SMA: a sentence without hope?
They are called SMA, but with a "smile", unfortunately, they are not associated, because the name is from the incurable diagnosis of SMA - spinal muscular atrophy. It affects 1 in 10,000 newborns, and every 40 inhabitants of the Earth are carriers of the gene, regardless of gender, nationality and age. The disease is rare genetic, associated with pathology of the nervous and muscular system, which restricts children’s movements, depriving them of the ability to walk, hold their head, move limbs, breathe and swallow. There is no treatment, there is only maintenance therapy with expensive injections, without which smilies do not live up to two years. The Ministry of Health of Ukraine does not keep a register of such children or mortality statistics, and de jure does not even have the right to set up a commission to finance such diseases. Therefore, the parents of Smileys can only rely on their own strength or with the help of charitable foundations,
The cost of children’s lives
Depending on the type of SMA in the world there are only 3 types of drugs:
"Solgensma" - suitable for type I SMA. A single intravenous injection worth $ 2.1 million to $ 2.3 million.
"Spinraza" - suitable for all types of SMA and for any age. Injection into the spinal canal, the first year - 6 injections, then 3 injections per year, one injection - 68 thousand dollars, 2.2 million dollars. for 10 years.
"Risdiplam" - suitable for all types of SMA, age - from 2 months. Probe nutrition gel, once a day, $ 213,000 per year and approximately $ 2.1 million. for 10 years.
Another option is home pediatric resuscitation, which is also a device (primarily to support breathing) costing tens of thousands of dollars and round-the-clock supervision of parents with knowledge of complex medical manipulations.
In some developed countries (Switzerland, Turkey, Spain, France, Germany, Poland, etc.), if parents fail to raise funds, the state provides free lifetime assistance in providing drugs. To date, Ukraine has only passed the stage of registration of drugs - "Spinraza" and "Rysdiplam". The inclusion of these drugs in the List of medicines purchased from the state budget is out of the question. Preliminary rhetoric of the Ministry of Health - a large number of patients, so to finance expensive treatment of each patient government agency will not pull.
Although, again, there are no official statistics, there is only a voluntary register approved by the Kharkiv Charitable Foundation "Children with SMA" , which has 267 children. According to the fund’s management, the annual amount of 1.8 billion UAH. from the state budget would be enough to treat all children with SMA in Ukraine.
Lotteries, foundations and courts as a chance to save young patients
In Ukraine, there are children who still receive treatment, and therefore have a chance to live. But these are only isolated cases, exceptions, fart.
In November 2020, the Zelenskys from the town of Bakhmut received the good news that their son, 14-month-old Elisha from the SMA, will receive a free injection worth $ 2.3 million. All thanks to the lottery for free distribution of 100 doses of Zolgensma, conducted by Novartis. The boy’s parents applied for participation and fate smiled on them.
Another family was recently helped by the Odesa Charitable Foundation "Monster Corporation" headed by Kateryna Nozhevnikova. We managed to collect UAH 790,000 in a few days. to the already collected 1 million 340 thousand UAH. for the girl Polina to be sent for surgery to Germany to install special rods along the spine, which can later be pushed apart by a magnetic device. Also, little Polina is a participant in an expanded humanitarian program and receives the drug "Risdiplam". This program accepted Ukrainian children until May 2021.
But at the end of 2020, the parents of a child with SMA Maxim Geary, in general, won the interim lawsuit "Gear against Ukraine" in the European Court of Human Rights and his decision obliged Ukraine to pay for the treatment of their sick child.
Support in the initiatives of those who care
According to the National Assembly of People with Disabilities of Ukraine, the Kharkiv Children’s Charitable Foundation "Children with WMD" is the only Ukrainian organization that is a member of the international community of people with disabilities and is recognized outside Ukraine. . For 15 years in a row, the foundation, headed by Vitaliy Matyushenko, the father of a 17-year-old daughter from the SMA, has been involved in educational activities, participating in legislative work, conducting research, trying to attract philanthropists to support such families. in such situations, provides psychological assistance. According to the president of the fund , the state is not interested in patients who do not bring it money, but need significant funds from it.
There are also many legislative gaps. For example, orphan diseases are not separated from palliative care. The inclusion of orphan patients in the list of those in need of palliative care makes it virtually impossible to develop and fund targeted programs to purchase drugs for people with orphan disease.
Is there hope?
At the end of January 2021, during a briefing, the head of the Ministry of Health, Maxim Stepanov, spoke openly about the existing problem and the procedural moment of its solution - controlled access agreements. This is a mechanism for purchasing innovative expensive drugs at the expense of the budget in the absence of such drugs in the country.
Concluding controlled access agreements is a multi-level and time-consuming process. But on the other hand, such official statements are already a big breakthrough for the last 10 years and the hope that children with SMA will still have a chance to be treated in Ukraine. Also, on January 28, the Nation’s Health Committee registered Bill 4662 "On Procurement of Medicines under Controlled Access Contracts."
