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Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Difference in proportions is frequently used to measure treatment effect for binary outcomes in randomized clinical trials. The estimation of difference in proportions can be assisted by adjusting for prognostic baseline covariates to…

Methodology · Statistics 2023-08-31 Jialuo Liu , Dong Xi

Marginal structural models (MSMs) are often used to estimate causal effects of treatments on survival time outcomes from observational data when time-dependent confounding may be present. They can be fitted using, e.g., inverse probability…

Methodology · Statistics 2023-12-27 Shaun R Seaman , Ruth H Keogh

In randomized experiments, regression adjustment can improve the precision of average treatment effect (ATE) estimation using covariates without requiring a correctly specified outcome model. Although well studied in low-dimensional…

Statistics Theory · Mathematics 2026-04-28 Dogyoon Song

We consider a regression modeling of the quantiles of residual life, remaining lifetime at a specific time. We propose a smoothed induced version of the existing non-smooth estimating equations approaches for estimating regression…

Computation · Statistics 2022-05-03 Kyu Hyun Kim , Daniel J. Caplan , Sangwook Kang

The mixture cure rate model is the most commonly used cure rate model in the literature. In the context of mixture cure rate model, the standard approach to model the effect of covariates on the cured or uncured probability is to use a…

Methodology · Statistics 2022-08-23 Suvra Pal , Yingwei Peng , Sandip Barui , Pei Wang

The quantile residual lifetime (QRL) regression is an attractive tool for assessing covariate effects on the distribution of residual life expectancy, which is often of interest in clinical studies. When the study subjects are exposed to…

Methodology · Statistics 2025-03-04 Tonghui Yu , Liming Xiang , Jong-Hyeon Jeong

Covariate adaptive randomization (CAR) procedures are extensively used to reduce the likelihood of covariate imbalances occurring in clinical trials. In literatures, a lot of CAR procedures have been proposed so that the specified…

Statistics Theory · Mathematics 2026-03-10 Zhang Li-Xin

In the context of right-censored and interval-censored data we develop asymptotic formulas to compute pseudo-observations for the survival function and the Restricted Mean Survival Time (RMST). Those formulas are based on the original…

Statistics Theory · Mathematics 2023-02-07 Olivier Bouaziz

Randomized controlled trials (RCTs) with binary primary endpoints introduce novel challenges for inferring the causal effects of treatments. The most significant challenge is non-collapsibility, in which the conditional odds ratio estimand…

Methodology · Statistics 2024-03-01 Yunfan Li , Arman Sabbaghi , Jonathan R. Walsh , Charles K. Fisher

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

We propose a nonparametric bivariate time-varying coefficient model for longitudinal measurements with the occurrence of a terminal event that is subject to right censoring. The time-varying coefficients capture the longitudinal…

Methodology · Statistics 2021-11-10 Yue Wang , Bin Nan , Jack D. Kalbfleisch

In randomized trials involving multiple treatments, bivariate survival outcomes present significant analytical challenges for making decisions. This paper addresses the problem of deriving optimal individualized treatment rules to maximize…

Machine Learning · Statistics 2026-05-29 Kun Ren , Yifan Cui , Wen Su

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

Covariate imbalance between treatment groups makes it difficult to compare cumulative incidence curves in competing risk analyses. In this paper we discuss different methods to estimate adjusted cumulative incidence curves including inverse…

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

Methodology · Statistics 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

We consider planning longitudinal covariate measurements in follow-up studies where covariates are time-varying. We assume that the entire cohort cannot be selected for longitudinal measurements due to financial limitations and study how a…

Methodology · Statistics 2024-07-03 Jaakko Reinikainen , Juha Karvanen

Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…

Methodology · Statistics 2021-03-24 Andrew Ying , Eric J. Tchetgen Tchetgen

The authors propose robust adaptive strategies based on stochastic minimax optimization for a series of simulated treatments on a one-dimensional patient phantom. The plan applied during the first fractions should be able to handle…

Optimization and Control · Mathematics 2018-10-01 Michelle Böck , Anders Forsgren , Kjell Eriksson , Björn Hårdemark

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li