Science

Alarming rise in prices for innovative medicines, Professor Alain Fischer

Spinal muscular atrophy is a genetic disorder responsible for the early degeneration of the nervous system. Announced in 2019, the development of a gene therapy for this pathology raised great hopes. However, it is available for around 1.9 million euros. If that makes it the most expensive drug on the market today, it’s not the only expensive drug! Innovative products introduced to the market in recent years to fight cancer, rheumatic or autoimmune diseases and genetic diseases are very expensive. A few examples: treatment of leukemia with CAR-T costs approximately 400,000 euros, medical treatment of cystic fibrosis approximately 200,000 euros per year per patient, treatment of Crohn’s disease with monoclonal antibodies 12,000 euros per year per patient … prices disproportionate to the prices of conventional medicines raise questions. Why are they so tall? Will they be available to everyone?

The pharmaceutical industry justifies these tariffs by the “transformative” nature of these innovations, i.e. curative in the case of gene therapy or cancer. Also because they are the result of complex and expensive research and development, or even because they (sometimes) target rare diseases. If these arguments can be heard, they do not stand up to analysis. These prices are indeed unrelated to the cost of research, development and marketing, even taking into account the costs associated with the development of drugs that have not come to fruition. As has been shown for anti-cancer drugs, they actually have little to do with their medical value. Moreover, the prospect of so-called healing therapies in most cases is only four or five years, and we already know that they do not avoid seeking medical help. According to the logic linking the price and the number of years of life saved, the treatment of sepsis with antibiotics should cost hundreds of thousands of euros!

“Availability of all these drugs is under threat”

This situation is not without consequences: the denial of reimbursement in some countries, the choice to focus on less expensive therapy when an alternative exists, and in the long run the inability of the payer, social security in France, to support the budget for health products. in a “reasonable” envelope. In other words, access to all these medicines is at risk. There seems to be an imbalance between the general interest and those of pharmaceutical companies, which we know have high profit margins. So what can be done to restore this balance while maintaining the ability of companies to innovate? Raising awareness is necessary and should lead to reform of the pricing regime. In France, the Economic Committee for Medical Products does its best to secure discounts, but its bargaining power is relatively limited. The group procurement of vaccines against Covid by the European Union has shown that he can speak with one voice. Here the door is open for European negotiations on the price of medicines, the situation is more favorable for obtaining lower prices.

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Can we, in full transparency, agree on pricing rules that integrate value added, R&D costs, marketing, and “acceptable” profits? It should be added that most, if not all, of these innovations come from public research. Thus, transfer agreements between public research and industry could include a reasonable price clause. Other paths are possible, such as the commitment of pharmaceutical companies to become companies with a public mission, or the creation, at least for rare diseases, of new drugs at cost, initially relying on public funds and on the work of foundations and philanthropic foundations.

None of these measures are easy to implement, but it becomes necessary to act, at least on a European scale, so that progress in research can be translated into medical advances for the benefit of all.

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Alain Fisher is professor emeritus at the College de France and co-founder of the Institute for Genetic Diseases (Imagine).

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