– 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 and Evrysdi from Roche are the only medicines 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) Pop the champagne One of those who is happy about today’s decision is Torstein Lerhol. He is head of department at Medvind Assistance. There was a lot of cheer when it became known that even adults can get medicine. – I’m at work and we’ve popped the champagne! It has been a five-year desert walk, and this is a seven-mile walk. We are a bit put out, and today it is for joy. Almost no matter what the criteria are, this is a saying, he says. Torstein Lerhol celebrated the decision in Beslutningsforum on Instagram on Tuesday. Facsimile: Instagram Lerhol has also received a phone call from his sister, who has the same diagnosis. – She is crying with joy. We have seen for five years how well it works on my niece. This is a big day for our entire environment, he says. Positive Ola Schrøder Røyset, who heads SMA Norway, which is the organization for patients with spinal muscular atrophy, also thinks there is reason to be happy today. – We are very positive that this has finally been decided. He thinks it is good that the same criteria are used for those over 18 as those used for those under 18 five years ago. Head of SMA Norway, Ola Schrøder Røyset. Photo: Trond Stenersen / news – We also hope that this innovative agreement with Spinraza can be used for other drugs. He points out that we pay for the effect the drug has, and that there are objective criteria that can be measured. Don’t dare to cheer Ane Berentsen is also happy that she can now get medicine that can slow down her illness, even if she doesn’t want to dare to cheer just yet. – I don’t dare to have too high hopes. The medicine has different effects on people. The most important thing for me is that I don’t get worse with age. That I don’t lose the ability to move my arms and don’t get weaker and weaker. She works as a dramaturg at Fyllingsdalen theater in Bergen. The medicine can enable her to continue at work. – I could risk that eventually I would have to have someone to dictate to. I am very happy that I can probably continue to be independent, she says to news. New agreement Adult patients with the disease spinal muscular atrophy (SMA) have been waiting for several years for clarification. In 2018, the Decision Forum agreed to give the medicine to young people under the age of 18. They thought the documentation for use by adults was too poor and weak. Director Terje Rootwelt in Health South-Aust. Photo: Knesia Novikova / news Now, new studies and negotiations with one of the manufacturers who make medicine have meant that the Decision Forum can also give the green light to 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. The agreement applies to the medicine Spinraza from the company Biogen. The negotiations with Roche, which manufactures the medicine Evrysdi, are ongoing, says Rootwelt. 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. First to give to adults There are currently around 70 adults who are qualified to start treatment with medicine. But Rootwelt say they do not know how many of these will accept the offer they are now receiving. He says the Decision Forum had to spend time ensuring that it was safe to give the medicine to adults as well. – There is a wholesome assessment behind this decision. We need to know that the treatment is safe and effective. The documented effect for adults is weaker than for children, but at group level we see that many patients either stabilize their function, and some even get a little better. At the same time, he believes that Norway is far ahead in the approval of this type of medicine. – None of our neighboring countries have introduced this medicine for adults. In Denmark, they have just said no.
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Decision forum says yes to Spinraza for muscular dystrophy in extra meeting – news Norway – Overview of news from different parts of the country
