Rare disease drugs to be reimbursed within 100 days

MOHW, MFDS, and KDCA announce measures to strengthen support for rare and intractable diseases

Over 10 ultra-low-demand rare drugs to be switched to emergency import status drugs every year

Government, medical community, and pharmaceutical companies to establish a made-to-order manufacturing system

Starting in the new year, the government will shorten the timeline for National Health Insurance (NHI) reimbursement listing of rare disease treatments from the current 240 days to 100 days, strengthening patient access to medicines.

To address frequent shortages of essential medicines, the government proposed ‘activating made-to-order manufacturing’ by establishing a public production and distribution network through collaboration between the government, pharmaceutical/distribution/medical associations, and pharmaceutical companies.

Under this model, the government requests production of frequently out-of-stock drugs from manufacturers and purchases the entire output for supply.

By expanding the emergency import of treatments and contract manufacturing, the government aims to create an environment where patients can obtain treatments without difficulty, even if private supply ceases due to low demand.

In addition, patient co-insurance payments under the NHI special reimbursement calculation scheme for rare and severe intractable diseases will be further reduced.

On the 5th, the Ministry of Health and Welfare (MOHW), the Ministry of Food and Drug Safety (MFDS), and other relevant agencies jointly announced measures to strengthen support for rare and severe intractable diseases. 

Rare Drugs to be listed within 100 days… approval-evaluation-pricing parallel review to continue

From the new year, the government will significantly reduce the time required for NHI listing of rare disease treatments from within 240 days to within 100 days.

Furthermore, the government will continue its pilot program for ‘parallel approval-evaluation-price negotiation’ for rare disease drugs, where it is difficult to establish robust efficacy and safety evidence due to extremely small patient populations. This program reduces the time required for drug price approval and listing from the current 330 days to 150 days, a reduction of 180 days.

The first pilot program included Qarziba and Bylvay Cap, while the second pilot program is currently underway with three selected drugs.

Prices for rare disease treatments will be set at a certain level relative to the average price in reference countries.

Expansion of emergency import and made-to-order manufacturing for rare essential and shortage drugs

Furthermore, to ensure access to treatments even if pharmaceutical companies halt manufacturing or imports due to low demand, the government is expanding emergency imports and custom manufacturing.

First, medications for self-treatment that patients previously had to purchase directly overseas will be converted into emergency import items for at least 10 products starting this year to stabilize supply.

Emergency import refers to a system in which the government directly procures drugs from overseas and supplies them when the domestic supply is disrupted.

While an emergency import system already exists through the Korea Orphan and Essential Drug Center, ultra-low-demand products have often been excluded. Expanding the scope of emergency imports aims to address these limitations.

If an emergency import drug was previously eligible for reimbursement, reimbursement price applications will be prioritized, and existing emergency import drugs will also be allowed to apply for reimbursement.

In particular, to ensure a stable domestic supply of essential medicines at risk of discontinuation, the government will activate made-to-order manufacturing through a public production and distribution network involving the government, pharmaceutical companies, distributors, medical associations, and industry groups.

Last year, KRW 810 million was allocated to manufacture 7 drugs through made-to-order manufacturing. This year, an additional KRW 500 million will be allocated to expand production to two more drugs.

Under this system, the government shares information on drugs scheduled for supply discontinuation with the pharmaceutical industry. The Korea Orphan and Essential Drug Center analyzes prescription and supply history, demand, and regulatory issues, after which pharmaceutical companies decide whether to proceed based on production intent and required budget. Public production projects are then implemented through product transfer, new approvals, or manufacturing contracts.

The government plan is to expand from the current 7 items to 17 items by 2030, adding 2 items annually starting this year.

This would mean 25% of the 40 essential drugs requested for emergency import by the medical field would be converted to public production.

When expanding emergency import and made-to-order-manufactured items, priority will be given to treatments for rare diseases.

Reduced patient co-insurance costs through enhanced special reimbursement support

To reduce the financial burden of high-cost care for patients with severe diseases, the government will strengthen special reimbursement schemes that lower NHI co-insurance payment rates.

For high medical expenses related to rare and severe intractable diseases, the patient's co-insurance payment rate will be further reduced from the current 10%.

Taking into account factors such as the need for continuous treatment and management and the burden of high medical costs, a reduction plan will be prepared in the first half of this year, followed by approval by the Health Insurance Policy Deliberation Committee, with implementation scheduled for the second half of the year.

Furthermore, starting this January, 70 additional diseases, including congenital functional short bowel syndrome, will be added to the list of rare diseases eligible for special billing exceptions, with continuous expansion planned.

The re-registration process for rare and intractable diseases will also be reorganized to be more patient-centerd. Until now, the government required separate test results for 312 specific rare and intractable diseases upon re-registration.

Reflecting field feedback that additional testing is unnecessary given the incurable nature of these conditions, unnecessary testing requirements will be eliminated during re-registration.

Support for low-income patients through the rare disease medical expense assistance program will also be expanded.

The income and asset criteria separately applied to households with dependent family members will be phased out starting in 2027, expanding support for low-income individuals.

Customized medical nutrition support based on disease-specific needs will continue to expand. The government currently provides special formula milk and low-protein instant rice to rare disease patients requiring dietary management. Since September last year, it has additionally provided special corn starch for patients with glycogen storage disease.

This year, based on a survey of the current status of special diet usage and additional demand, the government plans to review expanding the range of supported items and support the development of new products.