Fast-track listed rare disease drugs need to demonstrate performance

HIRA official presents operational plan at the Ministry of Health and Welfare public hearing

Annual data collection required from years 1 to 3…failure to submit may result in reimbursement removal

Drug prices to be set at around 90% of the lowest A8 country price…subject to expenditure cap agreements

The government will lower drug prices starting in the fourth year if rare disease treatments fail to meet their targets following post-marketing evaluation, following fast-track listing.

Evaluations will be based on real-world evidence (RWE), but if pharmaceutical companies fail to collect and submit the required data, the drugs may be removed from reimbursement coverage altogether.

Sook-hyun Lee, Director of New Pharmaceutical Benefits Listing Division at the Health Insurance Review and Assessment Service (HIRA)

On the 27th, Sook-hyun Lee, Director of the New Drug Listing Division at the Health Insurance Review and Assessment Service (HIRA), explained specific operational plans for the system during a public hearing on “Fast-Track Listing Strategies to Improve Access to Rare Disease Treatments.”

Until now, rare disease treatments underwent a 150-day approval negotiation process at HIRA and a 60-day process at the National Health Insurance Service (NHIS) following approval by the Ministry of Food and Drug Safety (MFDS) before official notification by the Ministry of Health and Welfare.

Under the revised drug pricing system, the review periods at both HIRA and the NHIS will be shortened to one month each. Instead, post-listing management will be strengthened through follow-up evaluations after reimbursement listing.

A general scope for drugs eligible for the fast-track listing pilot program has also been established. Drugs will be selected based on: ▲availability of alternative treatments ▲severity of the disease ▲need for rapid introduction ▲fiscal impact. The pilot project plans to begin by selecting two to three items based on these criteria.

Director Lee explained, “The primary criteria for selecting target drugs are treatments for rare diseases that have already received approval or are listed in at least three of the A8 countries.”

A performance-based evaluation model will be operated using real-world evidence (RWE) and linked to electronic medical records (EMR). Before reimbursement, pharmaceutical companies must submit clinical outcome evaluation plans for review by HIRA. The plan must include details such as ▲target patients and drugs ▲outcome measures ▲evaluation cycle and duration ▲reimbursement adjustment plan ▲methods for calculating clinical performance evaluation results ▲data collection procedures and quality control methods.

Afterward, pharmaceutical companies will be required to collect annual data during years one through three, and evaluations will be conducted based on those results. The findings will then be reported to the Drug Reimbursement Evaluation Committee.

Data collection and validation will continue for three years after fast-track listing, and reimbursement adjustments will begin in year four according to evaluation outcomes. Data collection and verification will be conducted for three years following fast-track listing, and reimbursement will be adjusted starting in the fourth year based on the evaluation results.

Lee stated, “If pre-established targets are not achieved, drug prices will be reduced, and if data are not submitted, we are reviewing measures to convert reimbursement to full out-of-pocket payment.” Obligations related to post-evaluation plans and follow-up measures based on results will also be included in fast-track reimbursement contracts.

The National Health Insurance Service will apply expenditure cap agreements to fast-track listed drugs. Negotiations will be conducted based on total expenditure figures proposed by pharmaceutical companies, and after one year of listing, actual claims will serve as the benchmark.

The expenditure cap mechanism is expected to prevent rapid increases in financial burden caused by the expansion of fast-track reimbursement listings.

Pricing standards are expected to be stricter than those applied to drugs exempt from pharmacoeconomic evaluations. Drug prices are expected to be negotiated at around 90% of the lowest price among the A8 countries.