Kuala lumpur: The Ministry of Health (MOH) today launched the National Policy for Rare Diseases, a reform initiative aimed at strengthening the country's healthcare ecosystem in addressing rare diseases in a more structured and sustainable manner. Health Minister Datuk Seri Dr Dzulkefly Ahmad said the policy reflects the government's commitment to ensure that rare diseases are no longer regarded as a marginal issue, but are instead elevated as a mainstream agenda.
According to BERNAMA News Agency, globally, the challenges in relation to rare diseases are immense. Out of nearly 8,000 types of rare diseases, only five percent have specific treatment options. At the launch of the policy in conjunction with the National Rare Disease Day 2026 observance at Tunku Azizah Hospital, it was detailed that about 12,000 patients involving nearly 500 types of rare diseases in Malaysia are currently receiving treatment at government hospitals. However, the actual figure is expected to be higher.
Many patients have yet to receive a diagnosis, including children who continue to suffer without medical answers, while their families face difficulties in obtaining certainty about the illnesses affecting them. Dzulkefly mentioned that MOH had previously implemented various initiatives in stages, such as the establishment of a Rare Disease List and a Trust Fund, but ad hoc approaches were insufficient to address what is essentially a systemic issue.
The policy marks a shift towards a more comprehensive and structured national framework, focusing on three main pillars: strengthening clinical governance, enhancing early detection and precise diagnostics, and expanding access to treatment, including the supply of orphan drugs and specialized nutrition. The policy was formulated based on a whole-of-nation approach, involving cross-ministerial synergy in areas such as education, welfare, and social protection, in addition to close collaboration with clinical specialists and industry partners.
Future priorities include the development of a national registry, sustainable treatment financing, and cross-border collaboration, aligning with Malaysia's role as ASEAN Chair in 2025 and host of the ASEAN Health Ministers' Meeting (AHMM) 2026. The government is committed to ensuring that no person living with a rare disease feels isolated, in line with the principles of inclusivity, solidarity, and sustainability in the development of the national healthcare system.