国外儿科药物研发的监管要求和研发策略

Global regulatory requirement and development strategy for pediatric drugs

从历史上看,药物用于儿童时通常没有获得像成人药物一样的安全性和有效性证据。随着人们对全球药品监管有关儿科药物研发互动协调的关注的不断增长,同时对儿童遗传性罕见病也给予了越来越多的支持和关注,尽早和监管机构接触的期望日渐增高。欧盟和美国的法律都强制性要求进行和支持儿科药物研发,同时包括随之而来的经济利益,即延长儿科用药的专利保护。在提交上市许可申请之前,欧盟儿科法规要求制药公司申请人与欧洲药品管理局(EMA)达成儿科研究计划的协议,而目前美国有4个儿科药物研发相关的法律,提出了儿科研究计划的要求。世界卫生组织推出了"量身定制儿童药物"的倡议。对于全球性药物开发项目,明智的做法是尽可能早地寻求欧盟EMA和美国FDA有关儿科药物研发计划的科学建议,并达成协议。

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 advisablefor global drug development programs to seek scientific advice timely from the EMA and FDA.