Building bridges and providing transparency to the hepatitis C virus drug approval process.
Building bridges and providing transparency to the hepatitis C virus drug approval process.
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为丙型肝炎病毒药物审批流程搭建桥梁并提供透明度。
DOI：
10.1053/j.gastro.2014.10.028
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发表时间：
2014
期刊：
影响因子：
29.4
通讯作者：
Liang,TJake
中科院分区：
文献类型：
--
作者：
Ghany,MarcG;Liang,TJake
慢性丙型肝炎是全球肝硬化和肝细胞癌的主要原因1，也是美国和欧洲成人肝移植的主要适应症。 2 尽管发达国家慢性丙型肝炎的发病率正在下降，但预计未来十年因感染并发症而死亡的人数将会增加。 3 美国治疗晚期慢性丙型肝炎并发症的费用预计将从目前的 65 亿美元增加到 2024 年的约 91 亿美元。 4 模型数据表明，实施与有效治疗相关的国家筛查计划将对减轻慢性丙型肝炎造成的疾病负担产生重大影响。 5 开发高效、安全的慢性丙型肝炎疗法有望改善慢性丙型肝炎患者的预后。 62013 年 12 月，美国食品和药物管理局 (FDA) 批准了一种可能改变游戏规则的药物索磷布韦（一种丙型肝炎病毒 NS5B 聚合酶核苷酸类似物抑制剂）。 FDA 授予该药物突破性地位（定义为治疗严重或危及生命的疾病的药物，并且初步临床证据表明该药物可能在临床显着终点上比现有疗法有实质性改善），从而使其能够接受快速审查。 7
Chronic hepatitis C is a major cause of cirrhosis and hepato-cellular carcinoma worldwide1 and the leading indication for adult liver transplants in the United States and Europe. 2 Although the incidence of chronic hepatitis C is declining in the developed world, the number of deaths owing to complications of the infection is estimated to increase over the next decade. 3 A parallel rise in the cost of managing complications of advanced chronic hepatitis C is projected to increase from a current $6.5 billion to approximately $9.1 billion in 2024 in the United States. 4 Modeling data suggest that implementation of a national screening program linked to effective therapy would have a substantial impact on reducing the burden of disease resulting from chronic hepatitisC. 5 The development of highly effective, safe therapies for chronic hepatitis C holds promise to improve the outcomes of patients with chronic hepatitis C. 6In December 2013, a potentially game-changing drug, sofosbuvir, a hepatitis C virus NS5B polymerase nucleotide analog inhibitor, was approved by the US Food and Drug Administration (FDA). This drug was afforded breakthrough status (defined as a drug that treats a serious or life-threatening condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint (s) over available therapies) by the FDA, which allowed it to receive an expedited review. 7