Colleagues from the World Health Organization, the Medicines Patent Pool and leading research and clinical institutions have published a new open-access paper in The Lancet Child & Adolescent Health on: “Paediatric Drug Optimization for Sickle Cell Disease: priorities for research and development in children.”

The paper presents the outcomes of the WHO-convened Paediatric Drug Optimization for Sickle Cell Disease (PADO-SCD) process, which reviewed existing treatments, pipeline candidates and potentially curative approaches to identify research and development priorities for children and adolescents.

The authors highlight six key priorities:

  • Hydroxyurea (hydroxycarbamide) remains the leading near-term priority for children with sickle cell disease. However, the absence of age-appropriate, affordable, and widely available formulations continues to constrain equitable access.
  • The Paediatric Drug Optimization for Sickle Cell Disease process identified age-appropriate hydroxyurea formulations as short-term priorities and developed a target product profile to guide formulation development, regulatory strategy, procurement, and implementation.
  • Among investigational therapies, pyruvate kinase activators—mitapivat, etavopivat, and tebapivat—and decitabine plus tetrahydrouridine (NDec) emerged as promising candidates for paediatric investigation, based on biological rationale, emerging efficacy signals, oral administration, and feasibility potential.
  • Gene therapies offer potentially curative options, but their development, pricing, infrastructure requirements, and restricted availability in high-burden countries raise major equity concerns that must be addressed from the outset.
  • The promise of potentially curative therapies must not delay investment in scalable disease-modifying treatments that can benefit far greater numbers of children in the near term. Scientific innovation in sickle cell disease should be guided by equity, feasibility, and public health relevance.
  • Paediatric inclusion should be embedded early in therapeutic development, with studies using validated, patient-relevant endpoints that capture long-term benefit for people, including children, in high-burden settings.

As the paper makes clear, scientific innovation in sickle cell disease should be guided by equity, feasibility and public health relevance, helping ensure that advances in treatment can reach the children who need them most.

Read the full paper in The Lancet Child & Adolescent Health.