The Russian Ministry of Health recommended including the drug for spinal muscular atrophy (SMA) "Spinraza" (nusinersen) in the list of vital and essential drugs (VED). The corresponding decision (nine votes "against", 11 "for") was made at a meeting of the departmental commission on the formation of lists of drugs.
Spinraza is considered one of the most expensive registered drugs in Russia. Taking the drug is required for life. The first course of therapy costs about 40 million rubles, and about 20 million rubles for each subsequent year. Without treatment, SMA patients gradually atrophy all muscles, including those responsible for breathing, while people remain conscious to the last.
Olga Germanenko, director of the SMA Family Foundation, who was present at the meeting, told RT that the discussion of the drug lasted about an hour and a half: "The discussion was extremely hot, there were many surprises, and it seems that the voting results surprised the commission itself."
Opponents of Spinraza's inclusion in the list suggested waiting until two other SMA drugs, Zolgensma and Evrisdi, were registered in Russia (the manufacturers of both submitted the relevant dossiers), compile a global list of patients and write down which drugs are suitable for whom, and also wait for the launch of the fund for financing rare diseases, which President Vladimir Putin spoke about at the end of June. In all cases, it would have been a minimum of a year of deferral, emphasizes Olga Germanenko.
In addition, representatives of the Ministry of Health and the Federal Antimonopoly Service, who opposed the inclusion of the drug in the VED list, referred to the high burden on the budget. At the same time, representatives of the Spinraza manufacturing company informed RT that the price offered for registration after the drug was included in the list would be lower than in the reference countries.
At a meeting of the commission of the Ministry of Health, a representative of Biogen noted that it is proposed for Russia to fix the cost of one injection at 5.6 million rubles, which is 25% lower than the current price of 7.8 million rubles, at which the regions buy the drug on average.
“In fact, the regions have to provide patients with medicine anyway, they just can do it two million cheaper for each ampoule. I think this was the decisive argument in the voting. It will be possible to reach more patients: every fourth patient will be able to receive the drug only at the expense of this discount, ”explained the director of the“ SMA Families ”foundation. “In addition, now the purchase price can rise, a package can cost both 9 and 10 million. And the inclusion of the drug in VED will fix the price ”.
Elena Maksimkina, Director of the Department of Pharmaceutical Provision and Regulation of Circulation of Medical Devices of the Ministry of Health, stressed that the conclusion of the commission is only a recommendation, the final decision is made by the Russian government. The timing of when the corresponding decree will be issued is still unknown. “Neither I, nor my colleagues will recall a case when, with a positive decision of the commission, the drug was eventually not included in the list. If this happens to Spinraza, it will be nonsense, ”says Olga Germanenko.
"Goosebumps"
A year has passed since the registration of Spinraza in Russia. Patients with SMA are entitled to free provision of medicines - for this, the conclusion of a medical council is sufficient. The inclusion of the drug in the list should give more reason to doctors to recommend Spinraza. However, in practice, cases when the drug could be obtained without much delay are extremely rare.
Dobryne, the son of Svetlana and Nikita Rukosuevs from the village of Boguchany, Krasnoyarsk Territory, has already given four Spinraza injections. He was given medicine only after his younger brother Zakhar died with the same disease, without waiting for the treatment he was entitled to. The Main Investigation Department of the RF IC for the Krasnoyarsk Territory and the Republic of Khakassia has opened two criminal cases under Art. 109 of the Criminal Code of the Russian Federation (causing death by negligence) and 293 of the Criminal Code of the Russian Federation (negligence).
The next Spinraza injection is scheduled for September 6, and Dobrynya's parents, despite assurances that it will take place on schedule, cannot hide their concern: “The coach said: we will continue to build up strength. But you must understand that if you stop taking the medicine, then everything will roll back. "
At the same time, the boy's parents did not expect to see such results from Spinraza so quickly. “Dobrynya has gained strength in his hands, he can push, pull, pinch my finger, quickly raises his hands - and earlier it could take him 10 minutes. There was a normal blood circulation in the muscles of the back, - Svetlana Rukosueva told RT. - He quickly and a lot rolls for a toy, started with five laps, and now he does 16. When you see this and compare it with what it was - goosebumps.
Svetlana calls the inclusion of the drug in the VED list “incredibly wonderful and important news”. But, according to her, there is still a lot of work to be done until 2021, including the development of a rehabilitation program for patients with SMA: "It is necessary to restore not only the musculoskeletal system, but also the respiratory system."
Hold out for six months
Another problem noted by parents of SMA patients: both the updated VED list and the allocation of funds from the increase in the personal income tax rate will start earning from 2021, while children should be treated immediately, emphasizes Yakov Redko, the father of two-year-old Vera. “So far, we, like many other parents, after a year of treatment as part of the early access program to Spinraz from the manufacturer, are collecting money to continue therapy,” he told RT.
Olga Germanenko says that the upcoming creation of a trust fund by increasing the tax was one of the arguments against the inclusion of Spinraza in the list: “The logic is this: next year there will be tax revenues, why should the regions now spend money, when then patients will be taken away and treated. But the mechanism proposed by the President is intended to complement, not replace, their assistance! If you wait until it starts working, then several dozen people, scampering through the courts, will irreparably lose their body functions, and some will die. "
According to her, patients are already faced with the fact that the regional authorities are trying in every possible way to stretch out the months remaining until the end of the year: "Up to administrative pressure on the judiciary, otherwise we cannot interpret what is happening."
However, some regions, on the contrary, are trying to find additional funds for the purchase of Spinraza from budgetary or extra-budgetary sources during these months, adds the director of the Family SMA Foundation: “There are few such entities, and it turns out to allocate 150-180 million, that is, assistance will receive a maximum of five patients. But this is better than nothing. "
“The medicine is very expensive, not all regions can afford it and are using all possible ways to postpone the purchase, including referring to the absence of the drug in the VED list,” Anastasia Gaponova, mother of two-year-old Varya, explained to RT. - Now doctors and officials will have one less excuse. It is as if we are waging a large-scale war, and we won one of the battles, although we won by a miracle. "