Photo: Victoria Odissonova/Novaya Gazeta »
On February 9, the State Duma held a meeting of the working group to amend the law on public procurement. The changes proposed by MP Dmitry Kuznetsov, employees of charitable foundations and doctors will affect the rules for the purchase of medicines. If they are adopted, they will quickly solve the problem of unavailability of original drugs: they can be purchased by trade names at the expense of the federal budget. This will also make it possible to provide those in need with pathogenetic therapy drugs that save the lives of patients with rare genetic diseases.
Changes to the law are dedicated to the memory of Eva Voronkova, a ten-year-old girl from Nizhny Novgorod, who died in November 2021 from cystic fibrosis. Eva's parents won the court, he ordered the regional Ministry of Health to immediately provide the girl with Trikafta medicine. But
the Ministry of Health was in no hurry, 51 days passed after the court decision, and Eva died without waiting for the drug.
Novaya Gazeta talked about the situation with the drug supply of patients with a leading Russian pulmonologist, head of the laboratory of cystic fibrosis of the Federal State Budgetary Institution Research Institute of Pulmonology of the Federal Medical and Biological Agency Elena Amelina. Using the example of the disease from which Eva Voronkova died and which affects almost four thousand people in Russia, she talks about what needs to be fixed in the system so that patients with rare diseases live long and full lives.
“The best investment of public money»
— In developed countries, people with cystic fibrosis live an average of 53 years. In Russia — 12 years. What is the reason for this gap?
— A patient with cystic fibrosis should, like any patient, receive adequate treatment when an exacerbation occurs. But the difficulty of living with cystic fibrosis lies primarily in the fact that treatment is required constantly, and not only in the [period of] exacerbation. And it is. Let's imagine the life of such patients, normal, not in exacerbation: a patient with cystic fibrosis gets up in the morning, inhales a bronchodilator, then he waits for this bronchodilator to work, then he inhales a mucolytic, a drug that dilutes sputum, then he spends an hour of kinesitherapy, coughing up this sputum, draining. Then, when he has already done all this, he makes another inhalation — he inhales an antibiotic. Then he has breakfast and drinks enzymes that allow him to digest food. And then he goes to college, to school, to work, because these are very active, very capable, very intelligent young people in most cases. And despite this treatment, they lead a very active life. And in the evening this procedure is repeated. This is if everything is fine and this is just the usual basic therapy, and the person feels good. And all this is joined by exhausting hours to achieve this treatment. If all over the world there is a problem of so-called compliance, the problem of the patient's readiness to follow the doctor's prescriptions, then here between the doctor and the patient there is still this huge problem — to get drugs.
Elena Amelina. Photo: ulmonology-russia.ru
— Where does it come from?
— In the nineties, when everything was very difficult, we knew for sure that we were prescribing an original drug to the patient, that is, a drug whose effect had already been tested on patients with cystic fibrosis. It was already known that these drugs help.
In the so-called «terrible dashing nineties» patients, at least in Moscow and the Moscow region, received these drugs,
only because the attending physician prescribed them. Now, unfortunately, we cannot recommend the drug to the patient under the so-called commercial name, that is, under the name given by the manufacturer. We can only recommend the chemical formula of this drug, name the active substance. And which company will reproduce this active substance? We have no influence on this choice.
FROM THE EDITOR
This mechanism has not always worked this way, and now this mechanism is conditioned by Federal Law No. 44, which regulates public procurement. It regulates the purchases, for example, of paper for printers at the MFC and chairs for some government agencies. And according to the same principle of preference for Russian manufacturers and the lowest price, they purchase medicines that are prescribed for health reasons.
— What does this mean for patients?
— For patients, this is fraught with the fact that these can be any drugs, of any production: Russian, Chinese, Indian, Pakistani. If, when choosing chairs for a cinema hall, it may be possible to proceed from the criteria of simply the optimal price, then this does not work here. Because the so-called «generics», not original drugs, [under current law] do not have to prove their clinical adequacy to the original. They only have to prove their chemical formula. Although the purification of the drug may be different. Not the same as the original. And if there is no study on a group of patients, in this case on patients with cystic fibrosis, then, of course, we do not know what effect it will have on our patient. Unfortunately, it is precisely in connection with the apparently low purity or low quality of production, which is not checked in this case, that we see a huge number of side effects. That is, in the midst of an exacerbation, when it is urgent to suppress the infectious process, instead, hemoptysis begins, which was not observed before with the same original drug, suffocation, itching begins, something that we cannot even imagine. Or the drug is well tolerated, but we do not observe the effect. We are waiting for the effect, and it does not come.
— In fact, the patient risks his life during an exacerbation?
“That’s how it works, that it is believed that we first need to fix the side effects of the drug. But the lack of effect is even more difficult to fix, and even original drugs may be ineffective in some severe cases. But first we must fix the side effects. That is, if this idea is thought through to the end, then the patient must first endure side effects, conduct an experiment on himself. And then again, in such a roundabout bureaucratic way, a medical commission will be held and, with the best outcome, the officials who are responsible for the supply of medicines and the institution will have the right to buy what this patient needs.
