Office of the Commissioner, US Food and Drug Administration, Silver Spring, Maryland.
Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland.
JAMA Netw Open. 2022 Nov 1;5(11):e2239336. doi: 10.1001/jamanetworkopen.2022.39336.
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.
DESIGN, SETTING, AND PARTICIPANTS: 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.
美国食品和药物管理局 (FDA) 有 4 个可以单独或联合使用的计划来加快药物供应：加速批准、突破性疗法、快速通道和优先审查。使用这些计划的药物可以包括新药物，这些药物不含有之前 FDA 批准的活性部分，以及孤儿药物，用于影响少于 200000 人的疾病或病症；迄今为止，尚无关于这些计划如何联合使用的综合评估。
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，加速计划单独使用和联合使用的频率，按孤儿药状态和适应证进行分层。
设计、设置和参与者：这项横断面研究评估了 2008 年 1 月 1 日至 2021 年 12 月 31 日期间所有获得 FDA 批准的新型药物。
主要结果是加速计划的使用频率和组合方式，按孤儿药状态和药物类型（小分子与治疗性生物制剂）进行分层。分析单位是新型药物-适应证对，因为一种药物可以批准用于多种适应证，每种适应证都可能使用不同的加速计划或在孤儿药状态上有所不同。
该研究包括 581 对新型药物-适应证对，在 14 年的研究期间获得批准；252 对（43.4%）为孤儿药，139 对（23.9%）为治疗性生物制剂，442 对（76.1%）为小分子药物。至少使用 1 个加速计划的药物数量从 2008 年的 26 对药物-适应证对中的 11 对（42.3%）增加到 2021 年的 55 对中的 41 对（74.5%）。在 363 对使用至少 1 个加速计划的已批准药物-适应证对中，225 对（62.0%）为孤儿药；在 139 对已批准的生物制剂药物中，至少有 1 个加速计划被使用 97 对（69.8%），在 442 对已批准的小分子药物中，至少有 1 个加速计划被使用 266 对（60.2%）。581 对新型药物-适应证对中的 82 对（14.1%）使用了加速批准计划；其中，65 对（79.3%）为肿瘤药物，70 对（85.4%）有孤儿药指定。
研究表明，美国使用 FDA 的加速计划将新型药物推向市场的数量从 2008 年增加到 2021 年。研究结果表明，这种趋势可能会继续下去。
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