There are no ones

The Ministry of Health spoke about the number of patients with SMA, palliative care and access to drugs. Parents and experts criticized the letter of the department

On February 20, New Gazeta sent an official request addressed to the deputy chairman of the government of Tatyana Golikova with a request to comment on the quality of medical care, which is in Russia to patients with spinal muscle atrophy (SMA), a rare genetic disease. We asked the Deputy Prime Minister whether the Ministry of Health plans to calculate patients and create an official register whether he intends to establish palliative help and facilitate the access of the patients to expensive imported drugs from the SMA-Spinras and Zulgensme.

The “new” also asked questions about the bill on the payment of drugs for patients of the SMA from the federal budget, which, according to the co -authors of the document, was rejected in the State Duma for formal reasons. The last question of the editorial office concerned the statement of Prime Minister Mikhail Mishustin that more than 1.1 trillion of budget rubles was not spent over the past year. We were interested in whether it is possible to request the unused money for the treatment of patients with SMA, and if so, how to do it most promptly.

What did the Ministry of Industry answer?
A week later, a letter from the Ministry of Health came to us - the department for the first time since the beginning of our campaign in support of patients SMA officially responded to the request of the editorial office. Referring to the data of the SMA Family charity foundation, it reported that in Russia there are about a thousand patients with such a diagnosis, but this information should be clarified.

“On February 14, 2020, the Ministry of Health of Russia held a meeting with the participation of relevant experts on the issue of providing patients with the cubes with the drug“ Spinraz ”, as a result of which a request was sent to the regions to clarify the number of patients and confirm the need for this drug,” the letter said.

About whether the Ministry of Health intends to create a register of patients is still not reported.

“As for the provision of palliative care, the subjects of the Russian Federation are provided with subsidies to provide patients with medicines and provide medical organizations with medical devices, including for use at home,” continued the Ministry of Health. “In particular, the problem of providing patients with Ivl apparatuses ( artificial lung ventilation - E.K. ) was solved for use at home.”

In addition, the Ministry of Health recalled that the registration of drugs in Russia “is declarative in nature”, and that the manufacturer himself must contact the department to begin this procedure.

In this regard, officials note, now Zulgensma is not registered in the country. There was no information from representatives of the Russian Ministry of Health from representatives of the Russian Ministry of Health from representatives of the Russian Ministry of Health.

Unlike spinras - according to the department, this drug is available to children as part of the open access program. Despite the fact that the medicine is registered and allowed for medical use, the Ministry of Health instructed specialists to submit a protocol of its clinical testing, since "the drug is not sufficiently studied by the drug and possible side effects and complications."

The situation with the abolition of the bill neither in the Ministry of Health or in the apparatus of Golikova did not comment. The press service of the Deputy Prime Minister redirected the issue of inexplicable budgetary funds to the Ministry of Finance.

We have not yet received the response of the department.

"New" turned to experts and parents of children with a request to comment on the letter of the Ministry of Health. In their opinion, the department did not understand the problem and tries to postpone the adoption of serious decisions that could help people with SMA.

Artem Dmitrienko

Timur's father (1 year and 1 month, diagnosis - SMA I and II, Zelenograd)

- This is a terrible letter, it would be better if they did not write anything. The Ministry of Health is still not aware of the problem. From this nonsense, the hair rises on end on children. It would be more honest to write: “Guys, there is a problem, we understand”, and not send such unsubscribes. A lot of time has passed so that the Ministry of Health could prepare and understand the issue, but he once again showed his incompetence.

Several times in a letter, officials contradict themselves. The first - they say that a thousand patients are allegedly registered in Russia. They made such conclusions on the basis of the data of the Family of the SMA, which keeps at least some accounting, and then only for themselves.

Below they write that only now they make a request to the subjects of the federation in order to calculate the exact number of patients. It immediately becomes clear that the Ministry of Health, as he did not deal with this issue, does not deal with. The disease is such that children can be born with this diagnosis every day.

It should be detected by perinatal screening, which has not yet been introduced for the lubricants.

The second - the Ministry of Health boasts that we have registered Spinraz, and by the fact that there is supposedly there is an open access program. And here they did not understand the problem. Most likely, we are talking about an extended access program, within the framework of which about 40 children received this drug for free [from the manufacturer] for one year.

Probably, the Ministry of Health had in mind this, because now no one “spinrase” gives patients to patients. Let them give at least one example. At the same time, in the letter, the department writes that the medicine requires an additional test. How could the Ministry of Health register it and give it to open access if it is unverified and needs clinical trials? In Europe, it has been successfully used for three years from birth after screening: firstly, for free, and secondly, in a timely manner.

Third, the “Zolgensme” has opened an extended access program, a lottery for children from around the world. We learned about this program in early December. But in order to take part in it, it was necessary that our Ministry of Health give an order to the doctors. For two and a half months we persuaded doctors to submit an application. Doctors under the pressure of parents themselves understood how to do it. No support from the state was provided. Already February, and we only now received an answer from the doctor that we can take part in the lottery. If the Ministry of Health was interested, then he would have caught this story for a long time, and all the doctors would be prepared.

About palliative assistance is not true.

In the case of Moscow, really, if you run and ask, they will help. Moreover, only hospices help quickly - those structures that have been working with this for many years. In order to get palliative assistance from the Ministry of Health, you need to run a million instances - even in Moscow. Patients with SMA in the regions often cannot get all this. If the child dies and practically cannot breathe, maybe Ivl will be given to him promptly.

But in order to in advance, this is not.

If, as the Ministry of Health writes, “the problem is solved”, then why are 30–40 families who are faced with SMA, gather on their own on the “spinrase” and on these same Ivl devices?

