The World Health Organization (WHO) has intensified efforts to improve access to quality-assured, affordable and child-friendly treatments for sickle cell disease (SCD), with a particular focus on sub-Saharan Africa, where nearly 80% of the world's sickle cell cases occur.

The initiative comes amid a significant burden of preventable childhood deaths associated with the inherited blood disorder. Sickle cell disease was estimated to contribute to approximately 81,100 deaths among children under five in 2021, highlighting persistent gaps in diagnosis, treatment and access to comprehensive care.

SCD is the world's most common inherited blood disorder and remains a major cause of childhood disability and preventable mortality, particularly in low- and middle-income countries. While effective treatments are available, many children continue to face barriers to timely diagnosis, appropriate treatment and access to medicines.

WHO prioritises access to child-friendly medicines

WHO's latest efforts bring together updated clinical guidance and initiatives aimed at increasing the availability of medicines specifically suited to children and adolescents living with SCD.

A key focus is hydroxyurea, a disease-modifying medicine that can reduce serious complications associated with sickle cell disease. WHO is working with partners to improve the availability of quality-assured formulations that children can safely and conveniently use.

"Having an effective medicine is not enough if children cannot get it, afford it or take it in a form designed for them," said Meg Doherty, Director of WHO's Department of Science for Health.

The initiative involves collaboration between WHO's Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing and the Global Accelerator for Paediatric Formulations (GAP-f). The partnership covers the pathway from clinical evidence and identification of priority medicines through to development of appropriate paediatric formulations and access to quality-assured products.

New clinical guidance strengthens the role of hydroxyurea

In May 2026, WHO issued its first consolidated guideline specifically addressing the diagnosis, prevention and clinical management of SCD among children and adolescents aged 0–19 years.

The guideline contains 15 recommendations across seven priority areas and strongly recommends hydroxyurea for children and adolescents with sickle cell anaemia aged 9 months to 19 years, regardless of disease severity.

WHO's work also recognises that clinical recommendations must be accompanied by reliable pharmaceutical supply. The organisation and GAP-f have therefore been working to identify the most appropriate medicines and formulations for children with SCD and establish pathways towards their wider availability.

A major opportunity for Africa's pharmaceutical sector

The initiative highlights a broader challenge facing African health systems: the availability of effective medicines does not automatically translate into access for patients.

For countries with a high burden of sickle cell disease, expanding access will require coordinated action across pharmaceutical manufacturing, procurement, regulatory systems, supply chains and health financing.

Strengthening local and regional production of quality-assured paediatric medicines could also help reduce dependence on imported products while improving the resilience and affordability of treatment.

For Africa, where the burden of SCD is disproportionately high, ensuring that children can access appropriate medicines could become an important test of the continent's efforts to strengthen pharmaceutical security and achieve universal health coverage.

Source: World Health Organization (WHO)