Contents
There were high hopes of parents of children affected by SMA, there is a hard clash with reality. There is no money to pay for gene therapy that could save the lives of children with spinal muscular atrophy. So, as it has been so far, there are online fundraisers, i.e. help from strangers.
- SMA, or spinal muscular atrophy, is a genetic disease
- Every year in Poland, about 50 newborns with SMA are born. It is one of the most common causes of death among children under 2 years of age.
- Gene therapy is financed, for example, in Italy, Germany, Slovakia and Hungary
- More information can be found on the Onet homepage
“No” to gene therapy for SMA in Poland
Recent months have raised hopes for children diagnosed with SMA. The commencement of negotiations between the drug manufacturer and the Ministry of Health was eagerly awaited. The aim was to reimburse, which turned out to be possible in Italy, Ireland, Germany or our neighbors – in the Czech Republic or Slovakia.
On October 14, it turned out that there is no chance for this in Poland.
– Deputy Minister of Health Maciej Miłkowski informed about the completion of negotiations on reimbursement of Zolgensma gene therapy with a negative result. This is devastating news for the Polish SMA community. We call for the continuation of the talks – then wrote the SMA Foundation.
This is practically the end of dreams for many parents whose children suffer from spinal muscular atrophy. Deputy Minister of Health Maciej Miłkowski argued the decision with financial reasons. The price for the drug was supposed to be “prohibitive”.
– The price is insanely high, but the progress in medicine is unprecedented – commented Dr. Mirosław Jasiński, a child neurologist for Newsweek. In 2020, the specialist participated in the process of administering the drug to an infant from Poland. – The disease, which until recently seemed a death sentence, turned out to be curable. Maybe not completely, because the administration of the preparation does not reverse the disease, some neurological deficits may remain, for example problems with movement, but this is incomparable to what was waiting for patients in the past.
The rest of the text is below the video.
Gene therapy for SMA is not for everyone. It is also not the only drug
Gene therapy requires only one dose, while another drug available in Poland (nusinersen) is taken for life. This one is reimbursed, but it works slower. And with SMA, time plays an important role.
– All treatments for SMA work in a similar way: they aim to raise the level of the protein responsible for the functioning of motor neurons – explains Kacper Ruciński of the SMA Foundation for Newsweek. – The advantage of gene therapy is a single dose and quick action of the preparation: the protein level is normalized after 1-2 days. With nusinersen, you need to take 2-4 doses and wait a few weeks. This is of particular importance in children in the first period after diagnosis, when the disease is progressing rapidly. A child, especially younger ones, loses neurons and muscle tissue rapidly, and the sooner the drug is administered, the more can be saved.
Two groups of children are eligible for gene therapy: those who have SMA type 1 symptoms and those who have presymptomatic symptoms and have no more than three copies of the gene. SMN2. Unofficially, there is also talk of a weight limit – a small patient should not weigh more than 13,5 kg. This means that in order to apply gene therapy, early diagnosis is necessary, and it is quite good in Poland. The only thing lacking is the possibility of receiving a therapy that will help to stop the development of the disease at this early stage. Not because such a drug does not exist, but because there is no money for it.
Diagnostics of SMA is possible thanks to genetic tests. At Medonet Market you can quickly and safely buy genetic screening test for newborns and children for SMA.
There are no refunds, collections remain
Gene therapy for children with SMA costs PLN 9 million without reimbursement. This amount is completely unattainable for the average Pole. And children with spinal muscular atrophy are born not only in the richest families for whom such an expense is real.
That is why the internet is full of heartwarming collections. Parents beg strangers to help raise funds for the treatment of their children. The topic itself is quite controversial, because not every little patient will be helped by gene therapy. Some of them are already taking the reimbursed drug, but do not believe in its effectiveness. Therefore, it is looking for an alternative. For some children, however, this is the best chance – and that is why, in this difficult situation, the most important thing is to be able to make a choice. Doctors just don’t have it these days. So if, in their opinion, gene therapy is the most appropriate in this particular case, collecting funds for treatment is the responsibility of the parent.
– Paying for gene therapy should be the responsibility of the payer, not the parents – admits prof. dr hab. n. med. Maria Mazurkiewicz-Bełdzińska.
What about the drug manufacturer? In its official position, the company does not hide its surprise that the deputy health minister disclosed the amount referred to in the negotiations. As he writes, this is “misleading the public”.
We have concluded agreements guaranteeing access to Zolgensma for patients in 22 countries around the world, including 13 countries of the European Union – just 16 months after the drug has been approved for sale. In discussions with health ministries and reimbursement bodies, we offer various options to reduce the cost of therapy, including rebates, deferred and installment payments, and risk sharing instruments based on treatment outcomes. Our goal is still to find the best solution so that Polish patients with SMA have access to this innovative, one-time therapy – we read in the statement.
Also read:
- COVID-19 despite vaccination – who is most at risk? A risk calculator was created
- New rules for declaring deaths. Changes in the regulations are coming
- BioNTech chief: a new COVID-2022 vaccine will be needed in 19
- The fourth wave is accelerating, but Poles are not afraid of infection [SONDAŻ]
- Norwegians started treating COVID-19 like the flu? There is a reaction from the local authorities
The content of the medTvoiLokony website is intended to improve, not replace, the contact between the Website User and their doctor. The website is intended for informational and educational purposes only. Before following the specialist knowledge, in particular medical advice, contained on our Website, you must consult a doctor. The Administrator does not bear any consequences resulting from the use of information contained on the Website. Do you need a medical consultation or an e-prescription? Go to halodoctor.pl, where you will get online help – quickly, safely and without leaving your home.