Updates to Pediatric Drug Development Guidelines in Japan

According to the news (小児用薬の計画策定推奨で通知  4月1日から適用、厚労省医薬局 | 日刊薬業 – 医薬品産業の総合情報サイト (jiho.jp)) released by NIKKAN YAKUGYO on March 29, 2024, the Ministry of Health, Labour and Welfare in Japan issued on March 29 an updated notification effective from April 1, asking pharmaceutical companies to provide a pediatric drug development plan before submitting J-NDA (new drug application) for adult use. The notification encourages using clinical data from adult and non-Japanese pediatrics, real-world data, and modeling & simulation to set appropriate pediatric dosages and formulations. PMDA will establish a “Pediatric Drug Development Plan Consultation Service and recommends using such a system. In response to a question on whether it is “mandatory” to formulate a development plan for pediatric drugs when developing drugs for adults, PMDA stated that it is desirable to formulate a development plan for pediatric drugs. Still, it is not mandatory, clarifying that it relies on a corporate decision. This policy aims to accelerate pediatric drug development in Japan.