The state fund created on the initiative of the President is slow with the purchase of medicines for children, patients of SMA. Their parents talk about the "unspoken prohibition" of the Ministry of Health

The State Circle of Good Fund, created in January at the initiative of the president to support children with rare diseases, did not purchase a single dose of Zulgensma. This is the most expensive medicine in the world. One ampoule costs $ 2.1 million, but it is enough to stop a serious genetic disease - spinal muscle atrophy (SMA). Last year, charitable foundations purchased Zulgensm for 25 children. People are privately transferred to their treatment in a total of more than 3.7 billion rubles. The “circle of good” was created so that the parents of children, sick SMA, do not collect such cosmic amounts on their own. Now at the Zolensm, according to approximate estimates, 40 babies need. But the State Fund is not in a hurry to purchase the drug for them.

Help "New"
Spinal muscle atrophy is a hereditary neuromuscular disease that affects the muscles of the whole body. First, the patient weakens the legs and arms, then the muscles are atrophy, which are responsible for swallowing and breathing. Most often, a person dies from suffocation, while in full consciousness - the disease does not affect intelligence.

Until recently, the lubricants were considered incurable. But a few years ago, the world's first drug appeared, which, even if it does not cure the disease, but stops its development. Now there are already three such drugs in the farm market.

Spinraz (Nusinersen) was developed by Biogen in 2016. The medicine is administered into the spinal cord. In the first year of therapy, six injections are put on the patient, and three in the next. And so until the end of life. The cost of one dose is 125 thousand dollars. The medicine was registered in Russia in August 2019.

“Eurisdi” (risdings) is another medicine for the treatment of SMA (manufacturer - Roche), it must also be taken for life. Every day, the patient needs to swallow a small amount of a solution of medicine. The cost of the annual reserve of the drug is 340 thousand dollars. Since November 2020, it has also been registered in Russia.

Zulgensmu (Overman's Abparvovets) was developed by Novartis. One dose of medicine costs $ 2.1 million. The drug is administered into the patient’s body once. Unlike Spinraza and Eurysdi, which can be prescribed to almost any patient, Zolgensma has restrictions on use. According to the American rules, it is necessary to make an injection until the patient reaches two years old, according to European ones, if the child’s weight does not exceed 21 kg. The medicine still has not been registered in Russia, although the manufacturer submitted documents to the Ministry of Health in July last year.

According to experts, there is no proven advantage of one drug over another. All three medicines have equally proved their effectiveness during clinical research.

Almost the entire last year, the “new” talked about how the parents of the Sma-detectors unsuccessfully tried to get from the state the receipt of “spinras”. At that time, she was the only medicine registered in the country for the treatment of SMA (risads, the trade name of Eurysdi, was registered in the Russian Federation only last fall).

The obligation to purchase Spinras, by law, is entrusted to the regions, but regional officials refused to purchase. The reasons were different: then the lubricants were absent in the list of life -threatening and progressive rare diseases (which means, according to the logic of officials, it does not progress and does not threaten the patient's life). Then in the region there are no specialists who could administer the medicine. Then this prevents, for example, the patient's scoliosis.

The real reason for the refusal is the lack of funds in the regional budget.

The Zulgensma is even more complicated. Parents of very young children, sick SMA, give preference to this drug primarily because the child needs to make only one injection, while Spinraz and risads are lifelong medicines. But, since Zulgensma is not registered in Russia, doctors can prescribe it to the patient only if officially approved therapy does not help. And therefore, the chance to get Zulgensm at the expense of the state is reduced to almost zero.

Parents of sick children had the only way out - collecting money for the medicine on their own.

Last year, Novaya received dozens of letters from parents of children who begged to support their charity fees. Twenty -five patients managed to collect the required amount of 150–160 million rubles - but only thanks to the huge public support and assistance of caring businessmen. For another 19 Russian children, the manufacturing company Novartis provided Zulgensm on the principle of lottery as part of a free program.

One baby with a smudden died, without waiting for the saving injection of Zulgensma. A few more children died while they were waiting for Spinraz.

