A lobby group for patients with a rare, brain-threatening disorder known as PKU is taking the rare step of filing a complaint that Ontario’s Health Ministry is practicing medicine without a license.
The complaint to the College of Physicians and Surgeons of Ontario alleges that the government is maintaining excessive restrictions to taxpayer funding of the drug Kuvan under an exceptional access program.
“The simple question is do you want the government to be your doctor? I don’t,” John Adams of Canadian PKU and Allied Disorders Inc. said Tuesday.
“I think this is forging new ground,” he added, speaking of the complaint.
His son, John, 29, suffers from the condition and gets Kuvan on compassionate grounds from the manufacturer, BioMarin.
Health Minister Eric Hoskins, a family physician, agreed in May to review the criteria for the drug, which costs an average of $70,000 a year per patient.
“Our decisions are based on science and evidence,” Hoskins said during the legislature’s daily question period, noting coverage of the drug is now considered on a “case-by-case basis.”
He said the government is still waiting for the manufacturer of Kuvan to make a key submission with further evidence of the drug’s efficacy so the review by a provincial panel of experts can proceed based on scientific data.
The Pan Canadian Pricing Alliance, a provincial drug consortium, has recommended against funding Kuvan, saying there is no evidence it’s any more effective than following a careful diet yet costs thousands more, said Hoskins spokesman Shae Greenfield.
Ontario and Saskatchewan are the only two provinces with some form of public coverage for the medication and have the same criteria.
Progressive Conservative MPP Gila Martow said PKU patients and activists are “frustrated” because “not one single patient” has been given taxpayer-funded Kuvan in Ontario.
“The criteria is too difficult.”
Adams, a former Toronto city councillor, said the clock is ticking because Kuvan’s three-year listing under the exceptional access program for drugs expires in February.
He said eight doctors, specialists in metabolic disorders, who treat PKU in Ontario have all urged the government to ease restrictions on Kuvan.
He estimates about 150 people in Ontario need taxpayer-funded coverage of PKU, for which newborns are screened at birth, because they do not qualify under private drug plans.
PKU patients live on severely restrictive vegan, low-protein diets because they can’t digest certain proteins. Without the diet, they face anything from mild cognitive impairment, including learning disorders, to severe intellectual problems.
They take special protein supplement in a formula that costs $10,000 a year, along with special foods including breads that cost $16 a loaf and cheese that sells for $20 a package. The government reimburses those costs, which some families have estimated at $20,000 a year.
John Adams Jr. said he felt like “a completely different person” after starting on Kuvan on his doctor’s recommendation in 2007, following his diagnosis of a learning disability in high school.
He recalls the sensation that his brain suddenly had “a high-speed internet connection instead of dial-up,” allowing him to dramatically improve his performance in school.
Groups representing patients with rare diseases are increasingly pressuring governments for better treatment. The provinces are studying ways to do better with a report due early in the new year to a meeting of health ministers.