中美政府资助罕见病与罕用药科研基金项目的比较分析  

Comparative analysis of government sponsored research fund projects of rare diseases and orphan drugs between China and USA

作　　者：陈昕[1] 熊潇磊 龚时薇[1] 

机构地区：[1]华中科技大学同济医学院药学院,湖北武汉430030

出　　处：《中国医院药学杂志》2016年第12期961-963,1002,共4页Chinese Journal of Hospital Pharmacy

基　　金：国家自然科学基金项目(编号:71373089)

摘　　要：目的:探索中美政府资助罕见病与罕用药科研基金项目差异性。方法:比较分析两国罕见病和罕用药科研项目的管理规定、数量、金额、研究机构和领域五大核心内容。结果:美国出台明确科研资助的罕用药和罕见病法案;2008-2013年间,美国NIH资助37 826项罕见病及罕用药科研基金项目,共计约147.3亿美元;同期,中国NSFC资助3 132项此类项目,共计约12.9亿元。中美的高校为罕见病及罕用药科研项目的主要承担机构。美国罕见病及罕用药项目的研究领域相对广泛,对重点研究采取持续性资助。结论:我国的罕见病和罕用药科研项目的管理存在定义界定不明确、缺乏专项科研基金项目、管理机构和激励机制,研究经费少和研究领域窄等问题,应针对性进行改进。OBJECTIVE To explore differences of government sponsored research projects of rare diseases and orphan drugs between China and USA.METHODS Comparative analysis was used to describe five contents of management principles, project numbers, funding amount, institutions and fields of Chinese and American research fund projects about rare diseases and orphan drugs.RESULTS US government had published two Acts to define research fund projects of rare diseases and orphan drugs. Total number of projects about rare diseases and orphan drugs that were sponsored by the FDA and NIH of USA was up to 37826 from 2008 to 2013（including orphan drug projects from 2008 to 2013 and rare disease projects from 2011 to 2013）, and the total sum of grants was about $14.73 billion. During the same period, total number of projects about rare diseases and orphan drugs that were sponsored by NSFC of China was 3132, and total amount of grants was about 1.29 billion RMB. Universities and colleges undertook most of projects of rare diseases and orphan drugs in China and USA. Research fields of fund projects in USA were relatively extensive, and grant support was more sustainable.CONCLUSION For the research fund projeats of rare disease and orphan drug in China, the definitions are not definite. No special research fund project, management institution or incentive mechanism are available. Research grants are insufficient. And the research fields are narrow. Target measures should be taken to solve these problems.

关 键 词：罕见病 罕用药 科研项目 比较分析 

分 类 号：R95[医药卫生—药学]