The Taiwan Foundation for Rare Disorders (TFRD) urges the NHIA to speed up the access to orphan drugs.  The NHIA mentioned in its response that the agency is currently in the process of evaluating 9 orphan drugs, including a gene therapy for SMA.  Up to now, the NHI has not yet included any gene therapy for rare diseases; therefore, this assessment posts a new challenge to the NHIA. 

Drugs for treating rare diseases are also known as “orphan drugs” because manufacturers encounter obstacles in drug development due to small patient populations and high R&D costs.  Therefore, orphan drugs are usually too expensive for most families.
According to a survey on rare diseases, 90% of the public expressed their support for better healthcare for rare diseases.  According to the TFRD, at present, there are 23 orphan drugs still waiting in the pipeline for the NHI listing.  The TFRD urges the government to speed up the process.

Chang Hui-Ping of the NHIA said that one of the mentioned 23 orphan drugs has actually been listed in the NHI Benefit Scheme since March.  It is a capsule for multiple sclerosis.  This new drug provides an alternative to existing NHI-reimbursed treatments.  It will benefit about 170 patients, said Chang.

In addition, another 3 of the 23 orphan drugs have already passed the assessment of the NHI Drug Dispensing Items and Fee Schedule Joint Establishment Meeting. They are treatments for SMA, Pompe disease and familial amyloid polyneuropathy.   Chang Hui-Ping expressed that the NHI is now negotiating with the drug companies to conclude a supply contract.  If everything goes well, these drugs are expected to be available by the end of the first half of this year.

There are still 9 orphan drugs in the evaluation process, including a gene therapy for SMA.  If approved, it would be the first NHI-reimbursed gene therapy for rare diseases. 

For the rest of the 23 orphan drugs, 5 cases were withdrawn by the manufacturers and 4 manufacturers have not yet submitted the applications.   Chang Hui-Ping pointed out that as the trial scale is usually small, the assessment of the effectiveness and safety of orphan drugs poses a challenge to the NHIA, not to mention the financial burden from the rising price of orphan drugs. 

The MOHW revised the drug reimbursement standards last year to make the reimbursement conditions more flexible, for example, including the concept of risk-sharing.  The NHIA hopes this will increase this year’s NHI new drug budget execution rate to 100%.

The TFRD urges the NHIA to address the urgent medical needs of patients with rare diseases by accelerating and expanding the reimbursement of orphan drugs.

【2023-03-02 / Central News Agent】