MOHW shortens rare disease drug review from 240 to 100 days

MOHW opens applications for pilot program through August 31

Selected products to receive pricing at 90% of the lowest adjusted A8 price

Five products to be chosen based on availability of alternatives, disease severity, budget impact, post-listing evaluation plans and patient access measures

The Ministry of Health and Welfare (MOHW) is preparing to launch the ‘Pilot program for fast-track listing of rare disease drugs’ designed to improve patient access to treatments for rare diseases by reducing the National Health Insurance reimbursement listing period for rare disease drugs from the current 240 days to as little as 100 days.

The ministry announced on June 30 that it will accept applications from pharmaceutical companies between July 1 and August 31 for the pilot program, which introduces a ‘list first, evaluate later’ approach, to enable patients to receive eligible therapies at the right time.

The core of the pilot program is the deferral of pre-listing assessments and the simplification of reimbursement negotiations. Cost-effectiveness analyses, which are currently mandatory before reimbursement listing, will instead be conducted after listing based on real-world clinical outcomes collected from patients receiving the therapy.

The negotiations over reimbursement price and expected pharmaceutical expenditure, which take the most time, will also be streamlined by applying pre-established contractual terms. Under the pilot program, selected products will receive a reimbursement price set at 90% of the lowest adjusted price among the eight reference countries (A8). The initial projected claims amount will be based on the manufacturer's proposed amount, up to KRW 30 billion. Companies may also opt to use Korea's flexible pricing agreement system, if desired.

Eligibility criteria will be strictly managed. Candidate products must be designated for reimbursement as a rare disease therapy for special exemption of calculation and must either already have marketing authorization or currently be under regulatory review. They must also apply for a reimbursement decision by Dec. 31 of this year. In addition, only products that are publicly reimbursed and have confirmed reimbursement prices in at least three of the eight A8 countries, including the United States, the United Kingdom, Germany and Japan, will qualify for the pilot program.

After the application period closes, the MOHW will comprehensively evaluate candidate products based on factors such as the availability of alternative therapies, disease severity, budget impact, and post-listing evaluation plans before selecting up to 5 products in September. The Health Insurance Review and Assessment Service (HIRA) will facilitate the collection of real-world clinical outcome data for the selected therapies.

Pharmaceutical companies wishing to participate must submit an application form and post-listing evaluation materials by 6:00 p.m. on Aug. 31 via email to the designated HIRA contact ([e-mail]). Additional inquiries regarding pricing and submission requirements may be directed to HIRA's New Pharmaceutical Benefits Listing Division or the National Health Insurance Service's New Drug Management Division.

Byung-ki Kwon, Director-General of the Health Insurance Policy Bureau at the MOHW, said, "This initiative is intended to substantially shorten the reimbursement listing period for rare disease therapies so that patients can gain earlier access to treatment. It is particularly significant because it reflects a long-standing request from patients with rare diseases."