[Reporter’s View] Rare disease drugs flock to 100-day listing pilot program

More pharmaceutical companies than expected have applied for the government’s fast-track pilot program aimed at listing rare disease treatments for National Health Insurance reimbursement coverage within 100 days.

A total of 14 rare disease treatments from Korean and multinational pharmaceutical companies were submitted for the recently closed pilot program. The government had initially planned to select five products. With applications reaching nearly three times the number of available slots, the program appears to have generated strong interest even before its launch.

Under the program, the cost-effectiveness assessment normally conducted before reimbursement listing will be shifted to a post-listing evaluation, while procedures related to drug pricing and expenditure caps will be substantially streamlined based on a price benchmark of around 90% of the lowest adjusted price in the A8 countries. Reimbursement procedures will also begin before regulatory approval, with the goal of completing final National Health Insurance listing within 100 days of approval. The plan is intended to cut the listing process, which can currently take up to 240 days, by more than half.

What deserves attention, however, is not simply that 14 products were submitted, but why pharmaceutical companies were so eager to participate.

The conditions were not entirely favorable to drugmakers either. Five years after early reimbursement listing, treatment outcomes will be reassessed based on real-world data (RWD), with the possibility of a price reduction or a switch to full out-of-pocket payment depending on the results. Companies are also required to submit a “patient treatment continuity assurance plan,” a new requirement not included in the existing reimbursement listing system. In other words, companies gain faster market access in exchange for taking on considerable uncertainty.

Nevertheless, 14 products were submitted. This suggests that demand for faster reimbursement access is substantial. At the same time, it also underscores just how long pharmaceutical companies and patients have had to endure the “wait” under the existing reimbursement process.

This is not the first attempt to shorten the reimbursement listing period for new drugs. The government previously introduced a regulatory approval-assessment-price negotiation linkage program, under which regulatory approval, reimbursement eligibility assessment and price negotiations proceed in parallel, with the goal of shortening a process that could take more than 300 days to around 150 days.

In practice, however, the process has not moved as quickly as intended. The first pilot program, launched in 2023, took about two years to complete, with some drugs taking more than a year to secure reimbursement coverage. The second pilot, launched in December 2024, has not been markedly different. Fintepla, the first drug to enter price negotiations, passed the Drug Reimbursement Evaluation Committee only about a year and a half after being selected for the pilot.

Winrevair, a treatment for pulmonary arterial hypertension, was also included in the second pilot of the linkage program, but prolonged delays in the reimbursement process prompted a patient advocacy group in March to publicly question the program’s effectiveness. In other words, there was a gap between the program’s stated goal of “fast-track” access and the point at which patients could actually receive the drug with reimbursement.

Of course, the new 100-day fast-track listing pilot program is not structured in the same way as the existing approval-assessment-negotiation linkage program. While the latter seeks to shorten timelines by running the existing approval, assessment and negotiation procedures in parallel, the new program goes further by substantially reducing pre-listing requirements themselves, including deferring the cost-effectiveness assessment until after listing.

This can be seen as a stronger signal of the government’s determination to address reimbursement delays. On the other hand, given the gap between target timelines and actual processing times under previous fast-track listing initiatives, the success of this program will ultimately be judged by its results rather than its design.

It is therefore too early to judge the program’s success based on the 14 applications alone. What matters is whether the selected treatments actually obtain National Health Insurance coverage within the timeframe promised by the government.

For patients with rare diseases, the time required to obtain reimbursement coverage is more than an administrative processing period. Even after a treatment receives regulatory approval, its high cost can restrict actual access to treatment until reimbursement coverage becomes available. The government’s push for faster listing is ultimately intended to narrow this gap between regulatory approval and actual treatment access.

The unexpectedly high number of applications for the 100-day fast-track listing program reflects strong expectations for the initiative. At the same time, the figure also highlights the “time delays” that the existing reimbursement system has failed to resolve promptly.

The 14 applications may be a measure of the program’s popularity, but they are not a measure of its success. This time, the true test of the “fast-track listing” program should be not its name, but how long patients actually have to wait before gaining access to treatment.