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The World Health Organization (WHO) has introduced a new pathway allowing manufacturers of childhood cancer medicines to submit their products for evaluation under the WHO Prequalification Programme. The move is aimed at improving access to quality-assured and child-friendly cancer treatments, especially in countries where essential medicines are not always available. The first Expression of Interest (EOI) includes 12 essential medicines used in childhood cancer care. These medicines have been divided into two categories. The first includes six treatments for which more suitable formulations for children are needed. The second covers six established medicines affected by continuing problems related to supply, access or formulation. WHO has emphasised that the 12 medicines have not automatically received prequalification. The EOI is an invitation for manufacturers to submit eligible products for assessment. Medicines that meet WHO's requirements may later be included in the WHO List of Prequalified Medicines.
Childhood Cancer Treatment Still Faces A Global Access Gap
An estimated 400,000 children and adolescents develop cancer worldwide every year. Nearly 90 per cent of these cases occur in low- and middle-income countries, where survival rates are considerably lower than those reported in high-income countries. WHO estimates that survival remains below 30 per cent in many low- and middle-income countries, while it exceeds 80 per cent in several high-income settings. Access to quality-assured medicines is one of the factors influencing this difference. However, the challenge extends beyond the availability of a medicine itself. Cancer medicines must be supplied regularly, remain affordable for health systems and be available in forms that children can take safely. Difficulties with tablet size, dosing and swallowing can make treatment more complicated, particularly for younger children. The new WHO pathway seeks to encourage the development of medicines that are both reliable in supply and suitable for paediatric use.
Six Medicines Identified For Better Paediatric Formulations
The first category includes six medicines that already play an important role in childhood cancer treatment. WHO has identified a need for formulations that are more appropriate for children. The medicines are:
- Cyclophosphamide
- Etoposide
- Mercaptopurine
- Methotrexate
- Procarbazine
- Temozolomide
These medicines were identified during WHO's first Paediatric Drug Optimization exercise for cancer medicines, conducted in January 2024. After the exercise, WHO prepared target product profiles describing the features needed in improved paediatric formulations. These include accurate dosing, acceptability, stability, safe handling and suitability for use in settings with limited healthcare resources. The EOI mentions formulations such as dispersible tablets, orodispersible minitablets and multiparticulate products. Such options may allow healthcare professionals to adjust doses more easily for children of different ages and body sizes. The focus is therefore not only on making cancer medicines available, but also on ensuring that children can receive them in a practical and safe manner.
Six Established Medicines Included Because Of Supply Concerns
The second category consists of six established essential medicines. These were selected because ongoing problems involving availability, supply or formulation may affect the safety and continuity of childhood cancer treatment. The medicines are:
- Pegaspargase
- Asparaginase
- Hydrocortisone
- Dactinomycin
- Vincristine
- Cytarabine
WHO said the medicines were selected after horizon scanning and consultations with government bodies, healthcare professionals, technical experts and partner organisations. This group addresses the challenge of ensuring that important medicines already used in childhood cancer care remain available when required. While the first category focuses on improving formulations for children, the second aims to strengthen the supply of established treatments. Together, the two groups are intended to support more consistent access to essential childhood cancer medicines.
How Will The WHO Evaluation Process Work?
Manufacturers responding to the EOI must submit the required documents to the WHO Prequalification Unit for assessment. Details about the submission process are available on the WHO Prequalification Unit's Medicines Assessment Team website:
The evaluation may involve reviewing evidence related to a medicine's quality, safety and efficacy. WHO may also assess manufacturing methods, quality-control procedures and production facilities. Manufacturing sites may be inspected to check compliance with Good Manufacturing Practices (GMP). Where applicable, clinical testing facilities may also be reviewed. The products may additionally be subject to random sampling and laboratory testing. If a product and its relevant manufacturing sites are found to meet WHO's recommended standards, the medicine may be added to the WHO List of Prequalified Medicines.
What Is The WHO List Of Prequalified Medicines?
WHO prequalification is a formal assessment process used to determine whether selected medical products meet international standards for quality, safety and efficacy. The current announcement does not mean that all 12 medicines have already been prequalified. Manufacturers must first submit eligible products, which will then undergo the relevant evaluation process. Products that successfully meet WHO's standards may be included on the WHO List of Prequalified Medicines. Such products can be considered for procurement by United Nations organisations and other eligible purchasers. The list can also help national procurement agencies identify medicines that have undergone an international quality assessment. For childhood cancer care, a larger number of prequalified products may help countries identify dependable suppliers and improve access to essential treatments.
Initiative Linked To Global Childhood Cancer Medicines Platform
The new EOI is part of the work being carried out through the Global Platform for Access to Childhood Cancer Medicines. The platform was established by WHO and St Jude Children's Research Hospital in collaboration with UNICEF and the Pan American Health Organization's Strategic Fund. It aims to connect medicine development and procurement with efforts to improve diagnosis, treatment, supply systems and the overall quality of childhood cancer care at the country level. The platform is expected to eventually reach approximately 120,000 children. Its work covers several areas, including medicine selection, product development, procurement and distribution. The new prequalification pathway is expected to support these efforts by encouraging manufacturers to develop and submit priority products for evaluation.
WHO Describes First EOI As An Important Step
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 would help move the process beyond identifying medicine needs and preparing target product profiles.
The next stage would involve creating a regulatory route through which manufacturers can develop and submit priority products for formal assessment. WHO has encouraged manufacturers of eligible products to study the invitation and contact the WHO Prequalification of Medicines Team for information about development requirements, eligibility and submission procedures.
Why Child-Friendly Cancer Medicines Are Important
Treatment for childhood cancer may include chemotherapy, surgery, radiotherapy, targeted medicines or a combination of these approaches. The treatment plan depends on the type and stage of cancer. Children often need doses that are carefully adjusted according to their age, weight and medical condition. If medicines are available only in unsuitable strengths or difficult-to-swallow forms, administering treatment can become more challenging. Child-friendly formulations may make it easier for healthcare professionals to provide accurate doses and for families to manage treatment. Consistent availability is also essential. If medicines are out of stock, treatment may be delayed or interrupted. This can affect continuity of care and add to the emotional and financial strain on families.
Improving the quality, suitability and supply of medicines is therefore a key part of addressing inequalities in childhood cancer outcomes. WHO has opened its first-ever Expression of Interest inviting manufacturers to submit childhood cancer medicines for evaluation under the WHO Prequalification Programme. The first EOI covers 12 essential medicines. Six have been identified for the development of more child-friendly formulations, while the other six have been selected because of continuing supply, access and formulation challenges.
The announcement does not mean that these medicines have already received WHO prequalification. Manufacturers must submit eligible products for assessment, and only those that meet WHO's standards may be added to the WHO List of Prequalified Medicines. The initiative aims to improve access to reliable, quality-assured and child-appropriate cancer treatments, helping ensure that children have a fairer chance of receiving life-saving care regardless of where they live.
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