A rare disease is any disease that affects a small percentage of the population and are frequently debilitating to patients. Timely access to the right treatment is crucial for effective disease management and delayed access contributes to poorer treatment outcomes and more severe disease complications. This policy paper “Improving access to orphan drugs in Malaysia” explores the challenges associated with accessing orphan drugs in Malaysia and proposes ways in which we can improve patient access to these drugs.

We explore the current situation, which includes difficulty in receiving diagnosis and obtaining treatment in time due to the lack of expertise along with limited treatment options. Orphan drugs are harder to obtain than other conventional drugs due to lack of economic incentive for manufacturers to develop drugs which can be attributed to the small market. This leads to orphan drugs being marketed at very high prices due to an absence of economies of scale in its sale. Low prevalence of the disease within healthcare settings and population in general results in a lack of understanding among physicians. The low prevalence makes it difficult to improve knowledge on the safety and efficacy of treatments through proper studies.

The paper explores different solutions to address these problems through two broader themes: controlling the high price of drugs and increasing the budget for orphan drugs. The solutions proposed under these themes are, controlling high prices of drugs and increasing the budget for orphan drugs.