在晚期随机对照试验中,何时应该使用因数设计?
Ian R White1, Alexander J Szubert1, Babak Choodari-Oskooei1
1MRC Clinical Trials Unit at UCL, London, UK.
Clinical trials (London, England)
|October 31, 2023
概括
因数设计可以有效地评估临床试验中的多种干预措施,从而节省资源. 研究人员应考虑成功实施的特定临床,实践,统计和外部因素,特别是当相互作用效应最小时.
科学领域:
- 临床试验的设计
- 医学研究方法论医学研究方法论.
- 医疗保健中的统计分析.
背景情况:
- 分数式设计同时评估多个干预措施 (例如,2x2设计).
- 这些设计可以比传统的随机对照试验更节省资源.
- 尽管有好处,但因数设计在临床研究后期阶段仍未得到充分利用.
研究的目的:
- 识别和讨论实施因数随机对照试验的关键问题.
- 通过解决潜在的挑战,鼓励更广泛地采用因数设计.
- 为在后期阶段设置中考虑这种设计的研究人员提供指导.
主要方法:
- 对2000年至2022年间发表的因数随机对照试验的期刊文章进行系统调查.
- 基于文献审查和专家经验,分析识别的问题.
- 将问题分类为临床,实践,统计和外部因素.
主要成果:
- 确定了15个有利于因子设计的关键问题,包括易于联合管理,低相互作用风险和类似的资格标准.
- 实际优势包括不受影响的招聘和坚持,以及简化的盲目化.
- 统计上的好处包括合适的分析尺度和不需要多重性调整;设计可以减少样本大小要求.
结论:
- 因数设计未得到充分利用,应在临床研究中更频繁地考虑.
- 成功实施取决于仔细考虑确定的临床,实践,统计和外部因素.
- 干预措施之间相互作用效应的低潜力是最佳使用的关键前提.
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