[Desk’s View] Rare disease pilot needs participation, not hesitation

Participation is essential if the pilot is to evolve into a workable permanent system

Fast-tracking the reimbursement listing process by reducing the review period for new drugs deemed necessary for patients. This is an objective few would oppose. That said, previous initiatives of this kind have consistently faced questions over their effectiveness.

In many cases, difficulties in demonstrating cost-effectiveness or prolonged review timelines meant that products were rarely listed within the target timeframe. Against this backdrop, the government has introduced a far more ambitious proposal.

The pilot program for the ‘expedited reimbursement of rare disease therapies’ aims to complete listing within 100 days. More significant than the timeline itself is the mechanism: both cost-effectiveness assessments and price negotiations will be waived, while eligible products will be listed upfront at 90% of the lowest adjusted A8 reference price without negotiations over projected budget impact.

Eligible medicines must treat diseases covered under Korea's Critical Illness Special Calculation program, excluding rare cancers. Products should already be approved, under regulatory review, or preparing for submission, and must already be reimbursed in at least three of the eight A8 reference countries. Applications will be accepted through August, with the pilot scheduled to begin in October.

By waiving pharmacoeconomic evaluation and adopting an upfront listing model similar to the waived products, the pilot effectively embraces the "list first, evaluate later" approach that the pharmaceutical industry has long advocated.

Despite achieving this long-sought goal, the industry reaction is lukewarm. The industry's greatest concern is in generating evidence for the post-listing reassessment. The government plans to establish real-world registries to generate real-world evidence (RWE) and reassess products after 5 years. Depending on the results, reimbursement status could remain unchanged, the price could be reduced, or patients could be required to pay the full cost.

With debate over the validity of RWE itself yet to be fully resolved, such reassessments could, in effect, cause a product to lose reimbursement status after 5 years. From the perspective of multinational pharmaceutical companies, this could mean that the Korean government officially labels one of their products as ‘ineffective.’

Another new requirement is the mandatory submission of a ‘patient treatment continuity assurance plan,’ a document that has never before existed in Korea's reimbursement system. With no detailed guidance yet available, this unknown requirement is adding to companies' hesitation. Because the pilot targets rare disease therapies, whose patients generally have longer life expectancies than cancer patients, concerns also remain over expenditure caps.

The industry needs to show courage. It was the one that had been persistently asking the government to "Open the door first and tighten the rules later." Bold reforms inevitably come with risks. A pilot program is, by definition, a testing ground that lays the ground for a permanent system. Pharmaceutical companies need to actively participate and voice their opinions to establish a stable framework for the final program. The government, too, is concerned that the Trump administration's most-favored-nation (MFN) pricing policy could affect access to innovative medicines. The rising awarness of the need for a change has triggered the pilot. Such a long-awaited project should not be left to wither away in calculated neglect."