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This paper reviews and compares methods to assess treatment effect heterogeneity in the context of parametric regression models. These methods include the standard likelihood ratio tests, bootstrap likelihood ratio tests, and Goeman's…

应用统计 · 统计学 2026-02-06 Yao Chen , Sophie Sun , Konstantinos Sechidis , Cong Zhang , Torsten Hothorn , Björn Bornkamp

Tasks requiring deductive reasoning, especially those involving multiple steps, often demand adaptive strategies such as intermediate generation of rationales or programs, as no single approach is universally optimal. While Language Models…

人工智能 · 计算机科学 2024-10-22 Rongxing Liu , Kumar Shridhar , Manish Prajapat , Patrick Xia , Mrinmaya Sachan

Randomized experiments are considered the gold standard for estimating causal effects. However, out of the set of possible randomized assignments, some may be likely to produce poor effect estimates and misleading conclusions. Restricted…

统计方法学 · 统计学 2025-08-28 Maggie Wang , René F. Kizilcec , Michael Baiocchi

Studies to compare the survival of two or more groups using time-to-event data are of high importance in medical research. The gold standard is the log-rank test, which is optimal under proportional hazards. As the latter is no simple…

统计方法学 · 统计学 2022-10-25 Ina Dormuth , Tiantian Liu , Jin Xu , Markus Pauly , Marc Ditzhaus

This paper presents the foundations of a computer oriented approach for preparing a list of random treatment assignments to be adopted in randomised controlled trials. Software is presented which can be applied in the earliest stage of…

应用统计 · 统计学 2015-02-12 N. S. Santos-Magalhaes , H. M. de Oliveira , A. J. Alves

Generalized linear mixed models (GLMM) are commonly used to analyze clustered data, but when the number of clusters is small to moderate, standard statistical tests may produce elevated type I error rates. Small-sample corrections have been…

统计方法学 · 统计学 2023-11-07 Hongxiang Qiu , Andrea J. Cook , Jennifer F. Bobb

Two different approaches to analysis of data from diagnostic biomarker studies are commonly employed. Logistic regression is used to fit models for probability of disease given marker values, while ROC curves and risk distributions are used…

应用统计 · 统计学 2013-12-02 Ying Huang , Margaret S. Pepe , Ziding Feng

Causal analyses for observational studies are often complicated by covariate imbalances among treatment groups, and matching methodologies alleviate this complication by finding subsets of treatment groups that exhibit covariate balance. It…

统计方法学 · 统计学 2021-04-26 Zach Branson

Group sequential design (GSD) is widely used in clinical trials in which correlated tests of multiple hypotheses are used. Multiple primary objectives resulting in tests with known correlations include evaluating 1) multiple experimental…

统计方法学 · 统计学 2021-03-22 Keaven M. Anderson , Zifang Guo , Jing Zhao , Linda Z. Sun

We present a probabilistic ranking model to identify the optimal treatment in multiple-response experiments. In contemporary practice, treatments are applied over individuals with the goal of achieving multiple ideal properties on them…

Sequential Multiple-Assignment Randomized Trials (SMARTs) play an increasingly important role in psychological and behavioral health research. This experimental approach enables researchers to answer scientific questions about how to…

统计方法学 · 统计学 2023-06-21 John J. Dziak , Daniel Almirall , Walter Dempsey , Catherine Stanger , Inbal Nahum-Shani

There is a growing interest in the implementation of platform trials, which provide the flexibility to incorporate new treatment arms during the trial and the ability to halt treatments early based on lack of benefit or observed…

统计方法学 · 统计学 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

What proportion of treated units actually benefited from an experimental intervention? What is the median or the largest individual treatment effect? This paper develops methods for answering such questions about the distribution of…

统计方法学 · 统计学 2026-05-11 David Kim , Yongchang Su , Jake Bowers , Xinran Li

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

Dynamic treatment regimes (DTRs) are sequences of decision rules to guide treatment assignments in response to a patient's evolving, time-varying disease status. Sequential multiple assignment randomized trials (SMARTs) are considered the…

统计方法学 · 统计学 2026-04-29 Xinru Wang , Meghna Bose , Bibhas Chakraborty , Robert Mahar

Delayed separation of survival curves is a common occurrence in confirmatory studies in immuno-oncology. Many novel statistical methods that aim to efficiently capture potential long-term survival improvements have been proposed in recent…

统计方法学 · 统计学 2022-01-26 Dominic Magirr , José L. Jiménez

We propose a new class of weighted logrank tests (WLRT) that control the risk of concluding that a new drug is more efficacious than standard of care, when, in fact, it is uniformly inferior. Perhaps surprisingly, this risk is not…

应用统计 · 统计学 2018-07-31 Dominic Magirr , Carl-Fredrik Burman

Neurodegenerative disorders such as Alzheimer's disease (AD) present a significant global health challenge, characterized by cognitive decline, functional impairment, and other debilitating effects. Current AD clinical trials often assess…

统计方法学 · 统计学 2025-02-07 Xiaoming Xu , Dhrubajyoti Ghosh , Sheng Luo

Reliable brain tumor segmentation in MRI is indispensable for treatment planning and outcome monitoring, yet models trained on curated benchmarks often fail under domain shifts arising from scanner and protocol variability as well as…

计算机视觉与模式识别 · 计算机科学 2025-09-23 Yuanhan Wang , Yifei Chen , Shuo Jiang , Wenjing Yu , Mingxuan Liu , Beining Wu , Jinying Zong , Feiwei Qin , Changmiao Wang , Qiyuan Tian

Single-arm studies in the early development phases of new treatments are not uncommon in the context of rare diseases or in paediatrics. If an assessment of efficacy is to be made at the end of such a study, the observed endpoints can be…

统计方法学 · 统计学 2026-03-02 Moritz Fabian Danzer , Rene Schmidt