The FDA's Final Rule on Expedited Safety Reporting: Statistical Considerations [0.03%]
美国FDA关于优先安全报告的最终规定：统计问题考虑因素
Janet Wittes,Brenda Crowe,Christy Chuang-Stein et al.
Janet Wittes et al.
In March 2011, a Final Rule for expedited reporting of serious adverse events took effect in the United States for studies conducted under an Investigational New Drug (IND) application. In December 2012, the U.S. Food and Drug Administratio...
Closed Testing in Pharmaceutical Research: Historical and Recent Developments [0.03%]
制药研究中的封闭试验：历史与近期发展
Kevin S S Henning,Peter H Westfall
Kevin S S Henning
In pharmaceutical research, making multiple statistical inferences is standard practice. Unless adjustments are made for multiple testing, the probability of making erroneous determinations of significance increases with the number of infer...
Sample Size Considerations in Clinical Trials when Comparing Two Interventions using Multiple Co-Primary Binary Relative Risk Contrasts [0.03%]
多重主要二分相对数对比情况下临床试验中针对两种干预手段的样本量考虑问题
Yuki Ando,Toshimitsu Hamasaki,Scott R Evans et al.
Yuki Ando et al.
The effects of interventions are multi-dimensional. Use of more than one primary endpoint offers an attractive design feature in clinical trials as they capture more complete characterization of the effects of an intervention and provide mo...
A simulation study evaluating bio-creep risk in serial non-inferiority clinical trials for preservation of effect [0.03%]
序列非劣效性临床试验中对保持疗效的生物延续风险的模拟研究
K Odem-Davis,T R Fleming
K Odem-Davis
In non-inferiority trials, acceptable efficacy of an experimental treatment is established by ruling out some defined level of reduced effect relative to an effective active control standard. Serial use of non-inferiority trials may lead to...
Group-Sequential Strategies in Clinical Trials with Multiple Co-Primary Outcomes [0.03%]
多重主要终点的临床试验中的序贯设计策略
Toshimitsu Hamasaki,Koko Asakura,Scott R Evans et al.
Toshimitsu Hamasaki et al.
We discuss the decision-making frameworks for clinical trials with multiple co-primary endpoints in a group-sequential setting. The decision-making frameworks can account for flexibilities such as a varying number of analyses, equally or un...
Estimation of Drug Effectiveness by Modeling Three Time-dependent Covariates: An Application to Data on Cardioprotective Medications in the Chronic Dialysis Population [0.03%]
基于三个时间依赖性协变量的建模估算药物疗效：慢性血液透析人群心脏保护类药物数据的应用分析
Milind A Phadnis,Theresa I Shireman,James B Wetmore et al.
Milind A Phadnis et al.
In a population of chronic dialysis patients with an extensive burden of cardiovascular disease, estimation of the effectiveness of cardioprotective medication in literature is based on calculation of a hazard ratio comparing hazard of mort...
John O&#x;Quigley,Alexia Iasonos
John O&#x;Quigley
The idea of bridging in dose-finding studies is closely linked to the problem of group heterogeneity. There are some distinctive features in the case of bridging which need to be considered if efficient estimation of the maximum tolerated d...
Statistical Methods for Selecting Maximum Effective Dose and Evaluating Treatment Effect When Dose - Response is Monotonic [0.03%]
单调剂量-反应时的效剂量选择和疗效评价的统计方法研究
Maiying Kong,Shesh N Rai,Roberto Bolli
Maiying Kong
The maximum effective dose (MaxED) is an important quantity for therapeutic drugs. The MaxED for therapeutic drugs is defined as the dose above which no improvement in efficacy is obtained. In this article, we propose two experimental desig...
Tony Sabin,James Matcham,Sarah Bray et al.
Tony Sabin et al.
The objectives of the phase 2 stage in a drug development program are to evaluate the safety and tolerability of different doses, select a promising dose range, and look for early signs of activity. At the end of phase 2, a decision to init...
Empirical Likelihood Approaches to Two-Group Comparisons of Upper Quantiles Applied to Biomedical Data [0.03%]
两类医学数据上端分位数的比较研究及其经验似然方法
Jihnhee Yu,Albert Vexler,Alan D Hutson et al.
Jihnhee Yu et al.
In many biomedical studies, a difference in upper quantiles is of specific interest since the upper quantile represents the upper range of biomarkers and/or is used as the cut-off value for a disease classification. In this article, we inve...