The National Health Care Institute advises the Minister of Health, Welfare and Sport (VWS) to reimburse the medicine risdiplam for the treatment of the hereditary muscle disease Spinal Muscular Atrophy (SMA) from the basic health insurance package. This is subject to the condition that only children and young people up to the age of 25 are eligible for reimbursement, because risdiplam has proven added value for them. Furthermore, the price has to be significantly reduced in negotiations with the manufacturer.
Effective in children and young adults
The drug risdiplam (Evrysdi®) has been proven effective in the treatment of children from 2 months to 25 years with the muscle disease SMA. These are people with type 1, 2 or 3 or people with 1 to 4 copies of the SMN2 gene who do not yet have symptoms of SMA. Treatment with the drug increases the chances of survival for young patients and significantly improves the movement and strength of the muscles. Patients must take risdiplam throughout their lives.
There is still insufficient evidence for the effectiveness of the drug for patients older than 25 years. The Zorginstituut will make an effort, together with the parties involved, to make risdiplam available to these patients through conditional admission.
Third drug for SMA
Risdiplam is the third drug to treat people with SMA. Onasemnogenic abeparvovec (Zolgensma®) is a single gene therapy. This therapy can be used in babies who have been diagnosed with SMA through the heel prick screening and in children with SMA type 1. Nusinersen (Spinraza®) is an SMA drug that is administered with an epidural. Because patients sometimes suffer from a deformity in the back, nusinersen cannot be applied to everyone. For them, treatment with risdiplam could be an option.
Price way too high
The cost of lifelong treatment with risdiplam is €4 to 7 million per SMA patient. The Zorginstituut concludes that the price is far too high to qualify for reimbursement from the basic package. This is partly due to uncertainties about the long-term treatment effects and because 2 other medicines are already available for SMA patients. That is why the Zorginstituut advises the Minister of Health, Welfare and Sport to only include risdiplam in the basic package if the price falls sharply in negotiations with the manufacturer. As a result, more other new, innovative medicines can be reimbursed from the basic health insurance package. And there remains sufficient financial scope to keep the existing good care from the basic package accessible to everyone in the Netherlands.
Over SMA
Every year 15 to 20 children are born with SMA in the Netherlands. About half of them develop the most severe form of this disease (type 1). These patients usually die before the second year of life without treatment. People with SMA have a deficiency of SMN proteins that are necessary for the nerves in the spinal cord to work due to a hereditary defect. As a result, their muscles die and patients are less and less able to move. Risdiplam increases the production of functional and stable SMN protein.
About National Health Care Institute
Everyone who lives or works in the Netherlands is obliged to have health insurance. The National Health Care Institute is a government organization whose main task is to compile the basic package of health insurance. We make complicated, but necessary choices to ensure that everyone in the Netherlands can receive good care. Now and in the future.