Novartis’ Zolgensma (onasemnogene abeparvovec), the world’s most expensive drug for the treatment of spinal muscular atrophy (SMA), will become reimbursable in August, the government has announced.
The government has set the maximum reimbursement price for the treatment at 2 billion won ($1.52 million), but out-of-pocket expenses will be only 5.98 million won ($4,558) with nationwide coverage. health insurance.
Zolgensma is the only gene replacement therapy to treat SMA with a single intravenous injection.
SMA is a rare disease that causes loss of muscle movement and motor neurons due to a mutation in the survival motor neuron 1 (SMN1) gene.
Infants with SMA type 1 who show symptoms within six months have an average life expectancy of 13 months. However, the disease is the number one killer of babies aged two or younger, as respiratory failure occurs before the age of two.
The Ministry of Health and Welfare on Wednesday approved reimbursement for Zolgensma at a meeting of the Health Insurance Policy Review Committee. In addition, it revised the list of reimbursed drugs and the maximum reimbursement rates.
According to the review, Zolgensma’s maximum refund rate is 2 billion won. Since the treatment is a one-time injection with an ultra-expensive price, the government said it would apply a risk-sharing agreement (RSA) to monitor the therapeutic effects and manage health insurance funding in a stable manner.
Zolgensma will be provided under the RSA in three types – a ‘reimbursement’ type where the pharmaceutical company reimburses a certain percentage of the claim amount to the National Health Insurance Service (NHIS); a “spending cap” type in which the excess amount is reimbursed when the cap exceeds a certain amount; and a ‘use cap/fixed cost per patient’ type where the drug manufacturer reimburses a certain percentage of the amount in the event of treatment failure.
In the first year of reimbursement, the government said 14 patients, including those treated with Biogen’s Spinraza (nusinersen), will receive treatment with Zolgensma. Among them, seven newly diagnosed SMA patients, and the remaining seven received Spinraza.
“In the first year, about 27.7 billion won had to be spent on the Zolgensma administration. But under the RSA, in the reimbursement, spending cap and usage cap types, spending will be less,” the Department of Health and Social Care said.
The ministry also said it would reassess the clinical utility and cost-effectiveness of Zolgensma to change the drug’s price and reimbursement rate in the fourth year of reimbursement.
A guardian of an SMA patient scheduled to receive administration of Zolgensma must consent to a five-year follow-up for regular assessments of response. Patients must also undergo a screening to determine if they meet Zolgensma’s reimbursement criteria prior to administration.
The government noted that if a patient receives Zolgensma and additional SMA treatment, the additional treatment will not be reimbursed.