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Double blind randomized controlled trials are traditionally seen as the gold standard for causal inferences as the difference-in-means estimator is an unbiased estimator of the average treatment effect in the experiment. The fact that this…

Methodology · Statistics 2021-08-25 Per Johansson , Mattias Nordin

We consider the problem of estimating covariance and precision matrices, and their associated discriminant coefficients, from normal data when the rank of the covariance matrix is strictly smaller than its dimension and the available sample…

Statistics Theory · Mathematics 2015-09-09 Didier Chételat , Martin T. Wells

Regression quantiles have asymptotic variances that depend on the conditional densities of the response variable given regressors. This paper develops a new estimate of the asymptotic variance of regression quantiles that leads any…

Econometrics · Economics 2019-09-27 Juan Carlos Escanciano , Chuan Goh

A new approach to adaptive design of clinical trials is proposed in a general multiparameter exponential family setting, based on generalized likelihood ratio statistics and optimal sequential testing theory. These designs are easy to…

Statistics Theory · Mathematics 2011-05-25 Jay Bartroff , Tze Leung Lai

Plausible identification of conditional average treatment effects (CATEs) may rely on controlling for a large number of variables to account for confounding factors. In these high-dimensional settings, estimation of the CATE requires…

Econometrics · Economics 2023-01-18 Adam Baybutt , Manu Navjeevan

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

Methodology · Statistics 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

In longitudinal panels and other regression models with unobserved effects, fixed effects estimation is often paired with cluster-robust variance estimation (CRVE) in order to account for heteroskedasticity and un-modeled dependence among…

Methodology · Statistics 2022-11-08 James E. Pustejovsky , Elizabeth Tipton

Composite endpoints are commonly used with an anticipation that clinically relevant endpoints as a whole would yield meaningful treatment benefits. The win ratio is a rank-based statistic to summarize composite endpoints, allowing…

Methodology · Statistics 2022-12-14 Di Zhang , Stephen R. Wisniewski , Jong-Hyeon Jeong

Baseline covariates in randomized experiments are often used in the estimation of treatment effects, for example, when estimating treatment effects within covariate-defined subgroups. In practice, however, covariate values may be missing…

Methodology · Statistics 2023-08-29 Gauri Kamat , Jerome P. Reiter

A population-averaged additive subdistribution hazards model is proposed to assess the marginal effects of covariates on the cumulative incidence function and to analyze correlated failure time data subject to competing risks. This approach…

Methodology · Statistics 2023-08-23 Xinyuan Chen , Denise Esserman , Fan Li

In randomized controlled trials, covariate adjustment can improve statistical power and reduce the required sample size compared with unadjusted estimators. Several regulatory agencies have released guidance on covariate adjustment, which…

Methodology · Statistics 2025-08-27 Takumi Kanata , Yasuhiro Hagiwara , Koji Oba

With advancement of medicine, alternative exposures or interventions are emerging with respect to a common outcome, and there are needs to formally test the difference in the associations of multiple exposures. We propose a duplication…

Methodology · Statistics 2024-12-04 Rikuta Hamaya , Peilu Wang , Lin Ge , Edward L. Giovannucci , Molin Wang

The win ratio is increasingly used in randomized trials due to its intuitive clinical interpretation, ability to incorporate the relative importance of composite endpoints, and its capacity for combining different types of outcomes (e.g.…

Methodology · Statistics 2026-02-02 Tracy Bergemann , Tim Hanson

Multi-arm bandit experimental designs are increasingly being adopted over standard randomized trials due to their potential to improve outcomes for study participants, enable faster identification of the best-performing options, and/or…

Methodology · Statistics 2025-06-04 Brian M Cho , Aurélien Bibaut , Nathan Kallus

Latent variable models (LVMs) are commonly used in psychology and increasingly used for analyzing brain imaging data. Such studies typically involve a small number of participants (n<100), where standard asymptotic results often fail to…

Methodology · Statistics 2020-06-15 Brice Ozenne , Patrick M. Fisher , Esben Budtz-Jørgensen

Randomized controlled trials generate experimental variation that can credibly identify causal effects, but often suffer from limited scale, while observational datasets are large, but often violate desired identification assumptions. To…

Econometrics · Economics 2023-12-27 George Z. Gui

In randomized clinical trials, adjusting for baseline covariates can improve credibility and efficiency for demonstrating and quantifying treatment effects. This article studies the augmented inverse propensity weighted (AIPW) estimator,…

Methodology · Statistics 2024-03-27 Marlena S. Bannick , Jun Shao , Jingyi Liu , Yu Du , Yanyao Yi , Ting Ye

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

Understanding variable dependence, particularly eliciting their statistical properties given a set of covariates, provides the mathematical foundation in practical operations management such as risk analysis and decision-making given…

Methodology · Statistics 2023-09-06 Yunyun Wang , Tatsushi Oka , Dan Zhu

Calibration is a vital aspect of the performance of risk prediction models, but research in the context of ordinal outcomes is scarce. This study compared calibration measures for risk models predicting a discrete ordinal outcome, and…