Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.
Institute of Law, University of Zurich, Zurich, Switzerland.
JAMA Health Forum. 2022 Aug 5;3(8):e222685. doi: 10.1001/jamahealthforum.2022.2685.
The number of drugs approved through the accelerated approval or conditional marketing authorization pathways has increased with unclear evidence of their therapeutic value.
To assess the therapeutic value of drug indications granted accelerated approval in the US or conditional marketing authorization in the European Union (EU) overall and for cancer indications.
DESIGN, SETTING, AND PARTICIPANTS: This cohort study used the public databases of the US Food and Drug Administration and the European Medicines Agency to identify all drugs (initial and supplemental indications) granted accelerated approval in the US or conditional marketing authorization (initial indications only) in the EU between January 1, 2007, and December 31, 2021. Therapeutic value ratings were obtained from national health authorities in Germany, France, and Canada.
Descriptive statistics were used to assess the proportion of accelerated approvals and conditional marketing authorizations overall and for cancer vs noncancer indications rated as having high added therapeutic value.
The cohort included 146 drug indications (94 first indications, 52 supplemental indications) in the US and 58 (all first indications) in the EU. Most drugs were approved for cancer (122 [83.6%] in the US; 40 [69.0%] in the EU). Therapeutic value ratings were available for 90 drug indications (61.6%) in the US and 56 (96.6%) in the EU. Overall, 35 drug indications granted accelerated approval (38.9%) and 21 granted conditional marketing authorization (37.5%) had high added therapeutic value in the US and EU, respectively, at the time of approval. The proportions of indications rated as having high added therapeutic value were 36.0% (27 of 75) for cancer vs 53.3% (8 of 15) for noncancer indications in the US and 30.8% (12 of 39) for cancer vs 52.9% (9 of 17) for noncancer indications in the EU.
In this cohort study, among new drug indications approved through the accelerated approval or conditional marketing authorization pathways in the US and Europe from 2007 to 2021, 38.9% and 37.5%, respectively, demonstrated high therapeutic value. A substantially lower proportion of cancer indications than noncancer indications were rated as having high therapeutic value. Policy makers and regulators should increase enforcement of timely postapproval study completion for drugs qualifying for these pathways.
通过加速批准或有条件营销授权途径批准的药物数量有所增加，但这些药物的治疗价值证据并不明确。
评估在美国获得加速批准或在欧盟获得有条件营销授权的药物适应证的总体治疗价值，以及癌症适应证的治疗价值。
设计、设置和参与者：这项队列研究使用美国食品和药物管理局和欧洲药品管理局的公共数据库，确定 2007 年 1 月 1 日至 2021 年 12 月 31 日期间在美国获得加速批准或在欧盟获得有条件营销授权（仅初始适应证）的所有药物（初始和补充适应证）。治疗价值评级来自德国、法国和加拿大的国家卫生当局。
使用描述性统计方法评估总体上以及癌症与非癌症适应证中获得加速批准和有条件营销授权的比例，并评估被评为具有高附加治疗价值的比例。
该队列包括美国的 146 种药物适应证（94 种初始适应证，52 种补充适应证）和欧盟的 58 种适应证（均为初始适应证）。大多数药物是为癌症批准的（美国 122 种[83.6%]；欧盟 40 种[69.0%]）。美国有 90 种药物适应证（61.6%）和欧盟有 56 种适应证（96.6%）可获得治疗价值评级。总体而言，在美国和欧盟，分别有 35 种获得加速批准的药物适应证（38.9%）和 21 种获得有条件营销授权的药物适应证（37.5%）具有高附加治疗价值。在美国，被评为具有高附加治疗价值的适应证比例为 36.0%（27/75），癌症适应证比非癌症适应证高 53.3%（8/15）；在欧盟，癌症适应证为 30.8%（12/39），而非癌症适应证为 52.9%（9/17）。
在这项队列研究中，2007 年至 2021 年期间，在美国和欧洲通过加速批准或有条件营销授权途径批准的新药适应证中，分别有 38.9%和 37.5%显示出高治疗价值。被评为具有高治疗价值的癌症适应证比例明显低于非癌症适应证。政策制定者和监管机构应加强对符合这些途径的药物进行及时完成批准后研究的执行力度。
