At the end of October, the HAS refused “early” access to Biogen’s Qalsody, indicated in a rare genetic form of the pathology, which aroused the anger of patients.
On October 25, the opinion of the High Authority for Health (HAS) had the effect of a cold shower for patients suffering from a rare form of Charcot disease: it had refused to grant access said “early” to the drug Qalsody, requested by the Biogen laboratory. This procedure allows, for patients in therapeutic impasse, the reimbursement of a supposedly innovative drug, even though ongoing studies have not yet fully demonstrated its benefit, sometimes even before their authorization on the market, or their reimbursement in the framework of common law. This medication is currently administered as part of “compassionate” access and at the expense of the laboratory. The treatment consists of a monthly injection in the spine, like a lumbar puncture, at an estimated cost of 30,000 euros per month.
Qalsody, based on the tofersen molecule, aims to slow the progression of Charcot disease, also called amyotrophic lateral sclerosis (ALS), a neurodegenerative pathology which manifests itself by progressive muscular paralysis due to degeneration of motor neurons, the cells which control muscle contraction. Incurable to date, its outcome is dramatic: life expectancy is on average three or four years after the onset of symptoms – although 10% of patients live more than ten years. Qalsody only targets patients with a particular genetic form, representing only a small percentage of patients. Namely, patients carrying a mutation in the SOD1 gene (around 1% of ALS cases).
Approved by US and EU health authorities
Tofersen had been approved in recent months by American and European Union (EU) health authorities, very rare news for this disease with almost no treatment. But the French HAS did not follow this example, due to the uncertain benefits of the drug in view of the study carried out by Biogen. This decision provoked the anger of the main French association fighting the disease, the Association for Research on Amyotrophic Lateral Sclerosis (ARSLA), describing it as “cruel and unacceptable”by calling on the President of the Republic, Emmanuel Macron.
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Faced with the outcry, the HAS published this Friday, November 8, an “update” to explain its decision. To be validated, the “early access” system must meet four conditions, she recalls: the medicine must be intended to treat a serious, rare or disabling disease; there is no appropriate treatment already available; the implementation of the treatment cannot be delayed; the medicine is presumed to be innovative, particularly with regard to a possible clinically relevant comparator.
A study that does not convince
Or, si “the first two criteria have been validated”continues the HAS, the data provided by the Biogen laboratory on the two other criteria did not allow it to “to conclude favorably”. “Indeed, the results of the main analysis of the phase III clinical study did not demonstrate a statistically significant difference between tofersen and placebo on the clinical state of patients after 28 weeks of treatment”. “Moreover, the data from the Biogen laboratory study were not sufficiently robust regarding a possible favorable development of a biological marker (neurofilament dosage)”adds the health authority. “Aware of the issues and expectations, HAS regrets not having conclusive data”and invites the Biogen laboratory “to provide such data”.
«So far, the published results have not shown the desired beneficial effect of this drug, but this could be due to the way the study was constructed», explained to Figaro Séverine Boillée, research director at Inserm and researcher at the Brain Institute, at the time of publication of the study, in September 2022.
A significant effect if taken very early?
HAS points out that “the evaluation of the drug Qalsody is continuing, at this very moment, during another procedure, the Biogen laboratory having also requested reimbursement under common law”and indicates that it stands ready to continue discussions with the laboratory in the coming weeks. “This procedure will lead the HAS transparency committee to adopt a new opinion”which will be followed by a decision from the Ministry of Health on the possible reimbursement of Qalsody, as for any medicine.
Experts in the field agree that Tofersen could have a significant effect if taken very early. “The ultimate goal would be to treat people carrying this mutated gene before they develop the disease.», continued in 2022 the scientist Séverine Boillée.
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