In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which affects no more than 1 in every 2,000 individuals. Throughout the world, clearly, there are different definitions of what a rare disease means. However, there is currently no standard of “rare” in Malaysia, a country located in Southeast Asia.
But according to The Star, Malaysia may soon be transforming its approach to rare disease. Datuk Dr. Noor Azmi Ghazali, the Deputy Minister of Health and MP for Bagan Serai, shared that he – and the Malaysian government – are working to develop a fund designed to help individuals with rare diseases and their families. In particular, the fund would work to provide financial assistance due to the higher cost of treatments, healthcare, and hospitalization associated with these conditions.
Most recently, Datuk Dr. Ghazali provided some funds to the family of Saffiyya Aisyah Sahrul Asyraf Sharuddin, a six-year-old girl who has a rare muscle cancer. While The Star did not define exactly what cancer Saffiyya has, types of rare muscle cancers include soft tissue sarcomas and rhabdomyosarcoma.
Moving forward, Datuk Dr. Ghazali shared that those with rare diseases and their families should call on the government for financial help if they could not afford treatment.
The Rare Disease Landscape in Malaysia
So what does the rare disease landscape look like in Malaysia? According to an article published in PLoS One, the authors explain that:
there are 13 rare disease specialists and a dozen medical doctors in genetic clinics around Malaysia, mainly in public health facilities. From the survey, 1,249 patients were diagnosed with rare diseases in public hospitals.
Additionally, shares the study, only around 60% of those with rare conditions in Malaysia were able to receive the necessary medications or supplements to aid in their treatment. The top three most prevalent rare conditions in Malaysia are believed to be Marfan syndrome, Prader-Willi syndrome (PWS), and osteogenesis imperfecta (OI). Other rare conditions found in Malaysia include Hunter syndrome (MPS II) and MPS IV, Pompe disease, Noonan syndrome, and Leigh syndrome.