Health minister Dzulkefly Ahmad said the National Policy for Rare Diseases in Malaysia was a critical step in strengthening clinical governance and improving early and accurate diagnosis.
It will also expand access to specialised treatment and orphan drugs, which are medicines developed specifically to treat rare medical conditions that would otherwise be ignored by manufacturers due to low profitability.
“The true measure of a healthcare system is not how well it serves the strongest, but how it protects and upholds the rights of the weakest and most vulnerable.
“Southeast Asia has 45 million people suffering from rare diseases. Malaysia is no exception – around 12,000 patients across nearly 500 types of rare diseases are currently receiving treatment in our hospitals,” he said at a World Rare Disease Day 2026 event at Tunku Azizah Hospital here today.
Dzulkefly said his ministry would prioritise the registry’s development, ensure continuity of care from hospital to the community, and improve sustainable financing mechanisms for treatment.
He also said Malaysia would push its agenda for rare diseases at the Asean level, including capacity building, research collaboration and clinical trials.
A patient support group yesterday called for a national rare disease registry that would consolidate fragmented data from across hospitals, genetics services and disease-specific records, making it easier to treat patients.
In an FMT report, Saida Abu Bakar, president of the Dystrophic Epidermolysis Bullosa Research Association Malaysia, said a registry would serve as the backbone of evidence-based care, research and policy.
She said that without it, patients would remain largely invisible, making it difficult to plan services, allocate resources or coordinate long-term care.
Saida, who was also present at the policy launch today, said while she welcomed the government’s move to set up a registry, it must be implemented with a clear scope, national coverage and sustained governance.
“This will translate into real planning, funding decisions and integrated care for patients,” she said.