The clauses confidential signed by the Ministry of Health and the pharmaceutical Novartis and Gilead to fund the costly therapy CAR-T against two types of cancer hematological provide for a system of deferred payment by the companies to not be charged for the second part —up to 64% of the total— if the patient does not survive beyond 18 months. This has been confirmed in THE COUNTRY, sources in the sector, which emphasize that this formula of “payment by results” has been designed to address the uncertainties that still weigh on the effectiveness of these treatments and to mitigate the impact that this will have for public health the high price, higher than 300,000 euros.

The desire to keep secret the content of these clauses has led Novartis to appeal in the courts for a resolution of the Council of Transparency, calling for Health to report on them to the platform Is Not Healthy, formed by several entities, medical, consumers, and promoters of transparency in the field of health. This type of conditions are common agreements about the CAR-T made in the last few months between pharmaceutical and european health systems, but this is the first time that they are published.

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The Kymriah (Novartis) is the first CAR-T included in the portfolio of the Spanish public health system. What was the last January with a maximum price —the hospitals are able to negotiate small discounts— 320,000 euros, while the Yescarta (Gilead) was in July by 327.000 euros. Both drugs are the most expensive against the cancer of public health and “the last therapeutic option for patients who have exhausted all available”, explains Ramón García Sanz, president of the Spanish Society of Haematology and Haemotherapy. “Are new therapeutic transcendental which combines the prior knowledge about the defenses of the organism against infectious diseases and the fight against the cancer cells,” he adds. The therapy CAR-T, applied in a single dose, are based on the reprogramming of cells of the immune system of the patient to learn how to eliminate the malignant cells.

the agreement of The Kymriah provides for a first payment of 160,000 euros (50%) to be administered to the patient. The drug is indicated for patients up to 25 years with acute lymphoblastic leukemia and for patients with B-cell lymphoma diffuse large cell. The second half is paid a year and a half if the patient has had a complete response to treatment and is “free of disease”.

In acute lymphoblastic leukemia that is considered reached if the tests reveal a presence of “residual leukemic cells, less than one in 10,000 cells analyzed”. In lymphoma, the response to treatment is measured with clinical criteria more complex and something different. The agreement of the Yescarta provides for a first payment of € 85,000 (36% of the total), and a second of 152,000. In this case, the criterion for completing the payment is based on the survival of the patient. The Yescarta is indicated for the above-mentioned B-cell lymphoma and another subtype of lymphoma.

The agreements include other demands for the pharmaceutical related to the first payment. Lymphocytes subjected to the CAR-T are extracted to the patient in any of the 15 large public hospitals selected Spanish for Health care, after which they are sent to the united States. “To collect the first part of the treatment, the hospital has to receive the cells with all the specifications in 30 days in the case of the Kymriah and 28 with Yescarta”, explain sources in the sector. “If you do not do so, as has already happened in some occasion, the first installment is not paid even though the drug is used”, add these sources. The industry stresses that firms assume “other risks”, such as staying without charge if the patient dies or ceases to be eligible to receive the drug while it is in production phase.

According to Novartis, clinical studies show that 62% of patients —children and adults-under 25 years— with acute lymphoblastic leukemia is disease free at 24 months, while 54% of those who suffer from the lymphoma B presents a complete response to the 19 months. Gilead provides studies with similar positive results published in journals such as The New England Journal of Medicine or The Lancet Oncology.

Price questioned

Despite these encouraging data, the extension of the first therapies CAR-T has been tarnished around the world due to the high prices charged by pharmaceutical companies, in a debate in which overlap between the two disputes. The first is the refusal of the companies to inform about the actual costs of research, development and production of drugs. An article published a year ago in the journal Health Affairs —described by The Washington Post as the “bible” of health policy— noted that the production of a dose of CAR-T costs no more than 36,000 euros. Taking into account all the costs of research, development and margins in the sector, the magazine estimated that the price of the Kymriah should not exceed the 144,000 euros. In Spain, the hospital pointer in these therapies —hospital Clínic of Barcelona— has managed to develop the drug with a lower cost of 60,000 euros, although the process is still in testing phase.

