A North Vancouver family says the province is unfairly denying them treatment for a rare disease that threatens to leave their son with devastating brain damage.
The Ministry of Health announced last month that it would not offer PharmaCare coverage for Kuvan, a new and costly drug for the treatment of phenylketonuria (PKU), a genetic disorder that causes the body to metabolize regular protein into a dangerous neurotoxin. The reasons given: too expensive without adequate proof of benefit.
For 18-year-old North Vancouver resident Svenga Forstrom, who is one of just 250 people in the province with the disease and one of only 60 who would reportedly respond to treatment with the drug, the decision means a much more difficult life ahead, according to his family.
PKU sufferers must adhere to an extremely strict low-protein diet that eliminates almost all regular food or face gradual brain damage.
Left unmanaged, PKU would lead to such severe mental incapacitation that the patient would have to be institutionalized, according to John Adams, president of CanPKU, a group lobbying provincial governments to cover the new drug.
Svenga has been taking Kuvan since being approved for a drug trial two years ago. He says it has made a dramatic difference in his life.
"(Kuvan) makes it easier for me to keep track of what I'm eating, or not have to worry so much about keeping track. It's great," he said. "It can be overwhelming to have to break down every single thing you eat into portions and then figure out how much phenylalanine was in each portion for everything."
"His tolerance for normal dietary protein has at least doubled - probably somewhat more than doubled - while being on this drug," said John Forstrom, Svenga's father. "That increase makes a huge difference in terms of his general quality of life."
The results are even more profound among some other patients, Adams said.
Svenga is one of a very lucky few to have access to the drug, which is 90 per cent covered by his father's private health insurance. But the day looms when he will not longer be covered by his father's private plan.
"There's no way I'm going to be able to afford to cover this drug myself. My diet would go back to being as restrictive as it was before," he said.
Usually, the only food options for PKU sufferers are specially-made low-protein alternatives, but they cost about 10 times more than their regular grocery store equivalents. Those alternatives are subsidized in every province but B.C. and P.E.I., Forstrom said.
But the Ministry of Health stands by its decision. "The ministry completed its Sapropterin (Kuvan) drug review this summer, and has decided to accept the Common Drug Review's and the B.C. Drug Benefit Council's recommendation to not fund Kuvan for PKU patients because of unclear benefits and high drug costs. No other provincial drug plan currently covers Kuvan," it said in a statement. "The annual cost per patient for the medication can be up to $180,000 per year for an adult, depending on dosing and the patient's weight. Manufacturers - and not taxpayers - should be responsible for demonstrating that the drug provides clear benefit."
But the ministry's cost estimate is way out of line, according to Eugenia Shen, spokeswoman for Kuvan's developer BioMarin.
"That's extremely high," she said with disbelief. "I'm not sure where the miscommunication is, but it's certainly not that high. . . . I don't know where that number is coming from."
The average cost is closer to $80,000 per year, she said - a price the company is currently negotiating with other provinces' health ministry drug plans.
The price is still high, Forstrom admits, but he remains committed to lobbying the government until it reverses its decision. "That's why we have socially supported medicine - to make sure those treatments are attainable and to make sure everybody gets the best healthcare that's available to them," he said.
