Strengths and weaknesses of 'real-world' studies involving non-vitamin K antagonist oral anticoagulants.
Strengths and weaknesses of 'real-world' studies involving non-vitamin K antagonist oral anticoagulants.
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DOI：
10.1136/openhrt-2018-000788
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发表时间：
2018
期刊：
影响因子：
2.7
通讯作者：
Fox KAA
中科院分区：
文献类型：
--
作者：
Camm AJ;Fox KAA
随机对照试验（RCT）为比较一种治疗或干预与另一种治疗或干预的疗效提供了参考标准。然而，随机对照试验具有严格的纳入和排除标准;因此，它们不能完全代表非真实世界的人群。真实世界证据（RWE）研究涵盖广泛的研究方法和数据来源，可大致分为非干预性研究、患者登记研究、索赔数据库研究、患者调查和电子健康记录研究。如果设计得当，RWE研究包括的患者人群比RCT更能代表COPD患者人群，但它们不能为比较治疗策略提供可靠的基础。RWE研究可以有非常大的样本量，可以提供通常被排除在RCT之外的患者群体的治疗信息，通常比RCT更便宜，更快，并且可以评估广泛的结果。RWE研究的局限性可能包括内部效度低、缺乏围绕数据收集的质量控制以及对比较结果的多种偏倚来源的敏感性。RWE研究可以通过提供关于治疗实践和患者特征的有价值信息来补充RCT的发现。这些信息对于指导治疗决策以及报销和支付决策是必要的。自2010年以来，RWE研究已广泛应用于非维生素K拮抗剂口服抗凝剂的上市后批准评估。然而，在解释研究结果时，必须仔细考虑与不同类型RWE相关的益处、成本、限制和方法学挑战。
Randomised controlled trials (RCTs) provide the reference standard for comparing the efficacy of one therapy or intervention with another. However, RCTs have restrictive inclusion and exclusion criteria; thus, they are not fully representative of an unselected real-world population. Real-world evidence (RWE) studies encompass a wide range of research methodologies and data sources and can be broadly categorised as non-interventional studies, patient registries, claims database studies, patient surveys and electronic health record studies. If appropriately designed, RWE studies include a patient population that is far more representative of unselected patient populations than those of RCTs, but they do not provide a robust basis for comparing treatment strategies. RWE studies can have very large sample sizes, can provide information on treatments in patient groups that are usually excluded from RCTs, are generally less expensive and quicker than RCTs, and can assess a broad range of outcomes. Limitations of RWE studies can include low internal validity, lack of quality control surrounding data collection and susceptibility to multiple sources of bias for comparing outcomes. RWE studies can complement the findings from RCTs by providing valuable information on treatment practices and patient characteristics among unselected patients. This information is necessary to guide treatment decisions and for reimbursement and payment decisions. RWE studies have been extensively applied in the postmarketing approval assessment of non-vitamin K antagonist oral anticoagulants since 2010. However, the benefits, costs, limitations and methodological challenges associated with the different types of RWE must be considered carefully when interpreting the findings.