Rare Diseases - Awareness and Funding Needed

In Malaysia, there is no official registry for rare diseases resulting in the absence of data as there is no tracking of the patients.

According to Rare Disease Malaysia website there are at least 79 types of rare diseases found in Malaysia.

Although the number of patients of each disease is small, rare disease patients as a collective is common.

The Health Ministry has yet to set an official definition for rare disease, complicating the monitoring and regulation of the management of rare diseases.

The Malaysian Rare Disorders Society (MRDS) has, therefore, taken it upon themselves to classify rare disease as a disease that affects less than one in every 4,000 people of the general population.

Malaysia does not have a National Rare Disease Policy.

Last year, a letter jointly written by three doctors from Universiti Sains Malaysia and University Malaya Medical Centre revealed that only 60% of rare disease patients in Malaysia are receiving treatment due to a lack of treatment options or a long waiting list.

On Dec 17 last year, Deputy Health Minister Dr Lee Boon Chye had told the Dewan Negara that the ministry would be formulating a National Framework for Rare Diseases but did not provide a timeline.

Dr Lee said the framework would include the setting up of a governance committee and a rare disease data system to facilitate policymaking, programmes, strategy and intervention.

Treatment costs for rare diseases are exorbitant, said Sunway Medical Centre Clinical Genetics external consultant Prof Thong Meow Keong.

“Drugs to treat neurological conditions such as spinal muscular atrophy may cost up to RM2mil yearly. Bone marrow transplants can come up to RM400,000, while growth hormones, up to thousands a month,” said Prof Thong.

The medical fees for rare disease sufferers are often well beyond the patients’ and their caregivers’ means, giving rise to the need for funding.

Malaysia Lysosomal Diseases Association (MLDA) president Lee Yee Seng said most parents have no choice but to rely on government aid and public generosity.

Lee himself has two daughters, Wei Ling and Yen Ling, diagnosed with Pompe disease, caused by an accumulation of glycogen in the lysosome due to a deficiency of acid alpha-glucosidase, an enzyme that converts glycogen to glucose. ERT for both of them can come to RM2mil annually.
Lee feels very grateful for the RM16mil budget allocated by MOH this year to Hospital Kuala Lumpur (HKL) for patients with rare diseases, including those suffering from LSD. Together with Zakat Selangor and corporate sponsorships, 50 LSD patients have benefited from these funds.

On Lee’s wish list are two key items: that the government come up with laws so that those with rare diseases can get prompt medical attention, and for stakeholders to come forward with funding to ease the suffering of those afflicted.

There are 90 other LSD patients registered with MLDA. The association was founded in 2011 and since then, 10 patients have died caused by complications brought on by their disease. For now, there is an urgent need for seven LSD patients to receive ERT treatments.

/theSTAR 28-02-201 

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