Taiwan's National Health Insurance Administration (NHIA) has decided to cover an expensive but effective gene therapy for a rare disease aromatic L-amino acid decarboxylase (AADC) deficiency.
According to a report by FocusTaiwan, the one-time therapy, which costs about NT$100m ($3.28m) per injection, will be covered on a temporary basis. It has, however, sparked a national debate over fairness and treatment access for patients with other rare conditions.
The NHIA has said that the effectiveness of the new gene therapy will be reassessed three years after treatment begins, and coverage terms may be adjusted as needed.
AADC deficiency affects infants and young children, causing severe developmental delays and autonomic dysfunction. Without treatment, many patients die between the ages of 2 and 5. The therapy, however, can significantly improve motor function and quality of life. The treatment was developed by National Taiwan University Hospital (NTUH) in 2007, with clinical protocols established in 2010, before being licensed to a US company.
NHIA estimates 13 patients will receive the therapy in its first year at a cost of about NT$1.3bn. From the second through fifth years, five additional patients are expected to be treated annually, costing roughly NT$500m per year.
The decision has sparked criticism from physicians, including pulmonologists. A leading pulmonologist Su Yi-fong said that drugs for other rare diseases treat dozens of patients for less than NT$100m yet are not fully covered.
Supporters counter that the AADC therapy offers life-saving benefits and meaningful developmental gains. A leading hospitalist said the investment could help position Taiwan as a key site for Asian genetic data, supporting future research collaborations and negotiations with international pharmaceutical companies.
Among the 31 patients who received the new therapy in a trial, however, 80% were able to sit up, and among those treated early with intensive rehabilitation, 40% were able to walk independently.