A month later, the lack of understanding persists. At the end of October, the French High Health Authority (HAS) ruled against the approval of “advance” reimbursement of Qasoldy, a drug that would supposedly fight a rare form of Charcot disease.
The reason, according to the study of health data: an inadequacy of the benefit-risk balance, according to an update published by the agency. For patients, powerless in the face of a disease as devastating as it is difficult to manage, the lack of understanding is strong.
“The data used by the HAS is the same as that used in Europe, except that the HAS did not accept this treatment. We have a rigid authority that today does not want to listen to anything,” annoys Sabine Turgeman, director of the association. for Amyotrophic Lateral Sclerosis Research, at the BFMTV microphone.
The drug developed by the Biogen laboratory aims to stop the progression of a very specific form of Charcot disease. Let us remember that this is a so-called degenerative disease, which causes a deterioration in the patient’s functions over a long period and then leads to death.
“I was desperate”
Patients saw hope in this revised treatment, especially since the drug was approved in recent months by American and then European health authorities.
“I was desperate, since I received this treatment, I have the impression of living normally! It works, this product works,” Sébastien, 48, sick and torn by severe pain, testifies to BFMTV.
Why is France different? Different indicators are analyzed, as François Salachas, AP-HP neurologist, explains to BFMTV:
“The main criterion of the study was the function of the patients. And when we look at the end of the study, at six months, there is no significant difference in functional deterioration. On the other hand, the study of a marker, neurofilaments, showed that from the second month onwards there was a significant drop in this rate of neuronal destruction.
Treatment promoters consider that this last point gives hope for an effect on symptoms if Qasoldy is administered earlier, before the first clinical signs of the disease appear. But this hypothesis remains uncertain in the absence of conclusive data.
Upon request, the Ministry of Health guarantees that it maintains current access to the treatment provided to around fifty people in France. The High Health Authority must issue a new opinion in the coming days on this treatment, its hypothetical reimbursement and its framework.
Source: BFM TV
