Implementing response-adaptive randomisation in stratified rare-disease trials: Design challenges and practical solutions [0.03%]
分层稀有疾病试验中响应适应性随机化的实施：设计挑战与实际解决方案
Rajenki Das,Nina Deliu,Mark R Toshner et al.
Rajenki Das et al.
Although response-adaptive randomisation (RAR) has gained substantial attention in the literature, it still has limited use in clinical trials. Amongst other reasons, the implementation of RAR in real world trials raises important practical...
Augmented two-stage estimation for treatment switching in oncology trials: Leveraging external data for improved precision [0.03%]
利用外部数据提高精度的肿瘤试验治疗转换的增强两阶段估计方法
Harlan Campbell,Nicholas Latimer,Jeroen P Jansen et al.
Harlan Campbell et al.
Randomized controlled trials in oncology often allow control group participants to switch to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment effects. When...
On identification and estimation for sufficient cause interaction through a quasi-instrumental variable [0.03%]
基于准工具变量的足因交互作用的识别与估计问题研究
Pei-Hsuan Hsia,An-Shun Tai,Shih-Chen Fu et al.
Pei-Hsuan Hsia et al.
Mechanistic interaction concerns how exposures affect the outcome. When investigating mechanisms, synergism is the most mentioned type in the fields of genetic study and pharmacology. Synergism is defined under the framework of sufficient c...
Stina Zetterstrom,Arvid Sjölander,Ingeborg Waernbaum
Stina Zetterstrom
Selection bias is a common type of bias, and depending on the causal estimand of interest and the structure of the selection variable, it can be a threat to both external and internal validity. One way to quantify the maximum magnitude of p...
Bayesian clustering prior with overlapping indices for effective use of multisource external data [0.03%]
基于重叠索引的贝叶斯聚类先验方法有效利用多源外部数据
Xuetao Lu,J Jack Lee
Xuetao Lu
The use of external data in clinical trials offers numerous advantages, such as reducing enrollment, increasing study power, and shortening trial duration. In Bayesian inference, information in external data can be transferred into an infor...
A robust Bayesian dose optimization design with backfill and randomization for phase I/II clinical trials [0.03%]
一种稳健的贝叶斯剂量优化设计（含补充和随机化）在I/II期临床试验中的应用
Yingjie Qiu,Mingyue Li
Yingjie Qiu
The integration of backfill cohorts into Phase I clinical trials has garnered increasing interest within the clinical community, particularly following the "Project Optimus" initiative by the U.S. Food and Drug Administration, as detailed i...
A comparison of semi-parametric statistical modeling approaches to dynamic classification of irregularly and sparsely sampled curves [0.03%]
半参数统计建模方法在不规则稀疏曲线动态分类中的比较研究
Ruben Deneer,Zhuozhao Zhan,Edwin Van den Heuvel et al.
Ruben Deneer et al.
This study describes and compares the performance of several semi-parametric statistical modeling approaches to dynamically classify subjects into two groups, based on an irregularly and sparsely sampled curve. The motivating example of thi...
Angela Carollo,Hein Putter,Paul Hc Eilers et al.
Angela Carollo et al.
Competing risks models can involve more than one time scale. A relevant example is the study of mortality after a cancer diagnosis, where time since diagnosis but also age may jointly determine the hazards of death due to different causes. ...
Implementing response-adaptive designs when responses are missing: Impute or ignore? [0.03%]
响应缺失时实施自适应设计：插补或忽略？
Mia S Tackney,Sofía S Villar
Mia S Tackney
Missing data is a widespread issue in clinical trials, but is particularly problematic for digital health interventions where disengagement is common and outcomes are likely to be missing not at random (MNAR). Trials that use response-adapt...
Exact power and sample size in clinical trials with two co-primary binary endpoints [0.03%]
二个主要二分类终点临床试验的样本量和功效计算公式
Gosuke Homma,Takuma Yoshida
Gosuke Homma
Binary endpoints are used widely to evaluate treatment effects during clinical trials. Although clinical trials in many therapeutic areas evaluate a single binary endpoint as the primary endpoint, clinical trials in certain therapeutic area...