Real-world evidence to support the FDA's regulatory review for new drug approvals

LI Ze-yu; YANG Yue

doi:10.20251/j.cnki.1003-3734.2025.10.007

Abstract：Objective:This study aims to analyze the utilization of real-world evidence(RWE)in the new drug application(NDA)process at the US FDA from January 2022 to July 2024,and to evaluate its impact on regulatory decisions.The findings will serve as a reference for RWE-based drug review decisions and new drug development by applicants.Methods:Through a comprehensive review of public resources on the FDA official website,we extracted and analyzed public documents of all new molecular entity(NME)NDA applications approved by CDER from January 1,2022 to July 1,2024,including proportion,type,data source and supporting role of RWE in NDA.The feedback from the FDA on RWE research was also summarized,including issues such as study design,data quality,and control of bias and confounding factors.Results:The study found that among the 77 NDA applications included in the review,36(47%)included RWE studies;19(53%)only supported safety,6(17%)only supported efficacy,and 11(30%)supported safety and efficacy;9(25%)were submitted as primary evidence,and27(75%)were submitted as supporting evidence;17(47%)of the 36 approvals were for rare diseases.The FDA's feedback on RWE studies mainly focused on research design issues,data quality issues,and insufficient control of bias and confounding factors.Conclusion:RWE is increasingly utilized in new drug applications,but the FDA has raised many limitations in the review process.In order to make RWE better support the review decisions,applicants should focus on the scientificity and rigor of the study design,the reliability and relevance of the data source,and the transparency of the research process,and adopt high-quality data and scientific research design to improve the applicability and reliability of RWE in NDA.