WHO Unveils Six New Target Product Profiles to Boost Child-Friendly Cancer Treatments. 

Launch marks major step toward equitable access to paediatric cancer medicines worldwide. 

Geneva:

The World Health Organization (WHO) has released six new Target Product Profiles (TPPs) for child-friendly formulations of essential cancer medicines, offering pharmaceutical manufacturers a clear and technical roadmap to develop optimized, age-appropriate treatments for children living with cancer across the globe.

Each year, an estimated 400,000 children and adolescents are diagnosed with cancer. Yet, while survival rates in high-income countries exceed 80%, they remain below 30% in most low- and middle-income countries (LMICs). One of the critical barriers is the lack of suitable, age-appropriate cancer medicines. Many children must rely on adult formulations that are difficult to administer, often leading to inaccurate dosing and increased risk of treatment-related complications.

The newly published WHO guidance — “Accelerating the Development of Priority Formulations in Childhood Cancer” — defines TPPs with both optimal and minimum standards for child-appropriate versions of six essential cancer drugs: cyclophosphamide, etoposide, mercaptopurine, methotrexate, procarbazine, and temozolomide.

These TPPs were developed through WHO’s standard consultation process, including a global expert meeting held virtually in December 2024, under the umbrella of the Global Accelerator for Paediatric Formulations Network (GAP-f). The process drew upon extensive collaboration with partners such as St. Jude Children’s Research Hospital, the European Paediatric Formulation Initiative (EuPFI), and the International Society of Paediatric Oncology (SIOP).

Following a public consultation in spring 2025 — which included input from paediatric oncologists, pharmacists, scientists, and product developers — WHO finalized the six TPPs. Together, they provide a practical blueprint for manufacturers, outlining the ideal characteristics for new, child-friendly formulations to improve safety, dosing accuracy, and acceptability.

“Every child with cancer deserves medicines that are safe, effective, and suitable for their age,” said Dr. Martina Penazzato, GAP-f lead in WHO’s Science for Health 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.”

Key Priorities for Manufacturers

The TPPs urge developers to focus on several essential criteria for paediatric cancer medicines:

• Flexible, child-friendly dosage forms such as dispersible or orodispersible tablets, minitablets, or multiparticulates;
• Stability in hot and humid climates, ensuring a shelf-life of at least 24 months;
• Palatable formulations with acceptable taste, verified through validated testing;
• Clear caregiver instructions for safe handling and administration, especially in low-literacy settings; and
• Affordable and sustainable production to ensure accessibility in LMICs.

The new TPPs also lay the groundwork for future inclusion of these optimized formulations in WHO’s Prequalification Expression of Interest list and, eventually, in the Model List of Essential Medicines for Children, once suitable formulations are developed.

Building Momentum: Upcoming GAP-f Dialogue

To sustain this momentum, WHO’s GAP-f network is inviting private-sector innovators and manufacturers to participate in the 2025 GAP-f Private Sector Entities Dialogue, scheduled for 11 November 2025. The virtual event, held in collaboration with the Access to Medicine Foundation, will provide a platform to explore technical solutions, shared challenges, and partnership opportunities aimed at accelerating paediatric formulation development and access.

Agenda highlights include discussions on strengthening collaboration between industry and public health partners, aligning with the GAP-f 2025–2030 Strategy, and a dedicated thematic session on childhood cancer focusing on identifying remaining gaps and joint solutions.

By setting clear scientific and technical targets, WHO’s new TPPs mark a pivotal advance in global efforts to improve childhood cancer care. The initiative complements the Global Initiative for Childhood Cancer and the Global Platform for Access to Childhood Cancer Medicines, both designed to ensure equitable access to safe, effective, and age-appropriate cancer treatments for children worldwide.

“This milestone takes us one step closer to ensuring that every child, no matter where they live, can receive the right treatment in the right form,” Dr. Penazzato added.