食品和药物管理局批准孤儿药和非孤儿药时可用的证据。

Health affairs scholar Pub Date : 2025-03-18 eCollection Date: 2025-04-01 DOI:10.1093/haschl/qxaf057
Amanda J Koong, Veronica L Irvin, Aditya Narayan, Sujin Song, Robert M Kaplan
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引用次数: 0

摘要

开发治疗罕见病的孤儿药有很大的经济激励,但对支持证据的质量和数量的担忧已经出现。我们比较了2016年至2023年美国食品和药物管理局(FDA)批准的孤儿药和非孤儿药的证据。本回顾性横断面分析利用FDA批准数据和ClinicalTrials.gov上的研究信息,比较2016年至2023年批准的孤儿药和非孤儿药相关研究的特征。在批准的368种药物中,50%是孤儿药。与非孤儿药物(2.4项研究/药物)相比,fda批准的孤儿药物的研究数量明显减少(1.5项研究/药物)。此外,在FDA批准孤儿药之前完成的研究比例明显较低(25%对41%)。孤儿药在随机临床试验(rct)中被评估的可能性明显较低(34%对63%)。在这些随机对照试验中,在批准前完成的(40%对54%)和公布结果的(35%对53%)明显较少。孤儿药和非孤儿药的现有证据有显著差异。随着像Cures 2.0这样的新立法的制定,检查加快审批时间表和临床证据标准之间的平衡至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Evidence available and used by the Food and Drug Administration for the approval of orphan and nonorphan drugs.

There are substantial financial incentives to develop orphan drugs for rare diseases, but concerns about the quality and volume of supporting evidence have emerged. We compare evidence used to evaluate orphan and nonorphan drugs approved by the Food and Drug Administration (FDA) between 2016 and 2023. This retrospective cross-sectional analysis utilizes FDA data on approvals and study information from ClinicalTrials.gov to compare characteristics of studies relevant to orphan and nonorphan drugs approved between 2016 and 2023. Of the 368 total drugs approved, 50% were orphan drugs. The FDA-approved drugs based on significantly fewer studies for orphan (1.5 studies/drug) compared to nonorphan (2.4 studies/drug). Additionally, a significantly lower proportion of studies were completed before FDA approval for orphan drugs (25% vs 41%). Orphan drugs were significantly less likely to be evaluated in randomized clinical trials (RCTs) (34% vs 63%). Of these RCTs, there were significantly fewer completed before approval (40% vs 54%) and that had results posted (35% vs 53%). There was a significant difference in the available evidence for orphan and nonorphan drugs. As new legislation like Cures 2.0 is developed, it is critical to examine the balance between an expedited approval timeline and the standard of clinical evidence.

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