Abstract
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.
Highlights
Research and development (R&D) on orphan drugs (ODs) for rare diseases has become an active field of research owing to the rapid advancement of biotechnology
OD, pharmacoeconomic evaluation (PE) OD, and weighted average price (WAP) OD) based on the results evaluated by the Drug Reimbursement
This study examined the change in the post-listing price of 48 new ODs listed between
Summary
Research and development (R&D) on orphan drugs (ODs) for rare diseases has become an active field of research owing to the rapid advancement of biotechnology. Most rare diseases are genetic in origin, and those who suffer from these diseases occasionally experience symptoms soon after birth or in their childhood. In some cases, these diseases have serious consequences that threaten patients’ lives [2,3,4]. These diseases have serious consequences that threaten patients’ lives [2,3,4] In this regard, the development of new ODs can bring about significantly positive changes in patients’ quality of life. The pricing and reimbursement (P&R) processes of ODs are perceived as the final stage for market access, as well as a crucial issue in terms of patient access to medical treatment [9,10]. That is, when new ODs are not reimbursed, patients are unlikely to use these
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