将参与者的福利纳入连续的多重分配随机试验
Xinru Wang1, Nina Deliu2,3, Yusuke Narita4
1Centre for Quantitative Medicine, Duke-NUS Medical School, 169857, Singapore.
Biometrics
|February 16, 2024
概括
新的SMART-EXAM试验设计通过个性化治疗任务来提高患者福利. 与传统方法相比,这种方法提高了动态治疗方案 (DTR) 和试验有效性.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 医疗保健服务研究 医疗服务研究
背景情况:
- 动态治疗方案 (DTRs) 指导基于患者状况的顺序治疗决策.
- 顺序,多重分配,随机试验 (SMART) 用于开发最佳的DTR.
- 传统的SMART设计引发了伦理方面的担忧,并可能影响试验有效性,因为参与者被分配到次优治疗.
研究的目的:
- 引入和评估SMART-EXAM框架,一个新的SMART设计.
- 通过纳入患者偏好和预测的治疗效果来改善临床试验参与者的福利.
- 与传统的SMART相比,评估SMART-EXAM在构建最佳DTR方面的性能.
主要方法:
- 该研究提出了SMART-EXAM框架,详细说明了其实施步骤.
- 它将SMART-EXAM的性能与传统的SMART设计进行比较.
- 评估包括评估参与者福利和构建最佳DTR的能力.
主要成果:
- 在临床试验中,SMART-EXAM显示了改善参与者福利的潜力.
- 设计显示了在参数定义良好时构建最佳DTR的可取能力.
- 绩效与传统的SMART进行评估,突出了以参与者为中心的结果的改进.
结论:
- 在伦理和有效的临床试验中,SMART-EXAM为传统的SMART设计提供了一个有希望的替代方案.
- 将患者偏好纳入随机化可以提高试验招募和有效性.
- 该框架具有实际应用,通过儿童ADHD的试验来说明.
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