– I wake up every day with a great uncertainty about “Is today the day I’m going to get a blood clot?” “Am I going to have a stroke tomorrow?” That’s what 25-year-old Trym Saugstad Karlsen says. In October last year, his life was turned upside down when he learned that he has contracted a very rare blood disease called PNH. There are only between three and five cases of this in Norway each year. The red blood cells are attacked by the body’s immune system, and are broken down faster than usual. This means that Karlsen, who will soon be fully trained as a jazz musician, quickly becomes exhausted. He needs a long time to recover from what used to be simple tasks. When he was healthy, he trained five times a week, climbed outside, jogged and cycled for long trips. Now he can’t do any of this. – After I played the exam concert on Monday, I had to lie in bed for two days, because I was so incredibly exhausted and tired, he says. Photo: Jøte Toftaker / news The disease also makes him extra vulnerable to several serious conditions. The chance that he may get a blood clot is 67 percent, and there is an increased risk of stroke, kidney failure and liver failure. But even if it exists, Karlsen does not receive treatment. – Differential treatment In autumn 2022, the Decision Forum decided that patients who get the disease after 26 September should no longer receive the drug Soliris – the most common treatment. This means that patients who were diagnosed with the disease before September will continue to receive the medicine, while Karlsen, and other patients who contracted the disease after the given date, will not receive it at all. – It is simply a desperate situation, which is difficult for the patient group and difficult to explain as a treating doctor – that there is discrimination in practice, says Anne Sophie von Krogh. She is a senior physician at St. Olav’s hospital, and a specialist in blood diseases and internal medicine. She is also Karlsen’s doctor, but speaks on a general basis. – For the group of patients who have the condition PNH, a collective professional environment, I would say, of hematologists in Norway, thinks that this is deeply problematic because we have an effective treatment that can change the lives of this group of patients, she says. Senior doctor Anne Sophie von Krogh says that the Decision Forum has put professionals and therapists in a difficult situation by refusing to use Soliris. Photo: Jøte Toftaker / news – Almost immediate effect Krogh says that there are several medicines that have the same effect, but that Soliris is the most common, and has been used the longest. – It works quite immediately as symptom relief and it works in the long term to reduce the risk of complications. The patients who receive it can potentially live almost symptom-free. – You get an almost immediate effect and it is effective in the vast majority of patients in terms of reducing the need for blood transfusions, increasing the quality of life, removing symptoms such as fatigue symptoms and reducing the risk of blood clot disease, she says. Anne Sophie von Krogh says there has been a lot of discussion in the professional community around the Decision Forum’s decision. Photo: Jøte Toftaker / news Costs NOK 4 to 6 million a year per person Soliris costs between NOK 4 and 6 million a year per person. In 2021, there were 22 people who received the medicine, according to Ulrich Johannes Spreng. He is a professional director at Health South-East and sits on the Decision-Making Forum. Spreng says that it is the pharmaceutical company that distributes Soliris that is the reason they have cut it. – I understand both patients and relatives, but it is the case that we are dependent on a collaboration with the pharmaceutical company. Here, the company has chosen not to provide documentation of effect or a price offer, and then it is difficult for the decision-making forum to introduce a method, he says. Ulrich Spreng is specialist director at Helse Sør-Öst. Photo: Vestre Viken HF The decision-making forum has three criteria they use when evaluating medicines: Severity, benefit and effect, and resource use. – Here it is important that resources are distributed equally and fairly. If you spend money and resources on one type of treatment, there are consequences for another type of treatment – because more money does not follow when you go in for a new method, says Spreng. – We want to find a solution here, but it also requires that the pharmaceutical company is on board, takes a social responsibility and does not demand an unreasonably high price. On Thursday, the specialist directors of the four healthcare organizations will meet for a meeting where they will discuss Soliris, among other things. Discussing price Annabel Cowper is communications manager at the pharmaceutical company Alxion Pharma. She confirms that they have been in dialogue about price with the Decision-Making Council since the end of 2022. – We are currently waiting for feedback on our latest offer, she writes in an e-mail to news. Cowper emphasizes that the medicine is approved for PNH patients in the USA, EU and Japan, and that it has been available in Norway for more than ten years. – We have a desire to deliver treatment to patients with PHN in Norway, and are in ongoing discussions with Sykehusinnkjøp to find a suitable and sustainable solution, she says. The only treatment Trym Slaugstad Karlsen receives is blood-thinning medicine and folic acid supplements. This is to reduce the risk of blood clots. Photo: Jøte Toftaker / news Will not die early As long as Karlsen does not get medicine, life is put on hold. He is going to work as a freelance jazz musician, but he can only work 50 percent and it is difficult to plan far in advance. – Now that I’m going out into working life, it’s completely impossible to plan with the band and work at all. I don’t have the capacity to book tours and play many concerts in a row. If nothing happens with the situation, he considers taking legal action against the state or the decision-making council. – It means so much to get that medicine, because then I know that I have a future. I have no desire to live until I’m 100, but I also don’t want to live only until I’m 30, he says.
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– Deeply problematic – news Trøndelag
