Adaptive Designs and Sequential Monitoring

自适应设计和顺序监控

• 批准号: 0907297
• 负责人: Feifang Hu
• 金额: $ 13万
• 依托单位: University of Virginia Main Campus
• 依托单位国家: 美国
• 项目类别: Standard Grant
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-01 至 2013-08-31
• 项目状态: 已结题
• 来源: https://www.nsf.gov/awardsearch/showAward?AWD_ID=0907297&HistoricalAwards=false
• 关键词: Adaptive; Designs; Sequential; Monitoring

Clinical trials are complicated and usually involve multiple objectives such as controlling type I error, increasing power of detecting treatment difference, assigning more patients to better treatment, and more. In literature, both adaptive designs (by changing design procedure sequentially) and sequential monitoring (by changing analysis procedure sequentially) have been proposed to achieve these objectives to some degree. In this project, the investigator combines these two sequential procedures and studies the advantages of sequential monitoring response-adaptive randomized clinical trial. The investigator first derives the asymptotic distribution of the sequential test statistics of the combined procedure. Based on the asymptotic properties, the investigator selects appropriate boundaries for the combined procedure to achieve multiple objectives. Further, he investigates the implementation of the combined procedure to some real clinical trials.Clinical trials usually involve multiple competing objectives such as detecting clinical difference among treatments, minimizing total cost and protecting more people from possibly inferior treatments. Adaptive designs dynamically use sequentially accruing data in decisions for collecting future data in order to achieve these objectives. In conducting clinical trials, sequential monitoring is a standard technique to balance the ethical and financial advantages of stopping a trial early against the risk of an incorrect conclusion. This proposal is concerned with combining adaptive designs and sequential monitoring. The investigator will first investigate sequential monitoring of a response-adaptive randomized clinical trial and will then study the advantages of this combined procedure. Upon completion of this project, one can apply sequential monitoring to an adaptive randomized clinical trial to achieve multiple objectives. The research projects will produce advanced statistical tools for analyzing data which is sequentially collected. These tools may be applied in many fields including drug development, medical studies, industrial experiments, economics and finance.

临床试验是复杂的，通常涉及多个目标，如控制I型错误，提高检测治疗差异的能力，分配更多的患者接受更好的治疗，等等。在文献中，已经提出了自适应设计（通过顺序改变设计过程）和顺序监测（通过顺序改变分析过程）来在一定程度上实现这些目标。在本项目中，研究者将这两种顺序程序结合起来，研究顺序监测反应适应性随机临床试验的优势。研究者首先推导出组合过程的序列检验统计量的渐近分布。基于渐近性质，研究者为组合过程选择合适的边界以实现多个目标。此外，他调查了一些实际临床试验中联合程序的实施情况。临床试验通常涉及多个相互竞争的目标，例如检测治疗方法之间的临床差异，最小化总成本以及保护更多的人免受可能较差的治疗。自适应设计动态地在决策中使用顺序累积的数据来收集未来的数据，以实现这些目标。在进行临床试验时，顺序监测是一种标准技术，可以在早期停止试验的伦理和经济优势与得出错误结论的风险之间取得平衡。该方案将自适应设计与顺序监测相结合。研究者将首先调查反应适应性随机临床试验的顺序监测，然后研究这种联合手术的优点。本项目完成后，可将序贯监测应用于适应性随机临床试验，以实现多重目标。这些研究项目将产生先进的统计工具，用于分析依次收集的数据。这些工具可以应用于许多领域，包括药物开发、医学研究、工业实验、经济和金融。