The World Health Organisation (WHO) has released six new target product profiles for child-friendly formulations of essential cancer medicines, giving pharmaceutical manufacturers a technical roadmap to develop optimised versions of the medicines specifically designed for use in children worldwide.
The agency says that about 400 000 children and adolescents develop cancer annually, yet survival rates remain below 30% in most low- and middle-income countries (LMICs), compared with more than 80% in high-income settings. A major obstacle is the lack of age-appropriate medicines, with these children frequently having to rely on adult formulations that are difficult or impractical to administer, and which leads to inaccurate dosing and unnecessary 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 earlier this year gathered additional feedback from industry experts, product developers, the scientific community including paediatric oncologists, pharmacists and formulations experts, implementers, clinicians, and health programme personnel all 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 optimised child-friendly formulations, and supports the goals of the Global Initiative for Childhood Cancer and 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.
These six TPPs provide clear guidance to manufacturers, and prioritise:
- 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 of more than 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.
See more from MedicalBrief archives:
WHO launches free cancer meds for children in low-income countries
Safer treatments urgently needed for child cancer patients
Africa’s $100m Global Hope project to save paediatric cancer lives
Early cancer diagnosis critical for children, say SA experts
SA med students failing to recognise childhood cancer’s early warning signs