University of Liverpool, Department of Women's and Children's Health, Institute of Translational Medicine, Liverpool Women's NHS Foundation Trust, Crown Street, Liverpool L8 7SS, UK.
University of Pavia, Italian Group for the Study of Pharmacoeconomics (GISF), Via Luigi Porta 14, 27100 Pavia, Italy.
Adv Drug Deliv Rev. 2014 Jun;73:2-13. doi: 10.1016/j.addr.2014.02.003. Epub 2014 Feb 18.
Children deserve medicines that are adapted to their needs. The need to include children in drug development has been recognised increasingly over the past few decades. Legal and regulatory frameworks are well established in the EU and US. The amount of work done to study medicines for children is significantly greater than it was 10 years go. Proof-of-concept has been demonstrated for all segments of the paediatric drug development pipeline. It is now time to examine how the practice of developing medicines for children has evolved within those frameworks and to determine how that work should be generalised. This review describes the development of medicines for children and critically appraises the work that has been done within those frameworks. Significant effort is needed to realize the potential provided by the current regulatory framework. Using the work programme of the Global Research in Paediatrics (GRiP) Network of Excellence as a template we outline current work and future growing points.
儿童应该使用适合其需求的药物。在过去几十年中，人们越来越认识到有必要让儿童参与药物开发。欧盟和美国已经建立了完善的法律和监管框架。与 10 年前相比，用于儿童用药研究的工作量显著增加。在儿科药物开发管道的所有环节都已经证明了概念验证。现在是时候研究在这些框架内开发儿童用药的实践如何演变，并确定如何推广这项工作。这篇综述描述了儿童用药的发展，并对这些框架内所做的工作进行了批判性评估。需要付出巨大的努力才能实现当前监管框架所提供的潜力。我们使用卓越全球儿科研究（GRiP）网络的工作计划作为模板，概述了当前的工作和未来的增长点。
