KUALA LUMPUR, July 28 — Twenty-six medicines for rare diseases had been registered in Malaysia as of April this year, from none before 2020, the Dewan Negara was told today.
Health Deputy Minister Datuk Hanifah Hajar Taib said the milestone reflects the Health Ministry's (MOH) ongoing efforts to improve access to medicines and treatment for patients with rare diseases.
“The ministry has introduced the Malaysian Orphan Medicines Guideline 2020, which sets out the procedures and criteria for evaluating new medicines. Compliance with these guidelines has helped expedite the approval of safe and effective medicines,” she said during the question-and-answer session.
Hanifah was responding to Senator Isaiah Jacob's query on the government's initiatives and latest developments in the management and treatment of rare diseases in Malaysia.
She added that 529 types of rare diseases have been identified and included in the Malaysian Rare Disease List, with about 80 per cent attributed to genetic factors.
These include lysosomal storage diseases, which impair the body's ability to break down certain substances due to enzyme deficiencies; spinal muscular atrophy, which affects nerve cells and muscle function; and several rare skin disorders.
The MOH has increased its annual allocation for the diagnosis and treatment of rare diseases to RM42 million this year from RM25 million previously, with treatment costs ranging from RM100,000 to RM1 million per patient.
Hanifah noted that the National Rare Disease Policy for Malaysia was formulated last year, while a national action plan is currently being finalised through collaboration among relevant ministries.
At the regional level, Malaysia hosted and spearheaded the Southeast Asia Rare Disease Policy Forum in 2025 and is working towards the adoption of an ASEAN Declaration on Rare Diseases.
