The National Health Care Institute advises the Minister of Health, Welfare and Sport (VWS) to reimburse the Libmeldy® gene therapy only under certain conditions from the basic health insurance package. The drug can cure children with the serious metabolic disease MLD. These are children who do not yet have symptoms of the disease. There are no other drugs for MLD yet. But Libmeldy is expensive and the long-term effects are still uncertain.
Libmelda
Libmeldy (atidarsagene autotem cell) is a new gene therapy for certain children with the rare, hereditary metabolic disease metachromatic leukodystrophy (MLD). In the Netherlands, this disease is found in 2 to 3 children per year. Due to a mistake in the genes, they have a reduced protection of the central nervous system and their nerve cells and brains are slowly damaged. Children with MLD develop an intellectual disability and are less and less able to move. Without treatment, children with the most severe form of MLD die within a few years of the onset of symptoms.
Only effective for children with MLD without symptoms
2 scientific studies show that Libmeldy has only been proven effective in children who do not yet have symptoms of MLD. Treatment with Libmeldy can prevent them from deteriorating mentally and physically and eventually dying from the disease. With the one-off gene therapy Libmeldy, genes are introduced into the body that the patient needs. These genes can divide and spread. In this way, the cause of MLD is tackled and children without symptoms can recover from the disease and develop in the same way as healthy children.
Conditions for reimbursement
The results of Libmeldy in the studied children are promising, but it is not yet certain whether the drug also works in the long term. Treatment with Libmeldy costs almost € 2.9 million per patient. That is why the Zorginstituut advises the minister to set the following conditions for reimbursement of the medicine:
- The Ministry of Health, Welfare and Sport makes a pay-for-performance– appointment with the manufacturer. This is a performance incentive, where payment to the manufacturer depends on how well the drug really works in practice. The risk of payment for patients for whom the treatment does not work lies with the manufacturer and not with society. A study into pay-for-performance at Libmeldy, conducted by Utrecht University on behalf of the Zorginstituut, can support making this agreement.
- The Zorginstituut draws up an orphan drug arrangement in consultation with the parties involved. This contains various agreements, including about which patients are eligible for Libmeldy (starting criteria) and about international data collection on the effects of the drug.
- The Ministry of Health, Welfare and Sport is negotiating with the manufacturer about a price reduction.
Different drug admission process for children with symptoms
The scientific studies show that it is important to treat patients before the first symptoms of the disease appear. For children who already have symptoms of the disease, there is insufficient scientific evidence whether treatment with Libmeldy has an effect. However, various parties, including the profession, have indicated that they would also like to have Libmeldy available for the treatment of a number of children with minimal symptoms of MLD. The Zorginstituut will therefore examine with these parties whether Libmeldy can still be made available for this group of children via conditional admission. This is a policy to make new, promising medicines accessible to patients with serious and rare diseases for which no effective treatment is yet available. The condition is that data is collected on the effectiveness of the drug during the term of the temporary authorization. Based on this, it can then be determined whether the drug can be definitively included in the basic health insurance package.
Libmeldy assessed in European collaboration
The Zorginstituut conducted the assessment of Libmeldy together with Belgium and Ireland. Like the Netherlands, these 2 countries are part of the Beneluxa Initiative partnership. The cooperation of these countries facilitates negotiations with pharmaceutical companies on the prices of innovative, but very expensive drugs. The Zorginstituut has previously also assessed other gene therapies in this regard. Including the drug Zolgensma for the treatment of muscle disease SMA (€2 million per patient) and the drug Zynteglo for the treatment of a form of hereditary anemia (€1.6 million per patient).
About National Health Care Institute
Everyone who lives or works in the Netherlands is obliged to have health insurance. Zorginstituut Nederland is a government organization whose main task is to put together 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.