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KMID : 1146320140020010035
Journal of Health Technology Assessment
2014 Volume.2 No. 1 p.35 ~ p.42
Methods on Improvement of Accessibility for Rare Disease Treatments: Providers¡¯ Perspective
Lee Ji-Yeon

Cho Yeong-Mi
Kim Sung-Tae
Kim Sung-Ju
Abstract
Objectives: Studies for policy proposal on orphan drug accessibility in Korea have been performed mainly in payers¡¯ perspectives so far. This study aimed to propose an improvement plan of orphan drug accessibility by considering current status and issues of orphan drug reimbursement in providers¡¯ perspectives.

Methods: We investigated publications on the distinctive features of orphan drug and the assessment process of orphan drug in other countries. For current reimbursement status and issues in Korea, Health Insurance Review & Assessment Service assessment documents were reviewed.

Results: The most distinctive characteristics of orphan drugs are rarity, disease severity, absence of alternatives and preference for equity rather than financial efficiency. In other countries, the assessment of orphan drugs tends to be more flexible than other drugs and the consideration of societal preference is emphasized in reimbursement decision. The reimbursement probability of orphan drugs is lower than other drugs in Korea and the main reason of rejection was uncertainty in cost-effectiveness. For improvement of orphan drug accessibility, maintaining consistency in drug assessment, mitigation of essential drug criteria, introduction of other assessment methods and special funding can be considered as solutions in current situation.

Conclusion: Recently Korean government also makes efforts to improve accessibility of orphan drugs by flexible assessment of drug cost-effectiveness considering disease severity. But in providers¡¯ perspectives, the barrier is still quite high and consideration of various institutional methods is essential to accelerate patient access to orphan drugs.
KEYWORD
Orphan drug, Rare disease, Accessibility
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