— In developed countries, a new word in the treatment of genetic diseases is targeted therapy. These are the most expensive drugs in the world, but they really do a miracle — they save lives. If in Russia it is so difficult with basic therapy, then how are things with pathogenetic therapy?
“The invention of targeted therapy began as very good news. And this is what we have all been waiting for for many years. For twenty years we have been saying to each other, to ourselves, and to patients that it is now necessary to improve symptomatic therapy as much as possible, because pathogenetic and genetic treatment will soon appear and this will completely change the lives of patients with cystic fibrosis. And it happened.
At first it was some incredible joy for us when we went to the convention and heard that yes, there were new drugs. The first such drug for a fairly narrow number of mutations, for a small number of patients, appeared in 2013. Now there is Trikafta, which is suitable for 90 percent of patients and is approved for use from the age of six. » />
Trikafta
— What can Trikafta do for a patient with cystic fibrosis? When should he receive it? For how long?
This product is really very easy to use. That is, if you imagine the treatment that I just talked about (frequent intravenous therapy, inhalation, and so on), then this drug can simply be taken in a pill twice a day.
Very rarely in medicine there are magic pills, but apparently, this is really the case.
But the patient must receive this therapy throughout his life. Now there are observational studies that say that this effect potentiates and increases over the life of the patient and with the continuation of this treatment. The number of exacerbations is significantly reduced, that is, the need for hospitalizations, intravenous therapy decreases, the number of complications that accompany cystic fibrosis decreases. And when using targeted therapy, the weight of patients increases by eight to ten kilograms, and very quickly: in a few months. Moreover, patients suffered from insufficient weight all their lives. We have a patient, she started targeted treatment in 2016, her health improved so much, and extrapulmonary problems resolved so much that she gave birth to a child without interrupting targeted therapy. Indeed, when we read about it, of course, we should feel a sense of joy…
— What darkens this feeling? Medicine price? Is it available to Russian patients?
— Yes, here this carriage very quickly turns into a pumpkin. The cost of the drug is more than 20 million rubles a year. And the patient must receive it throughout his life. If you stop taking it, the effect ends in a fairly quick time. And there may even be the strongest rollback in the state. And it is impossible to leave a patient who has already started targeted treatment without treatment. If you imagine, firstly, the impact that he has on the life of a very young person, a child, a teenager, then, of course, this makes a huge impression. And, besides, if we imagine the unimaginable costs, both human and economic, which require the treatment of a severe patient with cystic fibrosis without targeted therapy, an endless course of antibiotics, with mycobacterial infection, continuous intravenous courses that last a year, a year and a half, two years , endless hospitalizations, severe situations with hemoptysis, with embolization of bronchial arteries. I don't know, everything I listed is worth twenty million or not? I don't know.
Eva Voronkova. Photo from the family archive
— You can't calculate the price of life. But there is no need to do this in a country where there is money for such medicines, they are simply spent in the wrong place. But isn't there a huge difference in the quality and length of life without and with targeted therapy?
— It's crazy to compare it in general. If you imagine what a person and his relatives go through, faced with the complications of the disease, and imagine that taking two pills a day can prevent this, then, in general, I do not know what economic equivalents can be expressed. There is no better use for public money. Simply, if you imagine what the money is spent on, I will not list it now. Here
this is the most efficient use of public money imaginable.
That is, these are young, active, energetic people who will lead, live a normal life, will create their own families, will be high professionals or non-professionals, but, in general, this is really a great chance …
***
Starting from 2022, the provision of targeted drugs to patients with cystic fibrosis according to expanded criteria from 6 to 18 years old was taken over by the state fund for helping children with rare diseases “Circle of Goodness”. This is the beginning of a big change in the lives of hundreds of children with cystic fibrosis. However, many regional ministries of health do not fulfill their duties and refuse or hinder the execution of applications for the Circle of Kindness fund, delaying the process, thereby depriving little patients of a chance for life and health.
The most difficult situation is with the preparation of the necessary documents in Moscow and Moscow Region, Krasnodar Territory, Republic of Tatarstan, Rostov, Vladimir, Smolensk Regions, Stavropol Territory, Chechnya, Dagestan, Buryatia and Altai Republic. Some regions did not submit a single application at all — these are the Pskov region, the Komi Republic, the Tver region.
In adult patients, the situation with receiving pathogenetic therapy is even worse. Patients in serious condition, who have crossed the threshold of 18 years, are forced to sue the regional ministries of health for their right to life, receive endless refusals, rescheduling meetings and replies. Many of them travel to court sessions with oxygen concentrators, endanger their lives during hospitalizations in regional centers for illegal repeated consultations in general wards without observing sanitary standards for separating patients with different bacteria. And they cannot get the life-saving medicine that they are entitled to by law.
Patients, doctors and representatives of public organizations sent a collective letter to the Chairman of the Government of the Russian Federation Mishustin M.V. with the requirement to provide adult patients with Trikafta. They also launched a public campaign under the hashtag #TrikaftaChallange «Growing up scary», where they tell their stories of the fight against the disease and the healthcare system and demand that they be provided with a life-saving drug.