Patients group lobbies for rare drug
Ministry calls complaint over brain-threatening disorder ‘frivolous, inappropriate’
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 practising medicine without a licence.
The complaint to the College of Physicians and Surgeons of Ontario (CPSO) alleges the government is maintaining excessive restrictions to taxpayer-funding of the drug Kuvan under an exceptional access program. “This is forging new ground,” John Adams of Canadian PKU and Allied Disorders Inc. said Tuesday. “The simple question is: Do you want the government to be your doctor? I don’t.”
Adams’ son, also named John, 29, was diagnosed with a learning disability from PKU (phenylketonuria) in high school and gets Kuvan on compassionate grounds from the manufacturer, BioMarin.
He said it improved his performance in school dramatically within three days, making him feel like his brain had “a high-speed Internet connection instead of dial-up.”
Health Minister Eric Hoskins, who agreed in May to review the criteria for the drug, which costs about $70,000 annually per patient, called the complaint to the CPSO “frivolous and inappropriate.”
“Three times now — twice provincially and once nationally — this drug for this purpose has been reviewed and the recommendation was not to fund it,” Hoskins, a family doctor, told reporters.
“Notwithstanding that, we’ve provided it on an exceptional basis. There’s no other province that is doing more.”
The government is waiting for Kuvan’s manufacturer to submit more data expected later this month or early next year.
“Our decisions are based on science and evidence,” Hoskins said during question period in the legislature, noting coverage of the drug is now considered on a “case-by-case basis.”
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.
Ontario and Saskatchewan are the only two provinces with public coverage for the medication and have the same criteria reached in consultation with the manufacturer, but Hoskins spokesman Shae Greenfield said no patients meet the standards.
That leaves patients “frustrated . . . the criteria (are) too difficult,” Progressive Conservative MPP Gila Martow said in the legislature.
“There is something wrong when a drug exists and not one person has qualified for it,” New Democrat MPP France Gélinas told the Star, urging the government to “err on the side of patient care.”
Adams, a former Toronto city councillor, said eight doctors who treat PKU in Ontario, all specialists in metabolic disorders, have urged the government to ease restrictions on Kuvan and that governments need to find subsets of PKU patients who can gain a significant clinical benefit from the drug.
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.
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 in January at a meeting of health ministers.
“The high cost to treat rare diseases shouldn’t mean Ontarians are abandoned by their government to fund their own medical expenses,” said Conservative MPP Michael Harris, who has launched a website at treatraredisease.ca
He’s calling on the government to set up an all-party committee of MPPs on diagnosis and treatment of rare diseases, which Hoskins has admitted sometimes falls through the cracks of the health care system.
BioMarin said in a statement it is “disappointed that patients who could benefit from this treatment are still not able to access it through public funding” and it will resubmit Kuvan for approval in the new year.