Introduction to the New Regulatory Initiative
According to the news (小児薬の同時開発、新相談枠活用を PMDA、新センターが始動 | 日刊薬業 – 医薬品産業の総合情報サイト (jiho.jp)) released by NIKKAN YAKUGYO on 07/05/2024, on July 1st, the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan launched the “Pediatric and Rare Disease Drug Consultation Center.” This center aims to support the development of drugs for pediatric and rare diseases, addressing the drug lag in these critical areas. The center also offers consultation services for the simultaneous development of adult and pediatric drugs, as Japan’s Ministry of Health, Labour and Welfare (MHLW) encourages.
Importance of Simultaneous Development
Director Hiroshi Suzuki highlighted the significance of simultaneous development, stating “Promoting simultaneous development of drugs for adult and pediatric populations will increase the number of medications suitable for children. We urge companies to utilize this new consultation framework.”
Regulatory Support and Fee Exemptions
MHLW’s recent notifications urge companies to develop pediatric drug plans along with adult drug development. Additionally, PMDA introduced a mechanism in the 2024 drug pricing reform to offer higher add-on rates for pediatric drugs approved through simultaneous development plans confirmed by PMDA. Consultation fees at the center are partially or fully subsidized. Notably, the “Pediatric Drug Development Plan Confirmation Consultation” fee is entirely waived for requests submitted between July 1st and January 8th of the following year.
Additional Services and Monitoring
The center also provides consultations for orphan drugs and public solicitation drugs. PMDA monitors the consultation status and has noted initial interest in non-pediatric drugs, with expectations for future pediatric consultations.