"This medicine saved the life of the baby and the entire family, there is no other way to describe it."
This is how, excitedly, Hana Raginov of Kiryat Bialik describes the effect of the expensive and innovative drug that her one-year-old son, Osher, has been receiving for the past two months to treat his liver disease.
"Now Osher is able to function, his indicators have improved, and our lives, as parents, have also been saved. Since the treatment, Osher no longer suffers physically, and this gives him the freedom to simply be happy. He is finally able to do what a one-year-old baby is supposed to do - smile and laugh - and I This is all a mother wishes for.
"The problem is that today we receive the medicine as part of 'compassionate treatment' which is limited in time, and I really pray that the medicine goes into the basket, so that we can continue to live."
Osher the little is diagnosed with pifik, a rare genetic liver disease, which affects about five patients in Israel each year.
The disease is considered life-threatening and hereditary, and it affects the transfer of bile salts.
Its symptoms appear during the first year of the patient's life, and they include severe jaundice, hepatitis, developmental delay, and extreme itching that is intolerable to the point of bleeding - which leads to dysfunction, inability to sleep, and severe impairment of quality of life.
These days the patients and their family members are struggling to include in the state medicine basket a modern and expensive medicine called "BYLVAY", which is considered for the first time to be suitable for patients with the cruel disease, and whose cost is about NIS 400,000 per year.
However, in the meantime, until the drug basket committee finishes its deliberations at the end of the month, Osher receives the drug as "compassionate treatment" on behalf of the pharmaceutical company, temporarily and free of charge, as part of a special program of the company.
Prof. Ronan Arnon.
"The positive crime is clearly evident", photo: Michelle Dotcom
Prof. Ronan Arnon, a senior physician and head of the pediatric liver department at the Institute of Gastroenterology at the Rambam Government Medical Center in Haifa, has been accompanying Osher and his parents, Hana and Avner, for the past year. to him.
Osher did not respond to the available medications, and many times, while talking to his parents on the phone, I would hear him screaming in the background, and it would break our hearts.
"All this until 'Billway' arrived and changed the situation completely. The positive effect of the medicine, which is submitted to the medicine basket, is clearly evident, and Osher is now a calm, sweet and smiling baby, and his parents have also received significant relief."
Buy medicine in full money
Osher is not the only patient who receives expensive drugs that are not yet included in the basket.
According to the estimates of senior doctors and senior officials of the hospital associations, in Israel there are still hundreds of patients who are struggling with the basket committee with a request to include in the basket an expensive and new medicine, which they receive only as a temporary treatment - which is proven to be able to save them.
In doing so, they actually serve in their bodies as living proof of the critical - and urgent - necessity of putting the medicine in the basket.
The Medicines Basket Committee, photo: Yossi Zeliger
The patients receive the expensive drugs in various ways, including "compassionate treatments", studies that take place with the drug in Israel, donations from the drug companies, financing through commercial health insurances, and even buying the drug independently and in full.
Sometimes the medicine comes through the health insurance funds, through the exception committees that discuss requests to finance medicines that are not in the basket.
The State Medicines Basket Committee, chaired by Prof. Dina Ben Yehuda, discussed hundreds of new medicines and medical technologies costing about NIS 3.5 billion, which were submitted this year.
At the end of its deliberations, towards the beginning of March, the committee will choose which new drugs will be added to the public basket in 2024, with a budget of NIS 650 million.
"got life again"
Myasthenia gravis is a rare and life-threatening autoimmune disease, characterized by the weakening of the body's muscles, and it causes severe damage to the muscle nerve that leads to muscle fatigue.
Most patients begin with symptoms in the eye muscles, and later develop additional symptoms of impaired speech, swallowing and breathing.
Sometimes these patients reach difficult situations of breathing difficulties, up to the need for ventilation, or a physical disability that requires crutches or a wheelchair.
Some patients show cognitive impairment, and others will even suffer from depression and anxiety.
The disease can develop at any age, including children, but it is more common among women aged 20-40 and among men aged 60-70.
Now about 40 patients with myasthenia gravis are struggling to get a new drug called "Viogart" into the basket, which is given by intravenous infusion and causes the antibodies to be removed from the blood.
Its cost: about 750 thousand shekels per year.
Bat Sheva Plai-Sri.
"I started to feel like a human being", photo: Oren Ben Hakon
One of the patients is Batsheva Plai-Sari (58) from Alon Shavut, who is already receiving Viogart through her private health insurance.
"About six months ago I started taking the medicine, and this is the first time in decades that I started to feel like a human being. Suddenly I could do basic actions, like comb my hair, eat a carrot, be able to move my lips, talk even in the afternoon and evening and breathe without difficulty. These are things that used to be like climbing Everest for me.
"The treatment with Viogart allows me to keep my head above water, and I am currently receiving the treatment as part of the insurance, temporarily. The thought of the day when I will no longer be able to receive treatment terrifies me, and I pray that Viogart will enter the basket this year so that I can continue to live."
