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While Null Hypothesis Significance Testing (NHST) remains a widely used statistical tool, it suffers from several shortcomings in its common usage, such as conflating statistical and practical significance, the formulation of inappropriate…

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

Complementary features of randomized controlled trials (RCTs) and observational studies (OSs) can be used jointly to estimate the average treatment effect of a target population. We propose a calibration weighting estimator that enforces…

Methodology · Statistics 2022-02-16 Dasom Lee , Shu Yang , Lin Dong , Xiaofei Wang , Donglin Zeng , Jianwen Cai

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

Methodology · Statistics 2022-02-08 Melody Huang

Randomized control trials (RCTs) are the gold standard for estimating causal effects, but often use samples that are non-representative of the actual population of interest. We propose a reweighting method for estimating population average…

Methodology · Statistics 2022-02-09 Kellie Ottoboni , Jason Poulos

A practical limitation of cluster randomized controlled trials (cRCTs) is that the number of available clusters may be small, resulting in an increased risk of baseline imbalance under simple randomization. Constrained randomization…

Methodology · Statistics 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

Easy-to-interpret effect estimands are highly desirable in survival analysis. In the competing risks framework, one good candidate is the restricted mean time lost (RMTL). It is defined as the area under the cumulative incidence function up…

Methodology · Statistics 2024-09-13 Merle Munko , Dennis Dobler , Marc Ditzhaus

In a randomised clinical trial, when the result of the primary endpoint shows a significant benefit, the secondary endpoints are scrutinised to identify additional effects of the treatment. However, this approach entails a risk of…

Time-to-event data with long-term survivors (L-TS), subjects who never experience the event, have been reported in multiple areas of oncology as therapies have improved. Conventional two-sample tests ignore L-TS, but alternatives have been…

Methodology · Statistics 2026-05-06 Yu Bi , Durbadal Ghosh , Subodh Selukar

Epidemiologic studies often evaluate the association between an exposure and an event risk. When time-varying, exposure updates usually occur at discrete visits although changes are in continuous time and survival models require values to…

Background: Often when undertaking meta-analyses of time-to-event (TTE) outcomes, especially in a Health Technology Assessment context, a hazard ratio (HR) scale is used. However, issues arise when there is evidence of non-proportional…

Methodology · Statistics 2026-05-21 Rhiannon K Owen , Keith R Abrams

Survivorship analysis allows to statistically analyze situations that can be modeled as waiting times to an event. These waiting times are characterized by the cumulative hazard rate, which can be estimated by the Nelson-Aalen estimator or…

Statistics Theory · Mathematics 2019-07-04 Niklas Hohmann

Covariate adjustment is an important tool in the analysis of randomized clinical trials and observational studies. It can be used to increase efficiency and thus power, and to reduce possible bias. While most statistical tests in randomized…

Methodology · Statistics 2011-08-03 Xiaoru Wu , Zhiliang Ying

Credit risk assessment is a crucial aspect of financial decision-making, enabling institutions to predict the likelihood of default and make informed lending decisions. Two prominent methodologies in credit risk modeling are logistic…

Applications · Statistics 2026-04-30 Cheng Lee , Hsi Lee

One central goal of design of observational studies is to embed non-experimental data into an approximate randomized controlled trial using statistical matching. Despite empirical researchers' best intention and effort to create…

Methodology · Statistics 2022-06-22 Kan Chen , Siyu Heng , Qi Long , Bo Zhang

Randomized experiments or randomized controlled trials (RCTs) are gold standards for causal inference, yet cost and sample-size constraints limit power. We introduce CALM (Causal Analysis leveraging Language Models), a statistical framework…

Methodology · Statistics 2025-12-09 Xinrui Ruan , Xinwei Ma , Yingfei Wang , Waverly Wei , Jingshen Wang

Randomized clinical trials (RCTs) often involve multiple longitudinal primary outcomes to comprehensively assess treatment efficacy. The Longitudinal Rank-Sum Test (LRST), a robust U-statistics-based, non-parametric, rank-based method,…

Methodology · Statistics 2025-02-17 Dhrubajyoti Ghosh , Sheng Luo

This paper develops a framework for incorporating prior information into sequential multiple testing procedures while maintaining asymptotic optimality. We define a weighted log-likelihood ratio (WLLR) as an additive modification of the…

Methodology · Statistics 2026-02-24 Soumyabrata Bose , Jay Bartroff

Randomized clinical trials (RCTs) are ideal for estimating causal effects, because the distributions of background covariates are similar in expectation across treatment groups. When estimating causal effects using observational data,…

Methodology · Statistics 2019-02-27 Anthony D. Scotina , Roee Gutman

Synthetic control (SC) methods have gained rapid popularity in economics recently, where they have been applied in the context of inferring the effects of treatments on standard continuous outcomes assuming linear input-output relations. In…

Methodology · Statistics 2024-02-19 Alicia Curth , Hoifung Poon , Aditya V. Nori , Javier González