摘要
当前,在罕见病药物发展中,有诸多问题面临着挑战。本文通过介绍罕见病治疗的伦理基础、孤儿药经济学研究的特殊性、孤儿药报销对医保基金的风险、平衡孤儿药的可及性和费用控制的国际经验,以及中国香港特区颁布的罕见病药物目录和中国罕见病药物医保现状,基于中国已公布罕见病目录和临床急需进口的抗肿瘤和孤儿药物现状,以及国际对于罕见病高价药物控费的经验,探讨了其对未来医疗保险可能带来的影响。从不同角度分析美国孤儿药法案、英国国家卫生和临床技术优化研究所高特技术新概念、爱尔兰国家药物经济学中心对不含抗肿瘤孤儿药药物经济学回顾性评阅、瑞典药物评估中心对孤儿药评价原则、土耳其对进口罕见病药物的专题研究、韩国对高价药费用控制的4种措施等实践经验,以期为中国罕见病药物政策的制定以及相关药物经济学发展提供借鉴。
At present,we are facing challenges in the development of rare disease drugs.The paper introduces the ethical theories of rare disease treatment,the characteristics of pharmacoeconomic evaluation on orphan drugs,the risk of reimbursement in medical insurance funds,the international experiences on the balance between the accessibility and cost containment of high-cost orphan drugs,the orphan drug list promulgated by Hong Kong SAR of China in 2019 and the status quo of orphan drugs in China.The author also analyzes the international practice from different perspectives,such as,the Orphan Drug Act(ODA)in the United States,the new concept of high specific technology introduced by National Institute Health and Clinical Excellence(NICE)in the UK,the retrospective review of pharmacoeconomics of orphan drugs by National Centre for Pharmacoeconomics(NCPE)in Ireland,the principles of orphan drug evaluation by TLV in Sweden,a special research program on the cost of orphan drugs in Turkey,and the four measures to control the cost of high-priced drugs in the Republic of Korea.The aim of this paper is to introduce international experiences for reference on the formulation of drug policies in rare diseases and development of related pharmacoeconomics in China.
作者
胡善联
HU Shan-lian(School of Public Health,Fudan University,Shanghai 200032,China)
出处
《国际药学研究杂志》
CAS
北大核心
2019年第9期652-658,共7页
Journal of International Pharmaceutical Research
