罕见病治疗中的血浆蛋白制品  

Plasma protein products for the treatment of rare diseases

作　　者：皇甫超济 马玉媛 章金刚 HUANG-FU Chao-ji;MA Yu-yuan;ZHANG Jin-gang(Institute of Health Service and Transfusion Medicine,Academy of Military Medical Sciences,Academy of Military Sciences,Beijing 100850,China;Center for Disease Control and Prevention,Western Theater Command,Lanzhou 730020,China;NMPA Key Laboratory for Quality Control of Blood Products,Beijing 100850,China;College of Life Science and Technology,Xinxiang University,Xinxiang 453003,China)

机构地区：[1]军事科学院军事医学研究院卫生勤务与血液研究所,北京100850 [2]西部战区疾病预防控制中心,兰州730020 [3]国家食品药品监督管理局血液制品质量控制重点实验室,北京100850 [4]新乡医学院生命科学技术学院

出　　处：《国际药学研究杂志》2019年第9期691-698,共8页Journal of International Pharmaceutical Research

基　　金：国家自然科学基金资助项目（81803171）;国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”资助项目（2016YFC0901500）

摘　　要：血浆蛋白制品也称为血液制品。目前全球上市的血浆蛋白制品共30多个品种。鉴于其具有原料来源特殊、安全可靠、对部分疾病治疗不可替代等特性,很多产品在市场上处于供不应求的状态。罕见病常由遗传缺陷、自身免疫、变态反应等因素引起,给患者及其家庭造成巨大的负担。孤儿药是用于预防、诊断、治疗罕见病和罕见状态的诊断试剂、疫苗、药物、医疗器械等。自1983年美国通过《孤儿药法案》(ODA)后,已有多个国家或地区通过相关法案,以研究资助、税费减免、优先审批、市场独占等方式鼓励企业研发孤儿药,ODA的颁布极大地促进了相关血液制品企业的发展。本文总结了美国、欧盟、日本、澳大利亚药监部门以孤儿药身份审批的血浆蛋白制品,共包括四大类18个品种,占当前所有血浆蛋白制品品种数的56%。中国目前尚未立法定义罕见病和孤儿药,这在一定程度上制约了国内血液制品企业的发展。孤儿药相关法案颁布带来的政策红利将有助于国内血液制品企业发展壮大,缩小与国际血液制品巨头的差距。Plasma protein products are also called blood products.At present,more than 30 kinds of blood products have been issued worldwide.Because of their particular source of raw materials as well as the safety,reliability and irreplaceability in the treatment of some diseases,many blood products are in short supply in the market.Rare diseases are usually caused by genetic deficiency,autoimmunity,allergy and so on,which imposes a huge burden on patients and their families.Orphan drugs generally include the products of diagnostic reagents,vaccines,medicines and medical devices used for the prevention,diagnosis and treatment of rare diseases and rare conditions.Since the adoption of the Orphan Drug Act(ODA)in the United States in 1983,many countries or regions have passed the relevant laws aiming to encourage enterprises to develop orphan drugs by means of the research funding,tax relief,priority approval and market monopoly.The pass of ODA has greatly promoted the development of related blood product enterprises.This paper summarizes the plasma protein products approved by the pharmaceutical regulatory departments of the United States,the European Union,Japan and Australia as orphans,including four categories and 18 varieties,accounting for 56% of all plasma protein products in the world.At present,China has not yet enacted legislation to define rare diseases and orphan drugs,which has restricted to a certain extent the development of domestic blood product enterprises.The policy dividends from ODA will help the development of domestic blood product enterprises and narrow the gap between the domestic enterprises and the international blood product giants.

关 键 词：罕见病 血浆蛋白制品 孤儿药 

分 类 号：R954.4[医药卫生—药学]