Cancer gene therapy and pharmacoeconomics
Cancer: what is gene therapy really worth?
Cancer: que valent vraiment les thérapies géniques?
Pierre-André Juven, Catherine Bourgain, The Conversation
Translation: InoSMI.Ru
The report of the Accounting Chamber on the future of the health insurance system, published on November 29, first of all attracted attention by the proposed restrictions on the activities of freely practicing doctors. However, another important point went unnoticed. We are talking about "the cost of some new types of treatment," in particular in oncology, as indicated on page 26 of the material, and their potential burden on our healthcare system.
Gene therapy to fight cancer is cited in it as a concrete example of an expensive procedure. It consists in changing certain genes of our cells to counteract the disease. But let's look at what these new and supposedly promising treatments can give patients. And how they can threaten the health insurance system.
Gene therapy for oncological purposes is not yet available in France, as it is at the evaluation stage. But two methods have already been allowed in the US in cases of acute leukemia. The prospects they open up arouse the enthusiasm of the Food and Drug Administration (FDA).
On August 30, it gave the go-ahead for the launch of the drug Kymriah by the Swiss pharmaceutical company Novartis. FDA Director Scott Gottlieb made the following statement on this occasion: "This historic decision means overcoming a new frontier in medical innovation with the possibility of reprogramming patient cells to fight deadly cancer."
A few weeks later, permission was also granted to Yescarta of the Gilead California Laboratory. The head of the FDA called this method "a new significant stage in the development of a completely new medical paradigm in the treatment of serious diseases."
Fascination with gene therapy
To understand the reasons for this enthusiasm, one should look back to the 1990s. After the first successes in human genetics and the decoding of the genome in medicine, a new horizon opened up: gene therapy.
In theory, the idea is very simple. Since specific diseases are caused by genetic mutations, why not directly address the correction of these anomalies? A corrected version of the defective gene, the "medical gene", is introduced into the body, which is designed to restore the affected biological function.
In practice, the path turned out to be much more thorny than expected. Although the gene therapy industry enjoys broad government support, attempts to treat patients have not gone beyond clinical trials. Kymriah was the first drug of this kind to be approved in the USA. It was soon followed by Yescarta.
Medical and financial promises
But how could they achieve the necessary license to enter the market with an interval of only a month? Firstly, we are talking about a new kind of gene therapy. It uses cells of our immune system, T-lymphocytes, which are taken from cancer patients and then genetically modified in the laboratory. What is the peculiarity here? We are not talking about "repairing" the DNA of cells, but about introducing additional DNA into them so that they begin to produce a substance that is not isolated in its natural form.
This substance can in some way be characterized as a chimera, since it arises from the fusion of two elements, one of which is obtained from a mouse, and the second from a human. The lymphocytes created in this way (CAR-T from chimeric antigene response T) give the immune system the opportunity to cope with the disease. In other words, they can fight cancer cells.
Secondly, the United States gave the go-ahead for these techniques, because they allow us to fight cancers that are resistant to existing treatments: we are talking about acute lymphoblastic leukemia, in children in the case of Kymriah and adults in the case of Yescarta. Up to this point, serious side effects of gene therapy have inhibited its development. In France, experimental therapy for the treatment of immune system-deprived children was stopped after the occurrence of leukemia in some patients. Nevertheless, in the case of incurable fatal diseases, the issue of side effects recedes into the background. In addition, experimental methods are widespread in oncology, and highly toxic drugs have long been commonplace.
Accelerated verification procedure
Finally, Kymriah and Yescarta were checked in a priority, that is, accelerated order. In the event that we are talking about methods of treatment that allow us to fight a dangerous and potentially fatal disease, they can get the first permission based on preliminary data. Such measures, which significantly reduce financial risks in terms of commercial implementation, have significantly contributed to attracting investors (and they are especially motivated by the fact that the potential market is very large).
The main question is whether the preliminary results will be confirmed in practice. Moreover, it is all the more acute because several similar trials with other types of cancer were stopped due to the death of patients.
Kymriah and Yescarta are riding a wave of enthusiasm, both medical and financial. The hopes and anxieties associated with them form the basis of what sociologist Pierre-Benoît Joly calls the "regime of scientific and technical promises." It pushes government authorities, businesses, patients and clinics to invest in the absence of reliable and proven results.
Priceless treatment?
Medical and scientific changes caused by new methods are accompanied by serious economic and political transformations. The most acute disputes are conducted around the following point: the declared price of treatment.
Pharmaceutical laboratories cite a cost of $475,000 per patient when using Kymriah and 373,000 – Yescarta, not counting the fight against side effects. Such a high price calls into question the financial ability of healthcare systems in different countries to provide treatment to all patients. By the way, this issue is raised with all the "innovative" methods of cancer treatment.
The budgetary implications are still difficult to assess. In France, the report of the Council on Economic, Social and Environmental Issues, published in February this year, notes that in total, "innovative" anti-cancer medicines may require from 1 to 1.2 billion euros per year. To better imagine the scale, it is worth remembering that a total of 15 billion are currently being spent on cancer (2014 data). This disease accounts for a total of 10% of the expenses of the health insurance system.
Guarantee of the result
In response to these concerns, Novartis proposes to bring Kymriah to the market under a (relatively) new contractual scheme: payment by results. If no "improvements" are noticeable in the patient's condition within a month, the company will not bill for treatment.
This approach raises a number of questions. What exactly is meant by "improvement"? Complete and sustained remission? And why should a health assessment be carried out only for a month? Questions about these criteria arise from a number of French, European and American NGOs that work to ensure access to treatment.
At first glance, paying by results looks like a simple and even fair principle. In fact, he transfers the question of price to another sphere, the sphere of the effect obtained as a result of treatment. And his assessment is a rather unobvious matter. For example, the clinical trials that became the key to the success of Kymriah included 63 patients, which is by no means a large number. In addition, the assessment "in real life", and not in a clinical setting, has not yet been carried out at all.
Side effects and treatment
Side effects and the cost of their treatment represent another serious point. In the case of Kymriah, examples such as an inflammatory reaction have already been indicated. Do not forget about the costs of therapy, which some estimate at $ 150,000-$200,000.
Finally, it should be noted that the few examples of payment based on results that exist in the world, including in France, do not yet correspond to the hopes placed on them. For example, in Italy, the state received minimal financial returns.
This method of price regulation fits perfectly into the aforementioned "regime of scientific and technical promises". It has its own economy, which includes in its value what goes beyond the present and looks to the future. In this case, it is the hope that the first successes will be followed by even greater achievements. By the same logic, payment based on the result is designed to convince citizens and authorities that if the promise is not kept, nothing will be asked for it.
The new gene therapy in the treatment of cancer, apparently, will entail serious financial consequences for health systems in France and other countries. Be that as it may, it is necessary to discuss not only the prices, but also the quality of the treatment provided. These reflections do not concern only sanitary experts and central administrations, but directly affect citizens and patients.
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