The national drug formulary listing process for orphan drugs in South Korea: narrative review focused on pricing and reimbursement pathways

ABSTRACT Introduction: In addition to some countries facing challenges regarding patients with rare diseases, it has also become an important international topic. Through the efforts of many nations around the world toward providing benefits for the development of orphan drugs to improve the right to treatment of patients with rare diseases, various orphan drugs have been launched in the market. However, controversial issues such as impact on budgets due to high prices and concerns about the uncertainty of clinical outcomes are being raised. For this review, the official websites of government agencies related to the Korean national health insurance were investigated. Areas covered: This paper reviews key factors that should be considered in the process of development, regulation, and market access of orphan drugs in South Korea with a particular focus on the pricing and reimbursement review process. Expert opinion: It is necessary to understand the pricing and reimbursement system of orphan drugs in terms of regulations and market access from the perspective of stakeholders, namely research and development companies, patients, policymakers, and physicians. Stakeholder discussions regarding value-based pricing methodologies such as new outcome-based ris-sharing agreement schemes and multi–criteria decision analysis should be conducted more prospectively.