Rare disease patients suffer more due to the 'rarity' of their condition. Due to the small number of affected patients, even drugs that are already available cannot be easily listed for reimbursement due to difficulty in demonstrating cost-effectiveness or predicting their fiscal impact on the NHI budget.

Rare diseases are diseases that affect fewer than 20,000 people or those for which the number of affected patients cannot be estimated due to difficulties in diagnosis. As rare diseases are difficult to diagnose and treat and have a significant impact on the life expectancy of the patients, it is imperative to ensure patient access to the treatments. However, due to the small number of affected pati