根据患者偏好的治疗效益的复合终点
Ying Lu1,2, Qian Zhao1,3, Jiying Zou4
1Department of Biomedical Data Science, Stanford University School of Medicine.
Statistics in biopharmaceutical research
|November 20, 2023
概括
这项研究引入了一种新的复合结果排名 (DOOR) 的可取性,通过结合复杂疾病中多个结果的个体患者偏好来个性化治疗评估.
科学领域:
- 生物统计学 生物统计学
- 临床试验 临床试验
- 患者为中心的结果.
背景情况:
- 复杂的疾病涉及多种结果,对个体患者来说的重要性各不相同.
- 目前用于评估治疗效果的方法往往无法考虑患者特定的结果优先级.
- 在临床试验分析中,需要以患者为中心的方法.
研究的目的:
- 提出一种新的复合结果排名 (DOOR) 方法的可取性.
- 将个体患者对结果重要性的偏好纳入治疗效果评估中.
- 开发一种以患者为中心的指标,用于评估临床试验中的整体治疗效益.
主要方法:
- 开发了一个复合的结果排名可取性 (DOOR) 框架.
- 整合了个人级别的结果重要性排名.
- 使用威尔科克森-曼-惠特尼U统计学来平均对对的DOOR分数.
- 采用理论和经验分析来验证方法.
主要成果:
- 复合DOOR方法有效地整合了患者层面的偏好.
- 基于基线排名的分层随机化是可行的.
- 拟议的方法提供了以患者为中心的整体治疗效益衡量标准.
- 证明了统计性质,并与传统方法相比有利.
结论:
- 复合的DOOR准确地反映了复杂疾病中的患者特定优先事项.
- 这种方法适用于关键和比较有效性试验.
- 加强以患者为中心的治疗疗效和益处的评估.
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