WHO Pushes Child-Friendly Sickle Cell Medicines as Thousands of Young Lives Remain at Risk

The World Health Organization is responding with updated clinical guidance, clearer priorities for paediatric medicines and a pathway designed to bring quality-assured, affordable treatments closer to children living in high-burden countries.

Sickle cell disease contributed to an estimated 81,100 deaths among children under five in 2021, exposing a painful divide between the treatments already known to save lives and the care many families can reach. Nearly 80% of cases occur in sub-Saharan Africa, where children may face delayed diagnosis, medicine shortages, high costs and health services that lack the resources needed for lifelong care. The World Health Organization is responding with updated clinical guidance, clearer priorities for paediatric medicines and a pathway designed to bring quality-assured, affordable treatments closer to children living in high-burden countries.

Effective treatment remains beyond the reach of many families

Sickle cell disease is the world's most common inherited blood disorder, affecting the shape and movement of red blood cells and raising the risk of severe pain, anaemia, infections, stroke, organ damage and early death. Its heaviest burden falls on low- and middle-income countries, particularly across sub-Saharan Africa, though the condition affects families in the Eastern Mediterranean, the Caribbean, South Asia, Latin America and diaspora communities worldwide. Children can live healthier lives when diagnosis comes early, and care includes vaccination, infection prevention, regular monitoring and disease-modifying treatment, yet these services remain unevenly distributed and unaffordable for many households.

"Too many children with sickle cell disease are still dying or suffering devastating complications, even though we have treatments that can help them," said Dr Pascale Allotey, Director of WHO's Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing. She said the new package of guidance and practical tools is built around a clear principle: a child's birthplace should not decide whether treatment is available or whether that child has the chance to survive and grow into a healthy adult.

Hydroxyurea becomes a central part of childhood care

WHO published its first guideline dedicated to diagnosing, preventing and managing sickle cell disease in children and adolescents aged up to 19 years in May 2026. The document contains 15 recommendations covering seven priority areas, including a strong recommendation that hydroxyurea should be offered to all children and adolescents with sickle cell anaemia from nine months of age, regardless of how severe their symptoms appear. Hydroxyurea can reduce painful crises and other dangerous complications, making wider access to the medicine a major opportunity to prevent disability, hospital admissions and avoidable deaths.

A medicine cannot deliver its full benefit when a child cannot swallow it, families cannot afford it or health workers cannot adjust the dose safely as the child grows. WHO and the Global Accelerator for Paediatric Formulations identified hydroxyurea as an immediate priority during the first Paediatric Drug Optimization for sickle cell disease exercise in September 2025. A Target Product Profile published in July 2026 now sets out the preferred features of child-friendly hydroxyurea, including suitable dosage forms, flexible strengths, stability in difficult climates, practical packaging and pricing that reflects the realities of resource-limited health systems.

Quality-assured medicines and future therapies move into focus

The product profile has shaped WHO's first Prequalification Expression of Interest for sickle cell disease treatments, opening a route for manufacturers to submit paediatric hydroxyurea formulations and 500 mg capsules for quality evaluation. WHO is encouraging medicine producers to review the requirements and engage with its Prequalification of Medicines Team, a step that could give governments and procurement agencies greater confidence when purchasing products for national health programmes.

WHO is preparing for a treatment landscape that may eventually include new medicines, biologics and gene therapies. Its paediatric drug optimization exercise created a watch list of promising experimental treatments and identified research questions that need attention, especially around paediatric evidence, affordability and delivery in countries carrying the greatest burden. Progress will depend on cooperation among governments, manufacturers, regulators, researchers, health workers, funders, procurement agencies and affected communities. The GAP-f network and the forthcoming OneSCD Global Partnership are expected to help turn WHO's recommendations into coordinated action, giving more children access to early diagnosis, dependable care and medicines made for their needs.