Patients with rare diseases suffer due to the rarity of their disease. Even when treatments are available, the drugs have difficulty receiving reimbursement as it is difficult to prove cost-effectiveness and predict financial expenditures for the drugs due to the small number of patients.

The government is well aware of such difficulties. This is why it has attempted to improve access to rare disease treatments through various systemic improvements.

However, the situation has not improved much. Among the 6,000 to 7,000 rare diseases known worldwide, only about 6% have available treatments developed. This is because it is difficult to secure statistical significance of the data due to the sma