BANGKOK: Only 60% of rare disease patients in Malaysia are receiving treatment, says the head of the Genetics and Metabolism Unit at Universiti Malaya’s Paediatrics Department.
Dr Tae Sok Kun said in Malaysia, based on the findings of a recent study conducted by the School of Pharmaceutical Sciences at Universiti Sains Malaysia and Universiti Malaya Medical Centre, only 60% of rare disease patients were receiving treatment.
“For those rare disease patients who are diagnosed, funding for treatment remains a main obstacle as payment is contingent on charities, public and industrial subsidies, or out-of-pocket payments by patients,” she said.
Speaking during the second South-East Asia Rare Disease Summit 2024 here recently, Dr Tae said the inadequate clinical expertise, and lack of awareness, facilities for diagnosis and treatment of rare diseases, as well as high treatment costs and limited pathways for reimbursement, remained prevalent in Malaysia.
“The Rare Disease Summit highlighted the necessity for different stakeholders to come together to discuss tangible steps that can ensure rare disease communities in South-East Asia enjoy equitable and sustainable access to medicines.
“No single company or organisation can do this alone. It is important for all of us in society to create opportunities to broaden access to medicines for rare diseases patients to ensure that no one is left behind in Malaysia,” Dr Tae said, Bernama reported.
In South-East Asia, an estimated 45 million people or 9% of the region’s population suffer from a rare disease.
Meanwhile, the summit also saw five regional rare diseases patient advocacy groups – the Malaysia Rare Disorders Society, Thai Rare Disease Foundation, Philippines Society for Orphan Disorders, Indonesian Rare Disease Foundation and Rare Disorders Society (Singapore) – coming together in the Asean Rare Disease Consortium (ARdC).
Malaysia was represented by Rare Disorders Society president Nadiah Hanim Abdul Latif.
To address the challenges faced by the rare disease communities across the region, the ARdC will work with patient advocacy groups, top healthcare professionals and other key stakeholders to encourage best practices around patient support, resources, peer-to-peer exchanges, experiences around fundraising programmes and policy making.
The ARdC will also collaborate and hold discussions, including with governments to ensure sustainable funding for the reimbursement of medicines and treatments.
