To alleviate the financial burden on patients, the National Health Insurance Administration (NHIA) consistently implements reforms to the NHI payment system for relevant drugs. As part of this effort, the Center for Health Policy and Technology Assessment was established to strengthen the NHI payment policy by incorporating scientific evidence and values. Additionally, provisional listing was introduced to expedite the inclusion of new drugs and technologies into the NHI program.

Recently, the rare disease drugs that have been approved for NHI reimbursement include the following :

1. Crysvita injections for pediatric patients with X-linked hypophosphatemic disorder, over the age of one year and whose growth plates have not yet closed, have been included in the NHI payment program starting on May 1st. Compared to conventional treatment (i.e., phosphate oral solution with calcitriol), the new drug is capable of restoring renal tubules' ability to reabsorb phosphate and increasing the concentration of calcitriol in the blood. Crysvita injection treatment costs approximately NT$5.78 million per patient per year. The new policy is estimated to benefit 20 to 32 patients in the first 5 years, with a budget ranging from NT$110 million to NT$180 million.
2. Takhzyro injections, containing lanadelumab, used for preventing attacks of hereditary angioedema in patients over the age of 12, have been included in the NHI payment program starting on March 1st. The Takhzyro injection treatment costs approximately NT$7.97 million per patient per year. Provisional listing is applied, and treatment effectiveness is continuously tracked to serve as a reference for future evaluations of the payment policy. The new policy is estimated to benefit 2 to 7 patients in the first 5 years, with a budget ranging from NT$16 million to NT$36 million.