Patient and charitable organizations engaged in helping patients SMA claimed that the inclusion of the disease in the list of highly used nosologies (diseases) would help to solve the problem with the purchase of drugs at the expense of the budget. According to this program, drugs are purchased for federal money. But the Russian President solved the problem differently.

Last summer, Vladimir Putin proposed increasing income tax (personal income tax) from 13 to 15%. The initiative applies only to citizens who earn more than five million rubles a year. And only that part of their income that exceeds these five million is taxed. Such a system, according to Putin, will allow more than 60 billion rubles to the budget annually, which will dispose of the “circle of good”.

“The decision to use this money should be transparent and absolutely open. Only doctors and public figures can make moral right to make these decisions. People who have devoted their help to sick children all their lives, ”Putin said on the eve of voting on amendments to the Constitution.

Deputy Prime Minister Tatyana Golikova reported that most of the amount-47 billion rubles-will go to the purchase of drugs, including unregistered. The list of nosologies that the State Fund will be financed will include about three dozen diseases. The final list has not yet been defined, but there is a smell in this list. All three drugs existing on the market - Spinraz, Eurysdi and Zulgensma - can officially be purchased “a circle of good”. On the first two drugs, 9.4 billion rubles have already been spent (the first cash tran to the State Fund amounted to 10 billion).

But there has still been not a single purchase of Zulgensma.

"An unspoken ban from the Ministry of Health"

Martin Wulpe is a puffy, blue -eyed baby from Kashira near Moscow. In the photo and video that his parents publish on social networks, he seems completely healthy. It claps long eyelashes, smiles.

A week ago, Martin turned four months old. Usually by this time, the kids already hold their heads and begin to turn over. Martin does not.

Recently, parents learned that the cause of the boy’s weakness is a deadly disease, spinal muscle atrophy. Martin has the first type of SMA, the most severe form. Without treatment, children with such a diagnosis, as a rule, do not live up to two years.

“Time is the worst enemy,” says Margarita Wulpe, the boy’s mother. -

The disease is unpredictable. Every day I wake up in horror from the fact that he can lose another part of the skills.

He does not hold his head and does not try to roll over. Thank God he still eats on his own, without a probe. Now our most important task is to preserve the child as much as possible before the start of therapy.

Martin's parents are trying to get expensive treatment through the Kircular Circle Foundation. At the end of March, they turned to the National Medical Research Center for Children's Health (NCZZ). In a medical conclusion (has at the disposal of the “new”) pediatrician Lyudmila Kuzenkova recommended that Martina begin treatment with risad and. Next, she added: “Due to the early onset of the disease and the small age of the child, it is possible to carry out therapy with Zulgensma.

The federal medical centers, including NCZZ, have no authority to submit an application to the “circle of good”. Only the attending physician at the place of residence can do this. The doctor must transmit documents to the regional Ministry of Health, and he, in turn, to the federal. Further, the statement is considered by the expert council of the State Fund. He makes the final solution - which to buy the drug.

The Wulpe family lives in Kashira, so Margarita sent documents for the “circle of good” to Oleg Lapochkin, the head doctor of the neuropsychiatric hospital near Moscow for children with the defeat of the central nervous system and a violation of the psyche. There, Martin undergoes examinations. In the accompanying statement, Margarita repeatedly emphasized that it was precisely the “Zolgensma” who was seeking the appointment.

- I consider the one -time administration of the drug a huge advantage over other drugs. The child is very small, often regains. Risd and Plamon can also burst, but it cannot be given again. What will be effective then? - asks Margarita. - In addition, the “circle of good” was created for children.

When my child will be 18 years old, who will provide him with risadlasm?

On April 8, Margarita came to the hospital with her son. “We were told: come, we will see you, make sure that the child exists.”

Doctors of Alexander Smirnov and Nadezhda Shakhovskaya examined Martin, but did not give any conclusion to Margarita. They did not say that it was a medical consultation. Next week, doctors informed the mother of the child that they had already submitted an application to the “Circle of Good” for the receipt of risdlas. From a written answer, she later found out that this happened on April 7, that is, the day before Martin's examination.

