The worrying rise in the price of innovative drugs, by Pr Alain Fischer

Spinal muscular atrophy is a genetic disease responsible for early degeneration of the nervous system. The development of a gene therapy for this pathology, announced in 2019, has raised tremendous hope. However, it is accessible at a price of approximately 1.9 million euros. If this makes it the most expensive drug on the market today, it is not the only one to be expensive! Innovative products marketed in recent years to fight cancer, rheumatic or autoimmune diseases and genetic diseases all have a very high price. A few examples: the treatment of leukemia with CAR-Ts costs around 400,000 euros, the drug treatment of cystic fibrosis around 200,000 euros per year and per patient, that of Crohn’s disease with monoclonal antibodies 12,000 euros per year and per patient… These prices, disproportionate to those of conventional drugs, raise questions. Why are they so high? Will they be accessible to everyone?

The pharmaceutical industry justifies these tariffs by the “transforming” nature of these innovations, that is to say curative in the case of gene therapy or cancer. Also by the fact that they are the result of complex and costly research and development work, or even because they (sometimes) target rare diseases. If these arguments can be heard, they do not really stand up to analysis. These prices are indeed unrelated to the cost of research, development and marketing, even taking into account the expenses related to the development of drugs that did not succeed. As has been shown for cancer drugs, they actually bear little relation to their medical value. Moreover, the perspective on so-called curative therapies is in most cases only four to five years, and we already know that they do not avoid the pursuit of medical care. According to a logic that would link price and years of life saved, an antibiotic treatment for sepsis should cost hundreds of thousands of euros!

“Accessibility for all of these drugs is threatened”

This situation is not without consequences: refusal of reimbursement in certain countries, choice oriented towards less expensive therapy when an alternative exists, and in the long term, impossibility for the payer, Social Security in France, to contain the budget of the products of health in a “reasonable” envelope. In other words, accessibility for all of these drugs is threatened. It appears that there is an imbalance between the general interest and the interest of the pharmaceutical companies, which we know have high profit margins. So what can be done to restore this balance while preserving the ability of companies to innovate? Raising awareness is necessary and should lead to a reform of the pricing regime. In France, the Economic Committee for Health Products does its best to obtain discounts, but its negotiating capacity is relatively limited. The group purchase of Covid vaccines by the European Union has shown that it can speak with one voice. Here is a door ajar for a European negotiation of the price of medicines, a situation more favorable to obtaining lower prices.

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Could we not agree in complete transparency on pricing rules integrating an assessment of added value, the cost of research and development, marketing and an “acceptable” profit? We should add that most of these innovations, if not all of them, come from public research. Thus, a reasonable price clause could be introduced in transfer agreements between public research and industry. Other avenues are possible, such as a commitment by drug companies to become companies with a societal mission, or the establishment, at least for rare diseases, of production units for new drugs at cost price, initially relying on public funds and on the work of foundations and charities.

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