美国布列根和妇女医院和哈佛医学院Thomas J Hwang团队研究了FDA和EMA加急审批程序与新药治疗价值的相关性。2020年10月7日，该研究发表在《英国医学杂志》上。

为了评估美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）批准的新药的治疗价值，以及这些价值与通过加急计划进行的监管批准之间的相关性。

FDA和EMA在2007-2017年间批准了很多新药，研究组在2020年4月1日之前进行跟进，设计了一项回顾性队列研究。通过五个独立组织（加拿大、法国、德国和意大利的处方和卫生当局）使用新药的评级来评估治疗价值。

2007-2017年，FDA和EMA分别批准了320和268种新药，其中181种（57％）和39种（15％）至少符合一项加急计划的要求。在267种具有治疗价值的新药中，84种药物（31%）至少被一个组织被评为具有高治疗价值。
    
与非加急药物相比，加急药物中具有高治疗价值的比例更高，其中FDA批准药物中分别为45%和13%，EMA批准药物中分别为67%和27%。对于FDA而言，加急程序对于被单独评定为具有高治疗价值药物的敏感性和特异性分别为82%和54%。相比之下，EMA的比例则分别为25.3%和90.2%。

总之，FDA和EMA批准的新药中不到三分之一被评为具有高治疗价值，与非加急药物相比，加急药物更有可能获得高治疗评价。

附：英文原文

Title: Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study

Author: Thomas J Hwang, Joseph S Ross, Kerstin N Vokinger, Aaron S Kesselheim

Issue&Volume: 2020/10/07

Abstract:

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) v 13% (15/114); P<0.001) and EMA approvals (67% (18/27) v 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.

DOI: 10.1136/bmj.m3434

Source: https://www.bmj.com/content/371/bmj.m3434