评估与新瘤学药物和生物产品初步儿科研究计划相关的信息差距
Daphne Guinn1, Marilyn Gafford2, Gilbert J Burckart1
1Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Clinical pharmacology and therapeutics
|July 9, 2023
概括
针对儿童的治疗和公平 (RACE) 研究加速法案要求为新的癌症药物制定儿科研究计划 (PSP). 对最初PSP的分析揭示了常见的信息缺口,特别是豁免理由和临床研究细节.
科学领域:
- 在瘤学瘤学.
- 儿科药物开发 儿科药物开发
- 监管科学 监管科学
背景情况:
- 针对儿童的治疗和公平 (RACE) 研究加速法案要求提交针对成人癌症的新分子向药物和生物制剂的儿科研究计划 (PSP).
- 这些计划必须要么提出儿科调查,要么提供豁免或推迟的理由.
- 确保在药物开发中考虑儿科患者是一个关键目标.
研究的目的:
- 对2021年针对癌症新分子实体的初始儿科研究计划 (iPSP) 提交的信息缺口进行景观分析.
- 在iPSP审查过程中识别美国食品和药物管理局 (FDA) 评论中的趋势.
- 为赞助商提供指导,帮助他们准备适当的iPSP.
主要方法:
- 对2021年收到的癌症新分子实体的初始PSP (iPSP) 提交进行了景观分析.
- 来自FDA审查过程的评论使用九个不同的标志被分类.
- 分析的重点是确定常见的信息差距和趋势.
主要成果:
- 对于要求完全豁免的iPSP,主要信息缺口是关于分子标相关性的不充分理由.
- 其他iPSP计划 (延期,部分豁免或调查) 经常显示临床研究特征,临床药理学或缺少临床/非临床数据的信息缺口.
- 在初步审查期间FDA评论的趋势被确定.
结论:
- 最初的PSP提交往往含有信息缺口,需要赞助商的注意.
- 对分子标相关性的更明确的理由对于豁免请求至关重要.
- 赞助商需要确保临床研究特征和药理学的全面数据,以满足儿科药物开发的监管要求.
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