Drug Development for Pediatric Populations: Regulatory Aspects.
Drug Development for Pediatric Populations: Regulatory Aspects.
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DOI：
10.3390/pharmaceutics2040364
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发表时间：
2010-11-29
期刊：
影响因子：
5.4
通讯作者：
Dingemanse J
中科院分区：
文献类型：
--
作者：
Zisowsky J;Krause A;Dingemanse J
目前，儿科方面已纳入新药开发过程的早期阶段。近年来，监管机构加强了对儿科信息的执法力度，导致儿童试验数量增加。特别是欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）的具体指导方针和要求构成了监管框架。本文从行业角度总结了儿科药物开发的法规要求和策略。它涵盖了儿科研究规划和实施、儿童首次给药的考虑因素、适当的采样策略以及数据生成和分析的不同方法，以了解药物在儿童中的药代动力学（PK）和药效学（PD）。建模与仿真（M&S）在儿科的作用是突出的，包括监管基础和使用M&S的例子说明，以支持儿科药物开发。
Pediatric aspects are nowadays integrated early in the development process of a new drug. The stronger enforcement to obtain pediatric information by the regulatory agencies in recent years resulted in an increased number of trials in children. Specific guidelines and requirements from, in particular, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) form the regulatory framework. This review summarizes the regulatory requirements and strategies for pediatric drug development from an industry perspective. It covers pediatric study planning and conduct, considerations for first dose in children, appropriate sampling strategies, and different methods for data generation and analysis to generate knowledge about the pharmacokinetics (PK) and pharmacodynamics (PD) of a drug in children. The role of Modeling and Simulation (M&S) in pediatrics is highlighted—including the regulatory basis—and examples of the use of M&S are illustrated to support pediatric drug development.