Geneva, Switzerland – September 1, 2026
The World Health Organization has begun two coordinated efforts to close persistent access gaps in care for sickle cell disease and rare diseases — both of which disproportionately burden low- and middle-income countries and have historically been treated as orphan issues in global health policy.
The two initiatives, both announced in early September 2026, reflect a wider shift in WHO's approach: pairing updated clinical guidance with new efforts to make quality-assured, child-friendly medicines more widely available in the countries where the burden is greatest.
Sickle cell disease: closing the treatment gap
On 1 September 2026, WHO released a coordinated package to expand treatment and care for children and adolescents with sickle cell disease (SCD) — a condition that contributed to an estimated 81,100 deaths among children under five in 2021, with nearly 80 per cent of cases in sub-Saharan Africa.
The package builds on WHO's first normative guideline on SCD in children and adolescents, published in May 2026, which included 15 recommendations across seven priority areas and a strong recommendation for hydroxyurea for all children and adolescents with sickle cell anaemia aged 9 months to 19 years.
"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." — Dr Pascale Allotey, Director, Department of Sexual, Reproductive, Maternal, Child, Adolescent Health and Ageing, WHO
WHO followed the guideline with a Target Product Profile for paediatric hydroxyurea (July 2026) and the first-ever WHO Prequalification Expression of Interest for SCD therapeutics, which translates identified product needs into formulations of hydroxyurea eligible for WHO prequalification.
The work will be presented at the GAP-f #BetterMeds4Kids webinar on 2 September 2026 and feed into the forthcoming OneSCD Global Partnership.
Rare diseases: a 10-year action plan
On 31 August 2026, WHO outlined the process for developing a 10-year Global Action Plan on Rare Diseases, mandated by World Health Assembly resolution WHA78.11 (2025). The resolution treats rare diseases as a global health priority linked to equity and inclusion.
The plan will be developed through a structured consultative process: an online discussion paper published in the final quarter of 2026; a first draft in early 2027 informed by web-based Member State, UN agency and civil society inputs; web-based and virtual informal consultations on the first draft; and a revised draft for the WHO Executive Board's 162nd session in 2028, with submission for consideration by the Eighty-first World Health Assembly in May 2028.
"Rare diseases affect millions of people globally and are often complex, involving multiple organ groups and resulting in disabilities and premature death. Many people living with a rare disease frequently experience diagnostic delays, fragmented care, limited access to effective interventions, financial hardship, stigma and social exclusion." — WHO Departmental Update, 31 August 2026
The action plan will be the first time the agency has produced a comprehensive global instrument for rare diseases — a category that, taken together, affects an estimated 300 million people worldwide but has historically been excluded from mainstream health system planning because each individual condition is rare.
Why the two efforts are linked
Both efforts sit at the intersection of clinical guidance and access to medicines — the bottleneck that WHO has identified as the limiting factor for diseases that affect small populations. The agency's growing toolkit — prequalification, target product profiles, paediatric drug optimisation — is designed to be disease-agnostic and reusable.
What changes for affected countries
For countries with the highest SCD burden, the practical change is that hydroxyurea procurement can be planned around WHO-prequalified paediatric formulations rather than around whatever adult formulations are locally available. For rare diseases, the action plan's ten-year horizon gives health ministries a planning horizon within which to integrate rare disease services into universal health coverage frameworks.
What to watch
The sickle cell prequalification pathway is the more immediately visible. The first WHO-prequalified paediatric hydroxyurea product is expected to reach procurement channels in 2027. The rare disease discussion paper, due in late 2026, will be the first opportunity for civil society and patient organizations to influence the plan's strategic objectives and accountability framework.
Source: WHO News Release, "WHO moves to expand access to lifesaving sickle cell treatment and care for children", 1 September 2026. WHO Departmental Update, "Global action plan on rare diseases – process and next steps", 31 August 2026. WHA Resolution WHA78.11 (2025). WHO SCD Guideline, May 2026. WHO Prequalification Expression of Interest, SCD therapeutics, August 2026.
FIRAT Editorial Board
Institutional Research Desk · Foresight Institute of Research and Translation
The collective editorial and research translation board of FIRAT, synthesising peer-reviewed evidence, policy briefs, and division milestones across our seven foundational research pillars.
