The case in summary: Serenne Vikebø (22), who has the rare muscle disease spinal muscular atrophy (SMA), has experienced significant improvement after gaining access to an expensive medicine. The medicine Spinraza, which costs over NOK 2.3 million per patient per year, was approved for people under the age of 18 in 2018. Vikebø was one of the patients who received it. Vikebø got nerve root inflammation after four years of Spinraza treatment and started on a new medicine, Evrysdi, which she drinks once a day. This has had a great effect on her, but costs NOK 2.7 million per patient per year. Evrysdi is not available for adults with SMA, and many are still waiting for treatment with Spinraza due to capacity problems in the hospitals. The summary is made by an AI service from OpenAi. The content is quality assured by news’s journalists before publication. – I think it is so sad that not more of my friends can experience the wonderful effect of this medicine, says 22-year-old Serenne Vikebø. She shows us around the apartment in Bergen, which is adapted to her needs. Vikebø has the rare and serious muscle disease spinal muscular atrophy (SMA). It makes the muscles in her body gradually weaker, and Vikebø doesn’t have much mobility left – she depends on an electric wheelchair to get around. Therefore, the mirrors in the apartment are lowered, the bathroom is adapted to her, and the assistants who are with her around the clock have their own room inside her bedroom. But she controls the doors, the computer and the mobile phone herself with a button and a lever on the chair. She does this with tiny movements with two fingers in which there is still some movement. – If I had not received the medicine in time, perhaps these small movements would have disappeared. And then I would have had no privacy again – then my assistants had to do absolutely everything. It is a relief to know that I will not get worse than this, she says. Spinal muscular atrophy, SMA Spinal muscular atrophy is an inherited, neurological muscle disease that leads to muscle weakness. The disease affects the nerve cells in the spinal cord and the main symptom is paralysis which, to varying degrees, affects voluntary movements, breathing and swallowing. A gene error means that the nerve tissue cells in question make too little of the necessary SMN protein. SMA develops with increasing symptoms and worsening of the patient’s condition over time. Without treatment, children with type 1 lose most of their motor activity early and most die before they are 2 years old. Source: Rikshospitalet and Decision Forum for new methods. Serenne Vikebø has been given a new life with the medicine Evrysdi. The disease has stopped. Fight for Spinraza There are not that many people in Norway who have a diagnosis of SMA, they are around a hundred people. A small and close environment where they know each other well, and are used to fighting the system. And what they have fought for is the vital and very expensive medicine Spinraza, which costs more than NOK 2.3 million a year, per patient. In 2018, after waiting several years for a clarification, the Decision-Making Forum for New Methods decided that people under the age of 18 should, under a number of conditions, get it. It is the Decision Forum that decides which medicines patients in Norway should have access to. Vikebø was one of the patients who received Spinraza. – I was just under 18, and therefore qualified for Spinraza. But many of my friends had to wait until 2023, and some are still waiting, she says. Short of time And time is critical for those with this disease. Spinal muscular atrophy is an unpredictable diagnosis where things can happen quickly. In the last six months before Vikebø received the medicine, she suddenly became much worse. – If I had started the medication six months earlier, I might be able to write text messages with my hands today. It’s sad to think about, she says. While those under 18 now received treatment, the adults had to wait. Spinraza did not have the same visible effect on them. – The medicine has an effect on both adults and children, but it is often less visible on adults. In adults, you may see that they can move their finger one centimeter more, while children can suddenly move their entire arm. But the one centimeter for the adults means at least as much, says Vikebø. But last year, the Decision-Making Forum for New Methods decided that adults could also receive this treatment. To great cheers from the SMA patients who were finally able to receive treatment. ALSO FOR ADULTS: Spinraza became available to everyone with SMA last year. But there are some patients who cannot get Spinraza because it has to be injected into the back. Photo: Biogen / Presse Painful treatment It is, however, complicated to get treatment with Spinraza. The reason is that it is given by spinal puncture, with an 11 centimeter long syringe that is inserted into the back. – It is given every four months, and is resource-intensive for the hospitals as they need both nurses and radiologists to administer the injection. There were seven or eight people in the room every time I got it, says Vikebø. – Besides, it is incredibly painful. I myself was always given morphine to cope with the pain, she says. According to SMA Norge, an interest organization for people with SMA, there are more who are still waiting for treatment with Spinraza, because the hospital has not had the capacity to treat them yet. – A new life Vikebø received regular doses of Spinraza, but after four years of spinal punctures, she developed nerve root inflammation. She could no longer get this medicine. She thus became the only person with SMA in Norway over the age of 18 who was allowed to start a newer medicine, Evrysdi – a potion that she drinks once a day. – It has had an enormous effect on me. I have been given a new life. After I started on Evrysdi, my breathing has measurably improved, from 11 to 17 percent lung capacity. I use the breathing machine much less often, and I can eat and drink more than ever before, she says. – I don’t think people think about it, but when you’re social, it’s often about having a