Selection of Quantitative Decision-Making Criteria Using Weighted Decision Error Rates [0.03%]
基于加权决策误差率的定量决策准则选择
Daniel J Bratton
Daniel J Bratton
Quantitative decision-making frameworks provide objective criteria for advancing a drug development program. Within this process, two types of decision error can occur: proceeding to the next phase and failing (incorrect go) or not proceedi...
Adaptive Constrained Weighted Estimation for Incorporating Multiple External Information Sources [0.03%]
自适应约束加权估计方法及其在多源外部信息整合中的应用研究
Keita Takahashi,Kazufumi Okada,Shiro Tanaka et al.
Keita Takahashi et al.
There is growing interest in hybrid control approaches that augment concurrent control data in clinical trials. This study proposes a frequentist method based on constrained weighted maximum likelihood estimation with a concept of effective...
A Weighted Geometric Average Hazard Ratio Based Sample Size Formula for Non-Proportional Hazards and Its Application to a Smoothed Piecewise Model in Cancer Immunotherapy Trial Design [0.03%]
非比例风险下的加权几何平均数 hazard ratio 的样本量公式及其在癌症免疫治疗试验设计中的分段平滑模型应用
Qingyang Zhang,Zixing Wang,Allen Xue et al.
Qingyang Zhang et al.
Cancer immunotherapy trials often present unique challenges for time-to-event data modeling and analysis due to heterogeneous treatment effects across time or subgroups. A few known scenarios are delayed treatment effects and cure rates, wh...
Confidence Intervals for Validation of Analytical Procedures Under ICH Q2(R2) [0.03%]
ICH Q2(R2)下分析方法验证中置信区间的应用
Paul Faya,Chad N Wolfe,Adam P Rauk
Paul Faya
The International Conference on Harmonisation (ICH) adopted revision 2 (R2) of its Quality Guidance Q2 Validation of Analytical Procedures in 2023. The revision includes a new statement that confidence interval limits for validation perform...
Simulation-Based Bayesian Predictive Probability of Success for Interim Monitoring of Clinical Trials With Competing Event Data: Two Case Studies [0.03%]
基于模拟的贝叶斯预测成功概率在临床试验 interim 监测中的应用：两个案例研究
Chiara Micoli,Alessio Crippa,Jason T Connor;I‐SPY COVID Consortium;Martin Eklund et al.
Chiara Micoli et al.
Bayesian predictive probabilities of success (PPoS) use interim trial data to calculate the probability of trial success. These quantities can be used to optimise trial size or to stop for futility. In this paper, we describe a simulation-b...
Quantile Effect on Duration of Response: A Zero-Inflated Censored Regression Approach [0.03%]
分位数对响应持续时间的影响：一种零膨胀截断回归方法
Nan Sun,Jixian Wang,Ram Tiwari
Nan Sun
Duration of response (DOR) has been increasingly used as a useful measure of response to treatments in randomized clinical trials (RCT). Some estimands for DOR, such as the restricted mean DOR, although simple to use, may be sensitive to ou...
Statistical Consideration for Event-Free Survival With Cure Rate in Acute Myeloid Leukemia Studies [0.03%]
急性髓系白血病研究中具有治愈率的无事件生存的统计考量
Yuichiro Kaneko,Kentaro Takeda,Shufang Liu et al.
Yuichiro Kaneko et al.
In many acute myeloid leukemia (AML) studies, event-free survival (EFS) has been accepted as a primary efficacy endpoint. In those studies, the patients who do not achieve complete remission (CR) in the induction period are regarded as indu...
Addressing Non-Exchangeability in Hybrid Control Studies: A Variable Selection Approach [0.03%]
处理混合控制研究中的非可交换性：一种变量选择方法
Zhiwei Zhang,Jialuo Liu,Peisong Han
Zhiwei Zhang
There is growing interest in a hybrid control design for treatment evaluation, where a randomized controlled trial is augmented with external control data from a previous trial or a real world data source. The hybrid control design has the ...
A Unified Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials [0.03%]
随机试验中统一调整协变量的方法以生存终点指标为基础
Zhiwei Zhang,Ya Wang,Dong Xi
Zhiwei Zhang
Covariate adjustment aims to improve the statistical efficiency of randomized trials by incorporating information from baseline covariates. Popular methods for covariate adjustment include analysis of covariance for continuous endpoints and...
Great Wall: A Generalized Dose Optimization Design for Drug Combination Trials Maximizing Survival Benefit [0.03%]
长城：药物联合试验中最大化生存效益的剂量优化设计通用方法
Yan Han,Yingjie Qiu,Yi Zhao et al.
Yan Han et al.
Most phase I-II drug-combination trial designs assume that selecting the optimal dose combination based on early outcomes will also lead to maximum long-term survival benefits. However, this assumption is often violated in many clinical stu...