New global guidance and manufacturing initiatives focus on delivering age-appropriate sickle cell therapies to pediatric patients.
RT’s Three Key Takeaways:
- Global Pediatric Mortality: Sickle cell disease contributed to an estimated 81,100 deaths among children under 5 years of age in 2021, with nearly 80% of cases occurring in sub-Saharan Africa, according to the World Health Organization.
- Prioritizing Child-Friendly Formulations: The agency issued clinical guidance recommending hydroxyurea for young patients aged 9 months to 19 years and established product profiles to accelerate child-friendly drug options.
- Manufacturer Invitation: A newly launched expression of interest invites pharmaceutical manufacturers to submit pediatric hydroxyurea formulations and 500 mg capsules for international quality prequalification.
The World Health Organization (WHO) has initiated a series of global measures to expand access to lifesaving treatments and clinical management for pediatric sickle cell disease (SCD), according to the agency. SCD contributed to an estimated 81,100 deaths among children under 5 years of age in 2021, with nearly 80% of cases occurring in sub-Saharan Africa.
While the highest burden persists in sub-Saharan Africa, the condition also affects populations across the Eastern Mediterranean, South Asia, the Caribbean, Latin America, and expanding diaspora communities internationally. Inequities remain prevalent in early diagnosis, disease-modifying therapies, and specialized healthcare delivery, according to the WHO.
“Too many children with sickle cell disease are still dying or suffering devastating complications, even though we have treatments that can help them. Our goal is simple: to make sure that where a child is born does not determine whether they can get the treatment they need to survive and live a healthy life. The new WHO package of guidance and tools is aimed at improving the care and treatment of children and adolescents living with sickle cell disease, while accelerating access to medicines that are appropriate, quality-assured and affordable,” said Dr Pascale Allotey, director of the Department of Sexual, Reproductive, Maternal, Child and Adolescent Health and Ageing at the World Health Organization.
Clinical Guidance and Drug Optimization
To establish standard care practices, WHO published clinical guidelines in May 2026 covering the diagnosis, prevention, and clinical management of SCD in pediatric populations from birth to 19 years of age. The publication provides 15 specific recommendations across seven priority areas, headlined by a strong recommendation for the administration of hydroxyurea in all children and adolescents with sickle cell anemia aged 9 months to 19 years, regardless of clinical severity.
Because clinical recommendations require reliable supply pipelines, the agency partnered with the Global Accelerator for Pediatric Formulations to convene the Pediatric Drug Optimization for sickle cell disease exercise. The panel determined that hydroxyurea serves as an immediate priority for expanding treatment access in this patient demographic, leading to the July 2026 release of a Target Product Profile (TPP) for pediatric hydroxyurea formulations. The TPP outlines specifications for dosage forms, packaging, administration, stability, pricing, and weight-based dosing suitable for resource-limited healthcare environments.
“Having an effective medicine is not enough if children cannot get it, afford it or take it in a form designed for them. Together with our partners, we are working to change that for sickle cell disease, starting with hydroxyurea, a medicine that can prevent serious complications and save lives,” said Meg Doherty, director of the Department of Science for Health at the World Health Organization, in a news release.
Prequalification and Pipeline Therapies
Building on the product profiles, WHO launched its first-ever Prequalification Expression of Interest (EOI) for SCD therapeutics, according to the release. The initiative allows pharmaceutical manufacturers to submit pediatric formulations and 500 mg hydroxyurea capsules for evaluation by the WHO prequalification team to facilitate quality-assured procurement by international health agencies and national healthcare programs.
Beyond current pharmaceuticals, WHO reported that it is tracking investigational therapies, emerging biologics, and genetic therapies to ensure pediatric access considerations and the requirements of high-burden regions are factored into early clinical development. The agency stated that translating these recommendations into frontline clinical care will depend on international coordination across governments, regulatory bodies, healthcare providers, and the upcoming OneSCD Global Partnership.
