Presentation (CTI)

OBJECTIVES: To characterize how Taiwan's National Health Insurance manages access for high-cost spinal muscular atrophy (SMA) therapies and to describe how uncertainty is addressed across one-time gene therapy versus chronic disease-modifying treatments within a lifecycle reimbursement context.
METHODS: A descriptive policy review was conducted using publicly available Taiwan's National Health Insurance reimbursement price tables, rule-revision documents, and HTA-related materials for nusinersen, risdiplam, and onasemnogene abeparvovec (2020-2026). Each reimbursement listing or revision was defined as a decision episode. Data were systematically extracted from publicly available sources using a predefined coding framework informed by managed entry agreement (MEA) and HTA uncertainty domains. Extracted domains included eligibility criteria, financial controls, outcome monitoring, stopping and continuation rules, switching restrictions, and registry requirements. A structured comparison across these domains was used to describe differences in access control mechanisms over time across therapy types.
RESULTS: Across decision episodes, differences in managed-access approaches were observed across therapy types. For one-time gene therapy (onasemnogene abeparvovec), reimbursement was characterized by stringent eligibility and exclusion criteria, standardized post-treatment assessments, milestone-based monitoring, and long-term follow-up requirements. In contrast, chronic therapies (nusinersen and risdiplam) underwent multiple revisions that progressively expanded eligibility while maintaining requirements for periodic functional reassessment, stopping rules, and restricted switching. Reimbursement revisions for nusinersen and risdiplam were accompanied by price adjustments, occurring alongside of financial and utilization-related controls. These observations suggests differences in how access controls are applied across therapy types over the treatment lifecycle.
CONCLUSIONS: Taiwan's SMA reimbursement framework applies different managed-access mechanisms across therapy types. Access to gene therapy is primarily controlled through eligibility criteria and post-treatment follow-up, whereas chronic therapies are managed through periodic reassessment, switching restrictions, and price adjustments. These findings describe variation in access control approaches that may be relevant for planning evidence development and reimbursement for high-cost therapies in single-payer settings.