欧盟药品加速审评政策及实证分析

Empirical Analysis on the Policies of Drug Accelerated Assessment in the European Union

目的:为我国新药审评审批制度的深入改革提供参考。方法:系统分析欧盟药品加速审评政策的法律基础,并与其他同类政策进行比较;对加速审评流程的关键环节,尤其是加速审评申请的递交前准备、许可前检查、申请的递交和评估等详细流程进行介绍。以Maviret为实例进行个案剖析,实证分析欧盟加速审评政策的应用效果。结果:加速审评是欧盟促进患者尽早获得新药的几项法律条款之一,与条件上市许可、同情用药、优先药物计划、孤儿药认定、医院豁免等政策一样作为在欧盟尽早获得新药的主要途径之一。当药物具备充分的治疗数据、具有重大公共健康利益和治疗创新性时,即有望获得加速审评。在欧盟法规(726/2004/EC)的基础上,欧洲药品管理局(EMA)不断完善加速审评政策体系,相继发布了相关指导原则(指南)与加速审评程序的执行时间表,为该政策的切实落地提供了详细、具体的指导。一旦药物进入加速审评程序,则其审评时间将由标准审评程序的210日缩短至150日。治疗丙型肝炎病毒感染的新药Maviret从2017年1月20日开始加速审评,直到2017年6月22日获得EMA人用药品委员会(CHMP)发布批准其上市许可的肯定意见,整个流程不到半年。结论:欧盟药品加速审评政策既有完善的立法支持,又有详细具体的实施细则和执行时间表,能够加快一些公众急需的、具有特殊医疗优势的药品的上市速度。

OBJECTIVE:To provide reference for in-depth reform of new drug assessment and approval system in China.METHODS:The legal basis of drug accelerated assessment policies in European Union(EU)were analyzed systematically,and then compared with other similar policies.The key steps of accelerated assessment procedure were introduced,in particular,pre-submission preparation,pre-licensing inspection,submission and evaluation of accelerated assessment application.Taking Maviret as an example,the effects of EU’s accelerated assessment policies were analyzed empirically.RESULTS:Accelerated assessment is one of the EU’s several legal provisions to foster patients’early access to new drugs,as well as accelerated assessment and conditional market licensing,sympathetic drug use,priority drug program,orphan drug identification and hospital exemption,which were the main ways to obtain new drugs as soon as possible in the EU.The accelerated assessment was expected when the drugs had sufficient treatment data,significant public health benefits and innovation.Based on EU’s regulations(No.726/2004/EC),European Medicines Agency(EMA)had continuously improved the policy system of accelerated assessment,issued relevant guiding principle(guidelines)and timetables for the implementation of accelerated assessment procedures,providing detailed and specific guidance for the practical landing of the policy.Once the drug entered the accelerated assessment procedure,the assessment time would be shortened from 210 days of the standard procedure to 150 days.The assessment of Maviret,a new drug for the treatment of hepatitis C virus infection,was accelerated from Jan.20,2017 until Jun.22,2017.When it was approved by the EMA Committee for Human Medicinal Products(CHMP),the whole process was less than half a year.CONCLUSIONS:EU drug acceleration assessment has not only perfect legislative support,but also detailed and specific implementation rules and timetable.It can speed up the marketing of drugs which are required urgently by the public and have speeific medical advantages.