TAIPEI (TVBS News) — Taiwan's National Health Insurance Administration (NHIA, 中央健康保險署), the government agency overseeing the island's universal healthcare system, will cover a gene therapy for rare disease AADC deficiency priced at NT$100 million (around US$3.2 million) per dose. The decision sparked debates over social fairness given the treatment's extraordinary cost. NHIA Director Lian-Yu Chen (陳亮妤) stated Tuesday (Dec. 2) that the coverage followed a fair and transparent review process.
The NHIA will negotiate installment payments and reimbursement agreements with the manufacturer given the drug's high cost. The agency plans to evaluate the treatment's effectiveness over three years and reassess payment and coverage conditions. Infants with AADC deficiency, a rare genetic disorder, exhibit severe developmental delays and oculogyric crises by 3 to 4 months old, with potential fatal complications in childhood.
Meeting records reveal that 24 months after beginning AADC gene therapy, 50% of patients gained full head control, 38.9% sat independently, and 11.1% stood with support. Clinical trials showed children aged 2 to 6 who received treatment have a higher likelihood of sitting independently compared to those beginning treatment from ages 6 to 12. The NHIA will prioritize coverage for severe cases in the younger age group based on these findings.
The manufacturer applied for coverage in February 2024, and the NHIA, Taiwan's national health insurance agency, granted formal approval in August 2024. Dr. Wuh-Liang Hwu (胡務亮) from National Taiwan University Hospital (台大醫院), one of Taiwan's leading medical centers, developed the gene therapy for AADC deficiency in 2007 and later transferred the technology to a U.S. pharmaceutical company that now manufactures the treatment.