多臂,多阶段适应性和适应性平台随机对照试验的设计和修改中的经济考虑:系统性文献审查
Mwayi Kachapila1, Samuel Watson2, Thomas Pinkney3
1Health Economics Unit, Institute of Applied Health Research, College of Medical and Dental Sciences, University of Birmingham, Birmingham, England, UK; Global Health and Global Surgery Unit, National Institute for Health and Care Research (NIHR), University of Birmingham, Birmingham, England, UK.
概括
将健康经济学纳入适应性试验设计正在出现,但有限. 虽然成本效益分析可以为多臂,多阶段 (MAMS) 试验的决策提供信息,但它们在指导试验修改中的使用仍然受到限制.
科学领域:
- 临床试验设计 临床试验设计
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
背景情况:
- 关于将经济考虑纳入适应性试验设计的价值存在不确定性.
- 多臂,多阶段 (MAMS) 适应性和适应性平台试验为同时评估多种干预提供了复杂的框架.
研究的目的:
- 系统地审查用于将健康经济分析纳入MAMS适应性和适应性平台试验的分析方法和因素.
- 评估经济考虑如何影响这些先进的试验结构的设计和修改.
主要方法:
- 在主要数据库 (EMBASE,MEDLINE,科学网,Scopus,临床试验.gov) 进行系统的文献搜索,截至2023年8月.
- 两位盲目评论员对文章进行选.
- 在健康经济分析中使用的方法及其对试验设计的影响的叙述综合.
主要成果:
- 其中包括17篇文章,4篇经济评价和13篇议定书.
- 没有报告使用试验前经济评估为初始设计提供信息的试验.
- 在14篇文章中,可以进行临时成本和效益估计,其中有5篇临时成本效益分析,其中3篇是关于试验武器的知情决定.
结论:
- 卫生经济学正在纳入一些MAMS适应性和适应性平台试验,以指导修改.
- 在这些试验中,经济证据的应用受到设计和环境的限制,尽管它对决策的潜在价值.
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