Sleep, blindness and episodes: The rare diseases that struggle to enter the drug basket

Despite the government's paralysis, the Drug Basket Committee continues its dramatic discussions about subsidizing medical treatments for the coming year. Among other things, committee members are required to discuss three rare diseases that a tiny proportion of the population suffers from, but the introduction of basket treatments may improve and even save their lives.

Sleep, blindness and episodes: The rare diseases that struggle to enter the drug basket

As every year, the members of the Pharmaceutical Basket Committee are required to make decisions about the diseases for which the drugs will be subsidized. This year, in the midst of discussions, committee members will be required to discuss, among other things, three rare diseases, and decide whether by 2020 they will be admitted to health care. These diseases attack a few percent of the population, but they manifest themselves in acute health difficulties that do not allow for a healthy lifestyle without proper treatment - which, in private financing, is extremely high.

As every year, even in the basket discussions for 2020, questions are raised about the benefits and cost of drugs, but also among their contribution to the population: Does treatment for dozens of people, although seriously ill, exceed the value of a drug that will improve the lives of thousands? Either way, the decision of members and committee members, which relies on a broad spectrum of professional, economic and social views, is never fully accepted. However, this does not diminish the respectability of the decisions they make, and in the discussions themselves there were some regrets and concerns about drugs and treatments that were decided not to put them in the basket, and whether it was the right thing to do afterwards.

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He stated that he would extend the basket's coverage to 700 million. Litzman in the Knesset (Photo: Reuven Castro)

MK Jacob Litzman at the Knesset Plenum, May 29, 2019 (Photo: Reuven Castro)

The drugs are graded by committee members using a traditional method. The highest score that moves treatment to the next stage - which is about cost - is A10. This is a very rare ranking. Next come the A9, A8 and A8-9 which are also sufficient to reach the next round. The B7 rating is borderline, but B6 and C5 and below exclude the drugs that have been classified under them. At the end of the ratings phase, pharmaceutical companies and patients can challenge their location - and move them to the stage of economic discussions. There, the efficacy and prevalence of disease in relation to price are examined. This phase is expected to begin earlier this month.

The drug basket budget for this year is now only half a billion shekels, despite the statements of Deputy Health Minister Yaakov Litzman that he will extend the coverage to 700 million. However, at this stage it is not at all possible to know when the decisions will be made, as the 2020 budget has not been set due to the political deadlock. Thus, the budget amount remains vague and the committee members will not be able to make their final decisions until it becomes clear, hoping that the amount will not fall below NIS 500 million.

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The sleeping sickness that does not allow patients to go out

Only a few dozen patients in Israel suffer from narcolepsy, but 60% of them deal with severe conditions of the disease. These patients sleep on average about 18 hours during the day. In severe condition, they experience a sudden loss of muscle tone in the body - a condition called cataplexy. The great majority of seizures occur in moments of deep sadness or excitement. Thus, patients are at risk of daily living and unable to live a routine life, and in fact must be constantly supervised. Sleep attacks during the day actually require them to sleep wherever they are.

Recently, the US Food and Drug Administration, the FDA, approved a drug called Wakix. This year the treatment is being dealt with for the first time in drug basket discussions, earning an A8 \ 9 rating - and will continue to the next level of ratings. Absalom, 38, suffering from the disease, explained the difficulty, and the importance of treatment. "I am 38, married with a four-year-old girl. The disease first occurred to me during high school. In the military - although I was a good soldier - I would fall asleep on the tables several times a day. Of course, this was not accepted by my commanders, who instructed me to do tests. I was sent to a neurologist who referred I went to a sleep lab. In the lab, narcolepsy was diagnosed, and then I was prescribed medication. At first I did not want to take medication. I refused to believe there was a problem. One day I told myself I had nothing to lose and tried. I did not feel a change. Suddenly, I realized I had spent a full day without sleep attack. I couldn't believe it. I stopped the pills and then the seizures came back. That was the moment I realized that through therapy I was not alive. Sleep tolerate these attacks, I do not have to live like that. Since then, I'm taking medication aimed to reducing attacks of sleep as much as possible - a condition called balance. "

"The drug treatment changed my life. In fact, the treatment allowed me to live my life. Even my maths degree couldn't complete my situation without treatment. Among the pills I received were Ritalin, Adderle, Provigil and Atent. The problem is that the body adapts to the drug therapy, and At a time, most treatments lose their effectiveness and then I am required to increase doses and sometimes change treatment, "he explained.

