Parents in Canada are enraged at the cost of a lifesaving drug for their sick children. Cystinosis is a deadly genetic disorder that destroys the kidneys, and until now, children suffering from the disease have been surviving on the drug Cystagon. But recently, Cystagon was replaced by the newer medication, Procysbi. Both drugs have the identical active ingredient. The difference? Procysbi has a special coating which allows the medicine to be absorbed more slowly by the body. The result is that patients need only take it every 12 hours, instead of every six. For this “improvement” parents will need to spend 3,000% more, or about $300,000 per year. Cystagon was costing families about $10,000 per year and was covered by Health Canada, the government’s single-payer system.
According to CBC, “Cystagon…was never formally licensed in Canada but was available through a special access program operated by Health Canada. [I]f a drug gets approved for market in Canada, other unapproved forms of the drug are no longer allowed to be brought in through that program. The fact that the approved drug costs 3,000 percent more is irrelevant.” Since Procysbi is now an approved drug to treat patients with cystinosis in Canada, Cystagon can no longer be an option for them.
Horizon Pharma told CBC News that the price changes follow the rules for “a breakthrough medicine,” as set by a federal agency that ties Canadian prescription drug prices to the average price in eight other countries including the U.S., England, and Germany.
Families are particularly unhappy because both of the lifesaving drugs were developed by the same University of California San Diego team, using funds raised by parents doing grassroots fundraising events like bake sales and golf tournaments.
Horizon has responded by saying it would provide patients with Procysbi for free while price negotiations take place. One family, with two young daughters suffering from cystinosis, was offered the drug for three months. But they don’t know what they will do at the end of that time, since the cost of the lifesaving treatment will be comparable to the price of a new house every year.
Doctors who treat the rare disease would like the option to offer both drugs, depending on what might work better for a particular patient. But as of right now, that isn’t a possibility.