Sickle cell disease contributed to an estimated 81 100 deaths among children under five in 2021. WHO is working to close that gap, pairing updated clinical guidance with new efforts to make quality-assured, child-friendly medicines more widely available, especially in sub-Saharan Africa, where nearly 80% of cases occur.
"Too many children with sickle cell disease are still dying or suffering devastating complications, even though we have treatments that can help them. Our goal is simple: to make sure that where a child is born does not determine whether they can get the treatment they need to survive and live a healthy life. The new WHO package of guidance and tools is aimed at improving the care and treatment of children and adolescents living with sickle cell disease (SCD), while accelerating access to medicines that are appropriate, quality-assured and affordable," said Dr Pascale Allotey, Director, Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing, WHO.
The work brings together WHO's technical leadership on child and adolescent health and sickle cell disease from the Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing in close collaboration with the Global Accelerator for Paediatric Formulations (GAP-f), hosted by the Science for Health Department. Together, these efforts span the pathway from evidence-based clinical guidance and identification of priority medicines, to defining the characteristics of appropriate paediatric formulations, establishing a pathway towards quality-assured products, and anticipating future therapeutic options for children.
SCD is the most common inherited blood disorder worldwide and remains a leading cause of preventable childhood death and disability, particularly in low- and middle-income countries. The burden is greatest in sub-Saharan Africa, although SCD also affects populations in the Eastern Mediterranean, the Caribbean, South Asia, Latin America and increasingly diaspora populations worldwide.
"Having an effective medicine is not enough if children cannot get it, afford it or take it in a form designed for them. Together with our partners, we are working to change that for sickle cell disease, starting with hydroxyurea, a medicine that can prevent serious complications and save lives," said Meg Doherty, Director of the Department of Science for Health at WHO.
Despite the availability of effective interventions, major inequities persist in access to early diagnosis, comprehensive care and disease-modifying treatment.
Over the past year, WHO has taken a series of complementary steps to address these gaps.
From clinical guidance to better medicines for children with SCD
In May 2026, WHO published its first normative guideline specifically addressing the diagnosis, prevention and clinical management of SCD in children and adolescents aged 0–19 years . It includes 15 recommendations across seven priority areas, including a strong recommendation for the use of hydroxyurea for all children and adolescents with sickle cell anaemia aged 9 months to 19 years, regardless of clinical severity. Recognizing that clinical recommendations need to be matched by availability and access to medicines that children can actually receive and use appropriately, the WHO Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing and the Global Accelerator for Paediatric formulations convened the first Paediatric Drug Optimization for sickle cell disease (PADO-SCD) exercise in September 2025. The exercise identified priority medicines and formulations for currently available treatments, including hydroxyurea, and established a watch list of promising investigational therapies and related research priorities. The PADO-SCD exercise identified hydroxyurea as an immediate priority for expanding access for its use in this age group in line with the latest WHO guidelines and informed the development of a Target Product Profile (TPP) for paediatric hydroxyurea . Published in July 2026, the TPP defines the preferred and minimum characteristics for age-appropriate formulations, including dosage form, strengths, dosing flexibility, administration, stability, packaging and affordability, with an emphasis on paediatric-friendly forms suitable for flexible weight-based dosing and use in resource-limited settings.
The TPP has informed the first-ever WHO Prequalification Expression of Interest for SCD therapeutics (EOI), translating identified product needs into formulations of hydroxyurea eligible for WHO prequalification evaluation, including paediatric formulations as well as 500 mg hydroxyurea capsules. WHO encourages manufacturers to review the EOI and engage with the WHO Prequalification of Medicines Team regarding eligibility, development requirements and submission pathways.
Looking ahead to a changing SCD treatment landscape
WHO is also looking beyond today's treatment options. The therapeutic landscape for SCD is evolving rapidly, with new medicines, biologics and potentially transformative technologies, including gene therapies, under development.
Through the PADO-SCD exercise, WHO reviewed emerging products in the research and development pipeline and developed a watch list of promising investigational therapies together with priorities for future research.
This forward-looking work aims to ensure that paediatric evidence requirements, access considerations and the realities of high-burden, resource-limited settings are addressed early in development, while encouraging timely engagement with researchers, developers and funders.
Connecting guidance, innovation and access
Taken together, this body of work represents a strong coordinated effort by WHO to strengthen care and treatment for children and adolescents living with SCD.
The publication of these products is an important milestone, but implementation will require continued collaboration. Governments, manufacturers, regulators, researchers, funders, procurement agencies, health-care providers, affected communities and other partners all have a role in ensuring that evidence-based recommendations translate into medicines and services that reach children. The GAP-f network and the forthcoming OneSCD Global Partnership will help accelerate implementation by translating WHO guidance into coordinated action to improve access to quality SCD prevention, treatment and care in high-burden countries.
The upcoming GAP-f #BetterMeds4Kids webinar Advancing paediatric sickle cell disease treatment: new resources to support efforts on research, development and access , taking place on 2 September 2026, will provide an opportunity to launch and present these new WHO resources and reflect on how this body of work can collectively support efforts to improve treatment for children with sickle cell disease – from strengthening access to existing medicines to informing the research and development of future therapies.
