The Audit Chamber's report on the future of the health insurance system, released on November 29, first drew attention to 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 treatments”, in particular in oncology, as indicated on page 26 of the material, and their potential burden on our healthcare system.
Gene therapy for cancer is cited as a specific example of a costly procedure. It is about changing certain genes in our cells to fight off disease. But let's look at what these new and allegedly promising treatments can give patients. And how they can threaten the health insurance system.
Gene therapy for cancer purposes is not yet available in France as it is in the evaluation phase. But two methods have already been approved in the United States in cases of acute leukemia. The prospects they are opening are sparking the enthusiasm of the Food and Drug Administration (FDA).
On August 30, it gave the go-ahead for the market launch of Kymriah by the Swiss pharmaceutical company Novartis. FDA Director Scott Gottlieb made the following statement in this regard: "This historic decision marks a new milestone in medical innovation with the ability to reprogram patient cells to fight deadly cancer."
A few weeks later, permission was also granted to Yescarta by the Californian Gilead laboratory. The head of the FDA called this method "a significant new stage in the development of a completely new medical paradigm in the treatment of serious diseases."
Passion for gene therapy
To understand the reasons for this enthusiasm, one must look back to the 1990s. After the first advances in human genetics and the decoding of the genome in medicine, a new horizon opened up: gene therapy.
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In theory, the idea is very simple. Since specific diseases are caused by genetic mutations, why not directly address these abnormalities? A corrected version of the defective gene, the "medical gene", is introduced into the body to restore the affected biological function.
In practice, the path turned out to be much more thorny than anticipated. Although the gene therapy industry enjoys widespread government support, attempts to treat patients have not gone beyond clinical trials. Kymriah was the first drug of its kind to be approved in the United States. Yescarta soon followed.
Medical and financial promises
But how did they get the license they needed to go to market just a month apart? First, we are talking about a new type of gene therapy. It uses cells from our immune system, T-lymphocytes, which are taken from cancer patients and then genetically modified in the laboratory. What is special here? This is not about "repairing" the DNA of cells, but about introducing additional DNA into them so that they begin to produce a substance that is not naturally secreted.
This substance can in some way be described as a chimera, since it arises from the fusion of two elements, one of which is obtained from a mouse, and the other from a person. The lymphocytes created in this way (CAR-T for chimeric antigene response T) enable the immune system to cope with the disease. In other words, they can fight cancer cells.
Second, the US has given the go-ahead to these techniques because they can fight cancers that are resistant to existing treatments: acute lymphoblastic leukemia in children with Kymriah and adults with Yescarta. Up to this point, the serious side effects of gene therapy have stalled its development. In France, experimental therapy to treat immune-deficient children was stopped after leukemia occurred in some patients. However, in the case of incurable fatal diseases, the issue of side effects fades into the background. In addition, experimental methods are widespread in oncology, and highly toxic drugs have long become commonplace.
Expedited verification procedure
Finally, Kymriah and Yescarta passed the priority check, that is, the fast track. In the event that it is about methods of treatment that allow you to fight a dangerous and potentially fatal disease, they can get the first permission on the basis of preliminary data. Such measures, which significantly reduce financial risks in terms of commercialization, 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 underlie what the sociologist Pierre-Benoît Joly calls "the regime of scientific and technological promises." It pushes governments, businesses, patients and clinics to invest in the absence of reliable and proven results.
An invaluable treatment?
The medical and scientific changes brought about by new methods are accompanied by major economic and political transformations. The most heated debate revolves around the following point: the declared price of treatment.
Pharmaceutical labs cite a cost of $ 475,000 per patient for Kymriah and $ 373,000 for Yescarta, not counting side effects. Such a high cost casts doubt on the financial ability of healthcare systems in different countries to provide treatment for all patients. Incidentally, this issue comes up with all "innovative" cancer treatments.
The budgetary implications are so far difficult to assess. In France, a report from the Council on Economic, Social and Environmental Affairs, published in February this year, noted that in total, “innovative” anti-cancer drugs could require between 1 and 1.2 billion euros per year. To get a better idea of the scale, it's worth remembering that a total of 15 billion is currently being spent on cancer (2014 data). This disease accounts for a total of 10% of the costs of the health insurance system.
Guaranteed result
In response to these concerns, Novartis is proposing to bring Kymriah to market under a (relatively) new contractual scheme: pay-by-performance. If there is no noticeable "improvement" in the patient's condition within a month, the company will not bill for the 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 done only for a month? These criteria are being questioned by a number of French, European and American NGOs working to ensure access to treatment.
At first glance, performance-based pay 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 not obvious enough. So, for example, the clinical trials that became the key to Kymriah's success included 63 patients, which is by no means a large number. In addition, “real life” assessments have not yet been carried out in a clinical setting.
Side effects and treatment
Side effects and the cost of treating them are another major concern. In the case of Kymriah, examples such as the inflammatory response have already been pointed out. 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 pay-by-performance that exist in the world, including France, do not yet meet the expectations placed on them. So, for example, in Italy the state received the minimum financial return.
This method of price regulation fits perfectly into the aforementioned "science and technology promise regime". It has its own economy, which includes in the cost that which goes beyond the present and is directed to the future. In this case, it is the hope that even greater achievements will follow the first successes. By the same logic, pay by performance is designed to convince citizens and authorities that if the promise is not kept, nothing will be asked for it.
New gene therapy for cancer treatment is likely to have serious financial implications for healthcare systems in France and elsewhere. Be that as it may, it is necessary to discuss not only 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.
Pierre-André Juven, Catherine Bourgain