FDA 和 EMA 加速审批计划与新药的治疗价值之间的关联：回顾性队列研究。
Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study.
机构信息
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, MA, USA
Laboratory for Technology, Markets and Regulation, University of Zurich, Zurich, Switzerland.
出版信息
BMJ. 2020 Oct 7;371:m3434. doi: 10.1136/bmj.m3434.
OBJECTIVE
To characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited programs.
DESIGN
Retrospective cohort study.
SETTING
New drugs approved by the FDA and EMA between 2007 and 2017, with follow-up through 1 April 2020.
DATA SOURCES
Therapeutic value was measured using ratings of new drugs by five independent organizations (Prescrire and health authorities of Canada, France, Germany, and Italy).
MAIN OUTCOME MEASURES
Proportion of new drugs rated as having high therapeutic value; association between high therapeutic value rating and expedited status.
RESULTS
From 2007 through 2017, the FDA and EMA approved 320 and 268 new drugs, respectively, of which 181 (57%) and 39 (15%) qualified for least one expedited program. Among 267 new drugs with a therapeutic value rating, 84 (31%) were rated as having high therapeutic value by at least one organization. Compared with non-expedited drugs, a greater proportion of expedited drugs were rated as having high therapeutic value among both FDA approvals (45% (69/153) 13% (15/114); P<0.001) and EMA approvals (67% (18/27) 27% (65/240); P<0.001). The sensitivity and specificity of expedited program for a drug being independently rated as having high therapeutic value were 82% (95% confidence interval 72% to 90%) and 54% (47% to 62%), respectively, for the FDA, compared with 25.3% (16.4% to 36.0%) and 90.2% (85.0% to 94.1%) for the EMA.
CONCLUSIONS
Less than a third of new drugs approved by the FDA and EMA over the past decade were rated as having high therapeutic value by at least one of five independent organizations. Although expedited drugs were more likely than non-expedited drugs to be highly rated, most expedited drugs approved by the FDA but not the EMA were rated as having low therapeutic value.
目的
描述美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）批准的新药的治疗价值，并研究这些评级与通过加速审批程序获得监管批准之间的关系。
设计
回顾性队列研究。
地点
2007 年至 2017 年间 FDA 和 EMA 批准的新药，随访至 2020 年 4 月 1 日。
数据来源
使用五个独立组织（加拿大 Prescrire 和卫生当局、法国、德国和意大利）对新药的评级来衡量治疗价值。
主要观察指标
被评为具有高治疗价值的新药比例；高治疗价值评级与加速审批地位之间的关系。
结果
2007 年至 2017 年间，FDA 和 EMA 分别批准了 320 种和 268 种新药，其中 181 种（57%）和 39 种（15%）符合至少一种加速程序的条件。在 267 种具有治疗价值评级的新药中，有 84 种（31%）被至少一个组织评为具有高治疗价值。与非加速审批药物相比，在 FDA 批准的药物中，加速审批药物被评为具有高治疗价值的比例更高（45%（69/153）比 13%（15/114）；P<0.001），在 EMA 批准的药物中，这一比例更高（67%（18/27）比 27%（65/240）；P<0.001）。对于 FDA，加速审批程序预测药物被独立评为具有高治疗价值的敏感性和特异性分别为 82%（95%置信区间 72%至 90%）和 54%（47%至 62%），而对于 EMA，敏感性和特异性分别为 25.3%（16.4%至 36.0%）和 90.2%（85.0%至 94.1%）。
结论
在过去十年中，FDA 和 EMA 批准的新药中，不到三分之一被五个独立组织中的至少一个评为具有高治疗价值。尽管加速审批药物比非加速审批药物更有可能被评为高度评价，但在 FDA 批准的大多数加速审批药物中，被评为具有低治疗价值。
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