Statistical Tutorial for Cut-Point Determination in Immunogenicity Studies [0.03%]
免疫原性研究中统计学的割点确定方法
Yulia Mordashova,Xin Huang
Yulia Mordashova
Administration of therapeutic protein products might potentially elicit an immune response via production of Anti-Drug Antibodies (ADA). This immune response can cause some clinical consequences ranging from mild to harmful for the patient,...
Applying the Principal Stratum Strategy in Equivalence Trials: A Case Study [0.03%]
等效性试验中的主层策略应用案例研究
Jerome Sepin,Thomas P A Debray,Wei Wei et al.
Jerome Sepin et al.
The estimand framework, introduced in the ICH E9 (R1) Addendum, provides a structured approach for defining precise research questions in randomised clinical trials. It suggests five strategies for addressing intercurrent events (ICE). This...
Why "Minimal Clinically Important Difference" for Interpreting the Magnitude of the Treatment Effect Is Not Useful [0.03%]
为何用“临床重要差异最小值”来解释治疗效果的大小是没有用的
Jitendra Ganju
Jitendra Ganju
The term "minimal clinically important difference" (MCID), though defined as the smallest change in an outcome that is meaningful to the patient, is often used to interpret differences between treatment groups. It is in this context that th...
CUSUMIN Combination: A Cumulative Sum Interval Design for Phase I Cancer Drug-Combination Trials [0.03%]
CUSUMIN组合：一种用于I期抗癌药物组合试验的累积和区间设计
Tomoyoshi Hatayama,Seiichi Yasui
Tomoyoshi Hatayama
Recently, model-assisted designs, including a Bayesian optimal interval (BOIN) design with optimal thresholds for determining the dose for the next cohort, have been proposed for Phase I cancer studies. Model-assisted designs are useful owi...
Control of Unconditional Type I Error in Clinical Trials With External Control Borrowing-A Two-Stage Adaptive Design Perspective [0.03%]
借用外部对照的临床试验中控制无条件I类错误的两阶段自适应设计视角研究
Ping Gao,Xiao Ni,Jing Li et al.
Ping Gao et al.
Patient enrollment can be a substantial burden in rare disease trials. One potential approach is to incorporate external control (EC) into concurrent randomized trials, or EC borrowing, to reduce such burden. Extensive research has been con...
Chauhan Weighted Trajectory Analysis of Combined Efficacy and Safety Outcomes for Risk-Benefit Analysis [0.03%]
加权轨迹分析联合疗效和安全性结果的风险效益评估
Utkarsh Chauhan,Daylen Mackey,John R Mackey
Utkarsh Chauhan
Analyzing and effectively communicating the efficacy and toxicity of treatment is the fundamental basis of risk-benefit analysis (RBA). There is a need for more efficient and objective tools. We apply Chauhan Weighted Trajectory Analysis (C...
Detection of Outlying Correlation Coefficients in Multicenter Clinical Trials [0.03%]
多中心临床试验中相关系数异常值的检测
Lieven Desmet,David Venet,Laura Trotta et al.
Lieven Desmet et al.
Central statistical monitoring aims at finding centers whose data distribution differs significantly from the other centers in multicentric clinical trials. Such differences may point to data quality issues due to negligence, misconduct, or...
The Choice Between Pearson's χ2 Test and Fisher's Exact Test for 2 × 2 Tables [0.03%]
2×2列联表中Pearson’s χ2检验与Fisher’s确切概率检验的选择
Markus Neuhäuser,Graeme D Ruxton
Markus Neuhäuser
Pearson's asymptotic χ2 test is often used to compare binary data between two groups. However, when the sample sizes or expected frequencies are small, the test is usually replaced by Fisher's exact test. Several alternative rules of thumb...
Comparative Study
Pharmaceutical statistics. 2025 May-Jun;24(3):e70012. DOI:10.1002/pst.70012 2025
Randomization in Pre-Clinical Studies: When Evolution Theory Meets Statistics [0.03%]
进化理论遇到统计学时，在临床前研究中随机化的应用
Sofia Weigle,Davit Sargsyan,Javier Cabrera et al.
Sofia Weigle et al.
Randomization is a statistical procedure used to allocate study subjects randomly into experimental groups while balancing continuous variables. This paper presents an alternative to random allocation for creating homogeneous groups by bala...
Multiple Comparisons Procedures for Analyses of Joint Primary Endpoints and Secondary Endpoints [0.03%]
多重比较方法在联合主要终点分析和次要终点分析中的应用
Xiaolong Luo,Lerong Li,Oleksandr Savenkov et al.
Xiaolong Luo et al.
One of the main challenges in drug development for rare diseases is selecting the appropriate primary endpoints for pivotal studies. Although many endpoints can effectively reflect clinical benefit, their sensitivity often varies, making it...