The World Health Organization (WHO) is stepping up efforts to improve the availability of treatment for children and adolescents living with sickle cell disease, with a new focus on medicines that can prevent severe complications and products designed specifically for younger patients. The initiative combines new clinical guidance with measures aimed at improving the development, quality and availability of treatments in countries carrying the greatest burden of the disease.
The urgency is particularly high in sub-Saharan Africa, which accounts for nearly 80% of the global population living with sickle cell disease. WHO estimates that the condition contributed to around 81,100 deaths among children younger than five in 2021. Limited access to early diagnosis, medicines and comprehensive care continues to leave many children vulnerable to complications that could otherwise be managed.
The latest measures could also influence the global market for the sickle cell anemia drug segment, particularly pediatric formulations of hydroxyurea. WHO’s first guideline dedicated specifically to sickle cell disease in children and adolescents recommends hydroxyurea for patients aged nine months through 19 years with sickle cell anaemia, regardless of the severity of their symptoms. The recommendation is being accompanied by efforts to make the medicine easier and safer for children to use.
WHO Puts Hydroxyurea at the Center of Pediatric Sickle Cell Care
WHO’s new guideline covers patients from birth to 19 years and provides 15 recommendations across seven priority areas of sickle cell disease care. Its publication represents a significant step toward establishing more consistent approaches to diagnosing and managing the condition among younger patients.
Hydroxyurea is one of the established medicines used to reduce serious complications associated with sickle cell disease. Despite its long-standing role in treatment, access remains inconsistent across many low- and middle-income countries. Availability is only one part of the problem. Medicines also need to be supplied in forms that children can take accurately and safely.
That issue has become an important part of WHO’s latest work. In September 2025, WHO and the Global Accelerator for Paediatric Formulations, known as GAP-f, brought specialists together for a Paediatric Drug Optimization for Sickle Cell Disease exercise. The meeting examined medicines that should be prioritised for children and considered how formulations could better address the needs of younger patients.
Hydroxyurea emerged as an immediate priority from that exercise. The work subsequently contributed to a Target Product Profile for pediatric hydroxyurea published by WHO in July 2026.
The profile sets out characteristics that manufacturers should consider when developing hydroxyurea products for children. These include suitable dosage forms, the ability to adjust doses, product stability, appropriate packaging and affordability.
Such requirements have implications for both healthcare systems and pharmaceutical manufacturers. A medicine may be clinically effective, but difficulties with dosing, storage, administration or cost can prevent it from delivering its intended benefit in settings where healthcare resources are limited.
The focus on pediatric formulations is particularly relevant for younger patients. Children may require doses that differ considerably from adults, and formulations designed primarily around adult use may not always be practical for them.
WHO’s approach therefore extends beyond simply recommending a medicine. By defining the characteristics expected of a child-friendly product, the organization is attempting to encourage manufacturers to develop medicines that can be used effectively in the communities where they are most needed.
The organization has also begun using this work to strengthen its assessment of sickle cell treatments. WHO has issued its first Prequalification Expression of Interest for sickle cell disease therapeutics, covering pediatric hydroxyurea formulations as well as 500 mg hydroxyurea capsules.
Manufacturers interested in participating are being encouraged to work with WHO’s Prequalification of Medicines Team to understand the requirements for product development and evaluation.
For countries seeking to expand treatment access, prequalification can provide an additional reference when identifying medicines that meet internationally recognised standards for quality, safety and efficacy. This can be particularly useful where national regulatory resources are limited.
Sickle Cell Treatment Pipeline Gains Attention Beyond Hydroxyurea
WHO’s work also extends into the next generation of sickle cell disease treatments. Researchers and pharmaceutical developers are investigating new medicines, biologic therapies and gene-based approaches that could potentially alter the course of the disease.
The organization has established a watch list of promising investigational treatments through the Paediatric Drug Optimization exercise for sickle cell disease. The initiative is intended to ensure that the treatment needs of children and populations with a high disease burden are considered while potential therapies are still being developed.
This is important because the development of a new therapy does not automatically guarantee access in the countries where it could have the greatest impact. Cost, manufacturing capacity, regulatory requirements, healthcare infrastructure and the ability to deliver treatment safely can all influence whether an innovation reaches patients.
Gene therapies are attracting particular attention in sickle cell disease because of their potential to offer longer-term benefits for some patients. However, these approaches remain significantly more complex than established medicines and require specialised infrastructure and substantial investment.
WHO’s current strategy places these emerging treatments alongside immediate efforts to improve access to existing therapies. Expanding the availability of hydroxyurea while preparing for future treatment options allows the organization to address both present and longer-term needs.
The commercial implications are also becoming clearer. Pharmaceutical companies developing sickle cell therapies may face growing expectations to consider pediatric use, affordability and access early in the development process. WHO’s Target Product Profile provides manufacturers with a clearer indication of the characteristics required for pediatric hydroxyurea, while its prequalification initiative creates another pathway for quality-assured products to reach international markets.
For healthcare systems, the challenge will be converting global recommendations into routine care. Clinical guidance can establish what treatment should be offered, but governments and health providers still need reliable medicine supplies, trained healthcare workers, diagnostic services and systems capable of monitoring patients over time.
WHO has therefore emphasized collaboration among governments, manufacturers, regulators, researchers, funders, healthcare professionals and communities affected by sickle cell disease.
A GAP-f webinar scheduled for September 2, 2026, is expected to provide an opportunity to discuss the newly developed resources and their potential application. The broader objective is to ensure that progress in medicine development is matched by progress in access.
For the sickle cell anemia drug market, the latest WHO measures could encourage greater attention to pediatric formulations and quality-assured supply. More importantly, they place the needs of children in high-burden regions closer to the centre of treatment development.
The immediate priority remains making proven medicines available to children who need them. At the same time, WHO’s work signals that future sickle cell therapies will need to address not only clinical effectiveness but also practicality, affordability and access. That combination could shape how the global treatment landscape develops as new medicines and potentially transformative therapies move through research and development.
