Leqembi, a new treatment for Alzheimer's disease, will not be reimbursed immediately in France. Its High Authority for Health (HAS) is skeptical, citing side effects that are far too serious compared to the insignificant benefits.
"Early access to Leqembi was not retained," summarized Pierre Cochat, president of the HAS transparency commission, during an opinion issued Tuesday by the institution and eagerly awaited by specialists in Alzheimer's disease, the most common form of dementia with tens of millions of sufferers worldwide.
Leqembi (lecanemab), developed by Biogen and Eisai, is one of the main hopes that patient and family associations are clinging to, as is a treatment with a similar function, Kisunla (donanemab) from Eli Lilly.
In clinical trials, these drugs were able to slightly slow the decline of patients who were just beginning to develop the disease. Many experts therefore see this as a major breakthrough, as research into anti-Alzheimer's drugs has been stalling for decades.
But others regret an illusory hope, believing that the observed benefits are so meager that they do not make a difference for patients, especially since serious and sometimes fatal effects – hemorrhages and cerebral edema – are clearly proven.
– Lively controversy –
According to these skeptics, the low efficacy of these drugs means that research has been focusing for too long on an inadequate path, followed by both Leqembi and Kisunla: seeking to limit the formation of plaques of proteins called amyloid in the brains of patients.
The HAS, whose opinions are advisory but generally followed by the government, is therefore speaking out at a time when the controversy remains lively and when other health authorities have already spoken out.
The United States has already approved these treatments, and the European Union (EU) has done the same. But after initially refusing to give the green light, the EU only finally gave its approval last spring, restricting it to patients at the lowest risk of serious side effects.
Above all, authorization does not mean reimbursement, even though these drugs are expensive: they cost several tens of thousands of dollars per year in the United States. In a sign that the distinction is important, the United Kingdom disappointed the associations by authorizing these treatments in principle, but without validating their reimbursement.
It is on this last point that the HAS had to rule and, more specifically, on the merits of "early access." This means that the drug can, from now on, be reimbursed at a price set by its manufacturer.
This avoids waiting for the normal procedure. The benefit is for patients to have access to an innovative treatment, and for the laboratory to quickly market its drug.
– “Very insufficient” benefits –
This will not be the case for Leqembi. The HAS concludes, in view of the studies provided by the laboratories, that there is no justification for granting such preferential treatment.
"We know how hopeful everyone was," Cochat acknowledged. But he contrasts the benefits, which are "very insufficient compared to what we expected," with "side effects that are not at all minor."
This does not call into question the possibility that Leqembi will one day be reimbursed in France. The HAS, which will decide within a few months on the advisability of a normal procedure, assures that nothing is excluded.
But "it is obvious that we cannot expect a dazzling evaluation," warned Mr. Cochat.
Within the medical world, this decision has provoked contrasting reactions, between those convinced of Leqembi and those sceptical of it.
"There is still some disappointment," admits researcher Bruno Dubois, a neurologist at the Pitié-Salpêtrière Hospital (AP-HP), to AFP, calling on people not to overlook the benefits of "a few more months of being able to talk to their grandchildren or go to the theater."
On the contrary, "it's the best decision for Alzheimer's patients and their families," British psychiatrist Rob Howard, a specialist in old age at University College London, told AFP. More recent data confirms that Leqembi and Kisunla "do not actually modify the disease."
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