真实世界证据应用于儿科医药产品研发中的挑战及对策研究

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

目的:为促进真实世界证据在儿科医药产品研发中的应用提供参考。方法:介绍真实世界证据在欧美国家儿科领域应用的进展情况,分析真实世界证据在儿科医药产品研发中的应用障碍,并提出一系列可行性建议。结果与结论:真实世界证据已被广泛应用于儿科医药产品研发中,包括评估和验证生物标志物及预后指标、临床试验设计与优化、支持扩大适应证、上市后安全性监测/药物警戒等环节。但仍面临许多障碍,诸如真实世界证据来源与访问受限、真实世界数据标准化分析方法欠缺、儿科关键变量丢失、获得儿科患者知情同意困难等。建议今后应加强数据基础设施建设,扩大通用数据模型应用,开发收集儿科患者纵向数据的方法,提高研究设计的合理性等,以促进真实世界证据在儿科领域的应用。

OBJECTIVE:To provide 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.