Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, Maryland, USA.
J Clin Pharmacol. 2023 Mar;63(3):307-313. doi: 10.1002/jcph.2160. Epub 2022 Oct 19.
Pediatric extrapolation plays a key role in the availability of reliable pediatric use information in approved drug labeling. This review examined the use of pediatric extrapolation in studies submitted to the US Food and Drug Administration and assessed changes in extrapolation approaches over time. Pediatric studies of 125 drugs submitted to the US Food and Drug Administration that led to subsequent pediatric information in drug labeling between 2015 and 2020 were reviewed. The use of pediatric extrapolation for each drug was identified and categorized as "complete," "partial," or "no" extrapolation. Approaches to pediatric extrapolation of efficacy changed over time. Complete extrapolation of efficacy was the predominantly used approach. "Complete," "partial," or "no" extrapolation was used for 51%, 23%, and 26% of the drugs, respectively. This represents a shift in extrapolation approaches when compared to a previous study that evaluated pediatrics drug applications between 2009 and 2014, which found complete, partial, or no extrapolation was used for 34%, 29%, and 37% of the drugs, respectively. Pediatric extrapolation approaches may continue to shift as emerging science fills gap in knowledge of the fundamental assumptions underlying this scientific tool. The international community continues to collaborate on discussions of pediatric extrapolation of efficacy from adults and other pediatric subpopulations to optimize its use for pediatric drug development.
儿科外推在批准药物标签中提供可靠的儿科使用信息方面起着关键作用。本综述考察了儿科外推在向美国食品和药物管理局提交的研究中的使用情况，并评估了随时间推移外推方法的变化。审查了 2015 年至 2020 年间向美国食品和药物管理局提交的导致随后在药物标签中出现儿科信息的 125 种药物的儿科研究。确定了每种药物使用儿科外推的情况，并将其归类为“完全”、“部分”或“无”外推。疗效儿科外推的方法随时间而变化。完全外推疗效是主要使用的方法。分别有 51%、23%和 26%的药物采用“完全”、“部分”或“无”外推。与之前评估 2009 年至 2014 年儿科药物申请的研究相比，这代表了外推方法的转变，该研究发现，完全、部分或无外推分别用于 34%、29%和 37%的药物。随着新兴科学填补了这一科学工具基本假设知识空白，儿科外推方法可能会继续变化。国际社会继续就从成年人和其他儿科亚人群外推疗效的儿科外推进行讨论，以优化其在儿科药物开发中的使用。
