariates known to be at least moderately associated with the outcome increases the power of the trial. Recent work has shown particular benefit for more…

Methodology · Statistics 2023-11-27 James Willard , Shirin Golchi , Erica EM Moodie

There has recently been a growing interest in the development of statistical methods to compare medical costs between treatment groups. When cumulative cost is the outcome of interest, right-censoring poses the challenge of informative…

Methodology · Statistics 2017-05-25 Andrew J. Spieker , Arman Oganisian , Emily M. Ko , Jason A. Roy , Nandita Mitra

In this paper, we consider the problem of fair statistical inference involving outcome variables. Examples include classification and regression problems, and estimating treatment effects in randomized trials or observational data. The…

Machine Learning · Statistics 2018-01-23 Razieh Nabi , Ilya Shpitser

The Causal Roadmap outlines a systematic approach to asking and answering questions of cause-and-effect: define the quantity of interest, evaluate needed assumptions, conduct statistical estimation, and carefully interpret results. To…

Methodology · Statistics 2024-05-30 Nerissa Nance , Maya L. Petersen , Mark van der Laan , Laura B. Balzer

This paper considers robust modeling of the survival time for cancer patients. Accurate prediction can be helpful for developing therapeutic and care strategies. We propose a unified Expectation-Maximization approach combined with the…

Methodology · Statistics 2019-12-23 Yi Li , Muxuan Liang , Lu Mao , Sijian Wang

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

The problem of how to best select variables for confounding adjustment forms one of the key challenges in the evaluation of exposure effects in observational studies, and has been the subject of vigorous recent activity in causal inference.…

Methodology · Statistics 2021-12-02 Kelly Van Lancker , Oliver Dukes , Stijn Vansteelandt

A very classical problem in statistics is to test the stochastic superiority of one distribution to another. However, many existing approaches are developed for independent samples and, moreover, do not take censored data into account. We…

Methodology · Statistics 2023-06-19 Dennis Dobler , Kathrin Möllenhoff

Latent variable models are popularly used to measure latent factors (e.g., abilities and personalities) from large-scale assessment data. Beyond understanding these latent factors, the covariate effect on responses controlling for latent…

Methodology · Statistics 2026-01-12 Jing Ouyang , Chengyu Cui , Kean Ming Tan , Gongjun Xu

It is shown how to set up, conduct, and analyze large simulation studies with the new R package simsalapar = simulations simplified and launched parallel. A simulation study typically starts with determining a collection of input variables…

Computation · Statistics 2013-09-18 Marius Hofert , Martin Mächler

In the context of right-censored data, we study the problem of predicting the restricted time to event based on a set of covariates. Under a quadratic loss, this problem is equivalent to estimating the conditional Restricted Mean Survival…

Statistics Theory · Mathematics 2024-04-29 Ariane Cwiling , Vittorio Perduca , Olivier Bouaziz

We develop a multivariate cure survival model to estimate lifetime patterns of colorectal cancer screening. Screening data cover long periods of time, with sparse observations for each person. Some events may occur before the study begins…

Methodology · Statistics 2015-09-16 Yolanda Hagar , Danielle Harvey , Laurel Beckett

In situations where it is difficult to enroll patients in randomized controlled trials, external data can improve efficiency and feasibility. In such cases, adaptive trial designs could be used to decrease enrollment in the control arm of…

Methodology · Statistics 2020-10-02 Brian D. Segal , W. Katherine Tan

Many medical decisions involve the use of dynamic information collected on individual patients toward predicting likely transitions in their future health status. If accurate predictions are developed, then a prognostic mode can identify…

Methodology · Statistics 2018-02-22 Aasthaa Bansal , Patrick J. Heagerty

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,…

When choosing estimands and estimators in randomized clinical trials, caution is warranted as intercurrent events, such as - due to patients who switch treatment after disease progression, are often extreme. Statistical analyses may then…

Applications · Statistics 2023-03-13 Hege Michiels , An Vandebosch , Stijn Vansteelandt

The inverse probability weighting approach is popular for evaluating treatment effects in observational studies, but extreme propensity scores could bias the estimator and induce excessive variance. Recently, the overlap weighting approach…

Methodology · Statistics 2022-06-22 Chao Cheng , Fan Li , Laine Thomas , Fan Li

Factorial analyses offer a powerful nonparametric means to detect main or interaction effects among multiple treatments. For survival outcomes, e.g. from clinical trials, such techniques can be adopted for comparing reasonable…

Methodology · Statistics 2023-02-06 Takeshi Emura , Marc Ditzhaus , Dennis Dobler , Kenta Murotani

Predicting time-to-event outcomes in large databases can be a challenging but important task. One example of this is in predicting the time to a clinical outcome for patients in intensive care units (ICUs), which helps to support critical…

Computation · Statistics 2019-08-06 Yingying Xu , Joon Lee , Joel A. Dubin