与儿科专用药物相关的收入和标签变化,2013-2023年
Ian T T Liu1, Adam J N Raymakers2, Ameet Sarpatwari2
1Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA; Department of Pediatrics, University of Illinois at Chicago, Chicago, IL.
"儿童最佳药品法案" (BPCA) 的规定产生了宝贵的儿科药物信息和标签变化. 然而,它也通过推迟通用竞争而导致了大量制造商收入.
科学领域:
- 儿科药理学 儿科药理学
- 药物监管 药物监管 药物监管
- 制药经济学 制药经济学
背景情况:
- "儿童最佳药品法案" (BPCA) 制定了一项儿科专用条款,以激励为儿童开发药物.
- 了解这一条款的好处和成本对于评估其对儿科医学和市场动态的影响至关重要.
研究的目的:
- 评估BPCA儿科专利条款的好处 (临床试验,标签变化) 和成本 (延迟的仿制药竞争,制造商收入).
- 分析2013年至2023年的数据,以量化BPCA的影响.
主要方法:
- 使用公共FDA和ClinicalTrials.gov数据的回顾性队列研究.
- 确定获得BPCA儿科专用资格 (2013-2023) 的药物.
- 评估临床试验,标签变化和收入数据,使用SSR健康数据库对具有仿制药条目的药物进行评估.
主要成果:
- 在2013年至2023年期间,发生了229项临床试验和110项儿科独家资助.
- 97%的赠款导致了新的疗效标签,20%的新安全标签.
- 每项赠款的中位数过剩收入为1.338亿美元,总额为903亿美元,独家授予的中位数在仿制品进入之前为2.34年.
结论:
- BPCA有效地为儿科药物标签产生关键的临床信息.
- 该条款通过扩大市场独家权,显著增加了制药制造商的收入.
- 对BPCA实施的潜在修改可以提高获得儿科药物信息的及时性和成本效益.
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