The world marks Rare Disease Day on Feb 28 annually. Rare diseases affect 4%-6% of the population, and of the 8,000 recorded diseases, only about 200 have pharmaceutical treatment.
Malaysia has taken a step forward and defined rare diseases as life-threatening and/or chronically debilitating rare conditions listed in the Malaysian Rare Disease List. Increased awareness of rare diseases and recognition that they are a national healthcare issue would lead to improved understanding of the issue, clarity of the scope and stakeholders involved, increased access to healthcare resources and, consequently, the availability of crucial life-changing medication.