Exploring Stratification Strategies for Population- Versus Randomization-Based Inference [0.03%]
探索基于人群与随机化推断的分层策略
Marco Novelli,William F Rosenberger
Marco Novelli
Stratification on important variables is a common practice in clinical trials, since ensuring cosmetic balance on known baseline covariates is often deemed to be a crucial requirement for the credibility of the experimental results. However...
Hang Li,Tomasz M Witkos,Scott Umlauf et al.
Hang Li et al.
During the drug development process, testing potency plays an important role in the quality assessment required for the manufacturing and marketing of biologics. Due to multiple operational and biological factors, higher variability is usua...
Survival Analysis Without Sharing of Individual Patient Data by Using a Gaussian Copula [0.03%]
使用高斯 copula 进行生存分析无需共享患者个体数据
Federico Bonofiglio
Federico Bonofiglio
Cox regression and Kaplan-Meier estimations are often needed in clinical research and this requires access to individual patient data (IPD). However, IPD cannot always be shared because of privacy or proprietary restrictions, which complica...
Optimal Cut-Point Selection Methods Under Binary Classification When Subclasses Are Involved [0.03%]
涉及子类情况下二元分类下的最佳截断值选择方法研究
Jia Wang,Lili Tian
Jia Wang
In practice, we often encounter binary classification problems where both main classes consist of multiple subclasses. For example, in an ovarian cancer study where biomarkers were evaluated for their accuracy of distinguishing noncancer ca...
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint [0.03%]
基于替代终点的成功概率的无用性中期分析
Ronan Fougeray,Loïck Vidot,Marco Ratta et al.
Ronan Fougeray et al.
In clinical trials with time-to-event data, the evaluation of treatment efficacy can be a long and complex process, especially when considering long-term primary endpoints. Using surrogate endpoints to correlate the primary endpoint has bec...
G M Hair,T Jemielita,S Mt-Isa et al.
G M Hair et al.
Subgroup analysis may be used to investigate treatment effect heterogeneity among subsets of the study population defined by baseline characteristics. Several methodologies have been proposed in recent years and with these, statistical issu...
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines [0.03%]
稀释还是不稀释——基因药物的名义滴度剂量问题分析
Paul Faya,Tianhui Zhang
Paul Faya
Recombinant adeno-associated virus (AAV) has become a popular platform for many gene therapy applications. The strength of AAV-based products is a critical quality attribute that affects the efficacy of the drug and is measured as the conce...
Do You Want to Stay Single? Considerations on Single-Arm Trials in Drug Development and the Postregulatory Space [0.03%]
您想保持单身吗？药物研发和上市后监管中的单臂试验考量
Yulia Dyachkova,Cornelia Dunger-Baldauf,Nathalie Barbier et al.
Yulia Dyachkova et al.
Single-arm trials (SATs), while not preferred, remain in use throughout the drug development cycle. They may be accepted by regulators in particular contexts (e.g., in oncology or rare diseases) when the potential effects of new treatments ...
Jiaying Guo,Mengyi Lu,Isabella Wan et al.
Jiaying Guo et al.
Delayed outcome is common in phase I oncology clinical trials. It causes logistic difficulty, wastes resources, and prolongs the trial duration. This article investigates this issue and proposes the time-to-event 3 + 3 (T3 + 3) design, whic...
The partnership between statisticians and the Institutional Animal Care and Use Committee (IACUC) [0.03%]
统计学家与机构动物护理和使用委员会(IACUC)的合作关系
David Potter,Thomas Bradstreet,Davit Sargsyan et al.
David Potter et al.
In this tutorial we explore the valuable partnership between statisticians and Institutional Animal Care and Use Committees (IACUCs) in the context of animal research, shedding light on the critical role statisticians play in ensuring the e...