- How could they make this the participation of the child and parents? The doctors did not say why they submitted the application only on the risads, and did not become the Zulgensma, which the federal center also recommended us. This problem is not only ours. Parents of the other “emoticons” with whom I communicate, everyone says that doctors do not submit applications for “Zulgensma”, because there is an unspoken ban from the Ministry of Health.

Oleg Lapochkin did not answer the request of the “new” request.

"There are other doctors"

Margarita Wulpe was lucky: at least she has a medical conclusion in which Zulgensma is directly recommended. Many parents are unable to get this.

Dmitry Petrov from Moscow is the father of a six -month -old Cyril with the same diagnosis, the smell of the first type. The boy receives Spinraz from a “circle of good”. The fact that Cyril will be treated with this drug, parents learned after the attending physician in the Morozov children's hospital applied to the State Fund.

Now Dmitry is seeking the appointment of Zolgensma. He believes that one injection of this drug is “economically more profitable for the country” than the lifelong intake of “spinras”.

In mid-April, Dmitry came with his son to consult a neurologist Svetlana Artemyeva. She is a leading Russian specialist in neuromuscular diseases (including SMA), head of the children's neuropsychiatric department in nicknames of pediatrics named after Yu.E. Veltishchev. But he also takes patients in the Genomed private clinic.

In the conclusion from Genomed (is at the disposal of “New”) Artemyeva wrote: “Despite the fact that the child receives pathogenetic treatment with Spinraz, taking into account age, switching to simultaneous therapy with an unregistered drug Zulgensm with an informed consent of the parents.”

“But she added that the federal center, most likely, would not give such a conclusion,” Dmitry retells the conversation with Svetlana Artemyeva. - Because “there are other doctors” who need to be convinced that the drug will be more effective than already prescribed therapy.

Petrov submitted documents for examining his son in nicknames of pediatrics, but the answer from the medical center has not yet arrived.

Svetlana Artemyeva refused to comment on the prescription of drugs for Kirill Petrov, saying that "combined therapy around the world has not been approved and there are no methodological recommendations."

A neurologist who also examined Kirill Petrov (he asked not to mention his name), says that the child reacts positively to Spinraz. In order for the physician, “without croaking,” he could transfer the child to another type of therapy, the prescribed drugs should not help him or cause an allergic reaction, the interlocutor of “New” explains.

“If I had a choice of all three drugs, and if a small child had to be taken under three months, who had not received anything, I would start with Zolgensma,” the doctor argues. - Compared to other drugs, the side effects of Zulgensma are less pronounced in children under six months. And there is a more chance that the drug will have a good effect, which is enough for life. If it is missing, then the child can be prescribed tandem (combined) therapy. But these are just my dreams. The effectiveness of such a system has not yet been proven.

"You can at your own expense"

On April 29, Dmitry Petrov visited the admission at the NCR with a neurologist Sofia Popovich. Her signature, along with the signature of Lyudmila Kuzenkova, stands at the medical imprisonment of Martin Wulpe, who was recommended to Zulgensm. Dmitry, Kirill, Popovich, Dmitry, refused to prescribe the same medicine.

- I asked if suddenly $ 2.1 million in my pocket lies in my pocket, can she appoint Zulgensma? They answered me “Yes,” says Petrov. - I ask again: "Why don't you want to help your child for budget money?" “I have no right, since the drug is not registered.”

- The position of the federal clinics is this: if you are going to do it yourself, we do not mind. As soon as you demand the “Zulgensm” from the state, our position is immediately changing, ”confirms Dmitry Alexander Kurmyshkin, a neurologist and director of the SMA Help to the Families of the SMA, which protects the interests of more than 360 patients. - The state is thus trying to evade responsibility.

And this is not the first time.

In April, the Tekonsilium NCZD refused the “Zulgensm” of the 4-month Adis Garmaeva from Ulan-Ude. Doctors referred to the "lack of evidence in the efficiency and safety of tandem therapy." By that time, the girl had already received three spinras injections.

“The Ministry of Health of Buryatia told us that we will be able to put the“ Zulgensm ”and get an appointment for it only if we collect money and buy the drug,” the father of Adisa Arslan Garmaev told the publication “such things”. “We were directly told that from the state and from the“ circle of goodness ”it should not even be expected.”

