Presentation (CTI)

OBJECTIVES: To improve timely access to innovative drugs for life-threatening or rare diseases, South Korea introduced an approval-evaluation-negotiation linkage pilot program in 2023 that allows regulatory approval, reimbursement evaluation, and price negotiation to proceed in parallel. This study evaluated whether the pilot shortened the time to reimbursement.
METHODS: Five drugs selected for the first and second rounds of the pilot were analyzed. For each drug, the time from selection for the pilot to regulatory approval and National Health Insurance reimbursement listing was calculated. Drug characteristics, including therapeutic area and disease type, were analyzed.
RESULTS: The first-round pilot included Qarziba (dinutuximab beta), an anticancer agent for high-risk neuroblastoma, and Bylvay (odevixibat), a treatment for progressive familial intrahepatic cholestasis. The second-round pilot included Rimqarto (tislelizumab), an anticancer agent for relapsed or refractory diffuse large B-cell lymphoma, Fintepla (fenfluramine) for Dravet syndrome, and Winrevair (sotatercept) for pulmonary arterial hypertension. Both first-round drugs achieved reimbursement. The overall time from pilot selection to reimbursement listing was 17.5 months for Qarziba (12.0 months from selection to regulatory approval and 5.5 months from approval to reimbursement listing) and 27.6 months for Bylvay (14.1 and 13.5 months, respectively). Although Qarziba was listed rapidly after approval, its overall selection-to-listing interval did not show a significant reduction compared with previously reported timelines. As of the reference date, none of the second-round drugs, selected in December 2024, had been reimbursed.
CONCLUSIONS: The approval-evaluation-negotiation linkage pilot did not consistently shorten reimbursement timelines for the selected drugs. The 100-day expedited listing pathway for rare disease treatments, which the government plans to introduce to address the limitations of the pilot, may help improve timely patient access.