Newly approved treatment for rare children's disease can cost more than $300,000 annually
Dozens of families across Canada have expressed dismay over a recent decision by Health Canada that is forcing them to switch to a hugely expensive drug to treat their children’s illness.
"It's a ridiculous spot to put parents in," said Waterloo man Trevor Strauss, who had been using another drug – Cystagon – to treat his daughter Gabbie’s cystinosis. Left untreated, the genetic disease will destroy the child’s kidneys, reported CBC News.
The problem for Strauss, as well as numerous other families living with the disease, stems from Health Canada’s recent approval of a new treatment called Procysbi. It contains the same active ingredient as Cystagon, and was developed by the same scientists at the University of California San Diego. But Procysbi is formulated so that users have to take it every 12 hours instead of every six.
The new formulation offers some medicinal benefits, but its cost is a considerable burden for patients. In Gabbie’s case, Cystagon treatment costs $10,000 a year and had been covered by the Ontario government, but that’s being cut off. Procysbi, on the other hand, will reportedly cost $320,000 per year for the rest of her life.
Cystagon was never formally licensed in the country; instead, it was available through a special access program operated by Health Canada. But under the agency’s rules, the approval of a new drug — regardless of its cost — nullifies the availability of other unapproved forms of the drug through that program.
Even though Health Canada has approved Procysbi, provincial governments have not yet agreed to include it in their plans. That leaves parents like Strauss, who don’t have private insurance, in a bind. “There's no reason for the drug to be this expensive except for profit margins,” he said.
Horizon Pharma, which manufactures Procysbi, told CBC News that its Canadian price follows the rules for a “breakthrough medicine” as set out by the Patented Medicine Prices Review Board. The provinces, through the pan-Canadian Pharmaceutical Alliance, are trying to negotiate a lower price, and Horizon has informed some patients that it will provide the drug at no cost for a limited period.
Specialists who treat cystinosis patients would like the option to prescribe both drugs. But according to Health Canada, patients will no longer have access to Cystagon unless their doctor can provide a valid medical reason.
