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Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

Methodology · Statistics 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

The research described herewith is to re-visit the classical doubly robust estimation of average treatment effect by conducting a systematic study on the comparisons, in the sense of asymptotic efficiency, among all possible combinations of…

Statistics Theory · Mathematics 2020-06-01 Keli Guo , Chuyun Ye , Jun Fan , Lixing Zhu

The coefficient of determination, known as $R^2$, is commonly used as a goodness-of-fit criterion for fitting linear models. $R^2$ is somewhat controversial when fitting nonlinear models, although it may be generalised on a case-by-case…

Methodology · Statistics 2021-12-23 Mark Levene , Aleksejus Kononovicius

This paper considers conducting inference about the effect of a treatment (or exposure) on an outcome of interest. In the ideal setting where treatment is assigned randomly, under certain assumptions the treatment effect is identifiable…

Methodology · Statistics 2015-03-06 Amy Richardson , Michael G. Hudgens , Peter B. Gilbert , Jason P. Fine

The difference in restricted mean survival times between two groups is a clinically relevant summary measure. With observational data, there may be imbalances in confounding variables between the two groups. One approach to account for such…

Applications · Statistics 2014-10-17 Andrew Wey , David Vock , John Connett , Kyle Rudser

The Cox regression, a semi-parametric method of survival analysis, is extremely popular in biomedical applications. The proportional hazards assumption is a key requirement in the Cox model. To accommodate non-proportional hazards, we…

Methodology · Statistics 2022-06-13 Alexander Begun , Elena Kulinskaya

Time-to-event analysis often relies on prior parametric assumptions, or, if a non-parametric approach is chosen, Cox's model. This is inherently tied to the assumption of proportional hazards, with the analysis potentially invalidated if…

Methodology · Statistics 2023-03-15 Lucia Ameis , Oliver Kuß , Annika Hoyer , Kathrin Möllenhoff

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

In this paper we consider how to evaluate survival distribution predictions with measures of discrimination. This is a non-trivial problem as discrimination measures are the most commonly used in survival analysis and yet there is no clear…

Machine Learning · Statistics 2022-03-10 Raphael Sonabend , Andreas Bender , Sebastian Vollmer

Environmental epidemiologists are increasingly interested in establishing causality between exposures and health outcomes. A popular model for causal inference is the Rubin Causal Model (RCM), which typically seeks to estimate the average…

Applications · Statistics 2021-01-26 Keith W. Zirkle , Marie-Abele Bind , Jenise L. Swall , David C. Wheeler

In the last decades, it has been discussed the use of epidemiological prevalence ratio (PR) rather than odds ratio as a measure of association to be estimated in cross-sectional studies. The main difficulties in use of statistical models…

Sequential multiple assignment randomized trials mimic the actual treatment processes experienced by physicians and patients in clinical settings and inform the comparative effectiveness of dynamic treatment regimes. In such trials,…

Methodology · Statistics 2025-04-07 Zi Wang , Yu Cheng , Abdus S. Wahed

In paired randomized experiments individuals in a given matched pair may differ on prognostically important covariates despite the best efforts of practitioners. We examine the use of regression adjustment as a way to correct for persistent…

Methodology · Statistics 2017-11-27 Colin B. Fogarty

The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…

In many situations it could be interesting to ascertain whether nonparametric regression curves can be grouped, especially when confronted with a considerable number of curves. The proposed testing procedure allows to determine groups with…

Methodology · Statistics 2021-02-02 Nora M. Villanueva , Marta Sestelo , Celestino Ordóñez , Javier Roca-Pardiñas

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

Epidemiological investigations of regionally aggregated spatial data often involve detecting spatial health disparities among neighboring regions on a map of disease mortality or incidence rates. Analyzing such data introduces spatial…

Methodology · Statistics 2025-11-21 Kyle Lin Wu , Sudipto Banerjee

Standard measures of effect, including the risk ratio, the odds ratio, and the risk difference, are associated with a number of well-described shortcomings, and no consensus exists about the conditions under which investigators should…

Methodology · Statistics 2019-10-08 Anders Huitfeldt , Andrew Goldstein , Sonja A. Swanson

In recent years, cancer clinical trials have increasingly encountered non proportional hazards (NPH) scenarios, particularly with the emergence of immunotherapy. In randomized controlled trials comparing immunotherapy with conventional…

Applications · Statistics 2025-02-28 Takuto Iijima , Tomotaka Momozaki , Shuji Ando

In this paper we explore the very important case of finding a health measure in the lines of the survival curve but independent of the standard deviation parameter. This is done by estimating the health state curve and calculating the total…

Quantitative Methods · Quantitative Biology 2016-10-18 Christos H Skiadas , Charilaos Skiadas