Abstract
This study aimed to identify orphan drug accessibility and impact on pharmaceutical budgets in South Korea by analyzing the status of orphan drug designation, approval, reimbursement, and pharmaceutical expenditure. We analyzed the dataset on orphan drugs designated, approved, and reimbursed from 2007 to 2019 based on long-term real-world data. The designated and approved orphan drugs were 165 and 156, respectively, and 88 out of 156 approved products were reimbursed. Total expenditure on orphan drugs increased annually to account for about 1.44% of total pharmaceutical expenditure in 2018. Orphan drug expenditure per patient increased on average by 8.7% per year. The average annual cost of orphan drugs was USD 27,000–USD 47,000, with the maximum value of USD 260,000–USD 560,000. As there are a number of orphan drugs that have not yet been reimbursable after approval, a reimbursement policy should be established that considers the characteristics of orphan drugs. Since the rapid increase in orphan drug expenditure can be a potential threat to the insurance budget, budget management should also be considered. In conclusion, it is necessary to take preemptive measures to manage the health insurance budget efficiently while improving patient accessibility to orphan drugs.
Highlights
With improvements in medical and diagnostic technology, approximately 6000 to 8000 rare diseases have been identified, and the number of patients diagnosed with rare diseases has rapidly increased [1,2]
Of the 165 designated products, there may have been products that had not applied for a New Drug Application (NDA), and some products that were designated before 2007 may have been approved afterwards
Some of the 156 approved products may have not applied for reimbursement
Summary
With improvements in medical and diagnostic technology, approximately 6000 to 8000 rare diseases have been identified, and the number of patients diagnosed with rare diseases has rapidly increased [1,2]. Unmet needs for therapeutics are increasing as most rare diseases shorten the patient’s life expectancy by damaging the body and reducing the quality of life (QOL) [2,3]. The identification of many rare diseases and development of biotechnology promote research and development (R&D) and marketing approval of ODs [2,4,7,8]. Most rare diseases are characterized by high severity, low prevalence, and no alternative treatments, making it difficult to prove the safety and efficacy of ODs as it requires a longer time and higher R&D cost for clinical trials [9]. Due to high price and the small number of patients, pharmaceutical expenditure per patient is very high, and the total budget impact of individual ODs is relatively low [5,6,10,11]. Public Health 2020, 17, 2991; doi:10.3390/ijerph17092991 www.mdpi.com/journal/ijerph
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
