Rare Disease Treatment Costs Cut Sharply as Dravet Syndrome Drug Gains Coverage in 126 Days

Beginning Sept. 1, the Ministry of Health and Welfare will expand National Health Insurance coverage for treatments for severe and rare diseases and support the stable supply of drugs designated for supply stabilization.

The Ministry of Health and Welfare said Sept. 1 that it would expand drug reimbursement to reduce treatment costs for patients with rare and intractable diseases and patients with severe cancer, while also supporting the supply of essential drugs at risk of shortages.

National Health Insurance will cover Fintepla oral solution, a treatment for Dravet syndrome, a rare and intractable form of epilepsy that develops within the first year of life and causes severe seizures and developmental delays.

With an estimated 150 patients in South Korea, Dravet syndrome is difficult to treat and has involved high treatment costs because of frequent severe seizures. With the new coverage, the annual medical expense burden per patient will fall from about 100 million won to approximately 10 million won.

The new drug was selected for a pilot program that conducts approval, evaluation and negotiations in parallel, completing insurance coverage in 126 days—about half the 240 days previously required. The Ministry of Food and Drug Safety’s approval, the Health Insurance Review and Assessment Service’s reimbursement evaluation, and the National Health Insurance Service’s drug-price negotiations were conducted concurrently.

Polivy, a new first-line treatment for diffuse large B-cell lymphoma, for which no new treatment regimen had been available for an extended period, will also newly receive reimbursement coverage. It will provide a new treatment option for patients with malignant blood cancers caused by abnormal enlargement and rapid proliferation of B cells while reducing the burden of high drug costs.

Coverage will be expanded for Blincyto injection, a treatment for precursor B-cell acute lymphoblastic leukemia. Until now, insurance coverage was available only when the cancer had relapsed or existing treatments had failed to work. Going forward, patients will receive coverage beginning in the early stage immediately after initial chemotherapy, when residual minimal cancer cells are removed.

The incidence of this indication is high in children younger than 5 years.

The government will continue supporting the stable supply of drugs essential for patient treatment but at risk of production or importation discontinuation because of low profitability. Chugai Protamine 6% injection, essential for regulating blood pressure and stabilizing blood circulation in newborns and premature infants, will be newly designated as a drug subject to supply stabilization, and a system for compensating production costs will be established.

Tuberculin PPD AJV injection, a tuberculosis diagnostic reagent for which on-site supply disruptions were anticipated because of higher import prices, will have its increased import price promptly reflected in the drug price this year, addressing concerns about supply instability in advance.

Yoo Ju-heon, director general of health insurance policy, said, "We will continue to pursue the rapid application of National Health Insurance coverage for treatments for severe and rare diseases while actively supporting the stable supply of drugs essential for medical care."