2015年FDA批准的孤儿药统计分析

Analysis of orphan drugs approved by FDA in 2015

目的:统计分析2015年FDA批准的孤儿药情况,从作用机制出发重点介绍三大热点研发领域的孤儿药及其代表药物,让更多的医务人员了解近年来孤儿药的发展情况,为我国医药界提供参考信息。方法:查阅Drugs@FDA数据库、相关文献等,搜集2015年FDA批准的新药信息,对获批的孤儿药进行统计分析。结果:2015年FDA共批准了45个新药,其中有21个孤儿药获批,包括12个新分子实体和9个生物制品,是批准孤儿药最多的一年。近年来的热点研发领域分别集中于抗肿瘤、心血管、抗感染三大类,2015年孤儿药在此3个领域都占有一定比例,分别为11∶1∶1。结论:国外孤儿药研发蓬勃发展,我国可结合本国国情,从多方面提高孤儿药的可及性,缓解罕见病用药压力。

Objective： To make a statistical analysis of the general situation of orphan drugs approved by FDA in 2015,focusing on the mechanism research and development of orphan drugs and representative drugs in the field of three major hot spots,to let more medical personnel understand the development of orphan drugs in recent years and provide information for our medical profession. Methods： By searching Drugs @ FDA database and literature,etc,the information about the drugs approved by the FDA in 2015 was collected,and analysis was carried out for the orphan drugs. Results： FDA approved a total of 45 novel drugs in 2015,among which there were 21 orphan drugs,including 12 new molecular entities and 9 biological products. The number of approval was the highest among recent years. The hot spots in the research field included anti-tumor,cardiovascular,antiinfection drugs. The orphan drugs approved in 2015 in the three areas occupied a certain proportion,at a ratio of11 ∶ 1∶ 1. Conclusion： The research and development of orphan drugs are booming in foreign countries. Our country need to do more in this area,taking our national conditions into account,to improve the accessibility of orphan drugs and alleviate the pressure of treatment of rare diseases.