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Bayesian predictive probabilities of success (PPoS) use interim trial data to calculate the probability of trial success. These quantities can be used to optimize trial size or to stop for futility. In this paper, we describe a…

To precisely define the treatment effect of interest in a clinical trial, the ICH E9 estimand addendum describes that relevant so-called intercurrent events should be identified and strategies specified to deal with them. Handling…

统计方法学 · 统计学 2025-02-06 Camila Olarte Parra , Rhian M. Daniel , Jonathan W. Bartlett

The scheduling problem is a key class of optimization problems and has various kinds of applications both in practical and theoretical scenarios. In the scheduling problem, probabilistic analysis is a basic tool for investigating…

信息论 · 计算机科学 2024-01-30 Daiki Suruga

A fundamental limitation of causal inference in observational studies is that perceived evidence for an effect might instead be explained by factors not accounted for in the primary analysis. Methods for assessing the sensitivity of a…

统计方法学 · 统计学 2018-09-14 Colin B. Fogarty

Clinical trials often allow patients in the control arm to switch to the treatment arm if their physical conditions are worse than certain tolerance levels. For instance, treatment switching arises in the Concorde clinical trial, which aims…

应用统计 · 统计学 2023-09-06 Alessandra Mattei , Peng Ding , Veronica Ballerini , Fabrizia Mealli

Typical causal effects are defined based on the marginal distribution of potential outcomes. However, many real-world applications require causal estimands involving the joint distribution of potential outcomes to enable more nuanced…

统计方法学 · 统计学 2026-04-17 Peng Wu , Xiaojie Mao

In comparative studies, such as in causal inference and clinical trials, balancing important covariates is often one of the most important concerns for both efficient and credible comparison. However, chance imbalance still exists in many…

统计方法学 · 统计学 2018-07-30 Yichen Qin , Yang Li , Wei Ma , Feifang Hu

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

统计方法学 · 统计学 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

统计方法学 · 统计学 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Using the concept of principal stratification from the causal inference literature, we introduce a new notion of fairness, called principal fairness, for human and algorithmic decision-making. The key idea is that one should not…

计算机与社会 · 计算机科学 2022-03-28 Kosuke Imai , Zhichao Jiang

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

统计方法学 · 统计学 2026-03-31 Zhe Chen , Xinran Li

Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate…

机器学习 · 计算机科学 2025-11-03 Tien Huu Do , Antoine Masquelier , Nae Eoun Lee , Jonathan Crowther

The study of associations and their causal explanations is a central research activity whose methodology varies tremendously across fields. Even within specialized subfields, comparisons across textbooks and journals reveals that the basics…

统计方法学 · 统计学 2025-10-13 Sander Greenland

Stratification in both the design and analysis of randomized clinical trials is common. Despite features in automated randomization systems to re-confirm the stratifying variables, incorrect values of these variables may be entered. These…

统计方法学 · 统计学 2023-07-24 Neal Thomas

Estimation of treatment effect for principal strata has been studied for more than two decades. Existing research exclusively focuses on the estimation, but there is little research on forming and testing hypotheses for principal…

统计方法学 · 统计学 2021-12-20 Yongming Qu , Stephen J. Ruberg , Junxiang Luo , Ilya Lipkovich

The ICH E9 (R1) addendum on estimands, coupled with recent advancements in causal inference, has prompted a shift towards using model-free treatment effect estimands that are more closely aligned with the underlying scientific question.…

统计方法学 · 统计学 2024-10-11 Stijn Vansteelandt , Kelly Van Lancker

Motivated by learning from experience and exploiting existing knowledge in civil nuclear operations, we have developed in-house generic PSA models for pressurized and boiling water reactors. The models are computationally light, handy,…

物理与社会 · 物理学 2021-12-01 Ali Ayoub , Wolfgang Kröger , Didier Sornette

Simulation studies are computer experiments that involve creating data by pseudorandom sampling. The key strength of simulation studies is the ability to understand the behaviour of statistical methods because some 'truth' (usually some…

统计方法学 · 统计学 2019-01-18 Tim P Morris , Ian R White , Michael J Crowther

The ICH E9(R1) addendum provides guidelines on accounting for intercurrent events in clinical trials using the estimands framework. However, there has been limited attention to the estimands framework for meta-analysis. Using treatment…

Principal component analysis (PCA) is often used for analyzing data in the most diverse areas. In this work, we report an integrated approach to several theoretical and practical aspects of PCA. We start by providing, in an intuitive and…

计算工程、金融与科学 · 计算机科学 2021-06-09 Felipe L. Gewers , Gustavo R. Ferreira , Henrique F. de Arruda , Filipi N. Silva , Cesar H. Comin , Diego R. Amancio , Luciano da F. Costa