Two treatments for children with rare diseases -- Qarziba (dinutuximab) and Bylvay (odevixibat) -- have been selected as targets for the “approval- evaluation-negotiation linkage system,” a pilot program to shorten reimbursement screening for expensive drugs.
According to materials obtained by the healthcare journalists’ group covering the Ministry of Health and Welfare, the ministry recently sent a letter to related organizations, including the Health Insurance Review and Assessment Service (HIRA), which contained the results of the selection of drugs and other related matters for the pilot project.
Recordati’s Qarziba treats high-risk blastoma in children 12 months and older. Ipsen's Bylvay is indicated for treating cholestatic ichthyosis in children 12 months and older with alkaptonuria.
The ministry said it chose the drugs for the pilot program by comprehensively considering the diseases’ severity, alternative drugs’ presence, treatment effectiveness, and expert opinions.
The pilot project begins when the pharmaceutical company selling the drug applies for marketing authorization to the Ministry of Food and Drug Safety (MFDS). The company must then apply to MFDS for the target drug's GIFT (Global Innovative Products on Fast Track) designation.
“We have requested pharmaceutical companies to prepare for the GIFT designation process and submit related materials quickly,” the health and welfare ministry said. “We will convene a working group council to discuss the pilot project implementation in July or August.”
The MFDS’ approval, HIRA’s evaluation, and negotiations with the National Health Insurance Service (NHIS) will be conducted at the same level as standard new drug assessments.
The health-welfare ministry will also survey demand for the second half-year to select the second batch of targets. Drugs reviewed in the first demand survey may also be selected for the second round.
The approval-reimbursement-assessment linkage is a system that allows a new drug to apply for reimbursement if it proves safety and efficacy even before the official marketing authorization by MFDS to speed the use of new products.
Eligible drugs should treat a life-threatening condition with a life expectancy of less than one year or a rare disease for which no existing treatment exists or are clinically meaningful in efficacy over existing treatments.
The health-welfare ministry reviewed about 10 products from domestic and foreign pharmaceutical companies that met these criteria for selection based on a demand survey and selected the two products.