This suggests that there are other documents that can regulate such procedural issues. So, the problem now is not HOW, but - When? And while the necessary papers are being transferred from office to office, parents of children with SMA are forced to raise funds on their own, with the help of caring people and charitable foundations.
Author Kateryna Stebelska
Other news and notes
The cost of children’s lives
Depending on the type of SMA in the world there are only 3 types of drugs:
"Solgensma" - suitable for type I SMA. A single intravenous injection worth $ 2.1 million to $ 2.3 million.
"Spinraza" - suitable for all types of SMA and for any age. Injection into the spinal canal, the first year - 6 injections, then 3 injections per year, one injection - 68 thousand dollars, 2.2 million dollars. for 10 years.
"Risdiplam" - suitable for all types of SMA, age - from 2 months. Probe nutrition gel, once a day, $ 213,000 per year and approximately $ 2.1 million. for 10 years.
Another option is home pediatric resuscitation, which is also a device (primarily to support breathing) costing tens of thousands of dollars and round-the-clock supervision of parents with knowledge of complex medical manipulations. In some developed countries (Switzerland, Turkey, Spain, France, Germany, Poland, etc.), if parents fail to raise funds, the state provides free lifetime assistance in providing drugs. To date, Ukraine has only passed the stage of registration of drugs - "Spinraza" and "Rysdiplam". The inclusion of these drugs in the List of medicines purchased from the state budget is out of the question. Preliminary rhetoric of the Ministry of Health - a large number of patients, so to finance expensive treatment of each patient government agency will not pull.
Although, again, there are no official statistics, there is only a voluntary register approved by the Kharkiv Charitable Foundation "Children with SMA" , which has 267 children. According to the fund’s management, the annual amount of 1.8 billion UAH. from the state budget would be enough to treat all children with SMA in Ukraine.
Lotteries, foundations and courts as a chance to save young patients
In Ukraine, there are children who still receive treatment, and therefore have a chance to live. But these are only isolated cases, exceptions, fart.
In November 2020, the Zelenskys from the town of Bakhmut received the good news that their son, 14-month-old Elisha from the SMA, will receive a free injection worth $ 2.3 million. All thanks to the lottery for free distribution of 100 doses of Zolgensma, conducted by Novartis. The boy’s parents applied for participation and fate smiled on them.
Another family was recently helped by the Odesa Charitable Foundation "Monster Corporation" headed by Kateryna Nozhevnikova. We managed to collect UAH 790,000 in a few days. to the already collected 1 million 340 thousand UAH. for the girl Polina to be sent for surgery to Germany to install special rods along the spine, which can later be pushed apart by a magnetic device. Also, little Polina is a participant in an expanded humanitarian program and receives the drug "Risdiplam". This program accepted Ukrainian children until May 2021.
But at the end of 2020, the parents of a child with SMA Maxim Geary, in general, won the interim lawsuit "Gear against Ukraine" in the European Court of Human Rights and his decision obliged Ukraine to pay for the treatment of their sick child.
Support in the initiatives of those who care
According to the National Assembly of People with Disabilities of Ukraine, the Kharkiv Children’s Charitable Foundation "Children with WMD" is the only Ukrainian organization that is a member of the international community of people with disabilities and is recognized outside Ukraine. . For 15 years in a row, the foundation, headed by Vitaliy Matyushenko, the father of a 17-year-old daughter from the SMA, has been involved in educational activities, participating in legislative work, conducting research, trying to attract philanthropists to support such families. in such situations, provides psychological assistance. According to the president of the fund , the state is not interested in patients who do not bring it money, but need significant funds from it.
There are also many legislative gaps. For example, orphan diseases are not separated from palliative care. The inclusion of orphan patients in the list of those in need of palliative care makes it virtually impossible to develop and fund targeted programs to purchase drugs for people with orphan disease.
Is there hope?
At the end of January 2021, during a briefing, the head of the Ministry of Health, Maxim Stepanov, spoke openly about the existing problem and the procedural moment of its solution - controlled access agreements. This is a mechanism for purchasing innovative expensive drugs at the expense of the budget in the absence of such drugs in the country.
Concluding controlled access agreements is a multi-level and time-consuming process. But on the other hand, such official statements are already a big breakthrough for the last 10 years and the hope that children with SMA will still have a chance to be treated in Ukraine. Also, on January 28, the Nation’s Health Committee registered Bill 4662 "On Procurement of Medicines under Controlled Access Contracts."
This suggests that there are other documents that can regulate such procedural issues. So, the problem now is not HOW, but - When? And while the necessary papers are being transferred from office to office, parents of children with SMA are forced to raise funds on their own, with the help of caring people and charitable foundations.
Author Kateryna Stebelska
Other news and notes