College of Pharmacy, Sookmyung Women's University, Seoul 04310, Korea.
Department of Insurance Benefits, National Health Insurance, Wonju 26464, Korea.
Int J Environ Res Public Health. 2020 Apr 26;17(9):2991. doi: 10.3390/ijerph17092991.
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.
本研究旨在通过分析孤儿药的认定、审批、报销和药品支出情况，了解韩国孤儿药的可及性及其对药品预算的影响。我们分析了 2007 年至 2019 年长期真实世界数据中孤儿药认定、审批和报销的数据集。被认定和批准的孤儿药分别为 165 种和 156 种，其中 156 种获批产品中有 88 种获得了报销。孤儿药支出逐年增加，在 2018 年占药品总支出的 1.44%左右。孤儿药的人均支出平均每年增长 8.7%。孤儿药的年平均费用为 27,000-47,000 美元，最高值为 260,000-560,000 美元。由于批准后仍有许多孤儿药尚未可报销，应制定考虑孤儿药特点的报销政策。由于孤儿药支出的快速增加可能对保险预算构成潜在威胁，因此还应考虑预算管理。总之，在提高孤儿药可及性的同时，有必要采取先发制人的措施，以有效管理医疗保险预算。
