WHO advances access to quality-assured, child-friendly cancer medicines

The World Health Organization (WHO) has issued its first-ever invitation for expression of interest for manufacturers of childhood cancer medicines to submit products for evaluation by the WHO Prequalification Programme.

Each year, an estimated 400 000 children and adolescents develop cancer. Close to 90% live in low- and middle-income countries, where survival remains below 30%, compared with more than 80% in many high-income countries. Limited access to quality-assured medicines and a lack of formulations suitable for children contribute substantially to this survival gap.

The invitation for expression of interest (EOI) includes twelve essential childhood cancer medicines: six prioritized medicines for which child-friendly formulations are needed and six selected because of documented access and supply gaps.

The process of identifying these products was achieved through complementary prioritization processes that included global stakeholders: one coordinated through the Global Accelerator for Paediatric Formulations (GAP‑f), hosted by the WHO Science for Health Department; and the second by the Global Platform for Access to Childhood Cancer Medicines (Global Platform), an initiative hosted at WHO’s Department of Noncommunicable Diseases and Mental Health, which is a partnership between WHO and St. Jude Children’s Research Hospital in collaboration with UNICEF and PAHO Strategic Fund.

From identifying needs to stimulating product development

The EOI is an outcome of an end-to-end approach that connects country and programme needs with development and procurement of quality-assured, prioritized products.

WHO’s childhood cancer team and GAP‑f jointly convened the first paediatric drug optimization exercise for cancer medicines in January 2024. The process brought together further stakeholder groups including regulators, researchers and product-development specialists to identify priority medicines for development over the short, medium and longer term. Six medicines with established paediatric indications but without suitable child-friendly formulations were noted: cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine and temozolomide.

GAP‑f subsequently supported the development of target product profiles for the six medicines that define the minimum and optimal characteristics that new formulations should meet, including age-appropriate dosing, acceptability, stability, safe handling, affordability and suitability for use in resource-limited settings.

Additionally, after completing horizon scans and consultations with government agencies, health professionals, partners and technical experts, medicines with the greatest supply challenges were identified, specifically, pegaspargase, asparaginase, hydrocortisone (preservative-free), dactinomycin, vincristine and cytarabine.

Supporting the Global Platform and country access

The EOI is a landmark moment for market shaping efforts of the Global Platform. Established by WHO and St. Jude Children’s Research Hospital in collaboration with UNICEF and the PAHO Strategic Fund in 2021, the Global Platform is expected to reach approximately 120 000 children. Its work connects global product selection and procurement with country-led efforts to strengthen diagnosis, treatment, supply systems and quality of care.

“This first EOI is another major step towards ensuring that children with cancer can receive the quality-assured medicines they need, wherever they live,” said Alarcos Cieza, Unit Head, Management of NCDs, Department of Noncommunicable Diseases and Mental Health. “It brings together the Global Platform’s understanding of country and supply needs with WHO’s normative, technical and quality-assurance functions, creating a practical pathway from global priorities to medicines that can reach children.”

A shared call to manufacturers and partners

By opening a route for manufacturers to submit priority childhood cancer products for WHO evaluation, the EOI can help expand the pool of quality-assured medicines available for international and national procurement. WHO encourages manufacturers to review the EOI and engage with the WHO Prequalification of Medicines Team regarding product eligibility, development requirements and submission pathways.

“The EOI demonstrates the value of an end-to-end approach to paediatric medicines” said Martina Penazzato, GAP-f lead, WHO Science for Health Department. “Through GAP‑f, we have moved systematically from horizon scanning and drug optimization to target product profiles and now to a concrete regulatory pathway that can stimulate manufacturers to develop and submit the products children need.”