Simulation for Predicting Effectiveness and Safety of New Cardiovascular Drugs in Routine Care Populations.
Simulation for Predicting Effectiveness and Safety of New Cardiovascular Drugs in Routine Care Populations.
复制标题
预测新心血管药物在常规护理人群中的有效性和安全性的模拟。
DOI：
10.1002/cpt.1045
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发表时间：
2018
影响因子：
6.7
通讯作者：
Gagne,JoshuaJ
中科院分区：
文献类型：
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作者：
Najafzadeh,Mehdi;Schneeweiss,Sebastian;Choudhry,NiteeshK;Wang,ShirleyV;Gagne,JoshuaJ
在存在治疗效果异质性（HTE）的情况下，随机对照试验（RCT）的平均治疗效果可能不适用于不同患者，例如观察性环境中的患者。我们的目标是开发一种新方法，使用个体水平模拟将RCT结果扩展到存在HTE的目标患者人群。为此，我们比较了长期抗凝治疗随机评价（RE‐LY）试验的结果，以及比较达比加群与华法林在房颤患者中的获益和风险的两项观察性研究的结果。我们开发了一个模拟模型，使用已发表的结局风险模型和参与者的基线特征复制了RE‐LY中观察到的缺血性卒中和大出血的发生率。我们使用经过验证的模拟模型来预测如果在与观察性研究相似的人群中进行RCT，结果会是什么。
In the presence of heterogeneity of treatment effect (HTE), the average treatment effect from a randomized controlled trial (RCT) may not be applicable to different patients, such as those in observational settings. Our objective was to develop a novel approach that uses individual‐level simulation to expand RCT results to target patient populations in the presence of HTE. For this purpose, we compared the results of the Randomized Evaluation of Long‐Term Anticoagulation Therapy (RE‐LY) trial, and two observational studies that compared benefits and risks of dabigatran to warfarin in patients with atrial fibrillation. We developed a simulation model that replicates the rates of ischemic stroke and major bleeding observed in RE‐LY using published outcome risk models and participants' baseline characteristics. We used our validated simulation model to predict what the results of the RCT would have been had it been conducted in populations similar to those in the observational studies.