Experimental design considerations and statistical analyses in preclinical tumor growth inhibition studies [0.03%]
抗肿瘤药物临床前药效评价的实验设计与统计分析考虑因素
Vinicius Bonato,Szu-Yu Tang,Matilda Hsieh et al.
Vinicius Bonato et al.
Animal models are used in cancer pre-clinical research to identify drug targets, select compound candidates for clinical trials, determine optimal drug dosages, identify biomarkers, and ensure compound safety. This tutorial aims to provide ...
Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long-term endpoint: The non-Gaussian case [0.03%]
早期结果作为 interim 关于长期终点的决策的唯一信息来源：非高斯情形
Leandro Garcia Barrado,Tomasz Burzykowski
Leandro Garcia Barrado
In randomized clinical trials that use a long-term efficacy endpoint, the follow-up time necessary to observe the endpoint may be substantial. In such trials, an attractive option is to consider an interim analysis based solely on an early ...
Connor Celum,Mark Conaway
Connor Celum
This paper proposes a trial design for locating group-specific doses when groups are partially or completely ordered by dose sensitivity. Previous trial designs for partially ordered groups are model-based, whereas the proposed method is mo...
Covariate adjustment and estimation of difference in proportions in randomized clinical trials [0.03%]
协变量调整及随机临床试验中比例差的估计方法研究
Jialuo Liu,Dong Xi
Jialuo Liu
Difference in proportions is frequently used to measure treatment effect for binary outcomes in randomized clinical trials. The estimation of difference in proportions can be assisted by adjusting for prognostic baseline covariates to enhan...
Visualizing hypothesis tests in survival analysis under anticipated delayed effects [0.03%]
生存分析中假设有延迟效应时的假设检验可视化方法
José L Jiménez,Isobel Barrott,Francesca Gasperoni et al.
José L Jiménez et al.
What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has be...
Alexander Fierenz,Antonia Zapf
Alexander Fierenz
Since the introduction of the estimand in therapeutical studies, several adaptions have been developed. This short article highlights the important aspects of the estimand concept. A literature research was conducted to identify different e...
Lindsay R Berry,Joe Marion,Scott M Berry et al.
Lindsay R Berry et al.
Inferentially seamless 2/3 designs are increasingly popular in clinical trials. It is important to understand their relative advantages compared with separate phase 2 and phase 3 trials, and to understand the consequences of design choices ...
Comments on 'standard and reference-based conditional mean imputation': Regulators and trial statisticians be aware! [0.03%]
对“标准和基于参考的条件均值插补”的评论：监管机构和试验统计学家请注意！
Suzie Cro,Tim P Morris,James H Roger et al.
Suzie Cro et al.
Accurate frequentist performance of a method is desirable in confirmatory clinical trials, but is not sufficient on its own to justify the use of a missing data method. Reference-based conditional mean imputation, with variance estimation j...
Rejoinder to the letter: "Standard and reference-based conditional mean imputation: Regulators and trial statisticians be aware!" [0.03%]
对“标准和基于参考的条件均值插补：监管机构和试验统计学家请注意！”一文的回应
Marcel Wolbers,Alessandro Noci,Paul Delmar et al.
Marcel Wolbers et al.
Comparing various Bayesian random-effects models for pooling randomized controlled trials with rare events [0.03%]
比较各种贝叶斯随机效应模型以汇总稀有事件的随机对照试验
Minghong Yao,Yulong Jia,Fan Mei et al.
Minghong Yao et al.
The meta-analysis of rare events presents unique methodological challenges owing to the small number of events. Bayesian methods are often used to combine rare events data to inform decision-making, as they can incorporate prior information...