Lian-Yu Chen, M.D.,Ph.D.
Director-General
National Health Insurance Administration, Taiwan

With the rapid development of regenerative medicine, innovative therapies are gradually transforming disease treatment paradigms. To address high-cost therapies associated with significant uncertainty, Taiwan’s National Health Insurance (NHI) has established corresponding reimbursement and management mechanisms.

Cell and Gene Therapy: Opening a New Era of Medicine

In recent years, cell and gene therapies have made breakthroughs in disease treatment. Compared to traditional treatments by medicinal products, these therapies offer one-time treatment or limited treatment regimens with long-term efficacy, bringing new hope for patients. The Regenerative Medicine Act and the Regenerative Medicine Products Act were enacted in January 2026 comprising the legal basis for establishing a comprehensive system for clinical applications and product life cycle management. These acts ensure medical safety and quality while laying the foundation for the subsequent inclusion of innovative therapies into the NHI evaluations. However, these products are typically personalized, involving complex manufacturing processes and high costs. Therefore, balancing patient accessibility with the sustainability of Taiwan’s NHI system has become a critical issue.

From CAR-T to Gene Therapy: Expanding NHI Coverage for Breakthrough Therapies

As of 2023, the CAR-T therapy tisagenlecleucel, indicated for certain relapsed or refractory hematologic cancers, has been reimbursed under the “Provisional Payment”. This represents the first cell therapy reimbursed under Taiwan’s NHI and marks a milestone in personalized precision medicine. Meanwhile, a case registration system has been established for data collection. Assessments of efficacy and other outcomes will be conducted after a period of reimbursement. Taiwan’s NHI will evaluate three years of real-world data and clinical evidence to determine whether the therapy should be incorporated into regular reimbursement scheme. Additionally, only certified healthcare institutions meeting quality management requirements are authorized to provide the therapy, ensuring treatment effectiveness and patient safety.

In the same year, onasemnogene abeparvovec became the first gene therapy product covered by NHI for the treatment of spinal muscular atrophy (SMA), a rare disease. A single intravenous infusion can provide long-term therapeutic benefits; however, the cost of a single treatment course is extremely high. To alleviate the financial burden, the Taiwan’s NHIA has signed an “Outcome-based Installment Payment Agreement” with the manufacturer. Real-world data will be collected through the case registration system, and if the therapeutic effect does not meet expectations, payments will be halted.

The gene therapy eladocagene exuparvovec, indicated for the rare disease aromatic L-amino acid decarboxylase (AADC) deficiency, was also included under Taiwan’s NHI Provisional Payment in 2025, with efficacy to be evaluated after three years. This one-time gene therapy is designed to improve neural transmission, offering new therapeutic opportunities for affected children.

The Era of High-Cost Innovative Therapies: Balancing Innovation and Sustainability of NHI

In recent years, an increasing number of cell and gene therapies have been evaluated for NHI reimbursement, including treatments for hematologic cancers, hemophilia, and rare diseases. Given their high-cost nature—often ranging from tens of millions to over a hundred million New Taiwan Dollars per treatment—reimbursement decisions must carefully weigh clinical efficacy, safety, ethics, cost-effectiveness, patient populations, and financial impact. Strategies such as the Provisional Payment, Managed Entry Agreements (MEA), and real-world data collection have been implemented. Taiwan’s NHI will continue to establish sustainable reimbursement models aligned with precision medicine to ensure patient well-being.

Lian-Yu Chen, M.D.,Ph.D.
Director-General
National Health Insurance Administration, Taiwan