On August 21, 2025, the National Health Insurance Administration (NHIA) approved coverage for eladocagene exuparvovec, a gene therapy for severe cases of Aromatic L-amino acid decarboxylase (AADC) deficiency. Each patient is eligible for a one-time lifetime treatment at a reimbursement price of NT$99.99 million per dose, available to patients aged 18 months to under 6 years. The therapy is expected to benefit 13 patients in the first year and about 5 patients annually from the second to fifth year, with projected expenditures of NT$13 billion in the first year and NT$5 billion annually thereafter. Costs will be covered by the earmarked budget for “Rare Disease, Hemophilia Drugs, and Special Medical Devices for Rare Diseases,” ensuring no impact on the rights of other patients. NHIA will also negotiate phased payment arrangements with the manufacturer under a price-volume agreement.

The incidence of AADC deficiency in Taiwanese newborns is about 1 in 30,000—higher than in Japan, the US, and the EU. Developed with NTU Hospital, the drug is licensed in the US, EU, and Taiwan. Without gene therapy, children have a median survival of only 7 years and lack basic motor functions, while also suffering from frequent oculogyric crises and poor respiratory function, creating heavy burdens for families. Since 2010, 34 patients at NTU Hospital have received gene therapy, with follow-up up to 15 years. After 5 years, over 70% could sit independently, showed improved breathing, and had reduced oculogyric crises, marking a major therapeutic breakthrough.

Considering the high risks of intracranial injection, the NHIA has set strict requirements for qualified hospitals and surgeons. Each case must first be reviewed and approved by the NHIA before applying for special case-by-case reimbursement. Only patients deemed eligible may receive the therapy. The NHIA will negotiate supply arrangements with the manufacturer and, once an agreement is reached, coverage is expected to take effect on December 1 this year.

Gene therapy represents a major breakthrough in medical technology, offering unprecedented hope for patients with rare diseases. The NHIA will continue to assess and introduce emerging therapies with proven clinical benefits, ensuring through rigorous review and resource allocation that all citizens share the benefits of medical advances on a foundation of equity and sustainability.