Almost two million euros for an injection. Zolgensma, a treatment for children with spinal muscular atrophy (SMA), is considered the most expensive drug in the world. The laboratory that produces it, Novartis, has decided to offer it to a hundred randomly selected children.
Spinal muscular atrophy is a rare and deadly disease that weakens babies' muscles. Without treatment, their life expectancy is reduced to around two years. In France, there are approximately 1,500 cases. Globally, one in 10,000 babies is affected by the disease. "The AveXis unit of Novartis, which developed the drug, will deliver fifty doses of the treatment until June for babies under two years of age with up to 100 total doses to be delivered by 2020", reports Reuters. But the laboratory's method of distributing the beneficiaries, namely a draw, did not fail to react. The patient defense association SMA Europe was moved by the "Russian roulette" created by this device.
Indignation of the AFM-Théléton
The AFM-Telethon has also expressed its indignation at the Slate site, surprised that a "Such a program based on a draw could have been imagined and, a fortiori, proposed to parents whose child suffering from a severe spinal muscular atrophy is condemned to die in the short term. How can you imagine for a moment that a child's life could be the jackpot in a lottery? " The AFM-Telethon also participated in the funding of the initial research, before it was taken over and extended by AveXis, a start-up which was then bought by Novartis.
The laboratory for its part denies the qualifier of "lottery" and defends a device "Randomized, as in clinical trials". He also talks about a long matured process: "In designing this program, AveXis worked with an independent bioethics advisory committee and focused on the principles of equity, medical needs and global accessibility in order to best determine the overall distribution of a limited number of doses, avoiding favoring a child or a country compared to another ", Novartis said in a statement to Figaro. The laboratory does not however specify why it carries out this operation of free delivery of the drug.
The text further specifies: "Concerning more specifically France, we took the feedback of the various stakeholders with the greatest seriousness following the announcement of the program and understand the reactions (…). We have implemented the following decision: if a global access management program, making Zolgensma (AVX-101) available free of charge, is of interest in countries where it is not accessible, this logic does not apply in France."
The Minister of Solidarity and Health Agnès Buzyn reacted on Twitter. "Very concerned about the draw in place in certain countries for access to treatment with Zolgensma, I would remind you that France is not concerned: anyone who needs this treatment has access to it". French babies are actually eligible for this drug, subject to validation. The drug is currently dispensed to a few via temporary use authorizations (ATU) which are decided by an expert committee, confirms Novartis, "Which of course we do not attend".
Obtaining this medication remains to this day an unparalleled solution for parents. In recent months, one case has taken root in Belgium. A couple appealed to the generosity of internet users by opening participatory pots to be able to get it. Pia's parents, who had spinal muscular atrophy, had managed to raise the money overnight. The single injection of Zolgensma, produced by the pharmaceutical laboratory Novartis, is not approved by the European Medicines Agency (EMA). Pia had previously been treated with Spinraza, a drug that Belgian social security pays for.
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