The intention of the Russian authorities to create a fund to support children with severe and chronic, including rare diseases became known in June 2020.
According to Vladimir Putin, the funds received from the increase in the personal income tax rate on income over 5 million rubles a year will be used for these purposes.
It is expected that in 2021 this amount will be 60 billion rubles.
On January 6, a decree was signed on the creation of a fund called the Circle of Kindness.
The organization will provide support for approximately 30 diseases.
According to Deputy Prime Minister Tatyana Golikova, it is most important to purchase medicines for children with spinal muscular atrophy (SMA).
This is a fatal genetic disease in which the entire muscles, including the respiratory one, gradually atrophy.
In the register of the "SMA Families" fund, there are 1,070 people with a corresponding diagnosis, of which 840 are children.
Two drugs that help with SMA are registered in Russia - "Spinraza" and "Evrisdi" - they must be taken for life.
Only the first, so-called loading course, "Spinraza" costs about 48 million rubles.
The third drug, Zolgensma, is in the process of registration in the Russian Federation.
It is enough to apply it once, but the cost of this single injection reaches 160 million rubles.
466 children receive therapy with these drugs (either through regional purchases or directly from manufacturers).
According to the Minister of Health Mikhail Murashko, the "Circle of Kindness" is at the "final stage of its inclusion in the work."
Nyuta Federmesser, a member of the board of trustees of the foundation, founder of the Vera hospice charity foundation, said that the charter of the Circle of Kindness must be registered with the Ministry of Justice; RF, the procedure for the acquisition of drugs and medical devices, the procedure for maintaining the resource of those in need ”and other internal documents and regulations.
“And, alas, there are many nuances everywhere, and this bureaucracy will inevitably take precious time,” she added.
"The state will catch us"
Mikhail Murashko stressed that the regions will finance the treatment of sick children, while the Circle of Kindness has not yet started working.
Tatyana Golikova also previously stated that the volume of assistance to patients from the regions should not decrease.
However, in a number of constituent entities of Russia, patients were transferred to the federal balance before the foundation began operating.
The son of a resident of Stavropol Anastasia Boyko Savely is six months old.
The woman is fighting to give her child with type 1 SMA, "Spinraza": she has the appropriate medical opinion and a court decision, which she won on December 2.
However, a few days later, she received a letter from the local Ministry of Health that in 2021 Savely's treatment would be “provided by the government of the country”, and on December 31 she learned that the department had filed an appeal.
© Photo from personal archive
At the same time, Anastasia is gathering for Zolgensma.
“We raised 7.5 million in three months,” she told RT.
"Collections fell sharply for everyone, because donors believe that patients have already started receiving medicines from the fund."
Svetlana Gepalova, a resident of St. Petersburg, the mother of two-year-old Kostya, also speaks about the fact that "everybody has gotten the camps."
“We do not ask the fund to pay the entire cost of Zolgensma and, accordingly, wait until the entire amount, more than 160 million, will accumulate from the fund.
Many families already have savings, for example, we have 19 million, the family of Sasha Arkhipov - 100 million. We are ready to give this money so that the fund would pay less, add the missing amount and eventually buy this shot of life, ”she told RT.
The Gepalov family is the first in Russia to sue for the most expensive drug for SMA.
The court in the previous two instances refused to purchase the drug, although the family had the appropriate conclusions from the doctors.
The Gepalovs filed a second claim, but the case was suspended: by January 30, the family must complete and bring additional documents.
© Photo from personal archive
So far, Kostya is participating in the program of clinical trials of the experimental drug Branaplam, but it is not known how long they will last, whether the drug will be registered later and whether patients will receive it in the future, says Svetlana Gepalova.
Nika Aleksanyan participates in the same program.
Her mother Emma is also worried about the prospects of drug provision: “We have been on Branaplam since two months: Now Nika is two years and three months old.
At that time, this was the only chance in Russia.
Thanks to clinical trials, the child lives.
But our goal is to get Zolgensma to improve her motor skills.
We are only in favor if this drug is purchased by the fund.
But we cannot wait long, time is our enemy. "
In Russia, there is another project under which children gain access to medicines without government assistance.
One-year-old Veronica, the daughter of Evelina Safonova, a resident of Podolsk, participates in the one-year program of early access to the Eurydie drug.
It began when the medicine had not yet been registered in the Russian Federation and ends in May 2021.
“The effect of the drug is, when we just started, Veronica could not even raise her hand.
Now the hands are working, and the general condition has improved, - said Evelina RT.
“We really hope that when the program is over, the state will pick us up.”
"Every time on pins and needles"
However, some regions continued to purchase expensive drugs for SMA, although, according to families, this was preceded by a long struggle.
A resident of Barnaul, Lyudmila Panasenko, has two children with SMA: 17-year-old Daniil and 3-year-old Masha Yatsenko.
The elder was diagnosed correctly only on January 30, 2020.
Now both children receive Spinraza at the expense of the region, but it took the family a lot of time and effort to get treatment.
“In June, the court took our side, obliging the regional authorities to purchase the drug.
The decision was "with immediate effect."
However, the first injection to children was made only on November 16, - explained Lyudmila Panasenko RT.
- By that time, Marusya completely lost support in her legs, although before that she walked at the support.
The back began to weaken, so I could hardly sit.
Now, after the injections, the skills are gradually returning, it becomes stronger, the effect is noticeable.
But all the same, every time we sit on pins and needles, whether there will be the next injection or not. "
© Photo from personal archive
Another problem is that Daniel will soon turn 18, which means he will lose the right to help from the foundation.
His mother hopes that the region will not abandon its adult patients after the Circle of Kindness is fully operational.
According to Nyuta Federmesser, at the first meeting of the Board of Trustees of the Circle of Kindness, held on January 14, the problem of adult patients was raised (there are 230 of them in the SMA Family register alone).
“According to the instructions of the president, this is a fund to help children, which means that it will not be possible to go beyond 18 years,” she said on the ONF portal.
“But, of course, we need to develop some kind of mechanism for working with those patients who reach the age of 17, so that in a year, at the age of 18, help does not stop.”
Prior to the start of the fund's operations, they intend to purchase Spinraza in the Krasnodar Territory.
According to the response of the regional Ministry of Health to Elena Gorobinskaya, the corresponding application has been prepared, delivery is expected from March 2021.
“This is a very long time,” Elena sighs, “My son Sasha has already switched to non-invasive ventilation of the lungs, from 8 to 12 hours a day he has to be on an apparatus that helps him breathe.
It will be worse further - difficulties with nutrition, invasive ventilation, after which irreversible changes occur.
You pray every day that there will be no deterioration today, because they happen instantly in children. "
© Photo from personal archive
According to Olga Germanenko, head of the Family SMA Foundation, during the transition period hundreds of patients were in a suspended situation.
“The life and health of children largely depends on how quickly drug purchases are organized,” she explained to RT.
- On January 15, the Ministry of Health collected personalized applications from the regions for the centralized procurement of drugs.
We hope that their processing will be fast, and that the purchase will not be the only one, so that in 2021 people who, for some reason, were not included in the first list, could submit. "
Germanenko added that, following the logic of the letter sent out by the Ministry of Health, we are talking, at least at the first stage, about the purchase of only drugs registered in the Russian Federation, that is, Spinraza and Evrisdi.
For the transition period, in her opinion, the ideal scenario would be: “The regions collect and send applications for patients who are still left without treatment and who will later be picked up by the federal budget.
And while the fund has not started working, the regions are beginning to treat these patients at their own expense, realizing that this is not for long, ”summed up Germanenko.