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In this paper we present a data-adaptive estimation procedure for estimation of average treatment effects in a time-to-event setting based on generalized random forests. In these kinds of settings, the definition of causal effect parameters…

Methodology · Statistics 2021-04-28 Helene C. W. Rytgaard , Claus T. Ekstrøm , Lars V. Kessing , Thomas A. Gerds

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

As alternatives to the time-to-first-event analysis of composite endpoints, the {\it net benefit} (NB) and the {\it win ratio} (WR) -- which assess treatment effects using prioritized component outcomes based on clinical importance -- have…

Methodology · Statistics 2020-11-24 Roland A. Matsouaka , Adrian Coles

The problem of estimation of the proportion of units with a given attribute in a~finite population is considered. From the population a sample is drawn due to the simple random sampling without replacement. There are limited funds for…

Statistics Theory · Mathematics 2019-03-26 Dominik Sieradzki , Wojciech Zieliński

The primary analysis for longitudinal randomized controlled trials (RCTs) often compares treatment groups at the last timepoint, referred to as the landmark time. Assuming data are normally distributed and missing at random, the mixed model…

Methodology · Statistics 2026-03-18 Guangyong Zou , Shi-Fang Qui , Joshua Zou , Emma Davies Smith , Yun-Hee Choi , Yuhan Bi

In observational causal inference, in order to emulate a randomized experiment, weights are used to render treatments independent of observed covariates. This property is known as balance; in its absence, estimated causal effects may be…

Methodology · Statistics 2020-07-16 David Arbour , Drew Dimmery , Arjun Sondhi

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…

Effect size estimates are thought to capture the collective, two-way response to an intervention or exposure in a three-way problem among the intervention/exposure, various confounders and the outcome. For meaningful causal inference from…

Methodology · Statistics 2024-02-08 Orestis Loukas , Ho Ryun Chung

The Average Treatment Effect (ATE) is a global measure of the effectiveness of an experimental treatment intervention. Classical methods of its estimation either ignore relevant covariates or do not fully exploit them. Moreover, past work…

Methodology · Statistics 2013-11-05 Emil Pitkin , Richard Berk , Lawrence Brown , Andreas Buja , Ed George , Kai Zhang , Linda Zhao

Principal stratification is a framework for making sense of causal effects conditioned on variables that may themselves have been affected by the treatment. For instance, in an evaluation of an educational intervention, some subjects in the…

Methodology · Statistics 2026-05-05 Adam C. Sales , Kirk P. Vanacore , Erin R. Ottmar

A new matching method is proposed for the estimation of the average treatment effect of social policy interventions (e.g., training programs or health care measures). Given an outcome variable, a treatment and a set of pre-treatment…

Statistics Theory · Mathematics 2007-06-13 Stefano Iacus , Giuseppe Porro

Forecast of football outcomes in terms of Home Win, Draw and Away Win relies largely on ex ante probability elicitation of these events and ex post verification of them via computation of probability scoring rules (Brier, Ranked…

Applications · Statistics 2024-08-09 Jean-Louis Foulley

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

Methodology · Statistics 2022-11-10 Wenqi Shi , Xi Lin

Prognostic covariate adjustment (PROCOVA) is a two-sample two-stage estimation method used in randomized controlled trials. In the first stage, a prognostic score, defined as the conditional expectation of an outcome given covariates under…

Methodology · Statistics 2026-05-01 Nodoka Seya , Masataka Taguri

In this paper we propose and discuss variance reduction techniques for the estimation of quantiles of the output of a complex model with random input parameters. These techniques are based on the use of a reduced model, such as a metamodel…

Methodology · Statistics 2009-01-27 Claire Cannamela , Josselin Garnier , Bertrand Iooss

This paper focuses on the estimation of distributional treatment effects in randomized experiments that use covariate-adaptive randomization (CAR). These include designs such as Efron's biased-coin design and stratified block randomization,…

Econometrics · Economics 2025-06-09 Undral Byambadalai , Tomu Hirata , Tatsushi Oka , Shota Yasui

There are many different proposed procedures for sample size planning for the Wilcoxon-Mann-Whitney test at given type-I and type-II error rates $\alpha$ and $\beta$, respectively. Most methods assume very specific models or types of data…

Methodology · Statistics 2018-10-10 Martin Happ , Arne C. Bathke , Edgar Brunner

Background: It has long been advised to account for baseline covariates in the analysis of confirmatory randomised trials, with the main statistical justifications being that this increases power and, when a randomisation scheme balanced…

Methodology · Statistics 2021-12-09 Tim P. Morris , A. Sarah Walker , Elizabeth J. Williamson , Ian R. White

The policy relevant treatment effect (PRTE) measures the average effect of switching from a status-quo policy to a counterfactual policy. Estimation of the PRTE involves estimation of multiple preliminary parameters, including propensity…

Econometrics · Economics 2020-07-17 Yuya Sasaki , Takuya Ura

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