On April 12, the girl’s parents managed to close the gathering at Zulgensma. Most of the amount-130 million rubles-was transferred by the former top manager of the peak group Vladislav Sviblov, who is now engaged in the development of the lake deposit of polymetalls in Buryatia. After that, the federal clinic, which a month ago made a refusal, agreed to appoint the girl Zulgensma.

“When the Geography Foundation will purchase the drug, they will hold a consultation for permission to import medicines and immediately make an appointment,” said Adisa Darima Garmaeva. “That is, it is possible, but as if at your own expense.”

"There is no procurement mechanism"

The Gosfond “Circle of Good” says that they cannot purchase Zulgensma due to the “lack of developed procedure for providing an unregistered drug”. This follows from the official response of the doctor Oleg Lapochkin, who submitted an application for Martin Wulpe (is at the disposal of “New”).

“The leadership of the Fund refers to the fact that they do not have a regulation and a purchase mechanism, but what a snag, I, as a specialist, do not understand,” says Lilia Tsygankova, the head of social projects of the Geography of Good Foundation, who has repeatedly purchased an unregistered drug. - We are ready to provide complete support and share experience with colleagues how to make such purchases. To purchase Zulgensm, you need only two contracts: a donation agreement with the federal center where the child will undergo therapy, and an agent agreement with the supplier.

What is hidden behind the official “organizational” version is not yet clear.

- The doctor may not mind applying for unregistered therapy, but he will not go against his regional Ministry of Health. And he will not go against the federal. The one who jumps above his head will simply be removed, and they will put someone more convenient in his place-

suggests the interlocutor of the "new", familiar with the work of the State Fund.

Alexander Kurmyshkin connects the unwillingness to purchase Zulgensma and delay with the registration of the drug in Russia with the fact that the Russian company Biocad is developing domestic medicine for the treatment of SMA. It is assumed that, like Zulgensma, this drug will be administered once. Clinical research is underway. This year, the company plans to apply for clinical trials of the first phase.

Dr. Kurmyshkin says that he contacted the presidential apparatus with a detailed business plan to create another domestic drug for patients with SMA, which was developed by Kazan scientists. According to Kurmyshkin’s calculations, one dose of this medicine will cost 1.5 million rubles - this is 47 times cheaper than imported analogues. “As a result, the total annual budget savings will amount to more than 12 billion rubles,” the doctor writes.

The Presidential Directorate answered that Kurmyshkin’s appeal was considered, but then silence. Officials “additionally noted” that the release of the drug developed by Biocad is expected to pharmaceuticals in 2026.

“Not only do the authorities prefer the Russian manufacturer,” says Kurmyshkin, “so they also overlap the opportunity for a competitive struggle inside the Russian market.”

"People have no response"

Darina Sorokina, a chubby girl with brown eyes, was born in the city of Orekhovo-Zuevo near Moscow. She is 1 year and 11 months. The diagnosis is the first type, the most difficult.

Darina does not have a mother.

“She could not cope with the onslaught of such a heavy cargo - her daughter’s illness,” says Darina’s page on the BF website “Help Save Life”. - Her mother suffers from alcohol dependence and is in a serious psychological state. Она совершенно не способна заботиться о девочке».

21 апреля органы опеки забрали Дарину из семьи. «Сутки напуганная, измученная болезнью малютка провела в местной больнице под присмотром медсестер и волонтеров».

А потом о Дарине узнала Александра Франк, режиссер и основательница благотворительного фонда «Звезда на ладошке». Она стала законным представителем девочки и сейчас добивается для нее укола «Золгенсмы».

Франк рассказывает о ситуации, которая произошла после федерального консилиума, который проводила Елена Петряйкина, бывший главврач Морозовской больницы, ныне президент этого медучреждения и член Экспертного совета «Круга добра».

«Она сделала консилиум за нашей спиной, без лечащего врача, без представителей ребенка, без родителей и сообщила о решении консилиума по СМС. Сказала, что «Золгенсма» ребенку противопоказана, — рассказывает Франк. — Я отправила документ, который мы получили по почте в электронном варианте, на экспертизу.

