Abstract
Pediatric patients should be given medicines that have been appropriately evaluated for their use. However, the number of medicinal products currently labeled for pediatric use is limited. In EU and the United States, there are regulations aimed at promoting pediatric drug development, which have been significant success. In contrast, there is no specific regulation for promoting the development of medicines for pediatric use in Japan, and pediatric development is left essentially to the hands of pharmaceutical industries. Ministry of Health, Labor and Welfare (MHLW) has taken some measures to resolve this problem; such as extension of re-examination period of the drug, public knowledge-based marketing authorization application, and development promotion scheme for unapproved and off-label drugs for high medical needs. Pharmaceuticals and Medical Devices Agency (PMDA) has implemented several efforts to encourage pediatric drug development. PMDA established the pediatric working group in November 2011. In an effort to help develop medicinal products for children, the pediatric working group has, collaborated with foreign regulatory agencies for development of pediatric medicines, analyzed and clarified issues raised in the product reviews and the consultations, and exchanged views with domestic stakeholders including pharmaceutical industries, clinical investigators etc. Recently PMDA has established new strategies, Pharmaceutical Affairs Consultation on Research and Development Strategy and Advanced Review with Electronic Data Promotion. In Pharmaceutical Affairs Consultation on Research and Development Strategy, PMDA can advise academic researchers and small businesses on the development of novel drug and medical device candidates for pediatric use. PMDA believes these new strategies contribute to improve predictability of efficacy, safety and optimal dose of pediatric drugs and to accelerate efficient pediatric drug development.
