美国和欧盟儿科用药审评机构职责与作用探析

On Responsibilities and Functions of Review Agencies for Pediatric Drug in the United States and the European Union

目的:为提高我国儿科用药的审评质量和效率提供借鉴。方法:收集与整理美国、欧盟儿科用药审评、管理机构的职责与作用,并加以研究分析。结果与结论:近两年,我国成立了与两个儿科用药相关的专家委员会,分别隶属于药品审评中心及国家卫生和计划生育委员会,这两个专家委员会成员仍然都是儿科临床专家,相对于美国FDA和欧盟EMA,缺少了专门的药学、药理毒理、药物警戒、统计等方面的专家。目前,国家药品审评中心并没有专门的儿科用药审评团队。建议我国借鉴美国与欧盟的经验,在药品审评中心内部成立专门的儿科用药审评部门。为各审评机构在技术审评过程中遇到的与儿科用药相关问题提供日常内部咨询;为审评中心制定与儿科用药相关的技术规范、指导原则等做全面的统筹规划;为企业进行儿科用药研发提供科学建议。

Objective： To provide references for effective pediatric drug review in China. Methods： The FDA and EMA entities for pediatric medicines review and administration were sorted out, and their functions and responsibilities were analyzed. Results and Conclusion： In recent two years, two experts committees relevant to pediatric drug have been established in China, which are subordinate to CDE and NHFPC respectively. The members of these two committees are all pediatric clinicians. Compared to FDA and EMA, there are no CMC, nonclinical, pharmacovigilance, biostatistics experts. Currently, there is no special team to review the pediatric drug in CDE. It is suggested that China should learn from the US and EU for references, and establish the internal pediatric review committee within CDE. It will provide the routine consultation to other groups for the pediatric drug review, work on the overall planning for the technical guidelines about the pediatric drug, and offer scientific advice to the sponsors.