– I know that from a socio-economic point of view I should have had an abortion. Ola Schrøder Røyset tells how he feels that it is finances that determine whether he can get a medicine that can slow down the muscle wasting in his fragile body. Because while the state negotiates a price, the disease continues to make him worse. He has no time to wait. He knows that his life has a deadline. – I have lost some speech function, breathing capacity and I have almost completely stopped taking food by mouth. Weakness in the swallowing muscles makes swallowing risky. In addition, the strength in the fingers, which control the electric wheelchair, has become weaker. Ola Schrøder Røyset runs her own company and has several employees. He is waiting for medicine that can slow down the disease. Photo: Trond Stenersen / news Ola is 29 years old and head of SMA Norway, an organization for patients with the disease Spinal muscular atrophy. Although the disease has hit him hard, Ola is 100% at work. He owns his own company with several employees. For 2 1/2 years, he and 60 other adult patients have been waiting for price negotiations between the pharmaceutical companies and the state to be completed. In the meantime, the patients get worse: – When you don’t get either a yes or a no, you find yourself in a limbo, because you don’t know what to plan for. Ola really just wants an answer. 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, Decisions forum for new methods, Advokatfirmaet Lippestad) Because the knowledge that there is a medicine out there that can help is hard to live with. Nevertheless; he tries to have a positive mindset. – On the one hand, I am extremely happy that it exists. For anyone who was under 18 in 2018, that’s fantastic. I know that no one else in Norway is going to be like me, and have the same course of illness as me. It’s a wonderful feeling. Because that means that the experiences I have stop here, says Ola. Photo: Trond Stenersen / news 2018 was the last time there was a discussion about whether medicine should be given to patients. At that time, young people under the age of 18 received the medicine, partly because the price was high and that there was not good enough documentation at the time for adults. Documentation now shows that the medication can slow down development, also in adult patients. See fact box. Children were prioritized in 2018 In 2018, it was decided that young people under the age of 18 should receive the medicine, after long negotiations with pharmaceutical companies and media reports. According to the Decision-Making Forum for new methods, which are the ones who decide which medicine patients will receive, there was not enough good documentation for adult patients. In 2020, Biogen, which manufactures the drug Spinraza, reported to the Decision-Making Forum for new methods that the medicine can also have an effect on adult patients. But even now the price was too high, and the Norwegian Medicines Agency said that they would not assess a cost-benefit effect until the price had been negotiated down. Later, the pharmaceutical company Roche also developed a medicine for SMA patients, Evrysdi. Here, the Norwegian pharmaceutical company has drawn up a report, but says that the price is too high. According to the Decision-making forum for new methods (see answer further down in the case), negotiations are still ongoing about the price of the medicines. Is afraid of not lifting the children In the 5 years since 2018, it has become visible what the disease is doing to her body. In 2018, the mother of two, Line Nordahl, was able to go. Now she sits in a wheelchair, and is terrified that the muscles in her arms will also weaken so that she can no longer hold the children in her arms. Line Nordahl with the children on her arm in the wheelchair. She still has the strength in her arms to hold the children when she picks them up from the nursery. Photo: Øyvind Berge Sæbjørnsen / news – It does something to the self-esteem not to be the same mother, girlfriend and daughter. Not contributing in the same way as others. You feel less valuable. The mobile contains the memories from when she could walk, when her eldest daughter was small in 2018. Photo: Øyvind Berge Sæbjørnsen / news She is also waiting for an answer. – You feel that you are a bit on hold, that you don’t know what the future looks like. It is natural for me to think that since I was able to walk 5 years ago, I would have continued to walk if I had received the medicine then. Cuddling from mom after a day at kindergarten. Photo: Øyvind Berge Særbjørnsen / news Too much money for too little effect Today there are two medicines that are effective against spinal muscular atrophy, Spinraza and Evrysdi. The Norwegian Medicines Agency prepares a so-called method report, which shows whether medicines have a cost-benefit effect in relation to the price of the medicine. It is then up to the Decision Forum for New Methods to decide whether the medicine should be given to patients or not. The factors that the Norwegian Medicines Agency assesses are whether the medicine is effective, how serious the disease is, and the price of the medicine. – These medicines will have an effect, also on adult patients. What we don’t believe is that it will make the patients healthier, so the goal is to stabilize the disease, says acting area manager of the Norwegian Medicines Agency, Einar Andreassen. – How important is it to start as early as possible? – Since it can help to stabilize the disease, it is beneficial to start before the disease develops. For all patients, both adults and children, early treatment is beneficial. Einar Andresen of the Norwegian Medicines Agency says the price is too high compared to the effect of the medicine. Photo: Knesia Novikova / news The price the pharmaceutical company Roche has set for Evrysdi is NOK 2.7 million per year, shows the method assessment from the Norwegian Medicines Agency. In January, Evrysdi was introduced for young people under the age of 18 at a secret lower price. This is what the Norwegian Medicines Agency has taken as a starting point when they say that it is still too high for adult patients, says Andreassen: – We have assessed that patients can gain up to several quality-adjusted life years, and that is a good result, but it also costs a lot for the hospitals. It seems to be more than what the hospitals usually accept, says Andreassen. – So the price is too high in relation to the effect it has? – The price is clearly too high. – Then your assessment is that it will not pay off from a socio-economic perspective? – Because you could treat so many more with the same effect, for the same money. The price of Spinraza, which Biogen manufactures, is not known. Still negotiating Sykehusinnkjøp AS is negotiating with the pharmaceutical companies on behalf of the Decision Forum for new methods. They do not want to comment, and refer to the Decision Forum. They are the ones who decide which medicine patients should have access to. – We really want to introduce this medicine, and therefore everyone wants the ongoing process to be completed as soon as possible. But it must end with a result where the cost is consistent with the effect it has, says Terje Rootwelt, director of Health South-East. Terje Rootwelt says they have a good dialogue with the pharmaceutical companies, and hopes the process of negotiations will soon be finished. Photo: Knesia Novikova / news He says they have a good dialogue with the pharmaceutical companies. At the same time, he says that they must constantly make assessments based on criteria that the Storting has determined, namely the severity of the illness, the benefits of treatment and the price. – We fully understand that patients and their families are frustrated that this is taking time. We have to deal with the fact that we have to make a thorough assessment of the effect and price of the medicine, 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. The pharmaceutical companies Both Biogen and Roche say the dialogue with the Decision-Making Forum for new methods is good and that negotiations are still ongoing. – We at Roche are pleased that children and young people living with spinal muscular atrophy were given access to Evrysdi earlier this year. Adults living with this disease, on the other hand, have no offer of disease-modifying treatment in Norway today, writes communications manager Erlend Engh Brekke at Roche Norge AS to news. He confirms that adult patients with SMA have a different potential for health benefits than is the case for children and young people, because the disease has progressed further. For adults, it is about stabilizing the disease and preventing further loss of function. It is about maintaining the quality of life for people with SMA and their relatives. In Norway, children were given access to Evrysdi in January this year, and they have been negotiating with Sykehusinnkjøp since April about the medicine for adult patients. – We know that there are many people waiting for Evrysdi to become available, and we are doing everything we can from our side to make this process go as quickly as possible. Biogen says that they feel that the dialogue with Sykehusinnkjøp is good and hope that the medicine will soon be introduced, also for adult patients. They have been in dialogue with Sykehusinnkjøp since 2020 about whether adults should also receive Spinaza, which is currently given to young people under the age of 18.
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60 SMA patients are waiting for Spinraza and Evrysdi. Price negotiations are still ongoing. – news Norway – Overview of news from different parts of the country
