Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study.
Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study.
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DOI：
10.1136/bmj.m3434
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发表时间：
2020-10-07
期刊：
影响因子：
--
通讯作者：
Kesselheim AS
中科院分区：
文献类型：
--
作者：
Hwang TJ;Ross JS;Vokinger KN;Kesselheim AS
描述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，以及这些评级与通过加速计划获得的监管批准之间的关联。回顾性队列研究。FDA和EMA在2007年至2017年期间批准的新药，随访至2020年4月1日。使用五个独立组织（加拿大、法国、德国和意大利的处方和卫生当局）对新药的评级来衡量治疗价值。被评定为具有高治疗价值的新药比例;高治疗价值评定与加速状态之间的关联。从2007年到2017年，FDA和EMA分别批准了320种和268种新药，其中181种（57%）和39种（15%）符合至少一个加速项目的资格。在267种具有治疗价值的新药中，84种（31%）被至少一个组织评为具有高治疗价值。与非加速药物相比，在FDA批准（45%（69/153）vs13%（15/114）; P<0.001）和EMA批准（67%（18/27）vs27%（65/240）; P<0.001）中，更大比例的加速药物被评为具有高治疗价值。FDA对被独立评定为具有高治疗价值的药物的加速项目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EMA的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%）。在过去十年中，FDA和EMA批准的新药中，只有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。尽管加急药物比非加急药物更有可能被评为高等级，但FDA批准的大多数加急药物（而非EMA批准的药物）被评为治疗价值低。
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. Retrospective cohort study. New drugs approved by the FDA and EMA between 2007 and 2017, with follow-up through 1 April 2020. Therapeutic value was measured using ratings of new drugs by five independent organizations (Prescrire and health authorities of Canada, France, Germany, and Italy). Proportion of new drugs rated as having high therapeutic value; association between high therapeutic value rating and expedited status. 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. 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.
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影响因子：
51.1
作者：
Del Paggio, Joseph C.;Sullivan, Richard;Booth, Christopher M.
通讯作者：
Booth, Christopher M.
影响因子：
6.2
作者：
Molto, Consolacion;Hwang, Thomas J.;Tibau, Ariadna
通讯作者：
Tibau, Ariadna
DOI：
10.1136/bmj.j3837
发表时间：
2017-09-07
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Mostaghim SR;Gagne JJ;Kesselheim AS
通讯作者：
Kesselheim AS
DOI：
10.1136/bmj.h4633
发表时间：
2015-09-23
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Kesselheim AS;Wang B;Franklin JM;Darrow JJ
通讯作者：
Darrow JJ
影响因子：
45.3
作者：
Hwang, Thomas J.;Franklin, Jessica M.;Darrow, Jonathan J.
通讯作者：
Darrow, Jonathan J.