在2020-2022财政年度新药和生物制品许可证申请审批中的现实证据
Gabriel K Innes1, Kimberly A Smith1, Aida Kuzucan2
1Office of Medical Policy, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clinical pharmacology and therapeutics
|April 25, 2025
概括
现实世界数据 (RWD) 和现实世界证据 (RWE) 越来越多地用于支持医疗产品的批准. 美国食品和药物管理局 (FDA) 在2020-2022财年新药和生物制品许可申请中报告了RWE的使用情况.
科学领域:
- 监管科学 监管科学
- 制药医学 制药医学
- 医疗信息学 医疗信息学
背景情况:
- 常规收集的临床数据和先进的统计方法正在加强使用现实世界数据 (RWD) 来生成现实世界证据 (RWE).
- 作为处方药使用费法案 (PDUFA VII) 的一部分,美国食品和药物管理局 (FDA) 承诺每年报告在医疗产品监管决策中使用RWE.
- 第一个PDUFA VII报告 (FY2023) 强调了使用RWE批准的两种药物:托西利祖马布和拉克萨米德.
研究的目的:
- 描述2020-2022财年期间由药物评估和研究中心 (CDER) 批准的新药申请 (NDA) 和生物制品许可申请 (BLA).
- 确定NDA和BLA,其中RWE对有效性的证据做出了重大贡献,或为批准提供了必要的安全数据.
- 为了监管决策,向利益相关者介绍RWD在产生RWE中的应用.
主要方法:
- 审查CDER在2020年,2021年和2022财政年度批准的NDA和BLA.
- 确定RWE在建立实质性有效性证据方面发挥了关键作用的应用.
- 分析用于RWE生成的数据源,特别是注册表数据和医疗记录数据.
主要成果:
- 在2021财年和2022财年期间,RWE为五个NDA/BLA提供了实质性的有效性证据.
- 在RWE支持下批准的特定药物包括福斯登诺丁,塔克罗利斯,阿巴塔塞普特,沃索里提德和阿尔佩利西布.
- 总共有六项研究为这些批准提供了关键的RWE,其中四项使用注册数据,两项使用医疗记录数据.
- 在此期间,没有任何申请仅依靠RWE获得批准所需的安全数据.
结论:
- RWE是支持医疗产品监管决策的宝贵工具,特别是在证明有效性方面.
- 这些发现与PDUFA VII的目标一致,即提高药物批准过程中RWE的透明度和理解.
- 持续报告和分析RWE使用将进一步告知监管实践和行业参与.
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