Spinal Muscular Atrophy, or SMA, is a deadly disease: it kills young children and babies. It is milder in older patients, but over time they lose their ability to move independently. A very expensive drug for this disease will soon be reimbursed by the National Health Fund.
- Annual therapy with nusinersen costs nearly PLN 2 million and none of the Polish families can afford it
- The Minister of Health, Łukasz Szumowski, received a petition from the SMA Foundation signed by 62 people asking for the possibility of treating SMA
- Not only does the drug stop the progression of the disease, it can also help patients to develop new reflexes
Agata Roczniak, a 37-year-old photo model who has been suffering from SMA, i.e. spinal muscular atrophy since childhood, has special reasons for joy and pride. Together with the SMA Foundation, she achieved the goal she fought for: the only registered drug nusinersen (Spinraza®) will soon be reimbursed in Poland. This is what the Minister of Health, Łukasz Szumowski, promised in an interview with Robert Mazurek. On RMF FM, he made a declaration after he received a petition from the Foundation signed by over 62 people. The letter contained a request that the preparation be reimbursed, as the annual treatment with nusinersen costs nearly PLN 2 million and none of the Polish families can afford it.
SMA – the most common cause of infant death
SMA is a fatal disease. This neuromuscular condition is genetic and results in the death of neurons in the spinal cord. When neurons die as a result of disease, the muscles of the body weaken and gradually deteriorate. The muscles of the limbs, trunk, lungs and esophagus deteriorate most quickly, leading to respiratory failure and loss of swallowing ability. Spinal muscular atrophy is the most common genetically determined cause of death in infants and young children, and in adult patients it is longer and slightly milder.
The Minister of Health, Łukasz Szumowski, assured that thanks to the reimbursement, the nusinersen treatment program may cover from 500 to 800 patients with SMA. This means that practically all patients with spinal muscular atrophy registered in Poland have a chance to be treated with this preparation. It was registered in the USA in 2016.
Sit by yourself and even walk
After the ordinance is signed by Minister Łukasz Szumowski – which is to take place in the coming days – nusinersen will be included in the list of reimbursed drugs from January 1, 2019.
– This is wonderful news – Agata Roczniak is happy. – Because the drug not only stops the disease progression, but also allows patients to develop new reflexes. During therapy, processes occur on the nerve-muscle path that the patients did not have before. A colleague of mine had treatment with nusinersen. Before, he had tremendous trouble swallowing, it took him an hour to eat his breakfast. Now he eats normally, like a healthy person.
It is worth noting that many of the effects of this drug are not known yet, because it has only been used in the world for two years. So in some patients, nusinersen can cause spectacular changes, and in others, stopping the disease alone will be a success.
Children from outside Poland who were given nusinersen before the onset of SMA symptoms are known to have learned to sit on their own, and almost all of them learned to walk.
That there was something to get into
Szumowski explains that SMA is a very dangerous, deadly disease, especially for children.
– We do not want to extend their lives by three months – emphasizes the minister. – We do not want to improve the quality of life, we want to let these children live. All patients, without exception, will be entitled to a reimbursement.
The drug is administered by injection into the spinal canal, and according to US registration indications, it is administered every four months.
That is why Agata Roczniak already has plans for the coming weeks – I will organize a fitness campaign, of course under the watchful eye of doctors – says the 37-year-old. – Because in order to apply this drug, you need to rebuild your muscles, at least minimally, by exercising. I know that it is possible for me to punch my spine with nusinersen, but my husband jokes that he will not meet me anywhere else, only at the pool. There are a lot of people in Poland at different stages of the disease. They look forward to therapy. Although they are a bit worried, because they have never had such procedures in the lumbar region before. However, I know that medicine knows methods that allow you to pierce the most difficult and twisted spines of the disease.
Treatment of patients with SMA soon
After signing the ordinance by the minister of health, Andrzej Jacyna, the president of the National Health Fund, will issue a decision on launching the nusinersen treatment program within 30 days. Then the provincial branches of the National Health Fund will begin the process of contracting hospitals. The treatment program will be conducted in over a dozen centers in Poland. Previously, without reimbursement, the cost of a single dose of the drug was as high as EUR 90. However, Szumowski does not reveal what price he finally managed to agree with the producers.
– The cost has been negotiated to such an extent that it is acceptable for us. This drug is very, very expensive – emphasized the minister.
The SMA Foundation estimates that it will take approximately 2-3 months for drug qualification to begin and the first patient to receive the drug.