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      The use of real-world data in drug repurposing

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          Abstract

          Drug repurposing, or repositioning, is to identify new uses for existing drugs. Significantly reducing the costs and time-to-market of a medication, drug repurposing has been an alternative tool to accelerate drug development process. On the other hand, ‘real world data (RWD)’ has been also increasingly used to support drug development process owing to its better representing actual pattern of drug treatment and outcome in real world. In the healthcare domain, RWD refers to data collected from sources other than traditional clinical trials; for example, in electronic health records or claims and billing data. With the enactment of the 21st Century Cures Act, which encourages the use of RWD in drug development and repurposing as well, such increasing trend in RWD use will be expedited. In this context, this review provides an overview of recent progresses in the area of drug repurposing where RWD was used by firstly introducing the increasing trend and regulatory change in the use of RWD in drug development, secondly reviewing published works using RWD in drug repurposing, classifying them in the repurposing strategy, and lastly addressing limitations and advantages of RWDs.

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          Most cited references28

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          Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available.

          Ideally, questions about comparative effectiveness or safety would be answered using an appropriately designed and conducted randomized experiment. When we cannot conduct a randomized experiment, we analyze observational data. Causal inference from large observational databases (big data) can be viewed as an attempt to emulate a randomized experiment-the target experiment or target trial-that would answer the question of interest. When the goal is to guide decisions among several strategies, causal analyses of observational data need to be evaluated with respect to how well they emulate a particular target trial. We outline a framework for comparative effectiveness research using big data that makes the target trial explicit. This framework channels counterfactual theory for comparing the effects of sustained treatment strategies, organizes analytic approaches, provides a structured process for the criticism of observational studies, and helps avoid common methodologic pitfalls.
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            Drug repositioning: identifying and developing new uses for existing drugs.

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              Real-World Evidence - What Is It and What Can It Tell Us?

                Author and article information

                Journal
                Transl Clin Pharmacol
                Transl Clin Pharmacol
                TCP
                Translational and Clinical Pharmacology
                Korean Society for Clinical Pharmacology and Therapeutics
                2289-0882
                2383-5427
                September 2021
                27 September 2021
                : 29
                : 3
                : 117-124
                Affiliations
                Department of Pharmacology, Yonsei University College of Medicine, Seoul 03722, Korea.
                Author notes
                Correspondence to Kyungsoo Park. Department of Pharmacology, Yonsei University College of Medicine, 50-1 Yonsei-ro, Seodaemun-gu, Seoul 03722, Korea. kspark@ 123456yuhs.ac
                Author information
                https://orcid.org/0000-0002-6972-1143
                Article
                10.12793/tcp.2021.29.e18
                8492393
                34621704
                bbed029e-fdbf-47bc-b861-0bf5001aaf29
                Copyright © 2021 Translational and Clinical Pharmacology

                It is identical to the Creative Commons Attribution Non-Commercial License ( https://creativecommons.org/licenses/by-nc/4.0/).

                History
                : 13 September 2021
                : 24 September 2021
                : 24 September 2021
                Categories
                Review

                drug repurposing,real world data,electronic health record,21st century cures act

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