一个以患者为中心的范式和临床研究的工具:门是开放的
Toshimitsu Hamasaki1,2, Yijie He1,2, Qihang Wu1,2
1The Biostatistics Center, Milken Institute School of Public Health, The George Washington University, Washington, DC, USA.
结果排名的可取性 (DOOR) 范式为临床试验提供了以患者为中心的方法,改善了超越传统单一结果分析的利益:风险评估,以改善医疗决策.
科学领域:
- 临床研究方法论 临床研究方法论
- 生物统计学 生物统计学
- 患者报告的结果
背景情况:
- 随机临床试验 (RCT) 是至关重要的,但往往无法充分告知临床决策,因为在评估全面的患者益处:风险方面存在局限性.
- 标准RCT分析经常单独检查结果,忽视结果之间的关联,累积效应和竞争风险.
- 目前评估治疗效果异质性的方法通常依赖于单一的终点,限制了整体效益:风险的评估.
研究的目的:
- 引入结果排名可取性 (DOOR) 范式,作为临床试验设计,分析和解释的新框架.
- 通过全面评估益处和危害,解决对量化和比较患者体验的方法的需求.
- 将临床研究与医疗决策中面临的实际问题保持一致.
主要方法:
- 门的范式提供了一个以患者为中心的框架,用于临床试验中的益处:风险评估.
- 它涉及到仔细的研究设计和全面的统计分析,根据DOOR方法量身定制.
- 为实施DOOR提供了一个推的统计分析计划和说明性示例.
主要成果:
- 通过整合多种结果,DOOR范式可以对治疗替代方案进行更全面的评估.
- 它有助于评估整体的好处:风险,超越单一的终点分析.
- 这种方法提高了关于治疗有效性和安全性的研究结果的概括性.
结论:
- 门范式通过提供以患者为中心的益处:风险评估的强有力的方法来推进临床试验科学.
- 实施DOOR可以提高研究成果在临床实践中的适用性.
- 自由可用的工具支持使用DOOR范式的研究设计和分析.
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