Prof. Adi Vaknin-Mambinski, a senior neurologist at Hadassah Ein Kerem, reveals that she witnessed with her own eyes the effects of the new treatment that Batsheva is undergoing.
"Within a short time, she showed a significant improvement in her symptoms, to the point that today she can already go for long walks, breathe, and above all, function almost as before the disease. It would not be an exaggeration to say that she got her life back. That is why it is important that we, as doctors, have the tools to help In alleviating the severe symptoms, tools that may save patients from a life-threatening situation."
The new treatment for dwarfism
Achondroplasia is a genetic mutation that affects the growth of long bones in the body and causes dwarfism.
This is a rare genetic disease, which manifests itself in short limbs and large head bones, and it causes severe neurological problems, sleep apnea, cardiovascular diseases in old age and chronic pain that accompanies the patients throughout their lives.
The Vitus family, Ortal, Nir and Shahar.
The medicine helped the height, photo: no
Now about 50 families of patients with achondroplasia are struggling to get the drug "Vaxogo", which is considered the world's first treatment for the disease, into the basket.
The treatment is given from the age of 2-5 until the end of the height growth process - in the teenage years.
The cost: about one million shekels per year per patient.
One of the families struggling to get the medicine into the basket is that of Shahar Vitus, 13 years old from Hod Hasharon, who has been receiving the medicine for about two years through funding from the private medical insurance.
"Shahar is currently in the seventh grade, and he is 1.25 m tall, equivalent to the height of a child in the first grade," says his mother, Ortal.
"Shahar is the first in Israel to receive a 'Vaxogo', at the beginning of 2022, and since then we have seen a real improvement in his height. To our delight, Shahar has grown by almost 10 cm in two years, which is a very significant change, right in line.
"It is important to understand that a child who receives this treatment turns from dependent on others into an independent adult. It will affect him for life, in so many ways. There is a very short window of opportunity to receive the treatment, until the age of 16-17, and the meaning is that short children simply do not have time to wait."
Shahar himself is also moved by the effect of the medicine on his condition: "A few days ago, for the first time, I suddenly noticed the difference. I said to my mother: 'Look, I'm making it, I can't believe it.' I accepted, which restores the possibility to conduct oneself independently."
Dr. Zeitlin, photo: Gideon Markovich
Dr. Regev. "Breakthrough", photo: None
Shahar is treated and accompanied by Dr. Leonid Zeitlin, Director of the Achondroplasia Center in the Pediatric Orthopedic Department at the Dana Children's Hospital at the Ichilov Medical Center in Tel Aviv, and by Dr. Royat Regev, a senior physician in the Endocrinology and Diabetes Unit at Dana Hospital.
Dr. Regev: "This is a breakthrough, and a patient who starts treatment early enough will be able to reach a functional level and live an independent life in adulthood.
The inclusion of the treatment in the basket of medicines in Israel is critical for patients, because they cannot receive it in any other way."
The laser that restored function
One of the most important battles in Sel - in the field of medical technologies - is around the introduction of the "Visualize" technology, which enables laser burning of brain tissue in a minimally invasive method, while monitoring in real time under MRI.
The main advantage of the technology is the ability to surgically reach metastases or primary tumors in the brain without harming vital tissues that are in the access route, as is the case with the accepted traditional method of open brain surgery - a method that can lead to severe neurological damage that manifests itself in motor or cognitive damage.
At the moment, the technology is not included in the health basket, and the treatment is being used as part of a unique program only at the Ichilov Municipal Government Hospital.
After the technology is put into the basket, the experts estimate that the treatment will be intended for about 50 patients every year, at a total cost of about NIS 5 million per year.
The program at Ichilov is led by Dr. Ido Strauss, director of the functional neurosurgery unit in the hospital's neurosurgical system and one of the leading neurosurgeons in Israel.
Dr. Strauss. Surgery safely, photo: Gideon Markovich
Dr. Strauss recently wrote to the Hassel Committee: "This technology allows the surgeon to safely treat lesions located in deep parts of the brain, even near functional areas.
Also, since the procedure is minimally invasive, it reduces the risks and discomfort associated with open craniotomy surgery, and allows most patients to go home the very next day.
"Although the technology is already in use in more than 200 hospitals around the world, due to the economic barriers in Israel it is currently only available at the Ichilov Hospital in the center of the country, which contributes to the inequality in access to advanced medical services between the center and the periphery.
For patients who need surgery, there is no other good treatment solution, and I recommend adding it to the health care basket."
The patient Jacques Franz (70) underwent the operation with the new technology in Ichilov in March 2022. "In June 2023, my brain returned to its normal state, after the removal of the metastasis. I returned to who I was before. I was very lucky and also a miracle to know the right doctors, who know how to recognize the the situations quickly and to refer to the best treatment methods. Thanks to the new technology and thanks to them - I am alive today."
The Medicines Basket Committee stated: "The members of the committee, including senior doctors, professional officials and public representatives, are currently discussing hundreds of new technologies that have been submitted to the basket. The committee has not yet finished the discussion phase. The desire and desire of all of us is to include as many technologies as possible for the benefit of the public."
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