- In February 2025, the World Health Organization and St. Jude Children’s Research Hospital initiated the Global Platform for Access to Childhood Cancer Medicines, aiming to provide quality-assured cancer treatments to pediatric patients in low- and middle-income countries. The platform began distributing medications in Mongolia and Uzbekistan, with plans to expand to Ecuador, Jordan, Nepal, and Zambia. The initiative seeks to reach 50 countries over the next 5 to 7 years, ultimately aiming to treat approximately 120,000 children with cancer annually, thereby significantly reducing mortality rates in these regions
- In July 2024, Japan's Pharmaceuticals and Medical Devices Agency (PMDA) launched the Pediatric and Rare Disease Drug Consultation Center to support the development of drugs for pediatric (obesity) and rare diseases(cancer, etc). This initiative aims to address the drug lag in these critical areas and offers consultation services for the simultaneous development of adult and pediatric drugs, as encouraged by Japan’s Ministry of Health, Labour and Welfare (MHLW)
- In November 2024, the World Health Organization (WHO) emphasized the importance of improving access to better pediatric medicines on World Children’s Day. The initiative aims to address the lack of suitable medications for children, especially in low- and middle-income countries. WHO calls for more research and development to create child-specific treatments and improve the overall health and well-being of children worldwide
- In 2024, the FDA approved several significant treatments for pediatric care. One notable approval was for Dupilumab (Dupixent), making it the first treatment specifically indicated for children with eosinophilic esophagitis. Another key approval was for Epinephrine nasal spray (Neffy), marking it as the first nasal spray approved for treating anaphylaxis in both adults and pediatric patients aged 7 years and older
- In April 2024, the U.S. Food and Drug Administration approved Lutathera (lutetium Lu 177 dotatate) for treating pediatric patients aged 12 and older with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including those in the foregut, midgut, and hindgut. This approval marks Lutathera as the first therapy specifically reviewed and approved for pediatric patients with GEP-NETs. The decision was based on the NETTER-P trial, which demonstrated that Lutathera's safety profile in pediatric patients was consistent with that observed in adults
