罕见疾病治疗药物政策的循证评价  

An Evidence-based Evaluation on Orphan Drug Policies

作　　者：陈敏[1,2] 张伶俐[1,2] 李幼平[3] 全淑燕 

机构地区：[1]四川大学华西第二医院药学部/循证药学中心,成都610041 [2]出生缺陷与相关妇儿疾病教育部重点实验室,成都610041 [3]四川大学华西医院中国循证医学中心,成都610041 [4]四川大学华西药学院,成都610041

出　　处：《中国循证医学杂志》2015年第7期802-809,共8页Chinese Journal of Evidence-based Medicine

基　　金：国家自然科学基金面上项目(编号:81373381);美国中华医学基金会CP项目(编号:12-095)

摘　　要：目的了解各国(地区)罕见疾病治疗药物政策的制定情况和主要内容,为建立适合我国国情的罕见疾病治疗药物政策提供参考。方法计算机检索CBM、CNKI、VIP、EMbase、Pub Med、Web of Knowledge、National Library of Medicine、CRD database、The Campbell Library、The Cochrane Library,以及WHO、欧盟和美国、加拿大、英国、爱尔兰、荷兰、德国、西班牙、法国、澳大利亚、新西兰、中国、印度、韩国、日本、南非药品管理相关网站,搜集各国(地区)罕见疾病治疗药物政策相关文献,检索时限截至2014年2月。由2位研究者独立进行文献筛选和资料提取后,对各国(地区)罕见疾病治疗药物政策进行归纳总结和比较分析。结果最终纳入110篇文献。美国、新加坡、日本、澳大利亚、欧盟、中国台湾地区和韩国出台了孤儿药物激励政策。而南非、印度、加拿大、新西兰和中国香港则正在制订孤儿药物政策框架。孤儿药物政策的主要激励条款为:市场独占权、税费减免、协定帮助、基金赞助、快速审评程序和再审查时间延长。结论中国大陆尚无罕见疾病治疗药物管理政策,建议筹备专门组织机构和规范工作程序,推动国家孤儿药物政策相关立法工作,明确罕见疾病定义和患病率,制订生产企业激励机制促进孤儿药物研发,制订补偿机制保障罕见疾病患者权益,建立患者登记网络平台追踪罕见疾病进程。Objective To learn the development and implementation of orphan drug policies, in order to provide decision-making references for the establishment of orphan drug policy according with China＇s national conditions. Methods We electronically searched databases including CBM, CNKI, VIP, EMbase, PubMed, Web of Knowledge, National Library of Medicine, CRD database, The Campbell Library, The Cochrane Library and the drug administration websites of USA, Canada, UK, Ireland, the Netherlands, Germany, Spain, France, Australia, New Zealand, China, India, South Korea, Japan, and South Africa to collect studies about orphan drug policy. The search date was up to February 2014. Two reviewers independently screened literature, and extracted data. Then, all included orphan drug policies were summarized and a comparative analysis was performed. Results A total of 110 studies were included. USA, Singapore, Japan, Australia, European Union, Chinese Taiwan and South Korea had introduced orphan drugs incentive policies. South Africa, India, Canada, New Zealand and Chinese Hongkong were producing orphan drugs policy frameworks. The main items of orphan drug policy included marketing exclusivity, tax incentives, technical assistance, grant funding, expedite approval process and prolong re-evaluated time. Conclusion In China's Mainland, there is no orphan disease management policy. China should establish specific organization and working procedures, promote orphan drug policy related legislative work, clarify the definition and prevalence of orphan diseases, provide incentive mechanism to promote the research and development of orphan drugs, provide enterprises to develop compensation mechanism to safeguard the rights and interests of patients, as well as establish patients register network platform to track the processes of the diseases.

关 键 词：药物政策 罕见病 孤儿药 循证评价 

分 类 号：R95[医药卫生—药学]