The World Well being Group (WHO) has taken the following steps to enhance entry to lifesaving therapy and care for kids and adolescents dwelling with sickle cell illness (SCD)
Sickle cell illness contributed to an estimated 81,100 deaths amongst kids below 5 in 2021.
Nearly 80% of people living with the condition are in sub-Saharan Africa, where limited access to diagnosis, treatment and comprehensive care continues to contribute to preventable deaths and critical problems.
New steerage recommends wider use of hydroxyurea
As a part of its newest efforts, WHO published its first guideline specifically focused on the diagnosis, prevention and clinical management of sickle cell disease in children and adolescents aged from start to 19 years.
The rule incorporates 15 suggestions protecting seven precedence areas of care. One key suggestion is using hydroxyurea for kids and adolescents aged 9 months to 19 years with sickle cell anaemia, no matter symptom severity.
Hydroxyurea is a widely known therapy that may cut back critical problems related to sickle cell illness. Nevertheless, entry to the medication stays uneven, significantly in low- and middle-income international locations.
WHO is combining its scientific suggestions with efforts to make sure that medicines can be found in varieties that kids can safely and simply use.
Making medicines extra appropriate for kids
In September 2025, WHO and the World Accelerator for Paediatric Formulations (GAP-f) introduced collectively specialists by the primary Paediatric Drug Optimization for Sickle Cell Illness train.
The initiative recognized precedence medicines and formulations for kids, with hydroxyurea recognized as an instantaneous precedence. It additionally examined promising remedies nonetheless below growth and highlighted areas needing additional analysis.
This work led to the event of a Target Product Profile for paediatric hydroxyurea, printed in July 2026. The profile units out the traits wanted for a child-friendly medication, together with acceptable dosage varieties, versatile dosing, stability, packaging and affordability.
Opening the door to quality-assured remedies
The brand new product necessities have additionally knowledgeable WHO’s first Prequalification Expression of Curiosity for sickle cell disease therapeutics.
The initiative contains paediatric formulations of hydroxyurea in addition to 500 mg hydroxyurea capsules. Producers are being inspired to have interaction with WHO’s Prequalification of Medicines Staff to grasp the necessities for product growth and analysis.
Prequalification might help international locations establish medicines that meet recognised requirements for high quality, security and efficacy, supporting efforts to enhance entry to dependable remedies.
Getting ready for the way forward for sickle cell therapy
Researchers are additionally investigating new medication, biologics, and doubtlessly transformative approaches, together with gene therapies, for sickle cell illness.
Via the PADO-SCD train, WHO has established a watch listing of promising investigational remedies and recognized analysis priorities. The goal is to make sure that the wants of kids and international locations with a excessive burden of illness are thought-about early in growing future therapies.
WHO’s wider efforts convey collectively scientific steerage, medication growth and entry initiatives. Nevertheless, turning these suggestions into higher care would require continued cooperation between governments, producers, regulators, researchers, funders, health-care suppliers and affected communities.
A GAP-f webinar on 2 September 2026 will showcase the brand new sources and discover how they will help higher therapy and entry for kids dwelling with sickle cell illness.
