– We have decided that Spinraza can be used for the treatment of adult patients with spinal muscular atrophy from 15 May, with certain criteria, as we have had for use in children, says Terje Rootwelt, director of Helse Sør-Aust to news. The decision-making forum for new methods, which determines what medicines patients should have access to, was actually supposed to have made a new assessment of the medicine Spinraza at a meeting at the end of April, but at Easter they decided to set up an extraordinary meeting on Tuesday about the medicine. Facts about SMA and Spinraza The disease spinal muscular atrophy (SMA) belongs to a group of diseases characterized by the destruction of the motor cells in the spinal cord. It is the most common fatal and hereditary disease in children in our part of the world. Spinraza (nusinersen) from the company Biogen is the only medicine that can treat children with the very serious disease. The drug increases the production of a protein that enables many of the children to regain mobility. In the first year, the child needs six doses and then three injections annually. On 12 February 2018, the Decision-Making Forum for New Methods decided that people under the age of 18 should receive the medicine under a number of conditions. Preliminary findings from a new study from the medical faculty at Stanford University in the US show that the medicine also has an effect on adults. The patient association SMA Norge has demanded that the decision-making forum for new methods reverse the decision and offer the medicine to adults as well. A legal assessment from the Lippestad law firm concludes that the decision on 12 February was unlawful. Decision Forum chose to send the case to Bestillerforum, which on Monday 11 June chose to stop the case. As announced, SMA Norge is now suing the state because the decision is considered unlawful. (Sources: NTB, Decision-making forum for new methods, Advokatfirmaet Lippestad) New agreement Patients with the disease spinal muscular atrophy (SMA) have for several years been waiting for a clarification as to whether they can receive the life-saving medicine, which slows down the muscle disease. In 2018, the Decision Forum made an assessment. It was then decided that they would give priority to young people under 18. The reason for this was partly that they had insufficient documentation for adult patients, and partly that the price of the medicine was too high. This is the process when new medicines are to be introduced in Norway: The Government has proposed, and the Storting has adopted, frameworks for which medicines are to be approved in Norway. Frameworks have been set for both the budget and frameworks for how the medicines are to be distributed between different patient groups. This is laid down in Storting message no. 34: Values in the patient’s health service — Message on prioritization. Nye Metoder is a prioritization tool that will ensure that the framework is followed. This involves negotiating the price, as Sykehusinnjøp does, and then Method Assessment looks at whether the medicine fulfills the medical criteria. If the new medicine costs too much to satisfy the priority criteria, a negotiation is carried out between the hospitals at Sykehusinnkjøp and the medicine companies. The criteria for prioritization are how useful the treatment is, how much it costs and how serious the disease is. Finally, a forum, the Decision Forum, which is put together by the directors of the four health regions, looks at whether the drug should be approved and who should receive it. There are also some observers (including the Norwegian Medicines Agency and the Directorate of Health), present in this process. It is the various drug companies that are responsible for submitting the necessary documentation that the drug works as expected. Now the Decision Forum has come to an agreement with the manufacturer of the medicine, which means that they have now chosen to give the green light also for adults, says Rootwelt. – It is an alternative price agreement with criteria for start-up and possible termination. We will follow up the effect of the treatment by having the patients join a national register, so that we can see how the effect develops over time.
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Decision-making forum considered the medicine Spinraza for muscular dystrophy in an extra meeting – news Norway – Overview of news from different parts of the country
