How to increase patients’ access to new drugs without jeopardising the NHI finance?  Facing this dilemma, the NHIA tries to protect patients’ medication rights by regularly collecting the real-world data (RWD) for the evaluation of the efficacy and the cost-effectiveness of the NHI-reimbursed treatments.

Chang Hui-Ping, an NHIA official, pointed out the 3 measures that the NHIA has implemented to protect the medication rights of patients with rare diseases.  Firstly, the NHIA recognizes rare diseases as severe conditions.  Secondly, the NHIA accelerates the reimbursement of treatments for rare diseases by referring to the HTA reports and through expert discussion meetings.

Thirdly, from 2005, the NHIA has been setting a special annual budget ring-fenced for orphan drugs. This special budget increased from NT$5.17 billion in 2016 to NT$7.217 billion in 2020.  Last year, the budget usage rate was 98%.  For an orphan drug, it takes about 10-12 months from the application to the approval of NHI listing.

Chang pointed out that the NHIA wants to use the limited resources effectively, therefore, they usually refer to international treatment guidelines and trends, clinical literatures and expert comments to revise and update the reimbursement regulations for orphan drugs.  From September 2019, orphan drugs can only be prescribed to patients diagnosed with rare diseases.  Hospitals are required to upload the uses of orphan drugs and treatment results to a platform for monitoring and evaluating the cost-effectiveness of treatments. 

According to Chen Gau-Tzu, the Chairperson of the NHI Drug Dispensing Items and Fee Schedule Joint Establishment Meeting, if a drug whose effectiveness is yet to be confirmed has obtained the orphan drug certificate but has not yet obtained the drug license, the NHIA usually reimburses such a drug according to the international drug price or at the cost while taking into account the monthly reimbursement claims.  If the monthly claims are quite small, a bigger mark-up will be offered.   All reimbursement regulations are based on the consensus reached in the expert meeting.

If an orphan drug whose effectiveness is confirmed, the price management regulation is relatively relaxed after it has been listed by the NHI.  The NHIA used to exempt orphan drugs from price adjustment; however, as more and more new drugs become available on the market, the NHIA now subject some expensive orphan drugs to price adjustment.   The approach is lenient, for example once every two years instead of once every year.  Recently, experts suggest including orphan drugs into the scope of treatment agreement between the NHIA and drug manufacturers so that the NHIA could negotiate a better price with the suppliers.

【2021-11-23 / United Daily】