Victoria Zaitseva

Yegor's mother (13.5 years old, diagnosis - SMA III, Moscow)

- A thousand patients in Russia are only approximately. The register is maintained by the Family SMA Foundation, which includes far from all patients. Some do not know about this registry and even the fund at all. Not all of them are active Internet users.

Not all neurologists have heard about SMA, a rare disease.

There are still cases when the patient was incorrectly diagnosed: there are many neuromuscular diseases. In this case, it is necessary to do DNA analysis, but not everyone can pay it, but often get a direction.

As for the open access program, I do not really understand what is meant. In federal clinics, only 42 patients under the humanitarian program that the manufacturer pays for the humanitarian program pays for treatment at the moment. She was designed for a year. In May, the program ends, and what to do to these patients further is unknown.

What is the procedure for obtaining a “spinraise”: I go to the clinic, the doctor sends to a neurologist, I write a statement to conduct a medical commission where the prescription of the drug would be spelled out. Since Spinraz is the only possible pathogenetic treatment at the moment, doctors give me paper with indications for its use. I take this paper, go to the consultation or medical commission to the federal clinic, for example, to Morozovskaya. They confirm that my child really has such a diagnosis, and approve the prescribed medicine. I send all the documents to the Ministry of Health, they purchase it, and I stake the drug to the child.

But in fact, everything happens differently. Rather, it does not happen. In my hands now there is a conclusion of a consultation of the Morozov hospital. There is no more information.

From the moment of registration of Spinraza, seven children and several adults have already died, and without receiving it.

My son has a third type. For my son, Zulgensma is irrelevant, Spinraz would now help us. But what to do and where to go - I do not understand yet. Open access ... For whom is this access open? My child was diagnosed more than ten years ago, but he does not receive any treatment.

Thank God, we have no respiratory disorders. So far, Ivl is not shown and is not required.

But my son had already asked to promise him that if he reaches Ivl, he would not be able to swallow and have to use the gastrostal so that I would let him die.

It turns out that every family, every patient faced with SMA, should make a choice: to fight in all possible ways or to die for a long time and painfully. Our children and adults are well aware of what awaits them without treatment in the future. With this disease, deterioration is growing quite quickly. But if you start treatment as soon as possible, then this process can be completely stopped.

Alexey Krayukhin

Lisa's father (1 year 4 months, diagnosis - SMA I type, village Boyarka, Sverdlovsk region)

- The palliative service, at least in our case, really quickly issued the entire necessary set - we call it “resuscitation”.

In the case of relapse, the ambulance simply does not have time to get to the addressee, and constantly keeping the child in the hospital is not an option.

Regarding the effectiveness of “spinras”-this is still not a treatment, but therapy that allows the patient to receive the necessary protein. After the injection, the patient will again receive regression. In our case, you need to get a “spinraise” as soon as possible to fix the skills of Lisa and then “Zulgensm”. American clinics are talking about this.

Olga Germanenko

Head of the SMA Family Charitable Fund

- We see the Ministry of Health attempts to collect data on patients from last October. When we had a meeting in December, we were given an act that information was going to the regions: about 50 subjects were answered and counted about 700 patients. We see a low response, and I do not know how much these statistics are qualitatively prepared. I would like to hope that this will lead to the creation of accounting and setting patients for constant therapy.

I can’t say that the problem with obtaining devices for respiratory support and bickering is 100 %resolved.

In order to get equipment now, the regions must allocate subsidies, and this is always unreliable: this year they gave money, but in the next one. We are attached to certain limits. In addition, we have a rather inconsistent system of markings of medical devices, when completely ambiguous devices are in one category: Ivl for 150-200 thousand rubles and an outpatient bag for two thousand. The routing of equipment and documentation has not been developed. Sometimes the device has to wait six months, which the patient does not have. This is a reality by a kind of solved problem, which has a lot of thin, free issues. But I can’t say that she does not dare at all. In 2013-2015, for example, nothing was available at all.

As for Spinraza, we once again see uncertain statements that the drug is new and "we know nothing." Judging by this letter, clinical testing is the only option for the development of events that is now being considered.

This is not very happy with us, because the very fact of clinical testing suggests that there will be some additional testing of the effectiveness and safety of the medicine. As far as we understand, no one will say how the testing will take place, how many patients, how much time it will take - this raises a lot of questions. What is all this time to do people who need treatment right now?

As a rule, such things are carried out when there is already experience in applying the product and additional data are needed. In our situation, the regulator, which independently checks the data of manufacturers, approves and registers drugs. And then literally six months later he says: "Oh, we are not sure of something."

This is strange - you have already approved the drug and began to use. If you have any questions about security, it really makes sense to conduct clinical research. But at the moment, according to our data, patients who are involved in early access programs, no side effects that would not be described in clinical studies.

Worse than side effects than the disease itself and what it leads to, probably cannot be.

There is a feeling that real decisions that can help patients are trying to replace with simpler and more cheaper. Thus, once again, tightening the time, which patients with a grease do not.

PS

"New" decided to send a second request to clarify the information provided by the Ministry of Health.

We ask you to answer the following questions:

Why, when assessing the number of patients, the Ministry of Health relies on the data of the charity fund? When will the state register be created?
Given the number of complaints from patients about the speed of palliative care, was a study on the effectiveness of the current procedure for obtaining the necessary equipment?
What is the deadline set to clarify the needs of the regions in the Spinraz?
After receiving this information, from which budget, federal or regional, treatment will be financed?

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To consolidate in the Constitution the right of children with rare diseases for medical care: petition