最近在日本的儿科药物开发中利用儿科外推和建模和仿真方法
Akinori Nakashima1,2, Akihide Tsujimoto1,3, Masako Saito1,4
1Japan Pharmaceutical Manufacturers Association Clinical Evaluation Expert Committee, Drug Evaluation Committee, Tokyo, Japan.
日本的儿科药物开发进展停滞不前,但建模和模拟 (M&S) 提供了一种灵活的方法. 使用M&S和外推可以简化数据包,以更快地获得儿科药物批准.
科学领域:
- 药理学 药理学是指药理学的学科.
- 监管科学 监管科学
- 儿科 儿科 儿科
背景情况:
- 日本的儿科药物批准在2010-2020年间停滞在30%左右.
- 卫生,劳工和福利部提出了促进儿科药物开发的新策略.
- 建模和模拟 (M&S) 被引入为儿科药物开发的关键工具.
研究的目的:
- 分析在日本儿科药物批准数据包中对外推算和M&S的利用情况.
- 评估M&S在儿童群体剂量选择和治疗方案建议中的作用.
- 确定为更有效的儿科药物开发策略提供机会.
主要方法:
- 对日本95个儿科药物批准申请数据包的审查 (2019年1月 - 2023年3月).
- 根据儿科外推的程度对药物的分类 (完全,部分,没有).
- 在批准申请中分析M&S应用程序的剂量选择和剂量方案.
主要成果:
- 43.2%的药物使用了完整的抽象,30.5%的部分抽象和26.3%没有抽象.
- M&S被广泛使用 (60.0%) 来证明剂量选择和推方案的合理性.
- 除外推算策略与现有数据相结合,可以创建灵活和高效的批准包.
结论:
- 进一步整合现有数据和推断可以提高儿科药物批准效率.
- 将儿科发育安全纳入M&S对于未来的评估至关重要.
- 业界,监管机构和学术界之间的早期合作对于在日本及时提供儿科药物至关重要.
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