临床试验中的自适应设计:系统性审查-第一部分
Mohamed Ben-Eltriki1,2,3, Aisha Rafiq4, Arun Paul4
1Department of Pharmacology and Therapeutics, Max Rady College of Medicine, University of Manitoba, Winnipeg, MB, Canada. mbeneltriki@alumni.ubc.ca.
BMC medical research methodology
|October 4, 2024
概括
适应性设计 (ADs) 提高了临床试验的灵活性和效率. 本综述详细介绍了它们的使用,强调了报告方面的挑战,以及需要更好的指导方针和教育以实现更广泛的采用.
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
- 药物开发 药物开发
背景情况:
- 适应性设计 (ADs) 在临床试验中提供了灵活性,效率和成本节省.
- 研究界对阿尔茨海默病的特征,益处和局限性的广泛理解仍然有限.
- 本系统性审查 (第一部分) 探讨了ADS在已发表的临床试验中的应用.
研究的目的:
- 为适应性临床试验的方法特征提供全面的概述.
- 在适应性试验中确定常见的适应,统计方法和报告实践.
- 为未来的比较和建议奠定基础,特别是对于儿科研究.
主要方法:
- 在MEDLINE,Embase和国际制药摘要 (2010年至2020年4月) 的系统文献搜索.
- 包括临床试验协议,报告和二次分析,使用药物或疫苗的附加剂.
- 提取和总结有关研究特征,调整,统计分析和操作考虑的数据.
主要成果:
- 包括317项适应性试验,主要涉及瘤学 (53%) 和早期发育阶段 (87%).
- 剂量发现设计 (38.2%) 和自适应随机化 (16.7%) 是最常见的;频率主义方法 (64%) 比贝叶斯式 (24%) 更普遍.
- 报道的适应细节不一致,影响了解释和概括性.
结论:
- 适应性临床试验显示出效率的希望,但需要标准化的报告.
- 改善报告准则和教育策略对于有效实施至关重要.
- 应对科学,运营和伦理方面的挑战,将有助于更广泛地采用ADS.
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