A Review of Real-World Evidence (RWE)-Based Reimbursement Management Systems for High-Cost Medicines for Rare and Serious Diseases in Major Countries

As novel therapies for rare and serious diseases increasingly receive expedited regulatory approval based on limited clinical evidence, health technology assessment bodies face growing uncertainty regarding clinical effectiveness and cost-effectiveness.This uncertainty poses substantial risks to patient safety and healthcare budgets sustainability.Many countries have introduced reimbursement management frameworks incorporateing real-world evidence (RWE), whereas Korea remains predominantly reliant on financial risk-sharing, with limited outcomebased post-listing surveillance.This study conducted a narrative literature review of reimbursement management systems for high-cost medicines in six countries: the United Kingdom (NICE), the Netherlands (ZIN), France (HAS), Germany (IQWiG), Italy (AIFA), and Canada (CDA-AMC).The analysis focused on coverage with evidence development and conditional reimbursement approaches, examining eligibility criteria, RWE generation procedures, data collection infrastructure, outcome utilization, and cost-sharing mechanisms.All six countries combine conditional access with post-listing evidence generation and reassessment, though legal frameworks differ.The United Kingdom and France provide temporary access through dedicated funds or early access programs, whereas the Netherlands, Germany, Italy, and Canada grant reimbursement subject to post-listing reassessment.Data collection periods typically span 2-3 years.Common features include predefined evaluation criteria, substantial cost-sharing by pharmaceutical companies, and public or publicly coordinated data infrastructures.Outcome-linked policy actions include price adjustments, modifications to reimbursement conditions, and, patient-level financial clawbacks.These findings suggest that major countries have institutionalized RWEinformed reimbursement management through structured evaluation frameworks, systematic data collection, and outcome-linked mechanisms.Korea should therefore establish a legal basis for RWE-linked reimbursement reassessment and develop a public real-world data platform.Pre-listing agreements should standardize data collection protocols and result utilization while ensuring continued patient access following reimbursement adjustments.