For the first time, the World Health Organization (WHO) has opened the opportunity for manufacturers of pediatric cancer drugs to submit products for evaluation under the WHO Prequalification Program to increase access to quality-assured, child-friendly cancer treatments worldwide.
The first Expression of Interest (EOI) covers 12 key pediatric cancer drugs, divided into two groups: six drugs prioritized for development of age-appropriate formulations and six existing drugs selected due to persistent supply and access challenges.
WHO said the initiative aims to support national and global efforts to improve access and affordability of childhood cancer treatment, including through the Global Platform for Access to Children’s Cancer Medicines.
It is important to note that this announcement does not mean that the 12 drugs have recently received WHO prequalification. Instead, WHO invites manufacturers to submit suitable products for evaluation. Products that successfully meet WHO standards can subsequently be added to the WHO Prequalified Medicines List, making them eligible for procurement by United Nations organizations and other buyers.
Six drugs have priority forms that are convenient for children
The first group consists of six drugs that have already proven effectiveness in treating childhood cancer, but for which WHO has identified a need for more suitable forms for children:
- Cyclophosphamide
- Etoposide
- Mercaptopurine
- Methotrexate
- Procarbazine
- Temozolomide
These drugs were identified during the first WHO Pediatric Cancer Drug Optimization exercise, conducted in January 2024. WHO subsequently developed target product profiles describing the characteristics required for optimized formulations, including appropriate dosage, acceptability, stability, safe handling and suitability for use in resource-limited settings.
The WHO EOI specifies formulations such as dispersible tablets, orodispersible mini-tablets and multiparticulate formulations, with dosages designed to provide greater flexibility in pediatric dosing.
The need goes beyond simply having an active cure for cancer. WHO estimates that about 400,000 children and adolescents are diagnosed with cancer each year, with about 90% of them living in low- and middle-income countries. In many of these countries, survival rates remain below 30%, compared with over 80% in many high-income countries. Limited access to quality-assured medicines and the lack of dosage forms intended for children are among the contributing factors to this gap.
The six drugs tested were selected due to access and supply shortages.
The second group of drugs was included for a different reason. WHO has identified six recognized essential medicines for which continued access, availability or lack of prescription can affect the continuity and safety of childhood cancer treatment:
- Pegaspargase
- Asparaginase
- Hydrocortisone
- Dactinomycin
- Vincristine
- Cytarabine
The WHO said the products were selected following a horizon review and consultation involving government agencies, health professionals, partners and technical experts.
Together, these two groups enable a new prequalification pathway to address both sides of the childhood cancer drug challenge: the need for drugs specifically designed for children and the need for a more reliable supply of existing essential treatments.
What happens after manufacturers submit an application?
Manufacturers responding to the EOI must submit documentation to the WHO Prequalification Unit for assessment. To submit an expression of interest in product evaluation, the manufacturer must submit the required documentation, formatted according to the information provided on the WHO Prequalification Team – Medicines Evaluation Team (PQT/MED) website to:
WHO assessment may include analysis of data on product quality, safety and effectiveness; assessment of production processes and quality control systems; inspection of production sites for compliance with good manufacturing practices (GMP); and, where applicable, assessing the feasibility of conducting clinical trials. Medicines may also be subject to random sampling and laboratory testing.
Once WHO determines that the product and associated manufacturing sites meet recommended standards, the product can be added to the WHO List of Prequalified Medicines.
This route is therefore intended to increase the number of quality assured products for the treatment of childhood cancer that can be considered for international and national procurement.
The connection between medical development and access
The EOI is also part of the work of the Global Platform on Access to Children’s Cancer Medicines, created by WHO and St. Jude Children’s Research Hospital in collaboration with UNICEF and the PAHO Strategic Fund.
The platform is expected to eventually reach approximately 120,000 children, linking global drug selection and procurement with country-level efforts to strengthen diagnostic, treatment, supply and quality of care systems.
Alarcos CiezaHead of the NCD Management Unit, Department of Noncommunicable Diseases and Mental Health, WHO, described the first EOI as follows:
“Another important step towards ensuring that children with cancer can get the quality-assured medicines they need, wherever they live.”
Martina PenazzatoThe head of GAP-f, WHO’s Department of Science and Health, said the initiative moves the process from identifying drug needs and developing target product profiles to a regulatory pathway through which manufacturers can develop and submit priority products.
WHO encourages manufacturers of eligible products to consider the invitation and collaborate with the WHO Prequalification of Medicines Group on development requirements, eligibility and submission methods.
Read more on OncoDaily: WHO unveils 2026–2030 strategy to address childhood cancer drug shortages
