A national policy on rare diseases is needed so that there can be a clear framework for early diagnosis, documentation and treatment of rare diseases. It will also ensure better budget allocations for the treatment of all rare diseases.
“Without a national policy for rare diseases, funding will continue to be an issue and many patients will go without treatment. Many will be undiagnosed due to the lack of awareness about rare diseases and treatments that are available,” says Lee Yee Seng, president of the Malaysia Lysosomal Disease Association (MLDA).
Malaysia, at present, has no specific legislations pertaining to rare diseases, unlike countries in the region like Singapore, Japan, Taiwan, South Korea and the Philippines that have enacted laws and policies to recognise the needs of those with rare diseases and orphan drugs (drugs to treat rare diseases), observed UMMC's Prof Dr Thong Meow Keong in a letter to The Star late last year.
There is also no registry for rare diseases; as a result, there is no way to track the actual number of rare disease patients in the country.
Last December, Deputy Health Minister Dr Lee Boon Chye announced in parliament that the Health Ministry would be formulating a National Framework for Rare Diseases that would include the setting up of a governance committee and a data system to facilitate policy making, programmes, strategy and intervention.
MLDA’s Lee is hoping that this will lead to an actual policy.
“The ministry has set up three committees to look into rare diseases: a technical committee, another one for advocacy and the last one for drug formulary. The committees are meeting for the first time late this month and hopefully, this will lead to the formulation of a national policy,” he says.
The Malaysian Rare Disorders Society (MRDS) defines a rare disorder as a disease that is observed in less than 4,000 cases.
In the 2019 budget, the government allocated RM50mil for the treatment of rare diseases, Hepatitis C, stunted growth among children, haemodialysis treatments as well as for enhancing primary healthcare for Malaysians. Out of this allocation, RM16mil is for rare diseases, of which RM10mil is for patients with LSD.
“People may wonder why LSD gets such a huge allocation when there are so many other rare diseases that need funding for treatment. We (MLDA) have been knocking on the door (of the ministry) since 2011, lobbying for funding. In Malaysia, that’s how things work. If you don’t lobby and if you don’t keep knocking on the door, you won’t get anything,” says Lee.
Drugs for rare diseases are expensive because of the high cost of development and the relative rarity of these diseases. For these drugs to be more cost-effective, there needs to be health-financing models for rare diseases.
Other areas that need to be looked into are newborn screening programmes for rare diseases which can prevent severe learning disabilities and improve long-term outcomes for patients and their families.