The World Health Organization has launched a coordinated drive to widen access to child-friendly sickle cell medicines, focusing first on hydroxyurea in countries where diagnosis and treatment remain scarce. Announced on September 1, the package links clinical guidance, medicine specifications and a new prequalification pathway for manufacturers. WHO estimates that sickle cell disease contributed to 81,100 deaths among children under five in 2021, while nearly 80% of cases occur in sub-Saharan Africa.
Sickle cell disease is the world's most common inherited blood disorder. Abnormal haemoglobin can distort red blood cells, obstruct circulation and trigger severe pain, anaemia, infections, acute chest syndrome, stroke and organ damage. WHO says the heaviest burden falls on low- and middle-income countries, although the condition also affects the Eastern Mediterranean, Caribbean, South Asia, Latin America and diaspora communities. Unequal access to newborn diagnosis, comprehensive care and disease-modifying treatment leaves many preventable complications untreated.
The initiative builds on WHO's first dedicated clinical guideline for patients aged zero to 19, published in May. Its 15 recommendations across seven priority areas include a strong recommendation that all children and adolescents with sickle cell anaemia from nine months through age 19 receive hydroxyurea regardless of clinical severity. WHO says the medicine can reduce painful crises, acute chest syndrome, hospital admissions and transfusion needs, but safe delivery also requires blood-count monitoring, infection surveillance, vaccination, caregiver information and reliable referral systems.
WHO has also issued a target product profile describing preferred and minimum features for paediatric hydroxyurea, including dosage forms, strengths, flexible weight-based dosing, stability, packaging and affordability. That profile informed the agency's first prequalification expression of interest for sickle cell therapies, covering paediatric formulations and 500-milligram capsules. The aim is to give manufacturers and procurement agencies clearer standards for quality-assured products suited to children and resource-limited health systems.
The access challenge was also visible at a global sickle cell conference that opened in Nairobi on September 1. Kenya's Health Ministry renewed its call for universal infant screening and estimated that about 14,000 children are born with the disease nationally each year. The ministry said it is developing a national registry and revised treatment guidance after training more than 107,000 community health promoters and over 800 specialist care workers. Kenyan reporting noted that screening must connect families to medicines, vaccination, follow-up and referrals to save lives.
WHO is simultaneously tracking emerging medicines, biologics and gene therapies, but stressed that implementation of existing care needs cooperation among governments, regulators, manufacturers, researchers, funders, clinicians and affected communities. A WHO-hosted webinar on September 2 is scheduled to present the new resources, while the GAP-f network and forthcoming OneSCD Global Partnership are expected to support adoption in high-burden countries. The measure of success will be whether standards translate into affordable medicine and sustained care for children rather than remaining guidance on paper.
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