“Smot treatment is very expensive, not to cope without the federal budget” - an expert about the idea of ​​creating a fund for helping children with smudges

Vladimir Putin supported the idea of ​​creating a fund for helping children with spinal muscle atrophy (SMA) and other serious diseases that require expensive treatment. Help will be directed to children from those regions whose authorities cannot provide treatment at the expense of budget funds. The Insider talked with the director for several years of the working Family of the SMA Olga Germanenko about why the question of SMA is now on the agenda of the federal authorities and what can give timely therapy.

For some reason, everyone heard only the word "fund". This word in our country can mean many different institutions with different functions. It can be assumed that we are talking about creating a charitable foundation, but, on the other hand, there is a pension fund, a social insurance fund, an compulsory medical insurance fund - budget government structures. Depending on what exactly is meant, there will be different reactions and comments. I would like to get more detailed explanations from Golikova.

Golikova said the following: “There is a problem with the drug provision of patients with spinal-muscular atrophy. The fact is recognized as the government, and it requires a solution. ” These decisions may be related to budgeting from three different sources: funds of regional budgets, the federal budget and attracting socially responsible business by creating a fund. The good news is that the president gave a clear reaction to the fact that the problem is, and it must be solved. Here we fully support the president as a patient community. However, we do not understand how the government proposes to solve this issue.

In our country, for a year as there is registered drug therapy for the treatment of spinal-muscular atrophy. For this year, we clearly realized that the regional budget has not been possible. We continue to discuss the problem with different stakeholders and state authorities for a whole year, we ask for systemic decisions at the federal level with federal money. Patients should have certain guarantees that the process will be started and continued in the long run.

We voiced our proposal repeatedly. We believe that we need to include spinal-muscular atrophy in the program of high-cost nosologies, because today we have one registered therapy, two more will appear tomorrow. They are all at different steps either registered or prepared for registration in Russia, there will be several drugs using in different conditions, and they may be suitable for different categories. It is necessary to solve the issue based on these factors. All these drugs are expensive, and the disease is an orphans <rare - approx. The Insider>. Accordingly, this should be a program of highly used nosologies, due to which today 14 diseases are provided with drugs at the expense of the federal budget.

Today, the regions are not able to solve this problem due to the high cost. This is a disaster. Whatever the region you call now, in any of the 79, in which patients with SMA are identified, they will tell you that they do not understand how to solve the problem. Federalization will not only resolve the issue, but also reduce the cost of the purchase of drugs. Today, the region makes an individual purchase for each patient at a retail price: about 7800 for one ampoule of the registered drug, in the first year you need 6, and then 3 each year. This is a serious amount, and it can be reduced. For example, there is a registered SPINRAZA drug, an application for inclusion in the vital and most important drugs is applied to it - approx. The Insider>. This is the only mechanism that allows you to fix the price of the release of the drug. So far, the decision has not been made. If it is positive and the drug is included in the List of the GUNLP for 2021, then manufacturers promise a reduction in price up to 25%.

If nosology in the same 2021 years will enter the list of high -cost, it may allow this to reduce the cost due to centralized purchase and get additional discounts. Then we can talk not about 40 billion rubles, which are needed to provide almost 1000 patients, but about 30 - significant savings. We are in touch with the Health Committee of the State Duma, where this issue has been raised repeatedly, we know that at the level of the Federation Council and at the level of the Ministry of Health they are working on it. From the level of the government, we have not yet made contact with us.

We would like to ask the government to conduct working meetings to discuss possible decisions, to which people would be invited for whom the solution to this issue is key: representatives of patient organizations, doctors who work directly with patients. Today it is the regions that are thrown into the “advanced”, but they cannot do for various reasons. There are not so many of us, but we have needs, we do not cease to get sick because of Covid, we do not stop getting sick if there is no money for medicines.

We work with the regions and ask for information about how many patients, where they are observed, how route are, how much it is treated, whether budget financing is allocated. Sometimes, to our surprise, we know patients of our category much better than the relevant regional ministry, which can answer us that there are no patients in their region. We know that there are five patients who applied for treatment - why doesn’t the leadership know about this?

We know that there are five patients who applied for treatment - why doesn't the regional ministry know about this?

For a year we have hung up with registration: "Register us the drug." Now we have been hollowing a year so that we are provided with treatment with this registered drug. In Russia, SMA is already a painful topic, because until 2016 there was no treatment in the world for spinal-muscular atrophy. I remember this period well, then everyone talked about the MPS, mucopolysaccharidoses, which were also previously incurable. But now in the country the problem of mucopolysaccharidosis, if not completely, then at least partially resolved, because they are included in the same high -cost nosologies.

At the end of 2016, the first drug appeared in the world for the treatment of SMA. In Russia, it was registered after almost 3 years-in August 2019. Since then, we can say that in Russia SMA is theoretically not a sentence. From the moment when therapy appears in the country, patients, their families, patient organizations and the state arise another task: to provide it with patients. Treatment can significantly increase both quality and life expectancy.

Why is the SMA theme now? Because there are many of us. For rare diseases, there are many of us. This is one of the most common among rare diseases. The patient is faced with the months of the struggle within the region with the courts, the prosecutor's office, Roszdravnadzor. In recent months, I have not met a single person from power who would not hear about it.

About 80% of our patients are children, the remaining 20% ​​are those who survived until adulthood. These are people locked in their body, which weakens every day, stops breathing on its own, loses the opportunity to eat. At the same time, the intelligence is clean, and you understand that you can’t, it is terrible. Treatment allows people to be full -fledged members of society. Even in a stroller, you can be an accountant, programmer, lawyer, psychologist and continue to bring our favorite taxes, increase GDP. If we begin to treat patients early, from the moment of diagnosis, until the disease has taken too much, that is, a chance. Maybe these people will not be completely healthy, but they will be able to move, work, communicate, and not lie and breathe with the help of Ivl. We have no time to wait. Where does this desperate struggle of parents come from for children to be treated as soon as possible? Because every month we lose motor neurons that cannot be restored. The longer we pull with treatment, the less effective therapy will be. The faster we begin to treat the child, the greater the chances that Vasya, Petya and Masha will be able to cope with the disease and be physically as preserved.