WHO Opens New Path to Lifesaving Cancer Medicines for Children

Around 400,000 children and adolescents develop cancer each year, with close to 90% living in low- and middle-income countries.

The World Health Organization has invited pharmaceutical manufacturers to submit childhood cancer medicines for evaluation under its Prequalification Programme, marking the first time the agency has opened this quality-assurance pathway specifically for these treatments. The decision could increase the supply of trusted medicines for children and adolescents in countries where shortages, unsuitable formulations and inconsistent product quality continue to weaken cancer care.

Around 400,000 children and adolescents develop cancer each year, with close to 90% living in low- and middle-income countries. Survival rates in many of these countries remain below 30%, while more than 80% of young patients survive in numerous high-income nations. Late diagnosis and limited treatment capacity play a role, yet access to affordable, quality-assured medicines in forms that children can safely take remains one of the most persistent barriers.

Twelve Priority Medicines Enter the WHO Quality Pathway

The invitation covers 12 essential childhood cancer medicines selected through consultations involving governments, regulators, researchers, healthcare professionals, procurement partners and product-development specialists. Six require child-friendly formulations: cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine and temozolomide.

These medicines already have established uses in paediatric cancer treatment, yet available formulations may be difficult to measure, swallow, store or administer safely to younger patients. Adult tablets sometimes need to be split, crushed or mixed by caregivers and health workers, creating a risk of inaccurate dosing and unsafe exposure to powerful cancer drugs.

WHO's Global Accelerator for Paediatric Formulations has developed target product profiles for the six medicines, outlining the features manufacturers should consider when creating improved versions. The profiles cover age-appropriate dosing, taste and acceptability, product stability, safe handling, affordability and practical use in clinics with limited staff, equipment or refrigeration.

Six additional medicines were selected because health systems have reported serious access or supply difficulties: pegaspargase, asparaginase, preservative-free hydrocortisone, dactinomycin, vincristine and cytarabine. Unreliable availability of these products can delay chemotherapy, force doctors to change established treatment plans or leave hospitals without suitable alternatives.

Turning Treatment Needs Into Better Products

The initiative grew from work started in January 2024, when WHO's childhood cancer team and the Global Accelerator for Paediatric Formulations convened the first paediatric drug-optimization exercise focused on cancer medicines. Specialists examined which treatments required urgent formulation improvements and which products faced the greatest barriers to reliable supply.

Manufacturers can now bring eligible products into a structured assessment covering their quality, safety and efficacy. Medicines that meet WHO prequalification requirements become easier for international procurement agencies and national health programmes to identify and purchase with confidence, which may encourage more suppliers to enter underserved markets while supporting stronger competition and more dependable production.

This process creates a direct connection between the needs reported by doctors and health ministries, the development of better formulations, regulatory evaluation and eventual procurement. Children stand to benefit from medicines that are easier to administer, more accurately dosed and better suited to hospitals and households in resource-limited settings.

A Global Platform Designed to Reach 120,000 Children

The new pathway supports the Global Platform for Access to Childhood Cancer Medicines, created in 2021 by WHO and St. Jude Children's Research Hospital in collaboration with UNICEF and the PAHO Strategic Fund. Expected to reach about 120,000 children, the platform links medicine selection and procurement with country-led improvements in diagnosis, treatment, supply management and quality of care.

WHO is encouraging manufacturers to examine the invitation and speak with its Prequalification of Medicines Team about eligibility, product-development expectations and submission procedures. Strong participation could expand the number of quality-assured medicines available to national programmes, reduce damaging supply interruptions and give healthcare workers greater confidence that the treatments reaching their patients meet recognized standards.

For families facing childhood cancer, a medicine is only useful when it is safe, affordable, consistently available and suitable for the child who needs it. WHO's first invitation gives manufacturers and global health partners a practical opportunity to close that gap and bring more children within reach of effective cancer treatment.