量化和控制回归到平均值对随机对照试验中的影响
Daniel M Goldenholz1, Eliana B Goldenholz1, Ted J Kaptchuk1
1Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA.
Epilepsia
|July 28, 2023
概括
在药物试验中控制回归到平均值 (RTM) 减少了安慰剂效应. 从资格标准中排除基线期显著提高了试验有效性并降低了成本.
科学领域:
- 临床试验 临床试验
- 的研究研究.
- 生物统计学 生物统计学
背景情况:
- 对药物治疗的随机对照试验 (RCT) 是昂贵的.
- 高的安慰剂反应率,部分是由于回归到平均值 (RTM),使试验成本膨胀.
- RTM是随着时间的推移,发作频率自然下降到个体的平均水平.
研究的目的:
- 澄清症RCT中资格标准和RTM之间的关系.
- 评估RTM如何影响安慰剂反应和试验疗效.
- 确定优化RCT设计的战略,以降低成本.
主要方法:
- 使用了一个统计学上真实的日记模拟器.
- 在对抗药性的模拟II/III期RCT中,通过不同的资格标准探索了RTM的影响.
- 模拟分析了包括与排除基线时期在资格标准中的影响.
主要成果:
- 包括基准时期在资格标准中增加了RTM分数 (25%-47%).
- 较高的RTM分数与增加的安慰剂反应率 (RR50:2%-9%) 和减少的统计疗效 (RR50:47%-67%) 相关联.
- 除了基线标准,所需的患者人数减少了约30%.
结论:
- 资格标准显著和可预测地影响性RCT中的RTM和安慰剂反应.
- 调整资格标准可以提高药物效应的可检测性,提高试验有效性.
- 将基线排除在资格标准之外,可以大幅提高试验有效性和成本效益.
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