South Korea

The Korea Orphan & Essential Drug Center is the government-established body that imports and supplies orphan drugs and national essential drugs that are not commercially distributed in Korea. It supplies both clinical sites (emergency introduction) and individual patients (self-treatment drugs), and is for many rare-disease patients the only lawful supply route.

Approval alone does not make a drug affordable. Korea runs a positive list system: the Health Insurance Review and Assessment Service assesses clinical utility and cost-effectiveness, then the National Health Insurance Service negotiates price. Orphan and anticancer drugs may use risk-sharing agreements, and drugs for severe conditions with no alternative may be waived from submitting a pharmacoeconomic dossier.

Standard route to market. The Ministry of Food and Drug Safety grants marketing authorisation, and separately designates orphan drugs (희귀의약품). Designation requires a domestic patient population of 20,000 or fewer, or an equivalent unmet-need ground, and the Korea Orphan & Essential Drug Center issues the designation recommendation that supports the application.

Permits use of an unapproved investigational drug for treatment purposes where a patient has a life-threatening or seriously debilitating condition and no alternative therapy is available. MFDS operates two tracks: approval for an individual patient, and approval covering multiple patients.

Route for an individual patient to import a drug approved abroad but not in Korea. KODC reviews the application and issues an import-requirement exemption recommendation, which clears the consignment through customs. Processing takes 7 working days and there is no fee.