Research on Challenges and Policies of Real-world Evidence Applied in Pediatric Medical Product R&D / 中国药房

ABSTRACT

OBJECTIVE：To provid e reference for promoting the application of real-world evidence in pediatric medical product R&D. METHODS ：The progress of real-world evidence in pediatrics in the United States and Europe was introduced. The obstacles of real-world evidence in pediatric drug R&D were analyzed ，and a series of feasibility suggestions were put forward. RESULTS & CONCLUSIONS：Real world evidence had been widely used in pediatric medical product R&D ，including evaluation and validation of biomarkers and prognostic indicators ，clinical trial design and optimization ，support for extended indications ， post-marketing safety monitoring/pharmacovigilance ，etc. There were still many obstacles ，such as data fragmentation and access restrictions，lack of standardized analysis methods for real-world evidence ，lack of key pediatric variables ，and difficulties in obtaining informed consent from pediatric patients. In order to promote the application of real-world evidence in pediatrics ，it is suggested to strengthen the construction of data infrastructure ，expand the application of general data model ，develop methods for collecting longitudinal data of pediatric patients ，and carry out reasonable research design.