Taiwan NHI to cover costly gene therapy for rare disease

Taipei, Aug. 21 (CNA) Taiwan's National Health Insurance Administration (NHIA) announced Thursday that gene therapy for the rare genetic disorder aromatic l-amino acid decarboxylase (AADC) deficiency will be covered under the National Health Insurance (NHI) program.

Coverage for the treatment, which costs more than NT$100 million (US$3.28 million), is expected to begin as early as November, after the coverage review was approved, NHIA Deputy Director-General Parng I-ming (龐一鳴) told reporters.

AADC deficiency results in severe developmental delay, oculogyric crisis and dysfunction of the autonomic system, afflicting about one out of every 30,000 newborns in Taiwan, according to relevant research.

According to Health Promotion Administration data, 72 patients in Taiwan have been diagnosed with the disease as of July 31, 2025, with 27 having died before June 30.

The NHIA predicts that about 13 patients will receive the treatment within one year of coverage being extended.

The treatment is administered as a one-time injection, according to Hwu Wuh-liang (胡務亮), attending physician in the Department of Medical Genetics and Pediatrics at National Taiwan University Hospital, who was part of the hospital's development team for the treatment.

Children with AADC deficiency typically begin showing symptoms between the ages of 1 and 2 and, if untreated often die between the ages of 2 and 5.

However, with treatment most patients go on to live normal lives, the oldest observed patient being over 10 years old, Hwu said.

Parng said the therapy will be the most expensive drug covered by the NHI so far.

The agency will negotiate prices with pharmaceutical companies and pay in installments, he said.

Considering the treatment's high price, the agency is looking to gradually pay for the treatments after confirming the patient condition has improved and stabilized after treatment, Parng said.