says no to “hopeful” treatment and provokes anger among patients

says no to “hopeful” treatment and provokes anger among patients
France says no to “hopeful” treatment and provokes anger among patients

This medication, developed by the Biogen laboratory under the name Qalsody, “is not likely to meet an insufficiently covered medical need”concluded this week the High Health Authority (HAS).

The authority therefore refused so-called access “early” to this treatment, a procedure which allows reimbursement of a drug even though current studies have not yet fully demonstrated its benefit.

Qasoldy, based on the tofersen molecule, aims to slow the progression of Charcot disease (amyotrophic lateral sclerosis), but only targets patients with a particular genetic form, representing only a small percentage of patients.

However, it has raised great hope among patients because it was approved in recent months by health authorities in the United States and the European Union (EU), very rare news for this disease with almost no treatment.

The French HAS therefore did not follow this example, a divergence which can be explained by an uncertain benefit in view of the study carried out by Biogen.

A decision “cruel and unacceptable”

Published in 2022 in the New England Journal of Medicinethis study did not demonstrate a real slowing down of the symptoms of the disease, even if it had demonstrated an effect on its biological markers.

The promoters of the treatment consider that this last point gives hope for an effect on the symptoms if Qalsody is given earlier, before the first clinical signs of the disease. But this hypothesis remains uncertain.

However, the HAS’s decision provoked the anger of the main French association fighting the disease, the Association for Research on ALS (ARSLA), which regrets that plans[e] to deprive those affected of their only hope”.

https://www.whatsupdoc-lemag.fr/article/maladie-de-charcot-le-senateur-gilbert-bouchet-malade-emeut-le-senat-et-defend-une

In a press release published Friday, she described the HAS decision as “cruel and unacceptable”by calling on the President of the Republic, Emmanuel Macron.

With AFP

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