Debra Bellon
Strategic Engagement Manager, Rare Diseases International
Alexandra Heumber Perry
Chief Executive Officer, Rare Diseases International
People living with a rare disease face persistent challenges, but leaders have an opportunity to improve outcomes.
The 300 million people1 living with a rare disease worldwide face delayed diagnosis, limited access to treatment and care and significant financial burdens.
Urgent need for global action on rare diseases
Global health is being reshaped, restructured and redefined. At this time of shifting challenges and priorities, diagnosis, care and treatment of rare diseases offer valuable insights for global health. Designing systems that meet the complex needs of people living with rare diseases can improve health outcomes for everyone.
This requires a global, coordinated approach: expertise is often scarce and geographically
dispersed, and data, resources and care capacity are limited.
Global Action Plan and call for equity
This requires a global, coordinated approach: expertise is often scarce and geographically dispersed, and data, resources and care capacity are limited.
In 2025, the World Health Assembly took a historic step to address systemic gaps by adopting a Resolution recognising rare diseases as a global public health priority.
The Resolution calls on the WHO to develop a 10-year Global Action Plan on Rare Diseases: a coordinated framework that articulates targets, responsibilities and benchmarks. This will provide governments with a shared framework to structure policy and better integrate rare diseases into health systems and Universal Health Coverage. This framework is particularly vital for low- and middle-income countries, where WHO leadership and guidance can help ensure a more equitable and sustainable response to rare diseases.
The Coalition for Advocacy for Rare Disease Equity (CARE) brought together more than 300 organisations worldwide in support of the Resolution and remains committed to its effective implementation. Regional Task Forces convened by RDI stand ready to offer input from lived experience and practical knowledge, demonstrating the community’s commitment to advocacy and collaboration.
By engaging meaningfully with the rare disease community and advancing a timely and ambitious Global Action Plan, global policymakers have an unprecedented opportunity to transform the promise of the Resolution into lasting impact — making health systems fairer, stronger and more responsive for everyone.
[1] Wakap, S. et al. (2019). Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. European Journal of Human Genetics.
