KUALA LUMPUR, March 16 – The government is working to set up a trust fund and database for rare diseases by the end of next year as part of ongoing efforts to improve care.
Under the Ministry of Health’s (MOH) Rare Disease Programme Strategic Plan for 2022 to 2023, a Rare Disease Trust Fund will be established in partnership with patient advocacy groups to create sustainable funding for treatment of rare diseases in Malaysia.
The MOH is also in the midst of developing a database for rare diseases in the country as scheduled to be carried out in the 12th Malaysia Plan (12MP), Health Minister Khairy Jamaluddin told Batu Gajah MP V. Sivakumar in a written parliamentary reply on March 8.
The government has so far achieved three of its short-term objectives under the strategic plan, including establishing the definition of terminologies in rare disease, forming a guideline for easy access to orphan medications under the Malaysian Orphan Medicine Guideline.
An orphan medicine is a medicinal product that is primarily intended to treat, prevent or diagnose a rare disease.
The MOH has also established a Malaysian Rare Disease List containing 500 rare disease cases found in the country, Khairy said.
According to Rare Diseases Malaysia, among the top five rare diseases in Malaysia include Marfan syndrome; Prader Willi syndrome; Osteogenesis imperfecta; Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes (MELAS); and mucopolysaccharidosis type (MPS) type two.
In terms of funding allocation, the MOH has added another RM1 million in allocation to the Genetics Lab at Tunku Azizah Hospital in Kuala Lumpur this year to strengthen laboratory testing for patients with rare diseases.
MOH has allocated RM250,000 to HKL since 2008 for outsourcing of diagnostic tests for patients suspected with rare diseases. In 2019, HKL was given RM16 million to treat rare diseases for six patients, whose treatment began in 2018, and eight new patients for 2019.
The MOH allocated RM10 million for rare diseases in 2021 for follow-up treatment of rare disease patients and to begin treatment for patients on waiting lists. In 2020, MOH allocated RM16.5 million to treat patients with lysosomal storage disease, Prader Willi syndrome, cystinosis, and other rare diseases.