"Agreed"

What do experts and parents of children, patients of the SMA think about the proposal of the government supported by the president

People with rare diseases are one of the most vulnerable categories of citizens of the country. They need high -quality palliative help and expensive drugs. Now the patients are helped, first of all, charity funds that purchase the necessary drugs for the money of the donors. And what to do to patients with spinal muscle atrophy (SMA), who need the most expensive medicines in the world?

One injection of Zulgensma costs more than $ 2.1 million, spinras - 125 thousand dollars, but injections are necessary throughout life. Where to get that kind of money?

In Russia, only Spinraz has been registered. Its purchase should be carried out at the expense of the regions, but not all local authorities can do this due to a lack of budget funds. Charity funds have long been talking about the need to raise the problem of the disease to the federal level. Experts propose to introduce SMO to the list of highly voltage nosologies, which will allow financing treatment at the federal account.

This initiative was supported by regional Ministry of Health, in the State Duma and the Federation Council. Nosologies instructed the issue of expanding the list of the list in January the speaker of the Council of Council Valentina Matvienko. But there are no results.

However, the other day another proposal sounded: how to solve the problem of purchasing for children of expensive drugs, including for the treatment of SMA. Vice Prime Minister Tatyana Golikova proposed creating a special fund and replenishing it from three sources: regional and federal budgets, as well as by attracting a “socially oriented business”. The President of Russia agreed with Golikova - he first publicly responded to the need for drug provision of patients with SMA.

This initiative raised many questions in the patient community.

Olga Germanenko

Director of the Family Foundation SMA

- The word "fund" embarrasses us. It can be understood in different ways: for example, as a charity. In this case, this is not the decision that you want to see. It will be like a dialogue that we took place in the Ministry of Health at the end of December 2019.

Minister Veronika Skvortsova invited representatives of charitable organizations to an emergency meeting to say: there is no money, they know everything about the problem, but it cannot be solved, because it is very expensive. We were offered to "throw" for treatment. This is a dead end option.

Imagine that if the state does not have more than 40 billion rubles to solve this problem, then where did the funds get the money from?

There is a second version of interpretation the word "fund". The country has a pension fund, a social insurance fund and a compulsory medical insurance fund.

If Golikova had in mind the creation of a certain structure for Orphan diseases, and SMA in particular, which would distribute budget funds from various sources, then this decision could probably work. But then we must understand how this budget will take shape.

We do not understand the meaning of what we were told. We would like to know the details and get explanations. As an organization, we ask you to hold a meeting in order to understand what are the ways to solve the problem, and hear the opinion of different sides: the votes of patients, doctors, and regions.

We still believe that the best option would be to include SM in the list of highly used nosologies, the treatment of which is carried out at the expense of the federal budget. We live in a social state, and the responsibility for the health of orfanous patients lies on it.

Elena Zapolskaya

Mom Diana (6 years old, SMA 3 of type, Moscow). The family achieves Spinraza through the court

- I do not believe in such a fund. There should be a clear system of distribution, receipt and control of finance. The fund is limited funds.

Who will replenish it and in what terms if the funds are not enough?

The state is obliged to ensure treatment, regardless of the availability of funds in the budget of a constituent entity of the Russian Federation. It would be advisable to create a reserve federal fund and quickly take funds from there in case the region does not have them.

They are not going to make a list of high -cost nosologies financed from the federal budget. Namely, this would be the most optimal solution to the issue of financing treatment. This was discussed since the beginning of the year in both chambers of the Federal Assembly, and in the fall we have been waiting for this decision. But there is less and less strength - in every sense of the word.

The treatment of patients, regardless of the diagnosis and the cost of the drug, should be provided immediately if this is due to life indications.

It must be, as in Germany: today they made a diagnosis, tomorrow introduced Spinraz. And for years we have been trying to treat our children.

They grow up, turn into deep disabled people, die ... But they still do not treat them! This is cynicism and hypocrisy.

Today we had the next, third, court hearing, which the Depzdrav appeared. He requested all the medical documentation from the clinic, including the analyzes of feces and urine six years ago. As a result, the meeting was postponed again - on June 16. Why do they need this information, it is not clear. Not a single region requests that. We have all the basic documents confirming the diagnosis and purpose. It seems to me that they are taking time.

Anastasia Timukhina

Mom Milan (11 years old, SMA 1-2, Kemerovo). The family achieves Spinraza through the court

- I believe that the SMA should be in the list of highly used nosologies. So we are guaranteed to be able to receive treatment. The fund with three sources of financing will not solve the problem in which we now exist.

In Kemerovo, Spinraz was put to the first child, but not to Milan. The court sent her medical documents to the Novosibirsk Bureau of forensic medical examination. A month passed from the date of the court ruling, but they did not receive our documents in the bureau. Moreover, we were told that they would not conduct an examination.

The governor ignores my calls to fulfill his own promise: take Mila under personal control and acquire a medicine for her. In eight months that we seek treatment, its condition has worsened significantly. Handles practically do not work.

Stanislav Rostov

Dad Vari (1 year and 2 months, SMA of the 1st type, Georgievsk, Stavropol). The family collects money for Zulgensma

- The state should provide patients with medicines, not funds. He has enough money to ensure the treatment of everyone - children and adults. In other countries, the authorities agree with manufacturing companies, they make a discount on the drug.

Now our bosses are throwing these responsibilities to each other. Federal - on regional ones, and regional they say that they have no money.

Now the state, firstly, should at least clarify the number of patients with SMA. It does not know. Large fellows is the Family SMA Foundation, which calculated their approximate number. They are registered about a thousand people. But how many more parents do not know about this fund?

Secondly, it is necessary to fix perinatal screening at the state level in order to instantly identify the smoke and immediately inject the medicine.

In addition, we must bring to mind a medical component, because we happen like this: four and a half months Vara could not make a diagnosis. Then two months were prescribed the drug.

Imagine what would happen if we lived in a normal state in which we were diagnosed in time and administered the medicine? Our child would at least sit on his own.

I think that this is all - the reluctance of officials to work. Take, for example, our participation in the program of pre -registration access on the "Figlata". The application was supposed to submit a regional clinic.

When we asked our regional hospital to treat Varia, they answered us: “Are we like foreign agents?”

Since no one fussed in the Stavropol Territory, the application for participation submitted the clinic of Almazov. We had to go to St. Petersburg for two days to get a medicine. Because of this, the child was even more crushing. Varya fell ill with pneumonia, she disappeared a swallowing reflex. Now she cannot eat herself, we feed her through the probe. I had to buy a clearance at my own expense. We went for the medicine, but in the end the condition only worsened.

Alexey Demidov

Pope Polina (18 years old, SMA 2, Zmeinogorsk). The family won the "spinrase" court, but still has not received the medicine

- In fact, everything will remain the same as it is now. There will be no guarantees for providing patients with medicine.

Even if financing goes on a parity basis, it is jointly on all three sides, then in this case the regions will not have money for all patients. Surely the rule of subvention of the expenses of the region over the budget will probably not be applied. And the business in the regions is not the same and will not cover the needs of the fund.

My attitude? We are given false hope. Little Poland found funds for the treatment of her patients in insurance. And our country, which has 40% of all reserves of the planet’s natural resources, is not able to treat their own.

PS

"New" turned to Tatyana Golikova for explanations to the deputy prime minister Tatyana Golikova.