Presentation (CTI)

OBJECTIVES: In 2023, Korea enacted a pharmacoeconomic evaluation (PE) exemption pathway for pediatric rare disease drugs. This study compares reimbursement age criteria set under this pathway with pivotal trial age ranges, labeled indications, and age criteria from overseas Health Technology Assessment (HTA) agencies.
METHODS: Evaluation documents from the Health Insurance Review and Assessment Service (HIRA) Drug Reimbursement Evaluation Committee (January 2023-November 2025) were reviewed to identify drugs reimbursed under this pathway. For each drug, reimbursement age criteria, pivotal trial age ranges, and labeled indications were extracted. Criteria were compared with those of the National Institute for Health and Care Excellence (NICE) and HTA agencies from Australia, Canada, and Scotland.
RESULTS: Of 104 HIRA drug appraisals, four (3.8%) were reimbursed under the pathway: burosumab, selumetinib, maralixibat, and odevixibat. Trial-population-based: burosumab covered ages 1-12 years, matching its pivotal trial population, with continuation beyond age 12 requiring prior-authorization review of bone growth; selumetinib covered ages 3-18 years with discontinuation at 19, mirroring its pivotal trial. Label-indication-based: maralixibat and odevixibat were reimbursed from 3 months of age - below their pivotal trial lower bounds - reflecting the Korea label range. For burosumab, Korea applied a strict age ceiling while other agencies were age-agnostic or used clinical milestones. For selumetinib, Korea aligned with overseas agencies on treatment-initiation age; however, unlike NICE - which permitted initiation beyond age 19 - Korea requires objective justification to continue treatment past 19, whereas other agencies impose no such requirement.
CONCLUSIONS: Under Korea's PE exemption pathway for pediatric rare disease drugs, four therapies received reimbursement. Given the characteristics of pediatric therapies, age criteria were incorporated into reimbursement conditions in certain cases, using either pivotal trial populations or labeled indications. Korea applied relatively stricter upper age limits and treatment continuation requirements than overseas HTA agencies, highlighting the need for explicit and consistent age criteria principles.