新生儿及儿童药物疗法的发展：科学与监管要求
Development of Drug Therapies for Newborns and Children: The Scientific and Regulatory Imperatives.
作者信息
Mulugeta Yeruk Lily, Zajicek Anne, Barrett Jeff, Sachs Hari Cheryl, McCune Susan, Sinha Vikram, Yao Lynne
机构信息
Division of Pediatric and Maternal Health, CDER US Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20903, USA.
Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, 6710B Rockledge Drive Room 2334, MSC 7002, Bethesda, MD 20817, USA.
出版信息
Pediatr Clin North Am. 2017 Dec;64(6):1185-1196. doi: 10.1016/j.pcl.2017.08.015.
Abstract
Pediatric legislation has generated information about the efficacy, safety, and dosing of more than 600 products in children. Extrapolation of adult efficacy data has been an integral part of pediatric drug development. Advances in our understanding of physiology and pharmacology have improved the approach to pediatric dose selection. However, a high percentage of pediatric trials do not meet their primary efficacy endpoint. Delays in initiating completing pediatric studies persist. This article describes these advances and provides innovative approaches to optimize pediatric drug development.
摘要
儿科立法已产生了有关600多种儿童用药产品的疗效、安全性和剂量的信息。成人疗效数据的外推一直是儿科药物研发的一个组成部分。我们对生理学和药理学认识的进步改进了儿科剂量选择的方法。然而，很大比例的儿科试验未达到其主要疗效终点。启动和完成儿科研究的延迟仍然存在。本文描述了这些进展，并提供了优化儿科药物研发的创新方法。
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