И мы поняли: мало того, что там нет всех подписей и печатей, так еще и подписи наложены. Если подкрутить контрастность, то видно — там разная бумага».

Когда эту ситуацию предали огласке, Петряйкина признала, что «решение действительно было составлено с ошибками», и пообещала прислать «новую бумагу». Но через месяц по Дарине пришел тот же ответ — отказ.

Пока представитель девочки добивается жизненно важного лекарства, благотворительные фонды ведут денежный сбор. На сегодняшний день на «Золгенсму» собрано 25,9 млн рублей — это лишь 17,2% от необходимой суммы.

Мы хотим еще раз напомнить, что «Круг добра» был создан для того, чтобы облегчить получение дорогостоящей терапии, чтобы семьи не собирали космические суммы на лекарства в соцсетях и через СМС. Но, поскольку родители и представителей детей, больных СМА, не могут добиться закупки «Золгенсмы» через госфонд, они вынуждены снова возвращаться к сборам.

А сборы теперь почти не двигаются.

— В прошлом году мы нашли деньги для 15 детей. В этом году все сборы стоят, — рассказывает Лилия Цыганкова из фонда «География Добра». — Я звоню благотворителям, которые раньше жертвовали очень крупные суммы. Мне говорят: «Но мы уже в «Круг добра» деньги отчисляем». Считаю, что на этом этапе необходимо убрать информацию с сайта «Круг добра» о том, что фонд закупает «Золгенсму», чтобы не вводить в заблуждение родителей и благотворителей, поскольку ни одному ребенку помощь этим препаратом через госфонд не оказана. Семьи не могут даже подать заявку на «Золгенсму», не то что ее получить.

Маргарита Вулпе: «В телевизоре нам вещают, что «Круг добра» всем помогает. Но по факту это не так. Мы продолжаем сбор, а отклика у людей нет. Они думают, что родители мошенники. Нам остается надеяться только на неравнодушных граждан».

Alexander Tkachenko,

председатель госфонда «Круга добра»:

**— С начала своей работы «Круг добра» не закупил ни одного незарегистрированного препарата, хотя такая возможность фондом предусматривается. По какой причине незарегистрированные препараты не закупаются?**

— Закупка незарегистрированного препарата требует особой процедуры, и эта процедура регулируется специальным постановлением правительства. Это постановление вышло в конце апреля, и сейчас мы имеем право начать переговоры с производителем о цене. Препараты очень дорогие, и каждое решение о выделении таких объемов бюджетных ресурсов должно быть обосновано. Мы должны иметь не просто все основания, но и четкий ответ для общества, почему мы выбираем именно это лекарство. Потому что стоимость одного укола «Золгенсма» — более 100 млн рублей — сравнима по расходам со спасением 100 детей, например, с аномалией Эбштейна, гипоплазией левых отделов сердца и сложными комбинированными пороками. Стоимость самых сложных и дорогостоящих операций на сердце у новорожденных в условиях искусственного кровообращения — все лечение стоит 1,5 миллиона. И это спасает жизнь ребенка.

Более того, в разных странах производитель предлагает очень разные условия для закупки одного и того же препарата. Разница в цене одной дозы может быть в несколько миллионов. В некоторых странах производитель берет на себя ответственность за результат лечения, и в случае, если лечение препаратом не дало ожидаемого эффекта, стоимость существенно уменьшается. Мы сейчас прорабатываем условия взаимодействия с поставщиками.

Я понимаю, что детям некогда ждать, и мы стараемся максимально ускорить закупки. Но мы отвечаем перед обществом не только за быстрые решения, но и за эффективность лечения, и за то, чтобы условия поставок препарата в Россию были оптимальными и соответствовали современным требованиям.

**— Родители детей со СМА не могут добиться врачебного заключения на «Золгенсму». Они утверждают, что Минздрав запретил врачам федеральных клиник выдавать назначения на этот препарат. Можете ли вы подтвердить или опровергнуть эту информацию? Если запрет действительно существует, то что послужило основанием для его установления?**

— Вопрос о запрете лучше адресовать Минздраву. Я о таком запрете не слышал и сомневаюсь, что это в принципе возможно и законно. In my opinion, the problem lies in a different plane. Во-первых, врачи не выписывают «Золгенсму», потому что просто не знают, как работать с незарегистрированными препаратами, не обладают всей полнотой информации о самом препарате и во многих случаях боятся рекомендовать лекарство, действие которого для них еще пока не очевидно.

