Canadians with Fabry disease — many of whom have no access to drug therapy — will finally be able to obtain two staggeringly expensive medications under a new government deal.
Fabry patients will learn today that they must enroll in a research study to receive medicine to treat the rare disorder, which is caused by a faulty gene.
"Federal and provincial governments and two drug developers are about to enter into a three-year funding agreement to support an independent post-market study of enzyme replacement therapies for Fabry disease," said a draft patient fact sheet from the federal and provincial governments dated May 11 and obtained by The Globe and Mail.
"The purpose of this project is to contribute to a better understanding of this rare disease and the effectiveness of the enzyme replacement therapies that the two drug companies have developed to treat it."
An estimated 5,000 people worldwide, including more than 200 people in Canada, have Fabry disease, which primarily afflicts men. Those who inherit the abnormal gene cannot produce enough of a particular fat-eating enzyme. They typically experience pain, difficulty sweating and skin rashes.
In the latter stages of the disease, patients have strokes, heart failure and kidney failure. The life expectancy of untreated men is 60, said Dr. Michael West, who runs an adult Fabry clinic at the QEII Health Sciences Centre in Halifax. Women with the disease experience mild to moderate symptoms.
Although Health Canada has licensed two drugs to treat the condition — Fabrazyme and Replagal — provinces have struggled to pay for them.
It costs $275,000 to treat the average patient for a year with Fabrazyme. The drug has been approved for funding in 40 countries, where it is covered by governments or private insurance plans, said Susanne Courtney, spokeswoman for Genzyme Canada Inc., the company that markets the drug.
The Canadian Expert Drug Advisory Committee, a national advisory body that studies the cost-effectiveness of orally ingested drugs and makes recommendations to provincial government drug plans as part of the Common Drug Review, did not recommend either medication be funded, largely because it did not find them cost-effective.
The committee described Replagal as typically costing $239,200 for a year's supply before pharmacy markup.Under the new government deal, patients would be able to get treatment so long as they become part of a research protocol, Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, confirmed yesterday. However, she refused to divulge any details of that protocol, citing a confidentiality agreement.
However, the fact sheet — to be presented to patients at a Canadian Fabry Association conference in Halifax today — said the Canadian Institute for Health Research, a federal government agency, and the Fonds de la recherche en santé du Québec will oversee the study.
All patients in Canada are eligible for enrolment; those who meet the criteria will get the drug. No start date was given.
The federal and provincial governments are expected to announce the agreement, with funding details, as early as next week.
A spokesperson for federal Health Minister Tony Clement's office could not be reached yesterday.
Despite intense lobbying by patients, coverage is spotty in much of Canada.
The treatment is generally available in Alberta; in British Columbia it is funded through hospitals. In Ontario, some patients obtain it through a compassionate access program run by drug companies. While Ms. Wong-Rieger said she is pleased Fabry patients will get treatment, what is really required is a national orphan drug plan.
"I think the biggest thing that bothers me is that this country has not acknowledged that a public health system does not fund life-saving treatment for people with rare disorders, unlike the United Kingdom," Ms. Wong-Rieger said in a telephone interview yesterday. "Probably, we're the very worst developed country in the world to have a rare disease."
The European Union, the United States, Japan and Australia have orphan-drug policies. While the U.S. policy doesn't necessarily provide drugs, it has provided incentives to pharmaceutical companies to find treatments.
In a telephone interview from Halifax yesterday, Dr. West, who treats 60 patients with Fabry disease, said he hopes the drugs will halt or slow the disease.
He said he couldn't divulge details of the agreement, except to say that he was "extremely pleased."
"Patients have been waiting for over a year for this. I'm thrilled it's going to happen and in some ways, I can't believe it's going to happen."
