模拟随机临床试验与非随机现实世界证据研究:RCT DUPLICATE倡议的第一批结果
Jessica M Franklin1, Elisabetta Patorno1, Rishi J Desai1
1Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA (J.M.F., E.P., R.J.D., R.J.G., A.P., L.G.B., H.L., N.G., S.S.).
Circulation
|December 17, 2020
概括
现实世界证据 (RWE) 研究可以模拟随机对照试验 (RCT),但协议有所不同. 对心血管结果的RWE模拟与RCT有很好的一致性,支持RWE在医疗产品评估中的作用.
科学领域:
- 流行病学
- 卫生经济学与成果研究
- 临床试验设计
背景情况:
- 监管机构正在探索非干预性现实世界证据 (RWE) 研究以评估医疗产品的有效性.
- 该计划模拟使用现实数据进行随机对照试验 (RCT),以比较结果.
研究的目的:
- 通过使用现实索赔数据来评估模拟RCT的可行性和准确性.
- 通过将RWE研究与相应的RCT进行比较,评估抗糖尿病和抗血小板药物的心血管结果.
主要方法:
- 使用美国商业和医疗保险索赔数据复制了10项RCT (3项活性控制,7项安慰剂控制).
- 实施的RCT包括/排除标准,终点和对照群体.
- 使用了超过120个预先规定的混因子的倾向性得分匹配;前性定义的措施和注册的协议.
主要成果:
- 在10个模拟中达成6个监管结论.
- 在10项研究中的8项中,RWE的危险率估计在RCT的95% CI范围内.
- 在10个复制中,有9个符合监管或估计协议的成功标准,其中大多数对效果估计有很小的差异.
结论:
- 试验结果与RWE结果之间的一致性取决于所使用的指标.
- 使用具有相似指示和使用模式的活性比较剂可以提高RWE的有效性.
- 为了确定RWE-RCT协议的一致性和背景,需要进行进一步的试验模拟.
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