WHO sets new global standard for child-friendly cancer drugs, paving way for industry innovation

Geneva, October 2025 - World Health Organization (WHO) has released six new target product profiles for child-friendly formulations of essential cancer medicines. This publication provides pharmaceutical manufacturers with a clear, technical roadmap to develop much-needed, optimized versions of the medicines specifically designed for use in children worldwide.

Each year, an estimated 400,000 children and adolescents develop cancer, yet survival rates remain below 30% in most low- and middle-income countries (LMICs) compared with over 80% in high-income settings. A significant barrier is the lack of age-appropriate medicines. Children with cancer often rely on adult formulations that are difficult or impractical to administer, leading to inaccurate dosing and unnecessary treatment risks.

The "Accelerating the development of priority formulations in childhood cancer" publication defines targets product profiles (TPPs) with optimal and minimum standards for new, child-friendly formulations of six medicines: cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine, and temozolomide.

The TPPs were developed through a standard WHO procedure including an expert consultation held virtually in December 2024, leveraging expertise of partners and global experts in the WHO's Global Accelerator for Paediatric Formulations Network (GAP-f).

Since announcing its first-ever list of priority paediatric cancer formulations in January 2024, WHO has been leading the work on the development of TPPs in childhood cancer, working closely with GAP-f partners including St. Jude Children's Research Hospital, the European Paediatric Formulation Initiative (EuPFI) and the International Society of Paediatric Oncology (SIOP).

A public consultation in spring 2025 gathered additional feedback from industry experts, product developers, the scientific community including paediatric oncologists, pharmacists and formulations experts, implementers, clinicians, and health programme personnel currently involved in the management of childhood cancer.

These efforts led to the successful launch of the six TPPs providing a blueprint outlining the desired characteristics of optimized child-friendly formulations. This work directly supports the goals of the Global Initiative for Childhood Cancer and complements the efforts of the Global Platform for Access to Childhood Cancer Medicines by promoting equitable access to safe, effective, and easy-to-administer cancer medicines for children worldwide.

"Every child with cancer deserves medicines that are safe, effective, and suitable for their age," says Martina Penazzato, GAP-f lead in WHO's Science for Health, Science Division, "The work of WHO and its GAP-f partners on these TPPs serves as a reminder of the urgent need for investment and innovation in paediatric oncology drug development - a field that still trails adult oncology by nearly a decade."

These six TPPs provide clear guidance to manufacturers to address these issues by prioritizing:

• Flexible, child-friendly dosage forms such as dispersible or orodispersible tablets, minitablets, or multiparticulates;
• Stable formulations suitable for hot and humid climates, with shelf-lives over 24 months;
• Palatable and acceptable taste profiles, tested through validated assessments;
• Clear caregiver instructions for safe handling, including in low-literacy settings; and
• Affordable, sustainable production to ensure accessibility in LMICs.