Drug repurposing using real-world data. Issue 1 (January 2023)

• Record Type: Journal Article
• Authors: Tan, George S.Q.; Sloan, Erica K.; Lambert, Pete; Kirkpatrick, Carl M.J.; Ilomäki, Jenni
• Is Part Of: Drug discovery today. Volume 28:Issue 1(2023)
• Publication Date: 2023-01
• Subjects: drug repurposing -- drug repositioning -- big data -- real-world data -- real-world evidence -- scoping review; EHR Electronic health records -- RWD Real-world data -- RWE Real-world evidence -- RCT Randomized controlled trials -- FDA Food and Drug Administration -- EMA European Medicines Agency; Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2022.103422
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: 250 records, including 144 original studies, met the eligibility criteria for inclusion in this review. Studies evaluated repurposing mainly for cardiovascular disease, COVID-19 or cancer. A key limitation of the use of real-world data (RWD) in hypothesis generation is false-positive signals. A key limitation of RWD in hypothesis validation is potential bias and confounding. Recent support from regulators may accelerate RWD use in drug repurposing. Abstract : The use of real-world data in drug repurposing has emerged due to well-established advantages of drug repurposing in supplementing de novo drug discovery and incentives in incorporating real-world evidence in regulatory approvals. We conducted a scoping review to characterize repurposing studies using real-world data and discuss their potential challenges and solutions. A total of 250 studies met the inclusion criteria, of which 36 were original studies on hypothesis generation, 101 on hypothesis validation, and seven on safety assessment. Key challenges that should be addressed for future progress in using real-world data for repurposing include isolated data sources with poor clinical granularity, false-positive signals from data mining, the sensitivity of hypothesis validation to bias and confounding, and the lack of clear regulatory guidance.