Neurologists attack the drug basket committee: "Continued use of cheap substitutes for the appropriate drug will cause irreversible harm to patients" • Ethan's family, who suffer from a rare genetic disease: "The replacement therapy has led to deterioration in his condition"

• Finance Committee discusses drug basket // Photo: Oren Ben Hakon

In an unusual move, senior neurologists in Israel warned the Ministry of Health and the Pharmaceutical Basket Committee that a drug that the Ministry of Health has approved for months has caused side effects and serious damage to some patients.

This is an alternative drug that the Ministry of Health has exceptionally approved to treat a rare and incurable genetic disease called CTX instead of the overpriced drug for the disease. According to neurologists, the alternative drug has been causing side effects and very severe damage for months to some patients, and doctors have warned that many patients have already stopped taking the drug. This means that they suffer or are severely aggravated by the disease, deteriorating in their neurological and psychiatric condition and the serious danger of very severe disability and death at an early age.

"Preventing harm to patients"

The extraordinary reading is signed by two senior neurologists in Israel, Prof. David Tena, Director of the Stroke Institute at the Rambam Government Hospital in Haifa and Chairman of the Neurological Association of the Medical Association, and Prof. Sharon Hasin, Chair of the Neurological Association's Committee on Medicines and the Director of the Institute For high traffic and neurological disorders in the government hospital that will come to Tel Hashomer.

Senior neurologists recently wrote to senior health officials and Prof. Zeev Rothstein, chair of the Medicines Committee, that they wanted to include in the basket the expensive drug, "Ladiant," designed to treat the disease, and not settle for the cheaper alternatives. , Will cause patients' irreversible neurologic injury that causes severe disability to bed restraint, and finally premature death. We ask in every language of application to include care and prevent irreparable harm to our patients. "

The senior doctors also cautioned that only the expensive drug for inclusion in the basket is "the right treatment for disease that can prevent widespread and nerve damage in people suffering from the disease, and early treatment with the drug can even allow people with the disease to live a healthy life without any disability associated with the disease."

CTX is a rare hereditary disease involving some 50 Israelis, and is manifested by an abnormal accumulation of a fatty molecule in body tissues, which causes childhood to develop severely and with severe disability. In some cases it causes brain injury, blindness, psychiatric disorders, dementia, atherosclerosis, seizures and death at an early age.

The cure is expensive

Because of the rarity of the disease, it is usually diagnosed very late, after irreversible damage has already been discovered. The average age for diagnosis is 35, while patients have suffered since birth.

The cure for the disease, "Lianant", which is given in capsules, has been listed in Israel since 2017 as the cure of choice. However, after the registration, the Ministry of Health severely criticized the Israeli importer of the drug, MBI Pharma, which after registration increased the price of the drug by hundreds of percent to more than ten times its previous cost, so that the cost reached about NIS 1 million per patient.

The basket committee said it was "extremely overpriced" for the drug, and there were senior members of the basket committee who claimed it was a "pig price". This year, the drug was offered to the basket committee at a cost of about half a million shekels a year to the patient.

The drug has been discussed twice in the drug basket for the past two years, and during these discussions, the severe criticism of the drug price was raised and, consequently, it was not put in the basket. This year, it is nominated for the third time in the basket of 2020. This year, the basket committee was presented with new data from doctors, which indicate severe discrimination in drug delivery. This is because there are patients who cannot meet the high payment of hundreds of shekels of deductible for the drug, and as a result their condition has already deteriorated greatly.

Following claims of the drug's excessive price, the Ministry of Health in January granted special permission to import from Japan and Portugal the alternative drug, which is dramatically cheaper than the original drug submitted to the basket. Subsequently, a petition for the expensive drug importer has also been filed with the Supreme Court, but the petition has been rejected and the Ministry of Health's move to import the alternative drug can be approved.

Ethan's War

One of the patients struggling to get the drug is 39-year-old Ethan Shariki, who was diagnosed with illness at the age of 33, having already had a major deterioration and is suffering from severe disability and loss of the ingestion system. According to Eitan's brother, Zion Shariki, the alternative drug treatment given to him by a general health fund caused him serious problems and he stopped taking it. He now wants the disease drug to be put in the basket.

The Public Health Basket Expansion Committee for 2020 said in response to the doctors' letter that "this year, some 900 drugs and technologies worth more than NIS 3 billion have been submitted to the basket. The committee is currently holding hearings, at the end of which the list of recommended drugs and technologies will be added to the basket."

Ministry of Health response: "In view of the huge and extreme price gaps that exist between the registered preparation and the additional preparation, and in view of the fact that this is a product that has already been used for a considerable period of time and in order to allow continuation of the therapeutic continuum - the Ministry of Health approved the move. Alongside the commitment to ensure that the use of the product is effective and safe, The state must also consider economic and budgetary considerations that affect the public coffers, especially as in the case here, where price disparities are very high, and not for material reasons.

"The approval was given after receiving information from the HMO on the drug so far, the lack of side effects and the drug's effectiveness, and since a new drug is already a drug that has already been imported and marketed in previous years in Israel and found to be effective in treating the disease. We wonder about the timing of the announcement and the cynical exploitation For making profits at the expense of patients. "

The Health Ministry stated that they had approached the Competition Authority to handle the price, and that the importer was trying to attack the High Court decision, including allegations of drug quality, and its claims were rejected.