FDA加快新药审批程序及突破性治疗药物分析

FDA注重加快审批治疗人类严重疾病的新药,尤其是可填补空白或优于现有治疗的药物。为加快这类药物的审批,FDA已经建立了3种不同但很成功的方法,即快速通道指定、优先审评途径、加速批准指定。2012年7月9日,《FDA安全与创新法案》正式实施,法案中制定了一个新的加快药物开发审批的方式,即突破性治疗药物指定。突破性治疗药物指定自实施以来得到多方面的认可,截止到2014年5月5日,FDA共收到186项突破性治疗药物指定申请,其中授予48项,拒绝96项,另外42处正在审核过程中。这些药物涵盖小分子化学药、抗体、蛋白类、反义寡核苷酸类等。它们治疗多种疾病,尤其是癌症、丙型肝炎和囊性纤维化。授予突破性治疗药物指定资格后获得FDA批准上市的有5项。综述FDA的4种加快重要药物审批的程序以及突破性治疗药物指定的情况,以期为我国完善药物审批方式提供一定的参考。

Analysis on accelerating drug approval process and breakthrough therapies designation drugs by FDA

The Food and Drug Administration (FDA) has been interested in speeding the development and availability of drugs that treat serious diseases, especially the drugs that are the first available treatment or have advantages over existing treatments. To make such drugs available as rapidly as possible, FDA has developed three distinct and successful approaches: fast track, priority review, and accelerated approval. On July 9, 2012, President Obama signed into the law of Food and Drug Administration Safety and Innovation Act ("FDASIA"). FDASIA made a new way to accelerate the development of drug approval, namely breakthrough therapies designation drugs specified, which has been recognised by many since the implementation. By the end of May 5, 2014, FDA received 186 total requests for breakthrough designation, of which 48 requests were granted and 96 requests were denied. These drugs include small molecules, antibodies, proteins, antisense oligonucleotides, and so on. They can treat a range of illnesses, especially, cancer, hepatitis C, and cystic fibrosis. Five breakthrough designation drugs recieved full FDA approval. Four accelerating procedures for important drug approval of FDA and the situation of breakthrough therapies designation drugs are reviewed, in order to give some reference to improve the way for examination and approval of drugs in our country.