drink or a bite to eat. Now I can say yes to that. I have regained the joy of food, she says and smiles. Too expensive for the adults And not least, she avoids the painful injections in the back. Spinraza from the drug manufacturer Biogen is not the only drug approved for SMA patients. Evrysdi is therefore a potion that patients can take at home. But this is also very expensive. The price the pharmaceutical company Roche has set for Evrysdi is NOK 2.7 million per patient per year, according to the method assessment from the Norwegian Medicines Agency. In January 2022, Evrysdi was introduced for young people under the age of 18 at a secret lower price. – Valuable medicine becomes expensive Both medicines cost well over two million Norwegian kroner. But why are these drugs so expensive? According to the pharmaceutical company, the price is based on the value the treatment provides. – SMA is a very serious disease. Without specific treatment, some of the children die already in the newborn period, or during the first years of life. Others have a milder type of SMA. Treatment of SMA can be of great benefit to the patient, relatives and society at large, and the price is set based on the value the treatment provides, writes Audun Ohna, head of market access in Roche Norway. He also points out that there are large costs in developing the medicine, and that the costs must be shared among fewer people in the case of rare diseases. – The price also reflects the risk involved in developing medicines for rare diseases, he writes. EXPENSIVE MEDICINE: The price of the medicine is based, among other things, on the value the treatment provides, according to the head of market access in Roche Norway. Photo: CF-WESENBERG / CF-WESENBERG Reassessed Evrysdi is not available for adults with SMA. Only Vikebø has been granted a dispensation. Leader of the Ordering Forum for newer methods, Ulrich Spreng, says they understand that more people want this medicine, and that they also want to give it to those over 18. – Therefore, we have been in negotiations with the supplier for a long time to make this available at a price that is in a reasonable relationship to the utility. So far, the supplier has not wanted to lower the price to a level that enables the priority criteria to be met. The last consideration in the Decision Forum was in April 2023, writes Spreng in an e-mail to news. It will be reassessed in the Ordering Forum for new methods on 21 October. DISAGREE: Head of Bestillerforum, Ulrich Spreng says they still do not agree with Roche on a price for the medicine Evrysdi. Photo: Vestre Viken health company Read the full response from Ordering forum for new methods In January 2022, the decision-making forum agreed to introduce Evrysdi for the treatment of children with spinal muscular atrophy (SMA) from 2 months of age. In June this year, Nye Metoder received a request from the supplier for an assessment to introduce the medicine to a subpopulation, i.e. adults with SMA type 2 and genetic SMA type 3. We understand that adult patients with spinal muscular atrophy would like to have this treatment. The decision-making forum also wants to introduce the method to all adults with SMA and not just a subgroup that the provider has now requested. Therefore, we have been in negotiations with the supplier for a long time to make this available at a price that is in a reasonable relationship to the utility. So far, the supplier has not wanted to lower the price to a level that enables the priority criteria to be met. The last consideration in the Decision Forum was in April 2023. New methods have now reviewed the request and Sykehusinnkjøp HF has given feedback that the method is not comparable with other methods for the relevant population. The Directorate for Medical Products (DMP) must prepare a suitability assessment of the method, i.e. examine what documentation is available for the patient population covered by the request, and whether it is suitable for a method assessment. Professional input is also obtained from subject experts at the hospitals. The deadline for submissions is set for 1 October this year. Based on this, Bestillerforum will decide whether an assignment should be given, and whether DMP should carry out a method assessment and/or whether Sykehusinnkjøp HF should prepare a price note. This takes place in a meeting of Bestillerforum on 21 October. According to the plan, the request will be presented at the Orderers’ Forum meeting. Getting no treatment And while assessments and negotiations are ongoing, some sit and wait very impatiently. Several of the SMA patients cannot be treated with Spinraza partly because of the titanium that was inserted to stiffen their spines. That means they can’t get the shots required with Spinraza. Still, the doctors won’t give them Evrysdi. Subject director at Helse Bergen, Marta Ebbing, writes in an e-mail that they are waiting for a decision from the Decision-Making Forum for new methods. “We generally want more and better opportunities for our patients, but here we have to wait for a decision in the Decision Forum. In general, this is about prioritizing the resources we have available in the specialist healthcare service, whether it is for medicines, equipment or personnel, and the relationship between resource use and benefit.’ Hope everyone can get it Vikebø is immediately starting his final year at the Norwegian School of Economics. Next year she will graduate as a civil economist. She manages a lot with the little mobility she still has, thanks to the medication she has been given. All she wants now is for her friends with SMA to have the same opportunity. – It is so good that children with this disease get the medicine, and that they avoid being so badly affected. But I think it’s sad that so many of my best friends have to go through painful treatments, and that they risk permanent nerve pain like I got, when there is an alternative, says Vikebø. Published 04.08.2024, at 07.51
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Serenne is the only adult in Norway who receives Evrysdi instead of Spinraza for spinal muscular atrophy (SMA) – news Vestland
