Finding the Optimal Number of Splits and Repetitions in Double Cross-Fitting Targeted Maximum Likelihood Estimators [0.03%]
寻找双交叉拟合靶向最大似然估计器的最佳分割次数和重复次数
Mohammad Ehsanul Karim,Momenul Haque Mondol
Mohammad Ehsanul Karim
Flexible machine learning algorithms are increasingly utilized in real-world data analyses. When integrated within double robust methods, such as the Targeted Maximum Likelihood Estimator (TMLE), complex estimators can result in significant...
Comparing Estimation Methods for the Area Under the Bi-Weibull ROC Curve [0.03%]
双威布尔斯ROC曲线下的面积估计方法的比较
Ruhul Ali Khan,Musie Ghebremichael
Ruhul Ali Khan
In this paper, we carried out extensive simulation studies to compare the performances of partial and maximum likelihood based methods for estimating the area under the bi-Weibull ROC curve. Further, real data sets from HIV/AIDS research we...
Comparative Study
Pharmaceutical statistics. 2025 Sep-Oct;24(5):e70038. DOI:10.1002/pst.70038 2025
Design Considerations for a Phase II Platform Trial in Major Depressive Disorder [0.03%]
抑郁症二期平台试验的设计考量
Michaela Maria Freitag,Dario Zocholl,Elias Laurin Meyer et al.
Michaela Maria Freitag et al.
Major depressive disorder (MDD) is one of the leading causes of disability globally. Despite its prevalence, approximately one-third of patients do not benefit sufficiently from available treatments, and few new drugs have been developed re...
The Estimand Framework and Causal Inference: Complementary Not Competing Paradigms [0.03%]
估算法与因果推断：互补而非竞争的两种范式
Thomas Drury,Jonathan W Bartlett,David Wright et al.
Thomas Drury et al.
The creation of the ICH E9 (R1) estimands framework has led to more precise specification of the treatment effects of interest in the design and statistical analysis of clinical trials. However, it is unclear how the new framework relates t...
Drift Parameter Based Sample Size Determination in Multi-Stage Bayesian Randomized Clinical Trials [0.03%]
基于漂移参数的多阶段贝叶斯随机临床试验样本量确定方法
Yueyang Han,Haolun Shi,Jiguo Cao et al.
Yueyang Han et al.
Sample size determination in Bayesian randomized phase II trial design often relies on computationally intensive search methods, presenting challenges in terms of feasibility and efficiency. We propose a novel approach that greatly reduces ...
The Wilcoxon-Mann-Whitney Estimand Versus Differences in Medians or Means [0.03%]
威尔科克森-曼恩-惠特尼指标与中位数或均值的差异之间的关系
Linda J Harrison,Ronald J Bosch
Linda J Harrison
There is a renewed interest in defining the target of estimation when designing randomized trials. Motivated by design work in trials of HIV-1 curative interventions, we compare the Wilcoxon-Mann-Whitney (WMW) estimand to a difference in me...
Comparative Study
Pharmaceutical statistics. 2025 Sep-Oct;24(5):e70036. DOI:10.1002/pst.70036 2025
Hajime Uno,Lu Tian,Miki Horiguchi et al.
Hajime Uno et al.
Target Aggregate Data Adjustment Method for Transportability Analysis Utilizing Summary-Level Data From the Target Population [0.03%]
利用目标人群汇总数据进行外推性的靶标汇总数据分析的调整方法
Yichen Yan,Quang Vuong,Rebecca K Metcalfe et al.
Yichen Yan et al.
Transportability analysis is a causal inference framework used to evaluate the external validity of studies by transporting treatment effects from a study sample to an external target population by adjusting for differences in the distribut...
A Flexible Seamless Phase 2/3 Design With Biomarker-Driven Subgroup Enrichment and Sample Size Re-Estimation [0.03%]
一种具有生物标志物亚组富集和样本量重新估计的灵活连续二期三期设计
Zizhong Tian,Liwen Wu,Rachael Liu et al.
Zizhong Tian et al.
To support the expedited drug development addressing unmet medical needs, the seamless phase 2/3 design that makes the phase switching decision based on early surrogate endpoints is gaining popularity in practice. For also catering to poten...