New drugs eligible for approval and decision by June 2025
Three cancer therapies and six rare disease treatments seek expedited listing
Nine pharmaceutical products have applied for inclusion in the second pilot project by South Korea's Ministry of Food and Drug Safety (MFDS), which integrates the approval, evaluation, and negotiation processes of the Health Insurance Review and Assessment Service and the National Health Insurance Service.
According to industry sources, the Ministry of Health and Welfare (MOHW) concluded its recruitment for the second phase of the pilot project at the end of August. The project aims to streamline the inclusion of drugs that meet the following criteria:
● Ability to apply for approval and determination by June 2025.
●Significant efficacy in treating life-threatening or rare diseases, with no available alternative treatments.
● Clinically meaningful improvements compared to existing treatments.
● Eligibility for expedited listing by the MFDS.
Companies submitted detailed information on anticipated approval dates, efficacy, consistency with domestic standards, and the severity of conditions treated. The nine products proposed include treatments for small cell lung cancer, pulmonary arterial hypertension, ALS (Lou Gehrig’s disease), hemophilia A, HER2-positive cholangiocarcinoma, Dravet syndrome, diffuse large B-cell lymphoma, non-small cell lung cancer, and Duchenne muscular dystrophy.
In the first pilot project, nine products were also submitted. These included enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a hemophilia A treatment, and a therapy for acquired thrombotic thrombocytopenic purpura (aTTP). From these, the MOHW selected Qarziba (dinutuximab beta) from Recordati Korea and Bylvay (odevixibat) from Ipsen Korea as target drugs in June last year. However, neither has been included in the national reimbursement scheme to date.
Qarziba, a treatment for neuroblastoma, is under supplementary review after an initial non-reimbursable evaluation by the Drug Reimbursement Evaluation Committee.Bylvay, a treatment for pruritus in patients with the rare genetic disorder progressive familial intrahepatic cholestasis, received approval just last week.
With the reimbursement registration process for these prior drugs progressing slower than expected, there is growing interest in which drugs will be selected for the second pilot project.
Since January of last year, the government has implemented a combined evaluation-negotiation process, reducing the time required for reimbursement registration from 210 days to 150 days for drugs treating life-threatening diseases. The ongoing pilot project further links the MFDS's approval process with evaluation and negotiation efforts.