Penalized Variable Selection for Joint AFT Random-Effect Model With Clustered Competing-Risks Data [0.03%]
带有聚类竞争风险数据的联合AFT随机效应模型的惩罚变量选择
Lin Hao,Il Do Ha
Lin Hao
Clustered competing-risks data often arise in clinical studies, such as multi-center clinical trials, where the occurrence of an event within a cluster hinders the observation of other types of events. The correlation resulting from cluster...
Multiplicity Adjustment Methods for a Three-Way Crossover Bioequivalence Study [0.03%]
三周期四处理交叉生物等效性研究中的多重比较方法
David Hinds,Stella Grosser,Wanjie Sun
David Hinds
For generic drugs, a three-way crossover bioequivalence (BE) study is often used to compare two generic (T) formulations against the common brand-name (R) formulation. Adjustment for multiplicity in equivalence testing, however, is little r...
Leveraging Two-Stage δ Global Sensibility Analysis Method to Inform Parameter Estimation in PBPK Models [0.03%]
利用两阶段全局灵敏度分析方法改善PBPK模型参数估计问题
Marina Cuquerella-Gilabert,Alessandro De Carlo,Sergio Sánchez Herrero et al.
Marina Cuquerella-Gilabert et al.
Appropriately performing Global Sensitivity Analysis (GSA) and refining models through the estimation of key parameters on individual data are fundamental steps in PBPK modeling, yet they remain insufficiently addressed in current practice....
"Within-Trial" Prognostic Score Adjustment Is Targeted Maximum Likelihood Estimation [0.03%]
"时内"预后评分调整的目标最大似然估计方法
Emilie Højbjerre-Frandsen,Alejandro Schuler
Emilie Højbjerre-Frandsen
Adjustment for "super" or "prognostic" composite covariates has become more popular in randomized trials recently. These prognostic covariates are often constructed from historical data obtained from previous clinical trials or registries b...
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