美国应用真实世界证据辅助医药产品监管决策的研究

目的：回顾美国FDA真实世界数据(RWD)与真实世界证据(RWE)的发展过程，总结出促进 RWE应用所具有的关键要素，阐述RWE在医药产品监管决策中的机遇与挑战，为推动RWE在我国监管决策中的应用提出合理建议。方法：采用国内外文献研究，回顾美国FDA将RWE引入监管决策的相关立法及指南进程，分析在监管决策中引入RWE需考虑的关键问题。结果与结论：真实世界研究应当建立监管层面的通用数据标准，开发建设中国可以实际应用的患者登记系统、大型医疗数据共享系统等数据基础设施，遵循最佳方法标准进行研究设计。在医疗实践中结合以电子形式捕捉源数据、移动技术和远程医疗等先进技术，提高RWE在药品和器械审评、监管以及医保支付等决策中的应用。

Application of Real World Evidence in Supporting Drug and Medical Device Regulatory Decisions in U.S.A.

Objective: To review the development of FDA Real World Data (RWD) and Real World Evidence (RWE), summarize the key elements that promote the application of RWE, elaborate the opportunities and challenges of RWE in the regulatory decision-making of drug and medical devices, and to put forward reasonable suggestions to promote the application of RWE in China''s regulatory decision-making. Methods: By searching domestic and foreign literature and reviewing FDA''s relevant methods and guidelines for introducing RWE into regulatory decisions, this paper analyses the key issues to be considered in the introduction of RWE in regulatory decisions. Results and Conclusion: Real-world study should establish general data standards at the regulatory level, develop and build data infrastructures such as patient registration systems and large-scale medical data sharing systems that could be applied in China, and follow the best method standards for research design. In medical practice, advanced technologies such as electronic capture of source data, mobile technology and telemedicine are combined to improve the application of RWE in drug and device evaluation, supervision and health insurance payment, etc.