WHO Pushes Child-Friendly Sickle Cell Treatment Access

The World Health Organization has launched a coordinated push to make child-friendly sickle cell treatment easier to obtain, beginning with hydroxyurea formulations that younger patients can take and health systems can use safely.

The September 1 update brings several pieces of work into one pathway: new clinical guidance, a target product profile for paediatric hydroxyurea, and the first WHO invitation for manufacturers of sickle cell medicines to seek prequalification. Together, they are meant to narrow the distance between knowing what works and getting an appropriate, quality-assured medicine to a child.

This is not an announcement that every treatment gap has been solved. It is a practical attempt to align medical recommendations, product design, regulatory review and procurement around the needs of children, especially in high-burden countries.

What WHO announced

In its September 1 departmental update, WHO said sickle cell disease contributed to an estimated 81,100 deaths among children under five in 2021. Nearly 80% of cases occur in sub-Saharan Africa, although the inherited blood disorder also affects people in the Eastern Mediterranean, the Caribbean, South Asia, Latin America and diaspora communities worldwide.

WHO’s new package is designed to cover more than prescribing advice. It connects clinical recommendations with decisions about which medicines should be prioritised, what a usable paediatric formulation should look like, how products can enter prequalification review, and which emerging therapies should remain on the research watch list.

The immediate focus is hydroxyurea, a disease-modifying medicine that can reduce serious complications. Access remains uneven, and formulations available to health systems are not always easy to dose accurately for very young children.

Why a child-friendly formulation matters

Children are not simply smaller adults. Their medicine doses often change with body weight, and a tablet designed for adults may be difficult to divide or administer accurately. A formulation that works in a specialist hospital may also be impractical in a clinic with limited equipment, refrigeration or supply continuity.

WHO’s target product profile for paediatric hydroxyurea, published in July, describes the preferred and minimum characteristics developers and procurers should work toward. It covers dosage form, strength, dosing flexibility, administration, stability, packaging, affordability and the regulatory pathway.

The profile gives particular attention to soluble or dispersible tablets that can support flexible, weight-based dosing. WHO says scored tablets in child-appropriate strengths could make the medicine easier to give and more suitable for resource-limited settings. This is a product-design target, not a claim that the preferred formulations are already available everywhere.

The clinical guidance behind the access push

The foundation is WHO’s first normative guideline devoted specifically to sickle cell disease in children and adolescents aged 0 to 19. The May 2026 guideline contains 15 recommendations across seven clinical areas.

Those areas include early diagnosis, infection prevention, hydroxyurea treatment, pain management, acute chest syndrome, stroke prevention and screening for disease-related complications. WHO strongly recommends hydroxyurea for children and adolescents with sickle cell anaemia from nine months through 19 years of age, regardless of clinical severity.

The recommendation is intended to help countries create a consistent standard of care. Implementation still requires trained staff, reliable diagnosis, follow-up, laboratory capacity where needed, medicine supply and communication with families. A guideline can define good practice; it cannot by itself make that practice universally available.

How WHO prequalification could help procurement

On July 24, WHO issued its first expression of interest for sickle cell therapeutics. The invitation asks eligible manufacturers to submit hydroxyurea products for evaluation by the WHO Prequalification Unit.

The listed products include 500 mg capsules and soluble or dispersible tablets in 100 mg and 500 mg strengths. WHO identifies single-scored soluble or dispersible tablets as preferred because they can support safer administration and more flexible dosing for children.

Prequalification does not purchase medicine or approve it for every national market. It evaluates whether submitted products meet WHO standards for quality, safety and efficacy. A successful listing can give procurement agencies and health programmes an additional assurance when selecting products, which may make it easier to build dependable supply across multiple countries.

What patients and health systems should watch next

The next test is whether manufacturers submit suitable products and whether countries can convert the guidance into funded services. Governments, regulators, donors, procurement agencies, clinicians and affected communities all have distinct roles.

• Manufacturers need to develop or submit formulations that meet the preferred characteristics.
• Regulators and WHO evaluators need evidence that products consistently meet quality standards.
• Health ministries and buyers need procurement plans, budgets and reliable distribution.
• Clinicians and families need clear dosing guidance, follow-up and access to comprehensive sickle cell care.
• Researchers need to study implementation as well as new medicines, biologics and gene therapies.

WHO also plans to use the forthcoming OneSCD Global Partnership and the Global Accelerator for Paediatric Formulations network to coordinate implementation. A September 2 webinar is scheduled to present the resources and discuss research, development and access.

What this announcement does—and does not—change

The package provides a clearer route from evidence to medicine design and quality review. It also makes the needs of children explicit instead of treating paediatric access as an afterthought.

It does not mean hydroxyurea will immediately reach every child who needs it. WHO has not announced a universal delivery date, a global procurement budget or guaranteed manufacturer participation. Countries will still have to address diagnosis, supply, workforce capacity and long-term care.

The importance of the September update is therefore structural. It gives health systems and manufacturers a shared specification and a formal review pathway, while linking those tools to a clinical guideline that covers the broader realities of sickle cell care.

For readers following how international health institutions turn research into policy, Smashology Media also examined WHO’s renewed Science Council and its role in connecting evidence with public-health decisions.