日本拓展性临床试验制度对我国同情用药制度的启示  

Enlightenment of Japan expanded access clinical trial system to China’s compassionate drug use system

作　　者：王乾 尹玉 蒋丰 田侃[2] 喻小勇[2] 杨浩宇 WANG Qian;YIN Yu;JIANG Feng;TIAN Kan;YU Xiaoyong;YANG Haoyu(School of Health Economics and Management,Nanjing University of Chinese Medicine,Nanjing 210023,China;College of Elderly Care Service and Management,Nanjing University of Chinese Medicine,Nanjing 210023,China)

机构地区：[1]南京中医药大学卫生经济管理学院,南京210023 [2]南京中医药大学养老服务与管理学院,南京210023

出　　处：《中国药房》2023年第9期1038-1041,1075,共5页China Pharmacy

基　　金：江苏省社会科学基金后期资助项目(No.20HQ019)。

摘　　要：我国同情用药制度正处于初步探索阶段,尚未颁布正式的管理办法与具体的实施细则,有待进一步优化和完善。日本通过拓展性临床试验制度实现了未批准药品的提前使用,在信息获取、目标患者、知情同意、申请主体、实施计划书、拒绝给药的处理,以及药品费用、实施期限、意外损害补偿、拓展性临床试验批准后资料审查等方面有明确的规定。其中,当企业以“制度正当性事由”拒绝给药时,主治医师还可向日本厚生劳动省提出申请,由厚生劳动省进行许可评估,最大化为患者争取药品。这一“拒绝给药”的再处理,是日本独有的规定,尽最大可能保障了药品可及性。本文通过分析日本拓展性临床试验制度,建议我国可通过进一步搭建同情用药信息平台、发挥医师主导作用、保障企业利益、重视伦理审查,以及对药品费用支付问题进一步明晰,以完善和优化我国同情用药制度。The system of compassionate drug use in China is in the preliminary exploration stage,and the formal management methods and specific implementation rules have not been promulgated,which needs to be further optimized and perfected.Japan realizes the advanced use of unapproved drugs by expanded access clinical trial system,and makes clear provisions on information acquisition,target patient,informed consent,subject of application,implementation plan,handling of refusal to administer medication,drug expenses,implementation deadline,compensation for accidental damages,post-approval data review after expanded access clinical trials.When the enterprise refuses to give drugs because of the“legitimacy reasons of the system”,the attending physician can also apply to the Ministry of Health,Labor and Welfare,and the Ministry of Health,Labor and Welfare will conduct the licensing evaluation to maximize the drug for patients.This“refusal to administer”reprocessing is a unique regulation in Japan,which ensures the accessibility of drugs to the greatest extent possible.Based on the analysis of the expanded access clinical trial system in Japan,it is found that our country could further build the information platform for compassionate drug use,play the leading role of physicians,protect the interests of enterprises,pay attention to the ethical review,and make drug cost payment problems further clear in order to improve and optimize the system of compassionate drug use.

关 键 词：拓展性临床试验制度 同情用药制度 药品可及性 日本 

分 类 号：R95[医药卫生—药学]