The second controversy is the pricing model driven by pharmaceutical companies, so-called “price per value” and not in relation to the costs, but with an estimate of the years with quality of life that the patient gets. In a written response, Novartis argues that “fixing prices based on the value they bring to society is the mechanism most appropriate to define them in countries with a free market in most sectors”.

This approach has been met with a strong response in many countries. “With this criteria, how much would the antibiotic that saves the life of a baby with a whole life ahead of you? Or will the surgical process of appendicitis without the that a six year old child would die?”, says Ramón Gálvez, former hospitals, such as Peace and the 12 of October of Madrid and today he heads the Association for Fair Access to Medication, one of the six entities that form part of it Is Not Healthy.

In the united States, the first country to approve two years ago these therapies, pharmaceutical companies have freedom to set prices. Novartis set the Kymriah for the treatment of leukemia 426,000 kg euros and for lymphoma in 335.000 euros, the same price that Gilead set for Yescarta. In countries with public coverage, the agreements reached with pharmaceutical companies set prices disparate, although the existence of clauses confidential prevents an estimatedr the actual cost of the treatments. The Uk, for example, was the first european country to approve both treatments. Agreed to a price of 370,000 euros for Kymriah and 387.000 euros for the Yescarta, although the agreement “provides for discounts confidential,” according to the british press.

The experts consulted in Spain does not share the model of “price per value”, but it does highlight the advantages of the approach of “payment by results” applied for Healing. “It is true that the prices are very high and that there is transparency in the way in which it’s set,” says Olga Delgado, president of the Spanish Society of Hospital Pharmacy. “But the great virtue of the new system is that it requires a detailed evaluation of each treatment.”

System evaluation

The new evaluation system, called Valtermed and which came into force on Monday, “will allow to know the real results of treatments which are now spent large amounts of money without completely knowing the actual benefits they bring”. According to the available results, Kymriah and Yescarta provide clear benefits in a horizon of two or three years, but there are not yet enough data to evaluate its cost beyond. The two drugs were the first to be approved by way of urgency by the European Agency of the Drug, given its potential.
Ramón Gálvez highlighted that these first two therapies, CAR-T will add others soon. “There are some in a very advanced stage of development and researches focused on solid tumors, which would be the number of potential patients of a few hundreds to tens of thousands,” he says. “Will there be money to pay them all at these prices?”, is question.
The significance of this debate is what leads to the Not it Is Healthy to reject outright the “opacity of the system of approval and funding of medicines”. “It is a decision taken by Governments with public funds. Citizens have the right to know the content of these agreements and this may not be limited by the interests of the industry,” says Vanessa Lopez, director of another of the entities of the platform, Health Right.

The sector, for its part, argued that these agreements, which enable them to adapt to the low prices according to the reality of each country. Without confidentiality, they add, the tendency will be that the prices will match the rise and the access to the therapies of new patients is hindered.

The return of David to the football field after leukemia

David, who is now 16 years old, is the bright side of the new therapies CAR-T. The football is his big passion, but an acute lymphoblastic leukemia has kept him away from the pitch the greater part of his life. The first plague was that of the five years, but they got over it thanks to the chemotherapy. The second came at 11, his first relapse. “We did a bone marrow transplant. It was very tough. He recovered, but he had to learn to walk again,” recalls his mother, Lydia Maqueda. Two years later, a new hack: the second relapse exhausted the available treatments and strived to David to an experimental treatment with Kymriah, Novartis. In the Sant Joan de Déu Hospital of Barcelona withdrew lymphocytes, which by a process involving virus inactive, were reprogrammed in Philadelphia (USA) to learn to identify the antigens of the cancer cells. Back to your body, the white blood cells were able to eliminate the cancer cells. He returned to be admitted for two weeks, spent a few hard days, but for David it was almost like scoring a goal to empty door. Two months later, he returned to the institute. Shortly after, ran after a ball. “It is a wonderful process. There are No other words to describe it,” says Maqueda.

The treatment that has cured David is no longer experimental, but a reality funded by the Spanish public health system. The forecasts of the Ministry of Health are that 68 people will benefit from a year in Spain therapy CAR-T to leukemia and other 339 for the lymphoma, which raises the annual cost to the public purse of these treatments to 130 million.

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