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Lower Risk of Heart Failure and Death in Patients Initiated on Sodium-Glucose Cotransporter-2 Inhibitors Versus Other Glucose-Lowering Drugs: The CVD-REAL Study (Comparative Effectiveness of Cardiovascular Outcomes in New Users of Sodium-Glucose Cotransporter-2 Inhibitors).与其他降糖药物相比,起始使用钠-葡萄糖协同转运蛋白2抑制剂的患者发生心力衰竭和死亡的风险更低:CVD-REAL研究(钠-葡萄糖协同转运蛋白2抑制剂新使用者心血管结局的比较有效性)
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Effectiveness and safety of dabigatran and warfarin in real-world US patients with non-valvular atrial fibrillation: a retrospective cohort study.达比加群酯与华法林在美国非瓣膜性心房颤动真实世界患者中的有效性和安全性:一项回顾性队列研究。
J Am Heart Assoc. 2015 Apr 10;4(4):e001798. doi: 10.1161/JAHA.115.001798.
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Cardiovascular, bleeding, and mortality risks in elderly Medicare patients treated with dabigatran or warfarin for nonvalvular atrial fibrillation.老年 Medicare 患者在非瓣膜性心房颤动中接受达比加群或华法林治疗的心血管、出血和死亡率风险。
Circulation. 2015 Jan 13;131(2):157-64. doi: 10.1161/CIRCULATIONAHA.114.012061. Epub 2014 Oct 30.
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Design and rationale of Global Registry on Long-Term Oral Antithrombotic Treatment in Patients with Atrial Fibrillation: a global registry program on long-term oral antithrombotic treatment in patients with atrial fibrillation.《全球心房颤动患者长期口服抗栓治疗注册研究:一项关于心房颤动患者长期口服抗栓治疗的全球注册研究计划》
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Assessing the comparative effectiveness of newly marketed medications: methodological challenges and implications for drug development.评估新上市药物的比较疗效:方法学挑战及对药物开发的影响。
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An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies.倾向得分法在观察性研究中减少混杂效应的介绍
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Beyond the randomized clinical trial: the role of effectiveness studies in evaluating cardiovascular therapies.超越随机临床试验:有效性研究在评估心血管治疗中的作用。
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真实世界证据(RWE)——我们(对RWE)准备好了吗?

Real world evidence (RWE) - Are we (RWE) ready?

作者信息

Suvarna Viraj Ramesh

机构信息

Boehringer Ingelheim India Private Limited, Mumbai, Maharashtra, India.

出版信息

Perspect Clin Res. 2018 Apr-Jun;9(2):61-63. doi: 10.4103/picr.PICR_36_18.

DOI:10.4103/picr.PICR_36_18
PMID:29862197
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5950611/
Abstract

Real world evidence is important as it complements data from randomised controlled trials (RCTs). Both have limitations in design, interpretation, and extrapolatability. It is imperative one designs real world studies in the right way, else it can be misleading. An RCT is always considered higher in the evidence ladder and when there is discordance between a real world study and an RCT, it is the latter which is always considered pristine because of the way it is conducted, e.g., randomization, prospective, double-blind, etc. A real world study can also be done prospectively, and propensity score matching can be used to construct comparable cohorts but may not be able to account for certain biases or confounding factors the way an RCT can do. Nevertheless, comparative effectiveness research in the real world is being resorted to, especially for efficiency studies or pharmacoeconomic analyses, and with the advent of machine learning, the electronic healthcare database mining can result in algorithms that help doctors identify clinical characteristics that correlate with optimal response of a patient to a drug/regimen, thus helping him/her select the right patient for the right drug.

摘要

真实世界证据很重要,因为它补充了随机对照试验(RCT)的数据。两者在设计、解释和外推性方面都有局限性。必须以正确的方式设计真实世界研究,否则可能会产生误导。RCT在证据等级中总是被认为更高,当真实世界研究与RCT之间存在不一致时,由于其实施方式(例如随机化、前瞻性、双盲等),后者总是被认为是原始的。真实世界研究也可以前瞻性地进行,倾向得分匹配可用于构建可比队列,但可能无法像RCT那样解释某些偏差或混杂因素。尽管如此,尤其是在效率研究或药物经济学分析中,人们正在采用真实世界中的比较有效性研究,并且随着机器学习的出现,电子医疗数据库挖掘可以产生算法,帮助医生识别与患者对药物/治疗方案的最佳反应相关的临床特征,从而帮助他/她为合适的药物选择合适的患者。