Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison

This study aimed to analyze four current pathways affecting the listing and post-listing prices of new orphan drugs (ODs) in South Korea. These mechanisms were: (1) essential OD, (2) pharmacoeconomic evaluation (PE) waiver OD, (3) weighted average price OD, and (4) PE OD. We analyzed the ratio of the listing price of 48 new ODs to the average adjusted price (AAP) of seven advanced countries and examined the change in the post-listing price. Descriptive statistics were used to analyze the listing and post-listing price changes. The mean and median ratios of the listing price of total new OD to AAP were calculated to be 69.4% and 65.4%, respectively. Essential OD showed the highest mean (93.8%) and median (80.8%) ratios. The mean cumulative price discount rate of the new OD was 7.2% in the third year and 5.7% in the fifth year. The rarity of diseases impacts the listing price of OD, but the political effects of the benefits of OD on the post-listing price of these drugs could not be verified. Further research should be conducted to develop measures that facilitate the practical sharing of budget risks and increase patient access to new ODs.