Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints [0.03%]
加权复合时间结局的受限制均值分析的样本量和功效计算方法研究
Lu Mao
Lu Mao
As a new way of reporting treatment effect, the restricted mean time in favor (RMT-IF) of treatment measures the net average time the treated have had a less serious outcome than the untreated over a specified time window. With multiple out...
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials [0.03%]
新型抗癌药物一期组合试验设计的准确性及安全性分析
Rong Liu,Ying Yuan,Suman Sen et al.
Rong Liu et al.
Despite numerous innovative designs having been published for phase I drug-combination dose finding trials, their use in real applications is rather limited. As a working group under the American Statistical Association Biopharmaceutical Se...
Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods [0.03%]
临床试验中的假设赋值：因果推理和缺失数据方法的统一
Camila Olarte Parra,Rhian M Daniel,Jonathan W Bartlett
Camila Olarte Parra
The ICH E9 addendum introduces the term intercurrent event to refer to events that happen after treatment initiation and that can either preclude observation of the outcome of interest or affect its interpretation. It proposes five strategi...
A Bayesian decision-theoretic design for simultaneous biomarker-based subgroup selection and efficacy evaluation [0.03%]
基于生物标志物的亚组选择和疗效评估的贝叶斯决策设计
Zheyu Wang,Fujun Wang,Chenguang Wang et al.
Zheyu Wang et al.
The success of drug development of targeted therapy often hinges on an appropriate selection of the sensitive patient population, mostly based on patients' biomarker levels. At the planning stage of a phase II study, although a potential bi...
Planning for the Next Pandemic: Ethics and Innovation Today for Improved Clinical Trials Tomorrow [0.03%]
面向未来的伦理与创新：改善明日临床试验今日做起
Gianna McMillan,Cristiana Mayer,Rui Tang et al.
Gianna McMillan et al.
The coronavirus pandemic has brought public attention to the steps required to produce valid scientific clinical research in drug development. Traditional ethical principles that guide clinical research remain the guiding compass for physic...
Wenlin Yuan,Ming-Hui Chen,John Zhong
Wenlin Yuan
In this paper, we lay out the basic elements of Bayesian sample size determination (SSD) for the Bayesian design of a two-arm superiority clinical trial. We develop a flowchart of the Bayesian SSD that highlights the critical components of ...
Comparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants [0.03%]
贝叶斯自适应设计在罕见病中的效用研究：重复利用受试者的自适应随机化响应功效研究
Fengming Tang,Byron J Gajewski
Fengming Tang
Slow accrual rate is a major challenge in clinical trials for rare diseases and is identified as the most frequent reason for clinical trials to fail. This challenge is amplified in comparative effectiveness research where multiple treatmen...
Simulation-Based Pharmacokinetics Sampling Design for Evaluating Correlates of Prevention Efficacy of Passive HIV Monoclonal Antibody Prophylaxis [0.03%]
基于模拟的药代动力学抽样设计，用于评估被动HIV单克隆抗体预防的有效性关联因素
Lily Zhang,Peter B Gilbert,Edmund Capparelli et al.
Lily Zhang et al.
We address sampling design of population pharmacokinetics (popPK) experiments in the context of two ongoing phase 2b efficacy trials that evaluate the efficacy of VRC01 (vs. placebo) in reducing the rate of HIV infection among 4625 particip...
Efficient Multiple Imputation for Sensitivity Analysis of Recurrent Events Data with Informative Censoring [0.03%]
具有信息性截尾的反复事件数据敏感性分析的有效多重插补法
Guoqing Diao,Guanghan F Liu,Donglin Zeng et al.
Guoqing Diao et al.
Missing data are commonly encountered in clinical trials due to dropout or nonadherence to study procedures. In trials in which recurrent events are of interest, the observed count can be an undercount of the events if a patient drops out b...
Two-Stage Adaptive Design for Prognostic Biomarker Signatures with a Survival Endpoint [0.03%]
具有生存终点的预后生物标志物签名的两阶段自适应设计
Biyue Dai,Mei-Yin C Polley
Biyue Dai
Cancer biomarker discoveries typically involve utilizing patient specimens. In practice, there is often strong desire to preserve high quality biospecimens for studies that are most likely to yield useful information. Previously, we propose...