"Through therapy, I don't have to suffer." Absalom and his family

Absalom Kindil and his family (Photo: Courtesy of the photographers)

Absalom made it clear that sleep attacks are not fatigue attacks. "When you are tired, you can just splash water on your face and wake up more or less. In a sleep attack, nothing helps or wakes up; no water, no noise and no traffic. I once fell asleep standing in the bus and following this I fell. And still, I'm lucky. There are people Suffering from narcolepsy at such a level where the seizure comes in seconds. For me, once I feel a sleep attack - a feeling of very strong artificial fatigue - I have at most five minutes to find a bed. "

"Travel by bus and train is a nightmare," he shared. "During my studies at Tel Aviv University, I lived on the streets. Although there is a train that arrives directly from the streets to the university within half an hour, I preferred the bus. The last stop of the bus was at the university itself, so often, if I fell asleep, the driver at the last stop woke me at the end of the train. In contrast, I could find myself in another city. "

"During a bout you can't wake me up. The phrase 'fall asleep' is terribly deceptive. It's not like a normal person. The only way to have a bout is to just sleep. The bout can be several minutes or hours. At the end of the bout, you wake up as if after a night's sleep. "Narcoleptics are different. We go into the deepest sleep phase in minutes. This is unlike normal sleep, where dreams start after about an hour and a half. Today there is no cure for narcolepsy and the focus is on drug therapy that lowers the frequency of seizures, but there is no way to completely prevent them."

Drug treatment lowers seizure frequency (Photo: ShutterStock)

Nutritional supplements and vitamins on a supermarket shelf (Photo: ShutterStock)

Avshalom also said that "Narcolepsy disrupts life in many forms. A few years ago, I was a programmer, working and trying to build my life. Sleep attacks occur more during monotonous activity, such as working in front of a computer, watching TV and reading. These activities become difficult for me. "

"I recently learned from my neurologist about the new drug coming to Israel. Although very likely my body will eventually get used to this treatment, I am optimistic and hope that the new drug will bring me back to balance. Living with a disease like narcolepsy is living with a disability. It's only a handicap here, the drug treatment. He is the wheelchair. Balance therapy is life-changing, it is life-affirming. The last treatment allowed me to live in balance for six years. The new treatment can, too, and I hope it will. Even if it is for a few years, it will give me a horizon, I can help my wife, To work and support my family, and to be a father - less restricted - to my child. "

The precious cure for the rare eye disease

In recent years, gene therapy has gained momentum, in light of advances in encoding human genes alongside the development of safe technology for introducing healthy genes into the body in favor of its healing on viruses, as carriers. This technology has enabled the development of the world's first genetic engineering treatment for incurable genetic eye disease called Retinitis Pigmentosa - RP, which erupts in the proportion of one in 4,000 people in Israel in its first decade of life. The disease develops to full blindness in the second and third decades, following retinal degeneration.

Thanks to the new technology called Locksturn, with a single disposable injection, a proper gene can be introduced that improves vision, proportionally, and even stops retinal degeneration and prevents blindness. During the current discussions, the drug received an A9 rating, and in fact moved on to the next stage. The treatment has been approved by the FDA, and so far, dozens of people in the world, most of them in the United States, have been given impressive results in improving visual function.

Yossi Saidov, founder of the RP patients in Israel, explained the rarity of the disease, which not many people know about its existence - and the patients themselves are sometimes unaware of its consequences. "I set up an organization for Retinitis Pigmentosa patients called 'Seeing Far'. Our goal is to create a community of patients, to give them knowledge and to make them available. Locastorna is a treatment that fits one particular gene, out of about 200 genes that cause the disease, and is suitable for young people up to age 30 "That means that out of 4,000 patients, this drug is suitable for only ten people. It's a genetic drug, really science fiction."

