July 28 — A total of 26 medicines for rare diseases had been registered in Malaysia as of April this year, from none before 2020, the Dewan Negara was told today
Deputy Health Minister Datuk Hanifah Hajar Taib said the milestone reflects the Ministry of Health’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
She was responding to a question from Senator J. Isaiah on the government’s initiatives and latest developments in the management and treatment of rare diseases in Malaysia
Hanifah Hajar said 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, as well as several rare skin disorders
She said the ministry 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 Hajar also said the National Rare Disease Policy for Malaysia was formulated in 2025, while a national action plan is currently being finalised through collaboration among relevant ministries
At the regional level, she said 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
