FDA注重加快審批治療人類嚴(yán)重疾病的新藥，尤其是可填補(bǔ)空白或優(yōu)于現(xiàn)有治療的藥物。為加快這類藥物的審批，F(xiàn)DA已經(jīng)建立了3種不同但很成功的方法，即快速通道指定、優(yōu)先審評(píng)途徑、加速批準(zhǔn)指定。2012年7月9日，《FDA安全與創(chuàng)新法案》正式實(shí)施，法案中制定了一個(gè)新的加快藥物開發(fā)審批的方式，即突破性治療藥物指定。突破性治療藥物指定自實(shí)施以來得到多方面的認(rèn)可，截止到2014年5月5日，F(xiàn)DA共收到186項(xiàng)突破性治療藥物指定申請(qǐng)，其中授予48項(xiàng)，拒絕96項(xiàng)，另外42處正在審核過程中。這些藥物涵蓋小分子化學(xué)藥、抗體、蛋白類、反義寡核苷酸類等。它們治療多種疾病，尤其是癌癥、丙型肝炎和囊性纖維化。授予突破性治療藥物指定資格后獲得FDA批準(zhǔn)上市的有5項(xiàng)。綜述FDA的4種加快重要藥物審批的程序以及突破性治療藥物指定的情況，以期為我國(guó)完善藥物審批方式提供一定的參考。

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.