Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021.
Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021.
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DOI：
10.1001/jamanetworkopen.2022.39336
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发表时间：
2022-11-01
影响因子：
13.8
通讯作者：
Chahal, Harinder Singh
中科院分区：
文献类型：
--
作者：
Monge, Andrea N.;Sigelman, Daniel W.;Temple, Robert J.;Chahal, Harinder Singh
这项横断面研究考察了 2008 年至 2021 年间批准的孤儿药和非孤儿生物制品和小分子药物中使用美国食品和药物管理局 (FDA) 4 个加速开发和审评新药计划的频率和组合。美国食品和药物管理局 (FDA) 4 个加速开发和审评孤儿和非孤儿新药的计划的使用频率和组合是怎样的？在这项针对 581 个 FDA 批准的新药和适应症对的横断面研究中，加速开发和审评计划的使用从 2008 年的 42.3% 增加到 2021 年的 74.5%。在使用至少 1 个快速计划的批准药物适应症对中，62.0% 是孤儿药，69.8% 是生物制剂，60.2% 是小分子药物。研究结果表明，加急计划在将新药推向美国市场方面发挥着越来越大的作用，尤其是孤儿药和生物制品。美国食品和药物管理局 (FDA) 有 4 个项目可以单独或组合使用来加快药物供应：加速审批、突破性治疗、快速通道和优先审查。使用这些计划的药物可以包括不含 FDA 先前批准的活性部分的新药，以及旨在治疗影响少于 200 万人的疾病或病症的孤儿药；迄今为止，尚未发表对如何组合使用这些方案的全面评估。评估在已批准的新型生物制剂和小分子药物的开发和审查中使用加速计划的频率和组合，按孤儿药状态和适应症进行分层。这项横断面研究评估了 FDA 在 2008 年 1 月 1 日至 2021 年 12 月 31 日期间批准的所有新药。主要结果是加速计划的使用频率以及组合方式，按孤儿药状态和药物类型（小分子与治疗生物制剂）进行分层。分析单位是新药-适应症对，因为一种药物可以被批准用于多种适应症，每个适应症可能使用不同的加急计划或孤儿药状态不同。该研究包括 14 年研究期间批准的 581 个新药-适应症对； 252 种（43.4%）为孤儿药，139 种（23.9%）为治疗性生物制剂，442 种（76.1%）为小分子药物。使用至少 1 个加急项目的数量从 2008 年 26 个药物适应症对中的 11 个 (42.3%) 增加到 2021 年 55 个药物适应症对中的 41 个 (74.5%)。在使用至少 1 个加急项目的 363 个批准的药物适应症对中，225 个 (62.0%) 是孤儿药； 139 种批准的生物药物中的 97 种（69.8%）和 442 种批准的小分子药物中的 266 种（60.2%）至少使用了 1 个加急程序。 581 个新药-适应症对中的 82 个（14.1%）使用了加速审批计划；其中，65 种（79.3%）是肿瘤药物，70 种（85.4%）具有孤儿药资格。研究表明，从 2008 年到 2021 年，美国使用 FDA 加速计划将新药推向市场的情况有所增加。研究结果表明，这一趋势可能会持续下去。
This cross-sectional study examines how often and in what combinations 4 US Food and Drug Administration programs for expedited development and review of novel drugs were used among orphan and nonorphan biologics and small-molecule drugs approved between 2008 and 2021. How often and in what combinations are the 4 US Food and Drug Administration (FDA) programs for expedited development and review of orphan and nonorphan novel drugs used? In this cross-sectional study of 581 FDA-approved pairs of novel drugs and indications, use of expedited development and review programs increased from 42.3% of pairs in 2008 to 74.5% in 2021. Of approved drug-indication pairs using at least 1 expedited program, 62.0% were orphan drugs, 69.8% were biologics, and 60.2% were small-molecule drugs. The findings suggest that expedited programs have an increasing role in bringing novel drugs to market in the US, especially orphan and biologic products. The US Food and Drug Administration (FDA) has 4 programs that can be used alone or in combination to expedite drug availability: Accelerated Approval, Breakthrough Therapy, Fast Track, and Priority Review. Drugs using these programs can include novel drugs, which do not contain a previously FDA-approved active moiety, and orphan drugs, intended for diseases or conditions affecting fewer than 200 000 people; to date, no comprehensive evaluation of how these programs have been used in combination has been published. To assess how often and in what combinations expedited programs are used in the development and review of approved novel biologics and small-molecule drugs, stratified by orphan drug status and indication. This cross-sectional study evaluated all novel drugs that were FDA approved between January 1, 2008, and December 31, 2021. The main outcome was the frequency with which expedited programs were used and in what combinations, stratified by orphan drug status and drug type (small molecule vs therapeutic biologic). The unit of analysis was the novel drug–indication pair because a drug can be approved for multiple indications, each of which may use a different expedited program or differ in orphan drug status. The study included 581 novel drug–indication pairs approved during the 14-year study period; 252 (43.4%) were orphan drugs, 139 (23.9%) were therapeutic biologics, and 442 (76.1%) were small-molecule drugs. Use of at least 1 expedited program increased from 11 of 26 drug-indication pairs (42.3%) in 2008 to 41 of 55 (74.5%) in 2021. Of the 363 approved drug-indication pairs using at least 1 expedited program, 225 (62.0%) were orphan drugs; at least 1 expedited program was used by 97 of the 139 approved biologic drugs (69.8%) and by 266 of the 442 approved small-molecule drugs (60.2%). Eighty-two of the 581 novel drug–indication pairs (14.1%) used the Accelerated Approval Program; of those, 65 (79.3%) were oncology drugs and 70 (85.4%) had an orphan designation. The study showed that use of the FDA’s expedited programs to bring novel drugs to market in the US increased from 2008 to 2021. The findings suggest that this trend is likely to continue.
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影响因子：
7.3
作者：
Brown, Dean G.;Wobst, Heike J.
通讯作者：
Wobst, Heike J.
影响因子：
0.2
作者：
Kepplinger, Erin E.
通讯作者：
Kepplinger, Erin E.
影响因子：
2.7
作者：
Wallach, Joshua D.;Ross, Joseph S.;Naci, Huseyin
通讯作者：
Naci, Huseyin
影响因子：
6.7
作者：
Pregelj, Lisette;Hine, Damian C.;Darrow, Jonathan J.
通讯作者：
Darrow, Jonathan J.
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