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B.C. denies drug funding for brain-damaging disease
When last month the B.C. government denied medical-plan coverage for a new drug treatment for a rare, brain-damaging disorder, families like the Forstroms of North Vancouver were devastated.
Their 18-year-old son Svenga is one of only 150 British Columbians who suffers from phenylketonuria, a genetic metabolic disorder commonly referred to as PKU. The disorder occurs when the body is unable to process phenylalanine, or “Phe,” an essential amino acid found in the protein of most foods.
Without a special protein-restricted diet, that excess “Phe” can build up in the brain over time, causing mild impairment to severe retardation and brain damage.
But in 2010, Health Canada approved a brand new drug called Kuvan (sapropterin) which helps roughly one-fifth to one-half of PKU patients loosen those protein restrictions without suffering the amino-acid build-up. For a growing teenager on a protein-restricted diet, it was a breakthrough.
“I started the trial for Kuvan last September,” Svenga told The Outlook from the living room of his family home. “It’s doubled my tolerance for protein now.”
Where he used to be allowed only one bowl of Raisin Bran before maxing out his daily protein limit, now that same bowl puts him at just 50 per cent. Likewise, two cubes of cheese or one cup of rice used to push his protein levels right up into the danger zone, but today he can double those portions.
Svenga’s free trial of the drug, however, ended in March. And at a cost of $18,000-per-month for a 16-pills-per-day regimen, the Forstroms, like many others in B.C., couldn’t afford the drug if the family didn’t have private insurance to pick up 80 per cent of the tab.
When Svenga turns 25, however, health coverage under his parents’ plan stops. Without private insurance of his own, he could be out of pocket $216k a year for his prescription. That’s in addition to his already expensive diet of synthetic-protein foods which are on average priced at a tenfold mark-up over their non-synthetic counterparts.
“When that runs out, I really don’t know how I’m going to pay for it,” Svenga said.
Over the summer, B.C.’s Ministry of Health was in talks with the California-based drug manufacturer, BioMarin, trying to hash out a deal by which B.C.’s PharmaCare program would pay for Kuvan treatment for those estimated 20 to 50 per cent of PKU sufferers the drug can help.
But in a statement to The Outlook Thursday, the health ministry said it declined to cover the drug “because of unclear benefits and high drug costs.”
While the statement claimed that no other Canadian province’s drug plan covers Kuvan, Quebec’s drug plan does reimburse the cost of Kuvan treatment for women who are pregnant or may become pregnant. Eugenia Shen, spokeswoman for BioMarin said the California drug-maker is currently in talks with more than one other Canadian province to discuss providing the drug through their respective taxpayer programs.
So far, B.C. is the only province to outright reject it.
“The price we offered [B.C.] is pretty much consistent with that of the US,” Shen said in a phone interview with The Outlook Friday. “It’s weight-based therapy, so the average cost would be $80,000 per patient per year.”
B.C. and the rest of Canada have been carrying out mandatory screening for PKU on all newborn babies since the 1960s. Like other PKU parents, Svenga’s mother, Sally Broadbent, hadn’t heard of those initials until her son was diagnosed with them at birth. At the time, there was no known treatment for the disease aside from the restricted-protein diet. But today, Broadbent says she’s “shocked” the province can on the one hand require all newborns get screened for PKU, and yet throw its hands up when asked for help because the disease was found.
John Adams is the president of Ontario-based PKU patient-support group, Canadian PKU and Associated Disorders, and the father of a 21-year-old PKU sufferer.
“This is about the B.C. Minister of Health being unreasonable in the extreme,” Adams told The Outlook in a phone interview last week. “In Ontario, Quebec and New Brunswick, Nova Scotia and Manitoba, Saskatchewan and Alberta, the low-protein [food] is paid for by the public health program. But B.C. is really the only province that doesn’t do anything for PKU sufferers in terms of providing the low-protein medical food.”
While the B.C. health ministry declined The Outlook’s request for an interview with Minister Margaret MacDiarmid, the ministry said in a statement that it does provide, free of charge, a metabolic formula that helps PKU patients develop properly.
“The ministry does not fund food purchases as this is considered a personal responsibility,” the statement said, adding, “PKU patients who are on income assistance may also be eligible for a $40 monthly diet supplement from the Ministry of Social Development.”