“首届中国孤儿药研发论坛”专家观点  

Collection of Experts' View at First Forum for Research and Development of Orphan Drugs in China

作　　者：Editorial Office of Progress in Pharmaceutical Sciences 

机构地区：[1]<药学进展>编辑部

出　　处：《药学进展》2015年第5期321-334,共14页Progress in Pharmaceutical Sciences

摘　　要：对2015年4月25日召开的"首届中国孤儿药研发论坛"的专家报告内容进行归纳总结,旨在为从事罕见病诊断和孤儿药研发工作的人士提供信息参考。报告内容涉及国内外孤儿药研发现状和前景、中国孤儿药政策和审评状况、罕见病诊断以及中国孤儿药研发的机会等。The reports from specialists at First Forum for Research and Development of Orphan Drugs in China, which was held on April 25, 2015, have been summarized and introduced in this paper, so as to provide information references for practitioners involved in the diagnosis of rare diseases and RD of orphan drugs. These reports related to the status quo and prospect of RD of orphan drugs at home and abroad, current policies and regulations on orphan drugs in China, diagnosis of rare diseases as well as opportunities in RD of orphan drugs in China.

关 键 词：罕见病 孤儿药 临床研究 分子诊断 基因治疗 投资 

分 类 号：R97[医药卫生—药品]