“Knowing that there can be a single patient, a single child who does not have access to an innovative treatment, which can prevent him from dying, is simply unacceptable”was indignant on Monday February 21 on franceinfo Laurence Tiennot-Herment president of the AFM-Téléthon.
franceinfo: Which patients do you think are deprived of access to innovative medicines?
Laurence Tiennot-Herment: I have a specific example to cite, a gene therapy drug that can save the lives of children with a very rare disease, adrenoleukodystrophy, a disease that kills children when there is no donor compatible marrow. It is a drug which obtained its European marketing authorization last June or July, which is very good news for all families. And then in October, the American laboratory decided unilaterally to abandon its marketing to devote itself to the American market alone. This situation is unacceptable, because in addition this drug is the result of French research co-financed by Telethon funds.
Is this an isolated example or are there others?
There is another gene therapy drug from the same American laboratory, also from French research, for a rare blood disease, beta-thalassemia. The marketing authorization was in 2019, and this American laboratory decided to stop its marketing in October 2021. For this disease, there is a therapeutic alternative, since patients can undergo transfusions blood tests every three weeks, but hope was very important to the families. We know of many families who were waiting for this medicine to avoid these blood transfusions.
What do you think should be done to solve this problem?
There is a real fundamental problem: these innovations were born in France, with Telethon funds, with public research, hence the indignation. But it’s not just for rare diseases: we saw at the time of the Covid our fragility when we depend on industries outside France or outside Europe. This is also when we heard a lot about national sovereignty and reindustrialisation. On our side, for all the public innovations, or which come out of the Telethon laboratories, it has been years since we alerted the public authorities. We had organized a major symposium on securing our health independence in September 2019. It is clear that national sovereignty is not only about reindustrializing France with paracetamol or surgical masks, it must be about creating the conditions for that our therapeutic innovations do not leave our territory. And also so that the Biotechs, which carry these innovative projects, can raise investment funds with us, develop with us, and that we keep control of marketing.
What are the levers to achieve this?
We need ambitious health investment funds, with significant resources. They have to be proactive, support project leaders, and have a culture of risk. If we let innovation leave our territory very early, then we lose commercialization. The industrialist is then sovereign in his decision to continue, to stop, to choose his territories. It can be catastrophic: knowing that there can be a single patient, a single child who does not have access to this innovative treatment, which can prevent him from dying, is simply unacceptable.