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Effective decision making from randomised controlled clinical trials relies on robust interpretation of the numerical results. However, the language we use to describe clinical trials can cause confusion both in trial design and in…

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

Methodology · Statistics 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson

In a randomized control trial, the precision of an average treatment effect estimator can be improved either by collecting data on additional individuals, or by collecting additional covariates that predict the outcome variable. We propose…

Methodology · Statistics 2017-09-27 Pedro Carneiro , Sokbae Lee , Daniel Wilhelm

Synthetic control (SC) methods are commonly used to estimate the treatment effect on a single treated unit in panel data settings. An SC is a weighted average of control units built to match the treated unit, with weights typically…

Methodology · Statistics 2023-02-21 Xu Shi , Kendrick Li , Wang Miao , Mengtong Hu , Eric Tchetgen Tchetgen

Time-dependent Receiver Operating Characteristics (ROC) analysis is a standard method to evaluate the discriminative performance of biomarkers or risk scores for time-to-event outcomes. Extensions of this useful method to left-truncated…

Methodology · Statistics 2025-09-09 Kendrick Li , Mithun Kumar Acharjee

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent…

Methodology · Statistics 2025-09-19 Fan Li , Jiaqi Tong , Xi Fang , Chao Cheng , Brennan C. Kahan , Bingkai Wang

The paper considers two different designs; a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome, and a twoarm superiority cluster RCT with a binary outcome. From a Bayesian perspective, for the analysis…

Methodology · Statistics 2025-11-11 Abdullah Aloufi , Kevin Wilson , Nina Wilson , Lisa Shaw , Christopher Price

Platform trials allow treatment arms to enter and exit over time while maintaining a shared control arm, yielding concurrent and non-concurrent controls (NCC). Pooling NCC is often motivated as a strategy to improve statistical efficiency,…

Methodology · Statistics 2026-03-12 Antonio D'Alessandro , Samrachana Adhikari , Michele Santacatterina

There is a growing literature on design-based methods to estimate average treatment effects for randomized controlled trials (RCTs) using the underpinnings of experiments. In this article, we build on these methods to consider design-based…

Methodology · Statistics 2024-01-17 Peter Z Schochet

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

The Random Demodulator (RD) and the Modulated Wideband Converter (MWC) are two recently proposed compressed sensing (CS) techniques for the acquisition of continuous-time spectrally-sparse signals. They extend the standard CS paradigm from…

Information Theory · Computer Science 2011-10-11 Michael A. Lexa , Mike E. Davies , John S. Thompson

Long-term treatment effect estimation is a significant but challenging problem in many applications. Existing methods rely on ideal assumptions, such as no unobserved confounders or binary treatment, to estimate long-term average treatment…

Machine Learning · Computer Science 2025-10-23 Zeqin Yang , Weilin Chen , Ruichu Cai , Yuguang Yan , Zhifeng Hao , Zhipeng Yu , Zhichao Zou , Jixing Xu , Zhen Peng , Jiecheng Guo

In this work, we study the estimation of treatment duration effects in observational survival data, where treatment and covariate histories evolve over time and longer observed durations are only attainable among individuals who remain…

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

Methodology · Statistics 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

Receiver operating characteristic (ROC) analysis is a tool to evaluate the capacity of a numeric measure to distinguish between groups, often employed in the evaluation of diagnostic tests. Overall classification ability is sometimes…

The treatment assignment mechanism in a randomized clinical trial can be optimized for statistical efficiency within a specified class of randomization mechanisms. Optimal designs of this type have been characterized in terms of the…

Methodology · Statistics 2025-09-03 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Traditional causal inference approaches leverage observational study data to estimate the difference in observed and unobserved outcomes for a potential treatment, known as the Conditional Average Treatment Effect (CATE). However, CATE…

Machine Learning · Statistics 2022-09-22 Tianhui Zhou , William E Carson , David Carlson

Connectionist Temporal Classification (CTC) is a widely used method for automatic speech recognition (ASR), renowned for its simplicity and computational efficiency. However, it often falls short in recognition performance. In this work, we…

Audio and Speech Processing · Electrical Eng. & Systems 2025-02-17 Zengwei Yao , Wei Kang , Xiaoyu Yang , Fangjun Kuang , Liyong Guo , Han Zhu , Zengrui Jin , Zhaoqing Li , Long Lin , Daniel Povey