News item | 6/19/2023 | 1:00 pm
The drug risdiplam (brand name: Evrysdi) will be included in the health insurance package as of 1 July. The Ministry of Health, Welfare and Sport has reached an agreement with the marketing authorization holder about an acceptable price for treatment. The medicine is intended for the treatment of patients with spinal muscular atrophy (SMA). The reimbursement applies to patients who start treatment when they are 25 years or younger. For this group, the Zorginstituut has determined that the treatment meets the current state of science and practice. A decision will be made later on whether the drug also qualifies for conditional admission for the other patients.
The Healthcare Institute advised Minister Ernst Kuipers (Public Health, Welfare and Sport) to negotiate about the drug risdiplam because of its unfavorable cost-effectiveness. This means that the price asked before the negotiations was disproportionate to the treatment effect. The medicine is intended for the treatment of 5q Spinal Muscular Atrophy (SMA) in patients 2 months of age and older. The Zorginstituut concludes that risdiplam only meets the current state of science and practice for patients who start treatment when they are 25 years old or younger. This paves the way for reimbursement subject to conditions via health insurance for these specific patient groups:
- Patients 2 months through 25 years of age, at treatment initiation, with a clinical diagnosis of 5q SMA type 1, type 2 or type 3.
- Patients with a presymptomatic diagnosis of 5q SMA and 1 to 4 copies of the SMN2 gene.
The younger a patient is when the drug is first administered, the greater the chance of a better course of the disease. SMA has now also been included in the heel prick. Risdiplam is the first drug for SMA that can be drunk by the patient, rather than injected.
Minister Ernst Kuipers: “It is good news that there are now 3 medicines available for this terrible condition that are eligible for reimbursement from the basic insurance. That is not only good for patients who have been eagerly awaiting this treatment, but also for the treating physicians.”
Preparation treatment process
Before the advent of the 3 drugs for SMA, patients had no treatment options. The reimbursement of risdiplam is now another step towards the treatment of all patients with SMA. In the Netherlands there is 1 treatment center, the SMA Centrum Nederland in UMC Utrecht, where the best treatment option is determined for each individual patient and together with the patient. This is necessary because of the complex care needs of these patients. The multidisciplinary approach, intensive supervision, deployment of capacity and difficult choices for the individual patient means that several months are needed to get everyone started with treatment at the SMA Center in the Netherlands.
Conditional admission
Minister Kuipers will still make a decision on the candidacy of risdiplam for a conditional admission process for patients older than 25. If this process can be started, this means that the drug will be administered to patients in a research setting. In this way, the Zorginstituut can determine whether the product is also eligible for reimbursement for this group. For patients older than 25 years, this means that they currently do not have reimbursed access to risdiplam. As a result, no reimbursed treatment option is currently available for a small group of patients.
About SMA
Spinal muscular atrophy (SMA) is the collective name for a group of hereditary muscle diseases. The brain transmits signals to the muscles through the nervous system. In SMA, the nerves that are controlled from the spinal cord break down. As a result, the muscles are weakened. SMA is a rare hereditary disease.