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Outcome-dependent sampling designs are common in many different scientific fields including epidemiology, ecology, and economics. As with all observational studies, such designs often suffer from unmeasured confounding, which generally…

统计方法学 · 统计学 2020-10-13 Erin E. Gabriel , Michael C. Sachs , Arvid Sjölander

The analysis of adverse events (AEs) is a key component in the assessment of a drug's safety profile. Inappropriate analysis methods may result in misleading conclusions about a therapy's safety and consequently its benefit-risk ratio. The…

Most conventional risk analysis methods rely on a single best estimate of exposure per person which does not allow for adjustment for exposure-related uncertainty. Here, we propose a Bayesian model averaging method to properly quantify the…

应用统计 · 统计学 2020-04-07 Deukwoo Kwon , F. Owen Hoffman , Brian E. Moroz , Steven L. Simon

To investigate whether treating cancer patients with erythropoiesis-stimulating agents (ESAs) would increase the mortality risk, Bennett et al. [Journal of the American Medical Association 299 (2008) 914--924] conducted a meta-analysis with…

应用统计 · 统计学 2010-10-11 Rui Wang , Lu Tian , Tianxi Cai , L. J. Wei

Analysis of data from randomized controlled trials in vulnerable populations requires special attention when assessing treatment effect by a score measuring, e.g., disease stage or activity together with onset of prevalent terminal events.…

Successful pharmaceutical drug development requires finding correct doses that provide an optimum balance between efficacy and toxicity. Competing responses to dose such as efficacy and toxicity often will increase with dose, and it is…

应用统计 · 统计学 2024-01-26 A. Lawrence Gould

The causal dose response curve is commonly selected as the statistical parameter of interest in studies where the goal is to understand the effect of a continuous exposure on an outcome.Most of the available methodology for statistical…

In-vivo toxicological studies are characterized by multiple primary endpoints with quite different scales. Whereas guidelines and publications provide various statistical tests for normally distributed endpoints (such as organ weights) and…

应用统计 · 统计学 2022-01-11 Ludwig A. Hothorn , Klaus Weber

This paper proposes a new statistical approach for assessing treatment effect using Bayesian Networks (BNs). The goal is to draw causal inferences from observational data with a binary outcome and discrete covariates. The BNs are here used…

In this paper, a novel non-parametric method for estimation of expectation and maximum value of the variance function is proposed for recurrent events where intensity of event occurrence changes with the occurrence of each higher order…

统计方法学 · 统计学 2020-12-18 Sudipta Bhattacharya

Safety evaluation is an essential component of clinical trials. To protect study participants, these studies often implement safety stopping rules that will halt the trial if an excessive number of toxicity events occur. Existing safety…

统计方法学 · 统计学 2025-10-06 Michael J. Martens , Qinghua Lian , Brent R. Logan

We propose a Bayesian nonparametric (BNP) approach to causal inference using observational data consisting of outcome, treatment, and a set of confounders. The conditional distribution of the outcome given treatment and confounders is…

统计方法学 · 统计学 2025-12-01 Yongseok Hur , Joonhyuk Jung , Juhee Lee

Phase I dose-finding studies aim at identifying the maximal tolerated dose (MTD). It is not uncommon that several dose-finding studies are conducted, although often with some variation in the administration mode or dose panel. For instance,…

定量方法 · 定量生物学 2021-03-24 Moreno Ursino , Christian Röver , Sarah Zohar , Tim Friede

In observational studies, the identification of causal estimands depends on the no unmeasured confounding (NUC) assumption. As this assumption is not testable from observed data, sensitivity analysis plays an important role in observational…

统计方法学 · 统计学 2023-09-28 Md Abdul Basit , Mahbub A. H. M. Latif , Abdus S Wahed

Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events (AEs)…

统计方法学 · 统计学 2024-10-08 Anthony Sisti , Andrew Zullo , Roee Gutman

Network meta-analysis (NMA) is widely used to compare multiple interventions simultaneously by synthesizing direct and indirect evidence. The general fixed or random effects contrast-based NMA model can be applied to different outcomes and…

统计方法学 · 统计学 2026-03-03 Harlan Campbell , Jeroen P. Jansen

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

统计方法学 · 统计学 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Toxicologists are often concerned with determining the dosage to which an individual can be exposed with an acceptable risk of adverse effect. These types of studies have been conducted widely in the past, and many novel approaches have…

应用统计 · 统计学 2019-11-13 Faten S. Alamri , Edward L. Boone , David J. Edwards

The quantification and inference of predictive importance for exposure covariates have recently gained significant attention in the context of interpretable machine learning. Contemporary scientific investigations often involve data…

统计方法学 · 统计学 2024-12-31 Zitao Wang , Nian Si , Zijian Guo , Molei Liu

This article proposes a method of estimating benchmark dose (BMD) using a family of link functions in binomial response models dealing with model uncertainty problems. Researchers usually estimate the BMD using binomial response models with…

应用统计 · 统计学 2016-12-22 I. Das