The only treatment for his rare disease would cost nearly $ 200,000 a year
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Neither the RAMQ nor the insurer of a couple in the Laurentians want to reimburse a drug that costs $ 16,000 a month, but that could save their daughter’s life.
“We do not know where to turn, we do not know what to do […] Its life is limited if it is not treated and if a drug can help [my daughter], I want to try it, “pleads Julie Beauregard, her eyes full of hope.
But the mother of little Ellie, suffering from a rare disease, the family Mediterranean fever, says she is caught off guard.
“You feel lonely and it’s hard when you do not know that,” adds his spouse François D’Astous, who had nothing prepared to face the bureaucracy of drug claims.
Their 2 ½ year old daughter has a severe form of this little-known disease, which causes inflammation of the internal organs. Every month, she has intense fever, up to 41 ° C and last more than five days.
“The only thing she does is throw up,” her mother blows.
She already takes the maximum dose of anti-inflammatories, without result.
Doctors at CHU Sainte-Justine have prescribed injections of Canakinumab, a drug marketed by Novartis called Ilaris. They would cost $ 16,000 each month, or nearly $ 200,000 a year for the rest of their lives.
A huge sum for the Saint-Colomban couple, whose spouse is a manager at Bombardier and the part-time educator spouse.
The group insurance plan for the family, Industrial Alliance, rejected the request. In its letter, read by Le Journal , the company says “there is a lack of evidence regarding the effectiveness of this drug.”
This response rages Julie Beauregard, because in their application, the doctors are clear. Without the injections, their daughter may die. “I do not understand that a doctor writes that and [we] refuse [the medicine],” she says.
The family turned to the RAMQ and its exceptional drug program. But their request was rejected outright because the family is already insured.
To be part of it, the National Institute of Excellence in Health and Social Services (INESSS) must study and recommend it, which has not been done, confirms the INESSS.
Novartis Pharmaceutical spokeswoman Julie Schneiderman said the request was not made because the drug has already been reimbursed to Quebecers, both by the RAMQ and by private insurers.
Industrial Alliance was unable to respond to the Journal yesterday. The RAMQ did not confirm what Novartis said.
As a last hope, the family now hopes to get help from the pharmaceutical industry.
The drug is of a “formidable efficiency”
The medicine that the Laurentides family is trying to obtain to save their daughter suffering from familial Mediterranean fever is “terribly effective,” says a doctor at Sainte-Justine University Hospital Center (CHU).
According to the pediatrician, rheumatologist and immunologist Jean-Jacques De Bruycker, the injections would allow the little Ellie D’Astous to have a normal life. But without these, the repeated inflammation of its organs will make it stop working much sooner.
Familial Mediterranean fever is very rare in Quebec, but more widespread, as its name suggests, in countries around the Mediterranean Sea. Dr. De Bruycker says she sees it mostly in young Quebec patients whose parents are immigrants.
For some unknown reason, this disease causes inflammation of the organs, as if they were fighting a virus that does not exist.
“It looks like a big flu and it passes. These are brutal surges of fever, “he says.
The genetic disease, transmitted by a recessive gene in both parents, can not be cured. On the other hand, it is easily treated in 90% to 95% of cases with an inexpensive drug called colchicine.
But in a handful of cases, like Ellie D’Astous’s, an antibody like Ilaris is needed to block the immune system and inflammation.
“But the price is another pair of sleeves to accept,” says the doctor. On the other hand, the treatment is not experimental. “It is absolutely not new that there are patients resistant to colchicine,” he continues.
Without treatment, patients can not only die, but they must also endure a “quality of life that is truly horrible and mortgaged”.
But treated, patients can work and avoid costly complications, he concludes.