Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency, Amsterdam, The Netherlands.
Modelling and Simulation Working Party, European Medicines Agency, Amsterdam, The Netherlands.
J Clin Pharmacol. 2021 Jun;61 Suppl 1:S22-S27. doi: 10.1002/jcph.1863.
Getting the right dose regimen for children and adolescents is important but poses great scientific, practical, and ethical challenges. At the same time, the availability of data in adults is a huge advantage and needs to be used optimally when designing studies in children and analyzing pediatric data. Furthermore, the processes of maturation and growth are always key when selecting doses for children. All the above make study adaptations and model-informed approaches imperative for dose exposure-response characterization and dose selection in children. This article summarizes the experience gained in the European Medicines Agency on this topic and proposes some general guiding principles for defining objectives, study designs, and methodology tools for pediatric dose selection.
为儿童和青少年确定正确的剂量方案非常重要，但这也带来了巨大的科学、实践和伦理挑战。与此同时，成人数据的可用性是一个巨大的优势，在设计儿童研究和分析儿科数据时需要充分利用。此外，在选择儿童剂量时，成熟和生长过程始终是关键。所有这些都使得研究适应性和模型指导方法对于儿童的剂量-暴露反应特征和剂量选择至关重要。本文总结了欧洲药品管理局在这一主题上的经验，并为儿科剂量选择的目标定义、研究设计和方法学工具提出了一些一般性指导原则。
