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Although randomized controlled trials (RCTs) are a cornerstone of comparative effectiveness, they typically have much smaller sample size than observational studies because of financial and ethical considerations. Therefore there is…

统计方法学 · 统计学 2023-11-16 Lauren D. Liao , Emilie Højbjerre-Frandsen , Alan E. Hubbard , Alejandro Schuler

Analyses of randomised trials are often based on regression models which adjust for baseline covariates, in addition to randomised group. Based on such models, one can obtain estimates of the marginal mean outcome for the population under…

统计方法学 · 统计学 2017-07-17 Jonathan W. Bartlett

In this paper, we study the estimation and inference of the quantile treatment effect under covariate-adaptive randomization. We propose two estimation methods: (1) the simple quantile regression and (2) the inverse propensity score…

统计方法学 · 统计学 2020-02-26 Yichong Zhang , Xin Zheng

This paper investigates the size performance of Wald tests for CAViaR models (Engle and Manganelli, 2004). We find that the usual estimation strategy on test statistics yields inaccuracies. Indeed, we show that existing density estimation…

计量经济学 · 经济学 2021-02-03 Alain Hecq , Li Sun

In epidemiological cohort studies, the relative risk (also known as risk ratio) is a major measure of association to summarize the results of two treatments or exposures. Generally, it measures the relative change in disease risk as a…

统计方法学 · 统计学 2022-07-05 Gopal Nath , Krishna K. Saha , Suojin Wang

Propensity score (PS) methods are widely used to estimate treatment effects in non-randomized studies. Variance is typically estimated using sandwich or bootstrap methods, which can either treat the PS as estimated or fixed. The latter is…

统计方法学 · 统计学 2025-11-17 Baoshan Zhang , Sean M. O'Brien , Yuan Wu , Laine E. Thomas

Weighting procedures are used in observational causal inference to adjust for covariate imbalance within the sample. Common practice for inference is to estimate robust standard errors from a weighted regression of outcome on treatment.…

统计方法学 · 统计学 2025-07-29 Erin Hartman , Chad Hazlett , Arisa Sadeghpour

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

统计方法学 · 统计学 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

计量经济学 · 经济学 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

We consider the problem of testing for differences in group-specific slopes between the selected groups in panel data identified via k-means clustering. In this setting, the classical Wald-type test statistic is problematic because it…

统计方法学 · 统计学 2025-11-07 Chuang Wan , Jiajun Sun , Xingbai Xu

Random-effects meta-analyses have been widely applied in evidence synthesis for various types of medical studies. However, standard inference methods (e.g. restricted maximum likelihood estimation) usually underestimate statistical errors…

统计方法学 · 统计学 2019-05-13 Shonosuke Sugasawa , Hisashi Noma

Remarkable progress has been made in difference-in-differences (DID) approaches to causal inference that estimate the average effect of a treatment on the treated (ATT). Of these, the semiparametric DID (SDID) approach incorporates a…

统计方法学 · 统计学 2026-03-09 Takamichi Baba , Yoshiyuki Ninomiya

Two commonly used methods for improving precision and power in clinical trials are stratified randomization and covariate adjustment. However, many trials do not fully capitalize on the combined precision gains from these two methods, which…

统计方法学 · 统计学 2020-09-04 Bingkai Wang , Ryoko Susukida , Ramin Mojtabai , Masoumeh Amin-Esmaeili , Michael Rosenblum

The primary goal of randomized trials is to compare the effects of different interventions on some outcome of interest. In addition to the treatment assignment and outcome, data on baseline covariates, such as demographic characteristics or…

应用统计 · 统计学 2014-01-09 Alisa J. Stephens , Eric J. Tchetgen Tchetgen , Victor De Gruttola

Instrumental variables (IV) regression is widely used to estimate causal treatment effects in settings where receipt of treatment is not fully random, but there exists an instrument that generates exogenous variation in treatment exposure.…

计量经济学 · 经济学 2021-08-10 Stephen Coussens , Jann Spiess

Conditional power calculations are frequently used to guide the decision whether or not to stop a trial for futility or to modify planned sample size. These ignore the information in short-term endpoints and baseline covariates, and thereby…

统计方法学 · 统计学 2019-04-11 Kelly Van Lancker , An Vandebosch , Stijn Vansteelandt

Companies offering web services routinely run randomized online experiments to estimate the causal impact associated with the adoption of new features and policies on key performance metrics of interest. These experiments are used to…

统计方法学 · 统计学 2023-07-13 Lorenzo Masoero , Doug Hains , James McQueen

In confirmatory clinical trials, survival outcomes are frequently studied and interim analyses for efficacy and/or futility are often desirable. Methods such as the log rank test and Cox regression model are commonly used to compare…

统计方法学 · 统计学 2024-03-27 Peter Zhang , Brent Logan , Michael Martens

Adaptive designs for multi-armed clinical trials have become increasingly popular recently in many areas of medical research because of their potential to shorten development times and to increase patient response. However, developing…

应用统计 · 统计学 2017-03-16 Adam Smith , Sofia S. Villar

This paper presents a unified approach based on Wasserstein distance to derive concentration bounds for empirical estimates for two broad classes of risk measures defined in the paper. The classes of risk measures introduced include as…

统计理论 · 数学 2022-05-11 Prashanth L. A. , Sanjay P. Bhat