Translating evidence into practice: eligibility criteria fail to eliminate clinically significant differences between real-world and study populations
Translating evidence into practice: eligibility criteria fail to eliminate clinically significant differences between real-world and study populations
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DOI：
10.1038/s41746-020-0277-8
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发表时间：
2020-05-11
影响因子：
15.2
通讯作者：
Perotte, Adler
中科院分区：
文献类型：
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作者：
Averitt, Amelia J.;Weng, Chunhua;Perotte, Adler
随机对照试验（RCT）被认为是最有信誉的证据来源。在一些研究中，干预本身以外的因素可能会影响测量的效果，这种情况称为治疗效果异质性（HTE）。如果RCT人群与真实世界人群在诱发HTE的因素上存在差异，则试验效应将无法复制。随机对照试验的合格性标准应确定其证据将重复的亚群。然而，资格标准在多大程度上确定了适当的人口尚不清楚，这引起了对普遍性的关切。我们将来自RCT的报告数据与来自大型学术医疗中心的电子健康记录的真实数据进行了比较，这些数据是根据RCT资格标准策划的。我们的研究结果显示了RCT人群和我们的观察性队列之间的根本差异，这表明资格标准可能不足以识别RCT证据将重复的适用现实世界人群。
Randomized controlled trials (RCTs) are regarded as the most reputable source of evidence. In some studies, factors beyond the intervention itself may contribute to the measured effect, an occurrence known as heterogeneity of treatment effect (HTE). If the RCT population differs from the real-world population on factors that induce HTE, the trials effect will not replicate. The RCTs eligibility criteria should identify the sub-population in which its evidence will replicate. However, the extent to which the eligibility criteria identify the appropriate population is unknown, which raises concerns for generalizability. We compared reported data from RCTs with real-world data from the electronic health records of a large, academic medical center that was curated according to RCT eligibility criteria. Our results show fundamental differences between the RCT population and our observational cohorts, which suggests that eligibility criteria may be insufficient for identifying the applicable real-world population in which RCT evidence will replicate.