Drug repurposing using real-world data

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.

Automated summary

The use of real-world data in drug repurposing is advantageous for supplementing de novo drug discovery and incorporating real-world evidence in regulatory approvals. A scoping review analyzed 250 studies, including hypothesis generation, hypothesis validation, and safety assessment. Challenges include isolated data sources, false-positive signals, bias and confounding in hypothesis validation, and the lack of regulatory guidance.