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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 statistically analyze the situation of orphan drugs approved by the FDA in 2015, focusing on the mechanism of action to introduce orphan drugs and their representative drugs in the three hot research and development fields so that more medical personnel can understand the development of orphan drugs in recent years and provide medical services to China’s pharmaceuticals Community to provide reference information. Methods: Check the Drugs @ FDA database, relevant literature and so on. Collect the new drug information approved by the FDA in 2015 and make statistical analysis on the approved orphan drugs. Results: In 2015, the FDA approved 45 new drugs, of which 21 were approved, including 12 new molecular entities and 9 biological products, the highest year for orphan drug approval. In recent years, hot research and development areas are focused on the three major categories of anti-tumor, cardiovascular and anti-infectives. In 2015, orphan medicines accounted for a certain proportion of these three fields, at 11: 1: 1 respectively. Conclusion: The development of orphan drug research and development in foreign countries is booming. China can improve the accessibility of orphan medicine and alleviate the pressure of drug use in rare diseases in light of its own national conditions.