The World Health Organization (WHO) has opened its first-ever pathway for manufacturers of childhood cancer medicines to submit products for evaluation under the WHO Prequalification Programme, aiming to expand access to quality-assured and child-appropriate cancer treatments worldwide.
The first Expression of Interest (EOI) covers 12 essential childhood cancer medicines, divided into two groups: six medicines prioritized for development of age-appropriate formulations and six established medicines selected because of persistent supply and access challenges.
WHO said the initiative is intended to support national and global efforts to improve the availability and affordability of childhood cancer treatment, including through the Global Platform for Access to Childhood Cancer Medicines.
Importantly, the announcement does not mean that the 12 medicines have newly received WHO prequalification. Rather, WHO is inviting manufacturers to submit eligible products for assessment. Products that successfully meet WHO standards can subsequently be added to the WHO List of Prequalified Medicines, making them acceptable for procurement by United Nations organizations and other purchasers.
Six medicines prioritized for child-friendly formulations
The first group consists of six medicines that already have established roles in paediatric cancer treatment but for which WHO has identified a need for more suitable formulations for children:
- Cyclophosphamide
- Etoposide
- Mercaptopurine
- Methotrexate
- Procarbazine
- Temozolomide
The medicines were identified during WHO’s first Paediatric Drug Optimization exercise for cancer medicines, convened in January 2024. WHO subsequently developed target product profiles describing the characteristics needed for optimized formulations, including appropriate dosing, acceptability, stability, safe handling and suitability for use in resource-limited settings.
WHO’s EOI specifies formulations such as dispersible tablets, orodispersible minitablets and multiparticulate formulations at strengths intended to provide greater flexibility for paediatric dosing.
The need extends beyond simply having an active cancer medicine available. WHO estimates that about 400,000 children and adolescents develop cancer each year, with close to 90% living in low- and middle-income countries. Survival remains below 30% in many of these countries, compared with more than 80% in many high-income settings. Limited access to quality-assured medicines and the lack of formulations designed for children are among the factors contributing to this gap.
Six established medicines selected over access and supply gaps
A second group of medicines was included for a different reason. WHO identified six established essential medicines where persistent access, supply or formulation-related gaps can affect the continuity and safety of childhood cancer treatment:
- Pegaspargase
- Asparaginase
- Hydrocortisone
- Dactinomycin
- Vincristine
- Cytarabine
WHO said these products were selected following horizon scanning and consultations involving government agencies, health professionals, partners and technical experts.
Together, the two groups allow the new prequalification pathway to address both sides of the childhood cancer medicines challenge: the need for formulations specifically designed for children and the need for a more reliable supply of established essential treatments.
What happens after manufacturers apply?
Manufacturers responding to the EOI must submit documentation to the WHO Prequalification Unit for evaluation. To submit an expression of interest for product evaluation, the manufacturer must send the required documentation, arranged according to the information provided on the WHO Prequalification Unit – Medicines Assessment Team (PQT/MED) website at https://extranet.who.int/pqweb.
WHO’s assessment can include reviewing product data on quality, safety, and efficacy; evaluating manufacturing processes and quality-control systems; inspecting manufacturing sites for compliance with Good Manufacturing Practices (GMP); and, where applicable, assessing clinical testing facilities. Medicines may also undergo random sampling and laboratory testing.
Once WHO determines that a product and the relevant manufacturing sites meet its recommended standards, the product can be added to the WHO List of Prequalified Medicines.
The pathway is therefore intended to increase the number of quality-assured childhood cancer products that can be considered for international and national procurement.
Connecting medicine development with access
The EOI also forms part of the work of the Global Platform for Access to Childhood Cancer Medicines, established by WHO and St. Jude Children’s Research Hospital in collaboration with UNICEF and the PAHO Strategic Fund.
The platform is expected to ultimately reach approximately 120,000 children, connecting global medicine selection and procurement with country-level efforts to strengthen diagnosis, treatment, supply systems and quality of care.
Alarcos Cieza, Unit Head, Management of NCDs at WHO’s Department of Noncommunicable Diseases and Mental Health, described the first EOI as:
“Another major step towards ensuring that children with cancer can receive the quality-assured medicines they need, wherever they live.”
Martina Penazzato, GAP-f Lead at WHO’s Science for Health Department, said the initiative moves the process from identifying medicine needs and developing target product profiles toward a regulatory pathway through which manufacturers can develop and submit priority products.
WHO is encouraging manufacturers of eligible products to review the invitation and engage with the WHO Prequalification of Medicines Team regarding development requirements, eligibility and submission pathways.
Read further on OncoDaily: WHO Unveils 2026–2030 Strategy to Close the Childhood Cancer Medicines Gap
