Estimation of Treatment Policy Estimands for Continuous Outcomes Using Off-Treatment Sequential Multiple Imputation [0.03%]
基于序贯多重插补的连续型结局无治疗政策效应估计方法研究
Thomas Drury,Juan J Abellan,Nicky Best et al.
Thomas Drury et al.
The estimands framework outlined in ICH E9 (R1) describes the components needed to precisely define the effects to be estimated in clinical trials, which includes how post-baseline 'intercurrent' events (IEs) are to be handled. In late-stag...
Efficient Study Design and Analysis of Longitudinal Dose-Response Data Using Fractional Polynomials [0.03%]
基于分数多项式的纵向剂量反应数据的高效设计与分析方法研究
Benjamin F Hartley,Dave Lunn,Adrian P Mander
Benjamin F Hartley
Correctly characterising the dose-response relationship and taking the correct dose forward for further study is a critical part of the drug development process. We use optimal design theory to compare different designs and show that using ...
Steven Novick,Tianhui Zhang
Steven Novick
In preclinical drug discovery, at the step of lead optimization of a compound, in vivo experimentation can differentiate several compounds in terms of efficacy and potency in a biological system of whole living organisms. For the lead optim...
Sample Size Estimation Using a Partially Clustered Frailty Model for Biomarker-Strategy Designs With Multiple Treatments [0.03%]
使用部分聚类的脆弱性模型估算样本量，用于具有多种治疗方案的生物标志物策略设计中的样本量估计
Derek Dinart,Virginie Rondeau,Carine Bellera
Derek Dinart
Biomarker-guided therapy is a growing area of research in medicine. To optimize the use of biomarkers, several study designs including the biomarker-strategy design (BSD) have been proposed. Unlike traditional designs, the emphasis here is ...
Handling Partially Observed Trial Data After Treatment Withdrawal: Introducing Retrieved Dropout Reference-Base Centred Multiple Imputation [0.03%]
撤药后处理观察试验数据的多重插补方法：引入回收脱落参考基线中心法
Suzie Cro,James H Roger,James R Carpenter
Suzie Cro
The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires ...
Reparametrized Firth's Logistic Regressions for Dose-Finding Study With the Biased-Coin Design [0.03%]
参数化Firth_logistic回归在有偏硬币设计的剂量寻找研究中的应用
Hyungwoo Kim,Seungpil Jung,Yudi Pawitan et al.
Hyungwoo Kim et al.
Finding an adequate dose of the drug by revealing the dose-response relationship is very crucial and a challenging problem in the clinical development. The main concerns in dose-finding study are to identify a minimum effective dose (MED) i...
Yun Wang,Wenda Tu,William Koh et al.
Yun Wang et al.
In conventional subgroup analyses, subgroup treatment effects are estimated using data from each subgroup separately without considering data from other subgroups in the same study. The subgroup treatment effects estimated this way may be h...
Sin-Ho Jung
Sin-Ho Jung
Biomarkers are key components of personalized medicine. In this paper, we consider biomarkers taking continuous values that are associated with disease status, called case and control. The performance of such a biomarker is evaluated by the...
Comparative Study
Pharmaceutical statistics. 2024 Jul-Aug;23(4):557-569. DOI:10.1002/pst.2371 2024
Variable Duration Trial as an Alternative Design for Continuous Endpoints [0.03%]
连续型终点变量试验期临床试验替代设计方法探究
Jitendra Ganju,Julie Guoguang Ma
Jitendra Ganju
Clinical trials with continuous primary endpoints typically measure outcomes at baseline, at a fixed timepoint (denoted Tmin), and at intermediate timepoints. The analysis is commonly performed using the mixed model repeated measures method...
Timothy Schofield
Timothy Schofield
Chemistry, manufacturing, and control (CMC) statisticians play a key role in the development and lifecycle management of pharmaceutical and biological products, working with their non-statistician partners to manage product quality. Informa...