Debra Bellon
Strategic Engagement Manager, Rare Diseases International
Alexandra Heumber Perry
CEO, Rare Diseases International
Significant research and development investment in rare diseases has led to unprecedented breakthroughs in treatments and therapies. However, progress is inconsistent and unequal across the globe.
Persons Living with a Rare Disease (PLWRD) worldwide continue to face significant challenges. Clinical experts on rare diseases are few and geographically dispersed. PLWRD and their families face high out-of-pocket expenses. The shortage of financial incentives to develop drugs for the small and dispersed populations of PLWRD means that more than 95% of rare diseases do not currently have an approved treatment. 1
Coalition calls for a WHA resolution on rare diseases
Global challenges need a global response. That is why Egypt and Spain are sponsoring a resolution entitled ‘Rare Diseases: A Priority for Global Health Equity and Inclusion’ at the World Health Assembly (WHA) in 2025, with co-sponsorship from Qatar, Malaysia, France, Panama, Chile, the Philippines, Kuwait, Palestine, Luxembourg, Brazil, Romania and Jordan. This resolution is supported by a broad coalition of rare disease organisations, including Rare Diseases International’s 116 member organisations located across 50 countries and over 120 allied civil society organisations.
The primary proposed aim of the WHA Resolution on Rare Diseases is to call upon the World Health Organization to develop a comprehensive Global Action Plan on Rare Diseases (GAPRD), which would provide Member States with a tangible framework for action and a detailed roadmap. It would establish global targets and strategic goals, along with specific actions to achieve them.
It would be a tool for raising funds and aligning existing financial resources, policies and efforts to ensure more efficient and equitable outcomes. Further, it would establish a robust process for accountability and monitoring to track implementation progress.
Now is the time to shift the paradigm on
rare diseases by adopting long-term,
coordinated and sustainable solutions.
Crucial next step in rare disease advocacy
If successful, the WHA Resolution will build on important policy achievements, notably the 2021 UN Resolution on Addressing the Challenges of Persons Living with a Rare Disease, to provide a strong framework for improving the lives of PLWRD globally.
Now is the time to shift the paradigm on rare diseases by adopting long-term, coordinated and sustainable solutions. The WHA Resolution and the development and implementation of the GAPRD represent a unique opportunity for all stakeholders in the rare disease ecosystem to join together to improve the lives of PLWRD worldwide.
[1] Navarrete-Opazo, Angela A. et al. Can you hear us now? The impact of health-care utilization by rare disease patients in the United States. Genetics in Medicine, Volume 23, Issue 11, 2194 – 2201
