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FDA的突破性药物认定——4年经验
The FDA Breakthrough-Drug Designation — Four Years of Experience


Jonathan J. Darrow ... 其他 • 2018.04.12

FDA突破性疗法这四年

 

Ke Liu

Chinese American Hematology and Oncology Network

 

2018年4月12日,布列根和妇女医院及哈佛医学院的Darrow博士等发表了一篇标题为“FDA的突破性药物认定——4年经验”的卫生政策报告1。作者在原文中介绍了突破性疗法认定(breakthrough-drug designation,BTD)的发展历史以及FDA的其他加速项目,并进一步说明了BTD的优点、标准,列出了FDA之前批准的药物中,获得该认定的药物。在文末,作者就BTD提出了一些政策建议。

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2012年,美国国会推出了“突破性疗法”认定项目,旨在加快食品药品管理局(FDA)对以下药物的测试和审批:用于治疗严重或危及生命的疾病,且初步证据提示相对现有治疗方法,在一个或多个有临床意义的终点方面具有重大改进的药物1。促使该项目产生的原因是精确医疗的进步使得可以开发出起效早且疗效显著的治疗,因此随机分配患者接受安慰剂可能是不符合伦理的,且2期试验可以提供足够的证据供FDA批准药物2。该项目法案的发起者之一,迈克尔·本尼特(Michael Bennet)参议员(来自科罗拉多州的共和党人)解释道,该项目的目的是加速“对患者表现出卓越的结果”的药物审批3

制造商反应很强烈。根据FDA药品评价与研究中心负责人珍妮特·伍德科克(Janet Woodcock)的说法,该中心已被成百上千的申请“淹没”4,尽管预期每年只有约2种药物可以获得该认定5。2014~2016年,FDA批准的108种新型分子药物和原研生物制剂中,共有26种(24%)获得了突破性疗法认定。图1总结了这26种药物,以及2013年末批准的3种突破性认定药物和2种突破性认定疫苗。





作者信息

Jonathan J. Darrow, S.J.D., J.D., M.B.A., Jerry Avorn, M.D., and Aaron S. Kesselheim, M.D., J.D., M.P.H.
From the Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Boston.

 

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