Sickle cell disease (SCD) is the most common inherited blood disorder worldwide and a major cause of preventable childhood death and disability, particularly in low- and middle-income countries. WHO reports that SCD contributed to an estimated 81,100 deaths among children under five in 2021. The burden is concentrated in sub-Saharan Africa, where nearly 80% of cases occur, though populations in the Eastern Mediterranean, the Caribbean, South Asia, Latin America and diaspora communities are also affected.
In May 2026 WHO released its first normative guideline specifically for diagnosis, prevention and clinical management of SCD in children and adolescents aged 0–19 years. The guideline contains 15 recommendations across seven priority areas. Among these, WHO makes 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.
WHO recognizes that issuing clinical recommendations alone is insufficient if appropriate medicines are unavailable, unaffordable, or not formulated for children. To address this implementation gap, WHO’s Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing worked with the Global Accelerator for Paediatric Formulations (GAP-f), hosted by the Department of Science for Health, to coordinate actions spanning clinical guidance, medicine prioritization and product development.
As part of that work, WHO convened the first Paediatric Drug Optimization for sickle cell disease (PADO-SCD) exercise in September 2025. PADO-SCD identified priority medicines and age-appropriate formulations for currently available treatments, including hydroxyurea, and established a watch list of promising investigational therapies and related research priorities. The exercise highlighted hydroxyurea as an immediate priority for expanding access in line with the May 2026 guideline.
Building on PADO-SCD outcomes, WHO published a Target Product Profile (TPP) for paediatric hydroxyurea in July 2026. The TPP specifies preferred and minimum product characteristics for child-appropriate formulations, covering dosage form, strengths, dosing flexibility, administration, stability, packaging and affordability. The profile emphasizes paediatric-friendly formulations that support flexible weight-based dosing and are suitable for use in resource-limited settings.
The TPP has been translated into regulatory and procurement action: it informed WHO’s first-ever Prequalification Expression of Interest (EOI) for SCD therapeutics. The EOI identifies hydroxyurea product types eligible for WHO prequalification evaluation, including paediatric formulations and 500 mg capsules. WHO is encouraging manufacturers to review the EOI and to engage with the Prequalification of Medicines Team on eligibility, development requirements and submission pathways. These steps aim to create a pathway toward quality-assured products that can be procured and distributed at scale.
WHO leadership emphasizes that having an effective medicine is not enough if children cannot get it, afford it or take it in a form designed for them. The combined strategy—normative guidance, prioritization of paediatric formulations, and a prequalification pathway—seeks to align clinical recommendations with the practical availability of medicines that children can receive and use appropriately.
WHO is also preparing for a rapidly evolving therapeutic landscape for SCD. New medicines, biologics and transformative technologies, including gene therapies, are under development. Through the PADO-SCD exercise, WHO reviewed products in the research and development pipeline and produced 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 settings are taken into account as new therapeutics progress. By defining priorities early, WHO intends to help align research, regulatory expectations and product development with the needs of children and adolescents living with SCD, particularly in low-resource contexts.
WHO’s initiative brings together technical leadership on child and adolescent health and sickle cell disease with efforts to accelerate access to medicines that are appropriate, quality-assured and affordable. The collaboration spans clinical guidance, identification of priority medicines, definition of paediatric formulation characteristics, establishment of a pathway toward prequalification, and anticipation of future therapeutic options for children.
WHO leadership framed the work as an effort to reduce inequities in access to early diagnosis, comprehensive care and disease-modifying treatment. The package of guidance and tools aims to improve care and treatment for children and adolescents with SCD while accelerating access to child-friendly medicines, with a focus on the regions and populations that carry the greatest burden.
Details on specific manufacturer responses, numbers of products submitted for prequalification, procurement mechanisms, implementation timelines and country-level rollout plans were not reported in the source.