Assessment of Responder Analyses for Patient-Reported Outcomes With Varying Variability Between Intervention Groups: Maximum Responder Threshold and Effect [0.03%]
评估不同处理组之间变异度不同的患者报告结果的反应者分析：最大反应阈值和效果
Joseph C Cappelleri,Jia Ma,Manuela Di Fusco et al.
Joseph C Cappelleri et al.
Continuous outcomes are frequently dichotomized to classify subjects as "responders" or "nonresponders" with the difference in proportion of response between treatment and control groups defined as the "responder effect," which has been use...
Methodological Approaches for the Estimation of Confidence Intervals on Partial Youden Index Under Verification Bias [0.03%]
验偏情况下部分youden指数可信区间估计的方法研究
Sihan Jia,Shirui Wang,Gengsheng Qin
Sihan Jia
The advancement of precision medicine hinges on accurately tailored diagnostic strategies yet estimating reliable confidence intervals (CIs) for the maximal partial Youden Index under verification bias presents considerable challenges, espe...
Mauro Gasparini,Vincenzo Di Trani,Marco Ratta
Mauro Gasparini
In this work we illustrate a method to improve estimation of Vaccine Efficacy (VE), a vastly employed measure of effect in vaccine clinical research, with small and medium sample sizes. We introduce a comprehensive Bayesian approach that im...
A Statistical Concept for Conditional Marketing Authorisation Based on the Intermediate and Final Outcomes of a Single Confirmatory Randomised Clinical Trial [0.03%]
基于确证性随机临床试验的中间和最终结果的条件性营销授权的统计概念
Xiaofei Liu,Nele Henrike Thomas,Elina Asikanius et al.
Xiaofei Liu et al.
Conditional marketing authorisation (CMA) is a path to early market access of new medicines addressing an unmet medical need in the European Union (EU), and similar concepts exist in other regulatory regions. For justifying a CMA, the benef...
Handling Missing Data in Participants with Baseline but No Post-Baseline Data [0.03%]
具有基线但没有随访数据的受试者的数据缺失处理方法
Craig Mallinckrodt,Ilya Lipkovich,Samuel Dickson et al.
Craig Mallinckrodt et al.
Participants who are randomized to treatment but have no post-baseline data pose a unique challenge. These participants need to be included to preserve randomization. Because there is no information about the outcome or the intercurrent eve...
Evaluating Safety Rules in Ongoing Dose-Escalation Trials Using BOIN: A Comparative Analysis of Criteria and Their Impact on Dose-Level Safety Assessments [0.03%]
基于BOIN方法评估剂量递增试验中受试者安全界限的不同判停规则及对安全性的影响分析
Stefan Englert,Thomas J Prior,Anirban Mitra et al.
Stefan Englert et al.
Ongoing dose-escalation trials present unique challenges in assessing safety, all with the goal to establish the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D). Due to the extended duration of trials, it is common ...
Comparative Study
Pharmaceutical statistics. 2026 Mar-Apr;25(2):e70075. DOI:10.1002/pst.70075 2026
Using Off-Treatment Sequential Multiple Imputation for Binary Outcomes to Address Intercurrent Events Handled by a Treatment Policy Strategy [0.03%]
利用序贯多重填补处理间期事件的二分类结局：治疗政策策略应对方法
Sunita Rehal,Nicky Best,Sarah Watts et al.
Sunita Rehal et al.
The estimand framework proposes different strategies to address intercurrent events. The treatment policy strategy seems to be the most favoured as it is closely aligned with the pre-addendum intention-to-treat principle. All data for all p...
A Tobit Partly Linear Mixed and Mixture Cure Model for the Joint Analysis of Interval-Bounded Longitudinal Measurements and Survival Times With Cure Proportion [0.03%]
具有治愈比例的区间界定纵向测量和生存时间的Tobit部分线性混合和混合 cure 模型
Zhanfeng Wang,Zerui Shen,Ruixing Ming et al.
Zhanfeng Wang et al.
Motivated by the analysis of data from a clinical trial on patients with early breast cancer, we propose in this paper a new joint model that uses a Tobit partly linear mixed model for longitudinal measurements which are bounded in an inter...
Application of Causal Inference to Establish Assay Effect in the Absence of a Bridging Study: A Case Study of MenACWY-CRM Conjugate Vaccine Data [0.03%]
因果推断在缺乏桥接研究时建立检测方法效应的应用：MenACWY-CRM结合疫苗数据的案例研究
Meike Adani,Silvia Noirjean,Andrea Callegaro et al.
Meike Adani et al.
During a vaccine development program, if the assay used to measure immunological endpoints is changed, ideally, a bridging study is performed to establish the relationship between results obtained with the new and previous assay. However, t...
Multiple Comparisons With Overdispersed Multinomial Data: Methods, Properties and Application [0.03%]
过度离散的多项式数据的多重比较：方法、性质和应用
Sören Budig,Charlotte Vogel,Frank Schaarschmidt
Sören Budig
Overdispersion, a common issue in clustered multinomial data, can lead to biased standard errors and compromised statistical inference if not adequately addressed. This study describes a comprehensive procedure for constructing multiple com...
Comparative Study
Pharmaceutical statistics. 2026 Jan-Feb;25(1):e70073. DOI:10.1002/pst.70073 2026