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Strengths and weaknesses of 'real-world' studies involving non-vitamin K antagonist oral anticoagulants.

Camm, AJ; Fox, KAA (2018) Strengths and weaknesses of 'real-world' studies involving non-vitamin K antagonist oral anticoagulants. Open Heart, 5 (1). e000788. ISSN 2053-3624 https://doi.org/10.1136/openhrt-2018-000788
SGUL Authors: Camm, Alan John

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Abstract

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.

Item Type: Article
Additional Information: © Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted. This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Keywords: anticoagulant, limitations, real-world evidence
SGUL Research Institute / Research Centre: Academic Structure > Molecular and Clinical Sciences Research Institute (MCS)
Journal or Publication Title: Open Heart
ISSN: 2053-3624
Language: eng
Dates:
DateEvent
21 April 2018Published
6 March 2018Accepted
Publisher License: Publisher's own licence
PubMed ID: 29713485
Go to PubMed abstract
URI: https://openaccess.sgul.ac.uk/id/eprint/109791
Publisher's version: https://doi.org/10.1136/openhrt-2018-000788

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