Global regulatory requirement and development strategy for pediatric drugs
HUANG Fang-hua
WANG Qing-li
Jim Ridings
Susan Watts
WANG Ying
ZHANG Yun
Abstract：Historically,drugs have been used in children without the same levels of evidence for safety and efficacy that have been obtained in adults.Pediatric legislation has been implemented in the US and EU to help address this pediatric knowledge gap.There is an ever increasing focus on global harmonisation of regulatory interaction and increasing attention on rare genetic diseases of children,and the need for early engagement has never been higher.Both EU and US legislation to support pediatric drug development involves both mandated requirements and financial benefits from extended patent lives associated with pediatric use.The EU pediatric regulation requires the applicants to agree to a Pediatric Investigation Plan (PIP) with EMA and meet those binding elements before submission of a Marketing Authority Application (MAA).There are currently four US regulations in force which are of relevance to pediatric drugs development,including the requirement for a Pediatric Study Plan (PSP).The World Health Organisation (WHO) launched the "Make Medicines Child Size" initiative.Integrating pediatric drugs into development plans is critical to efficient development and regulatory engagement.It is advisable for global drug development programs to seek scientific advice timely from the EMA and FDA.
Keywords：pediatric drugresearch and developmentregulations requirementdevelopment strategyPediatric Investigation Plan (EMA)Pediatric Study Plan (US)
Publication Date：2016-01-01
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)