"The goal - to create a community of patients and make them accessible." Yossi Saidov

Yossi Saidov, Founder of RP Patients in Israel (Photo: (Courtesy of Interviewee), Image Processing)

"Retinitis pigmentosis is an eye disease whose first manifestation is poor night vision, from there to narrowing the field of vision - which may deteriorate to blindness," he explained. "A year ago, the drug was nominated for the drug basket and received the same rating, but it fell at the last minute. The cost is $ 850,000 for one-time treatment. This makes people who are certainly blind - people see. We were able to make the treatment available to two patients in Israel, and report Our goal is for the drug to go into the basket this year. We know of at least another five to six people that care is right for them, including three children. "

The treatment of arthritis - defined less important

Gout is a severe chronic arthritis caused by metabolic disruption in the body, which is manifested by an increase in blood uric acid levels. In normal condition, uric acid is excreted, but increased production or reduced clearance from the body causes an increase in blood acid concentration and, as a result, crystals that sink into different body tissues and joints, especially those in the feet and hands. The crystals sink into the joints which causes sharp and unbearable pain attacks accompanied by bloating and redness, which can last for several days. Beyond the pain attacks that occur for a while, the accumulation of uric acid can cause lasting damage such as kidney stones and lumps under the skin, bone and cartilage, which are called toffee, leading to bone erosion, deformity and joint damage.

An extreme gout event includes recurring bouts of acute arthritis whose most common location is in the toes but spreads to many more joints, urinary stones and tissue crystals. The seizure culminates within 12-24 hours, and may take several days to two weeks.

KRYSTEXXA is based on an enzyme given intravenously once every two weeks. The enzyme acts on the light acid and makes it a molecule that is easily excreted through the urine. The result is a dramatic and rapid drop in blood uric acid levels, maintaining this low level over time. Consistent treatment with KRYSTEXXA leads to a decrease in seizure frequency, until the gradual disappearance of the signs of the disease. The drug is based on advanced technology because it contains a unique enzyme capable of decomposing the light acid.

KRYSTEXXA is given as a third line when the patient is durable or due to therapeutic failure of the existing medications, even when administered at the highest dose. Chronic levels of uric acid have been associated with a wide range of life-threatening diseases, including cardiovascular disease, high blood pressure, diabetes, and blood lipid balance. For this reason, treatment with KRYSTEXXA may prevent secondary medical complications that shorten life expectancy. This year, the drug was rated B6, and if the appeal is not accepted - it will not proceed to the next stage of the hearing.

"Our goal is for the drug to enter the basket this year" (Photo: Police spokeswoman)

Doctor and son are suspected of smuggling drugs that are dangerous drugs abroad, September 11, 1919 (Photo: Police spokeswoman - Merhav Negev, official website)

Amnon Feldman, pianist and piano teacher and voice development, discovered the disease at the age of 21. "I was in the middle of a performance, and suddenly my hand swelled," he said. "The elbow swelled up in a matter of minutes, and when I went to the doctor, I was informed that gout had found me. Today I am 75 years old, and I have been suffering ever since. The only thing that really helps is this medicine. I came to a state where I couldn't walk, I crawled to the bathroom, and I didn't exaggerate. ".

"This drug really helps," he added. "It takes a while for it to start working, but when it starts to work it lowers the suffering to indescribable levels. I've been to hospital many times throughout my life because of the disease, and although I've been in the United States all these years, they didn't realize that this disease was acting like all other diseases. "I got attacks in my neck and all the parts of my body that didn't get it. They always thought I had something else, and they put me in the hospital and released me. Nobody had a solution."

Feldman asked to address the committee members. "It's a shame that they don't approve of this drug. I know it's a very expensive drug, around NIS 45,000 for treatment, it's a fortune. But it helps. Except for this drug, nothing helped. It frustrates me that young people can't I would like it to be more popular, Israelis invented the drug. I would tell the basket committee that it's a shame to get people undergoing such torture. They have no idea what this horrible disease is, what the pain is and how it disables the human being. No quality of life when it comes to such levels. If you could help the public get this drug it would have saved a lot of people. A way to leave it. "