Подобные препараты могут быть рекомендованы только коллегиальным врачебным решением, таким, например, как решение федерального консилиума. Такие вопросы должны рассматриваться с учетом всех особенностей, и важно, чтобы рекомендацию давали врачи разных специальностей. Я сомневаюсь, что кто-то или что-то может помешать врачам, работающим в федеральных центрах, выписать ребенку лекарство, которое спасет ему жизнь.

Минздрав России не ответил на вопросы «Новой» до публикации материала.

How to help

Мартину Вулпе:

PAO Sberbank
Номер карты: 4276 4000 4366 4854
(получатель Маргарита Сергеевна Вулпе — мама)

АО АЛЬФА БАНК
Номер карты: 5559 4938 0532 9639
Карта привязана к телефону: +7 (985) 843-78-00
(получатель Маргарита Сергеевна Вулпе — мама)

ТИНЬКОФФ БАНК
Номер карты: 5536 9139 0125 4959
Карта привязана к телефону: +7 (962) 928-84-71
(получатель Мариус Вулпе — папа)

ВТБ БАНК
Номер карты: 5368 2902 3346 5311
Карта привязана к телефону: +7 (985) 843-78-00
(получатель Маргарита Сергеевна Вулпе — мама)

PayPal
margherita.tarasova@yandex.ru
Благотворительный фонд «География добра»

СМС на короткий номер: 7522
«Живи» и сумма пожертвования.
Например: Живи 500

MOLDINDCONBANK
Номер карты: 5574 8402 6849 1382 — MDL
(получатель Marius Vulpe — папа)

Кириллу Петрову:

Все карты оформлены на маму
Марина Владимировна ПЕТРОВА
+79269332294

СБЕРБАНК
по номеру телефона +7 (926) 933-22-94

ТИНЬКОФФ
по номеру телефона +7 (926) 933-22-94

АЛЬФА-БАНК
4584 4328 4992 0096

VTB
по номеру телефона +7 (926) 933-22-94

PayPal:
mospetrova@mail.ru

Яндекс кошелек:
4100116627350424

QIWI кошелёк:
+7 (926) 933-22-94

БФ «География Добра»
СМС «Кирилл ₽» на номер 7522

БФ «Алёша»
СМС на номер 2325 с любой суммой
(сумма делится поровну между всеми «смайликами»)

БФ «Милосердие»
СМС «Кирюша ₽» на номер 3434

БФ «Сострадание»
СМС «Кирюша ₽» на номер 3443

Служба помощи «Милосердие»
СМС с суммой пожертвования цифрами на номер 5275

Дарине Сорокиной:

Благотворительный фонд помощи детям, детям-сиротам и детям с тяжелыми заболеваниями «Помоги спасти жизнь»:

Наименование получателя платежа: Благотворительный фонд «Помоги спасти жизнь»
ИНН получателя платежа: 7736260191
КПП получателя платежа: 772501001
Номер р/c получателя платежа: 40703810338000004033
Наименование банка получателя платежа: ПАО «Сбербанк» г. Москва
BIC: 044525225
Номер к/с банка получателя платежа: 30101810400000000225
Назначение платежа: Благотворительное пожертвование для Сорокиной Дарины

Осуществить пожертвование можно через Сбербанк-онлайн: https://pszh.ru/pozhertvovaniya/onlayn/
Назначение платежа: Благотворительное пожертвование для Сорокиной Дарины

Осуществить пожертвование на сайте фонда с помощью банковской карты:
https://pszh.ru/pomoshch/detyam/perechen/sorokina-darina/

Осуществить пожертвование с помощью СМС-платежа:
Чтобы помочь Дарине, отправьте SMS на номер 7545 со словом «Дарина» и любой суммой пожертвования (например: Дарина 300).