Missing data in clinical trials: from clinical assumptions to statistical analysis using pattern mixture models [0.03%]
临床试验中的缺失数据：从临床假设到模式混合模型的统计分析
Bohdana Ratitch,Michael O&#x;Kelly,Robert Tosiello
Bohdana Ratitch
The need to use rigorous, transparent, clearly interpretable, and scientifically justified methodology for preventing and dealing with missing data in clinical trials has been a focus of much attention from regulators, practitioners, and ac...
Stratification of randomization is not required for a pre-specified subgroup analysis [0.03%]
无需分层随机化即可进行预设亚组分析
Lee D Kaiser
Lee D Kaiser
Published literature and regulatory agency guidance documents provide conflicting recommendations as to whether a pre-specified subgroup analysis also requires for its validity that the study employ randomization that is stratified on subgr...
On the practical application of mixed effects models for repeated measures to clinical trial data [0.03%]
混合效应模型在临床试验重复测量数据中的应用研究
Scott W Andersen,Brian A Millen
Scott W Andersen
The use of mixed effects models for repeated measures (MMRM) for clinical trial analyses has recently gained broad support as a primary analysis methodology. Some questions of practical implementation detail remain, however. For example, wh...
Comparative Study
Pharmaceutical statistics. 2013 Jan-Feb;12(1):7-16. DOI:10.1002/pst.1548 2013
Evaluating dynamic treatment strategies: does it have to be more costly? [0.03%]
动态治疗策略的评估：是否需要更高的成本？
Jozefien Buyze,Els Goetghebeur
Jozefien Buyze
Dynamic treatment strategies are designed to change treatments over time in response to intermediate outcomes. They can be deployed for primary treatment as well as for the introduction of adjuvant treatment or other treatment-enhancing int...
Comparative Study
Pharmaceutical statistics. 2013 Jan-Feb;12(1):35-42. DOI:10.1002/pst.1546 2013
A structured framework for assessing sensitivity to missing data assumptions in longitudinal clinical trials [0.03%]
纵向临床试验中评估缺失数据假设敏感性的结构化框架
C H Mallinckrodt,Q Lin,M Molenberghs
C H Mallinckrodt
The objective of this research was to demonstrate a framework for drawing inference from sensitivity analyses of incomplete longitudinal clinical trial data via a re-analysis of data from a confirmatory clinical trial in depression. A likel...
Haochuan Zhou,Gengsheng Qin
Haochuan Zhou
Comparison of accuracy between two diagnostic tests can be implemented by investigating the difference in paired Youden indices. However, few literature articles have discussed the inferences for the difference in paired Youden indices. In ...
Comparative Study
Pharmaceutical statistics. 2013 Jan-Feb;12(1):17-27. DOI:10.1002/pst.1543 2013
Sample size determination for clinical trials with co-primary outcomes: exponential event times [0.03%]
复合主要疗效指标的临床试验样本量计算：事件呈指数分布的情形
Toshimitsu Hamasaki,Tomoyuki Sugimoto,Scott Evans et al.
Toshimitsu Hamasaki et al.
Clinical trials with event-time outcomes as co-primary contrasts are common in many areas such as infectious disease, oncology, and cardiovascular disease. We discuss methods for calculating the sample size for randomized superiority clinic...
The challenges of evaluating dose response in flexible-dose trials using marginal structural models [0.03%]
边缘结构模型在灵活剂量试验中评估剂量反应的挑战
Ilya Lipkovich,Craig H Mallinckrodt,Douglas E Faries
Ilya Lipkovich
Assessing dose response from flexible-dose clinical trials is problematic. The true dose effect may be obscured and even reversed in observed data because dose is related to both previous and subsequent outcomes. To remove selection bias, w...
Comparative Study
Pharmaceutical statistics. 2012 Nov-Dec;11(6):485-93. DOI:10.1002/pst.1540 2012
A generalisation of T-optimality for discriminating between competing models with an application to pharmacokinetic studies [0.03%]
T型优化在竞争模型鉴别中的推广及其在药代动力学研究中的应用
Pavan Vajjah,Stephen B Duffull
Pavan Vajjah
The T-optimality criterion is used in optimal design to derive designs for model selection. To set up the method, it is required that one of the models is considered to be true. We term this local T-optimality. In this work, we propose a ge...
Comparative Study
Pharmaceutical statistics. 2012 Nov-Dec;11(6):503-10. DOI:10.1002/pst.1542 2012
Paola Rebora,Stefania Galimberti
Paola Rebora
In some exceptional circumstances, as in very rare diseases, nonrandomized one-arm trials are the sole source of evidence to demonstrate efficacy and safety of a new treatment. The design of such studies needs a sound methodological approac...