MONTREAL — People with amyotrophic lateral sclerosis (ALS) may benefit from additional treatment to slow the progression of the disease. A new drug that might save them some time.
The National Institute for Excellence in Health and Social Services (INESSS) recommends under certain conditions that the drug Albrioza be paid for by the public drug insurance plan.
In a new notice published Friday, INESSS recommends that the Minister of Health include Albrioza on the list of exceptional medications provided that its “manufacturer contributes to reducing the economic burden on the health system”.
This drug is marketed by the American pharmaceutical company Amylyx.
According to the information contained in the institute’s report, it is estimated that treatment with Albrioza during the first three years of the disease would cost the public plan an additional $134.7 million to treat 476 patients.
Last June, INESSS recommended refusing registration of the drug due to doubts regarding its effectiveness. Additional studies, however, have reassured the experts. This time, the institute believes that “Albrioza, taken for 6 months, slows the loss of physical functions (eg, writing, speaking, walking) in people”.
However, it is warned that “the magnitude of this effect is considered to be modest”. In the longer term, the drug might possibly extend the life of patients by regarding five months, indicates INESSS. In addition, the scientific committee considers the cost of treatment to be high. The cost-effectiveness ratio would also be “unfavorable” compared to the two other drugs available on the market, we can read.
Furthermore, INESSS suggests restricting access to this treatment to patients who have been diagnosed with ALS, also known as Lou Gehrig’s disease, for less than 18 months. Patients must also enjoy a certain autonomy and not have had a tracheotomy.
It is added that the authorization for treatment must not exceed a maximum of six months. A request for continuation of treatment might then be filed, always demonstrating that the patient has not undergone a tracheotomy, insists the scientific committee.
Win time
For Dr. Angela Genge, director of the amyotrophic lateral sclerosis clinic at the Montreal Neurological Institute-Hospital, ALS should be seen as pancreatic cancer that leaves patients only a short time to live.
“In such a case, a gain of five or six months is huge!” she pleads.
Thus, it fervently hopes that the Régie de l’assurance maladie du Québec (RAMQ) will not delay in acceding to the INESSS recommendation to make the drug available to Quebecers.
This is also what Amanda Tam wishes wholeheartedly. The 22-year-old Montrealer, a student at McGill University, was diagnosed with ALS in October 2021.
Since then, she has been taking the three drugs available on the market to slow the progression of the disease. In addition to Albrioza, there is riluzole, which would prolong life by a few months, and edaravone (or Radicava), which would slightly slow the progression of the disease.
“My doctors told me that it was definitely better to take it all than to take nothing. I am therefore a body force-fed with drugs, hoping that it will work”, she confides, saying that she observes a certain slowing down of her decline.
However, if the first two treatments are covered by the RAMQ, she has access to the Albrioza thanks to her mother’s private insurance. The young woman therefore fears losing her medication when she graduates at the end of the session this spring.
Amanda Tam also wants to speak on behalf of other patients with the disease or even those who will soon receive the terrible diagnosis.
“The thing regarding ALS is that you have to take advantage of all available treatments because the prognosis can sometimes vary between a life expectancy of six months to two years. If you have to wait a year before having access to an approved drug, it’s ridiculous!” denounces the one who studied psychology and marketing.
Dr. Genge adds by saying that the hope of patients rests on the promise of new treatments, new scientific discoveries. The problem is that you have to buy them that time so that they can still be there when the next drug comes out in six months, a year or even two years.
Albrioza is a combination of two drugs, sodium phenylbutyrate (PB) and ursodoxicoltaurine (TURSO). We do not know precisely its mechanism of action, concedes INESSS, but it would have the effect of reducing the death of neuronal cells.
This medicine is consumed in the form of a powder which is mixed in a glass of water.
ALS is a relatively rare disease that attacks the nerve cells responsible for muscle movement. As it progresses, it leads to paralysis of the body, even affecting the ability to speak, eat and breathe. Life expectancy rarely exceeds five years from the appearance of the first symptoms.
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