Three new national plans for rare diseases
In the wake of the World Health Assembly (WHA) Resolution on Rare Diseases increasing the visibility of rare diseases on the global health agenda, three countries have taken concrete measures to affirm their commitment to meeting the needs of the rare disease community. Last month, Malaysia, Ireland, and Luxembourg each published new national plans for rare diseases, providing policy frameworks for the three nations to better address the challenges faced by people living with a rare disease (PLWRD) and their families.
On 27 August 2025, Malaysia’s Ministry of Health published the National Policy for Rare Diseases in Malaysia 2025. This is the nation’s first ever strategy for rare diseases, and aims to address critical gaps and strengthen the delivery of comprehensive healthcare solutions for PLWRD in Malaysia. The policy defines a disease as rare if it affects fewer than 1 in 4,000 people, and identifies 9 key pillars of action to improve rare disease care, diagnosis and awareness.
In total, the plan identifies 30 targets across the pillars outlined above. These targets are informed by the specific challenges facing Malaysia’s rare disease community, such as the lack of a national rare disease registry, no regulatory incentives for orphan drugs, and limited access to comprehensive genetic diagnostic testing.
Moving forward, the new National Policy outlines the steps which must be taken to address these challenges and meet the plan’s targets. These include the establishment of multi-stakeholder technical committees to oversee an integrated rare disease management program under the purview of the Ministry of Health; the creation of a national rare disease registry; and facilitating the manufacture and importation of affordable orphan products.
In addition to the full strategy, the Malaysian Ministry of Health also published a summary fact sheet to help patients, families, healthcare professionals and the general public understand the policy’s goals and impact.
Also on 27 August, the Irish Department of Health unveiled their National Rare Disease Strategy 2025-2030. This strategy builds on the foundation of the previous National Rare Diseases Plan for Ireland 2014-2018, and addresses issues encountered by PLWRD throughout all stages of life. The result of multi-stakeholder collaboration between patients, clinicians, researchers, and government representatives, the strategy puts forward 11 recommendations to reinforce rare disease care and awareness in Ireland.
By implementing these recommendations, the Irish strategy seeks to establish a comprehensive framework designed to improve diagnosis, treatment, and support for PLWRD. Overall, the actions outlined in the strategy aim to create a more inclusive healthcare system in Ireland which address the unique challenges posed by rare diseases.
Finally, in September, Luxembourg’s Ministry of Health adopted the country’s second national plan for rare diseases, the Plan National Maladies Rares Luxembourg 2025-2029 (PNMRL). This plan represents a continuation of the first national plan, which was in place from 2018-2023, and aims to promote a reinforced, coordinated healthcare system for people living with a rare disease (PLWRD) in Luxembourg. It takes a holistic, person-centred approach to the challenges facing the rare disease community across five thematic areas.
Across these themes, the plan contains a total of 23 objectives, 64 measures and more than 139 specific actions. The PNMRL’s implementation is intended to complement and work alongside other national plans, such as the implementation of the Convention on the rights of people with disabilities, the second national cancer plan and Luxembourg’s newborn screening programme. Overall, the hope is that implementing the PNMRL will lead to more efficient resource utilisation in the rare disease field, as well improved access to diagnosis, treatment and care for PLWRD in Luxembourg.
Together, these new national plans for rare diseases demonstrate the growing recognition of rare diseases worldwide as a major public and global health challenge. This is in line with the recent adoption of the WHA Resolution on Rare Diseases, which recognises the global challenges faced by PLWRD and calls on Member States to reinforce rare disease care. By introducing policies specific to rare diseases, Malaysia, Ireland, Luxembourg, and many other countries are setting an example for the rest of the world, and are taking steps to ensure that PLWRD are not left behind in national and international health policy ecosystems.
