The Canadian Organization for Rare Disorders (CORD) is calling on all Canadians to come together and ask the government to help save lives by implementing its own Rare Disease Strategy.
CORD did have a rare disease strategy already prepared in 2015, but was never adopted. CORD has launched a letter campaign on the Canada4Rare website, encouraging Canadians to write to their local MPs to encourage collaboration between provincial and federal governments in implementing Canada's Rare Disease Strategy.
The federal government has committed $1 billion to set up Canada’s first rare disease drug strategy, promised to be rolled out this year. But even after two years of planning, details are not yet finalized.
One of the nearly three million Canadians suffers from a rare disease, two-thirds of whom are children. CORD says those with a rare disease have poorer access to effective medicines than Canadians with more common conditions and are far less likely to get an essential drug than rare disease patients in many other countries.
"We are trying to get support and get people to understand what is happening in Canada as far as rare diseases go," said local rare disease patient advocate Beth Vanstone.
Beth and her daughter, cystic fibrosis (CF) warrior Madi Vanstone, know this to be true as they have been fighting for the CF community and rare disease patients for the past decade in an effort to get them the life-saving medications they need.
The pair first met with Health Minister Christine Elliott, who was the provincial health critic at the time, nine years ago when they convinced the Ontario government to approve coverage for CF drug Kalyedco after a lengthy public battle. Had Kalydeco not been funded by OHIP, the Vanstones would have had to pay $350,000 yearly for the drug. Even with private insurance and participation in a drug study, they were looking at a cost of about $60,000 per year.
But that was just the beginning of their legacy, as they continue to advocate for others in the rare disease community, who still don't have access to the medications they need.
They want to know why, in one of the wealthiest countries in the world with a highly developed health-care system, there is so much red tape and restrictions preventing patients' access to life-saving medications. They believe Canada has been ignoring the rare disease community for too long and the time is now to implement a rare disease strategy similar to countries like the United States and the United Kingdom.
Beth says the plan for rare disease patients needs to be a sustainable method that includes patient and doctor engagement in order to develop realistic policies and strategies.
"They (doctors) are the ones that know the patients and understand the disease, bureaucrats...do not understand these rare diseases and how they affect patient families," she said.
According to CORD, Canada remains the only developed country without a national plan for rare diseases and without an orphan drug policy. It can take Canadians with a rare disease more than seven years to get a diagnosis and Canada’s public drug plans provide only 30 to 40 per cent of rare disease therapies approved by Health Canada.
“We believe there is genuine commitment and even consensus at the federal and national level, but there has been relatively little visible dialogue at the level of provincial government and health systems,” said Durhane Wong-Rieger, president and CEO of CORD.
“We suspect there are behind-the-scenes conversations between the federal and provincial governments, and these need to be moved into the public discourse so all parities will be aligned when that national Rare Disease Drug Strategy is implemented," Wong-Rieger added.
While the Vanstones are aware that rare disease medicines come at a high cost, they believe there are ways to get them into the hands of patients at a fair price.
"I do think though that collaboration is the answer to getting these drugs to patients in Canada and I think if Health Canada would partner with the manufacturers and alongside patients, that there can be solutions to the high costs," said Beth.
She also suggested the cost for treat patients with orphan drugs would be economically beneficial for society as a whole, allowing them to live a healthy life, and contribute to society, as opposed to filling up hospital beds.
She notes the pandemic has exposed the 'sour' relationship between Health Canada and big pharma.
"I would hope it's opened the eyes of a lot of people to how bad our (health-care) system is," said Beth. "I am hoping we can use what has happened to highlight all the holes in the system Canada has right now.
"I think if Health Canada genuinely wants better for their rare (disease) patients, they need to change their attitude and begin to look for solutions with pharma," she added. "And look for different ways to get drugs to the patients faster, get a price that's affordable."
The mother-daughter duo have taken part in a number of seminars and webinars in the past, advocating for the rare disease community. The Vanstones will be heading to Ottawa in June for a conference on rare diseases, and working on fleshing out the Rare Disease Strategy from 2015.
"We need to make it the best it can be and hopefully have flexibility for change as it comes because science is changing all the time," said Beth.
Last summer, CF drug Trikafta was approved by Health Canada, helping approximately 1,100 eligible patients over the age of 12 with CF.
Beth commended Elliott for her efforts in getting the drug approved for patients in Ontario.
"Trikafta's struggle really highlights how things can happen," said Beth, noting the lengthy process it had to go through before patients were able to access it. Some patients were able to have gain access quicker due to varying coverage approvals in each province.
Beth and the rare disease community refer to this as the "postal code lottery," when only some patients get access to medications depending on where they live, something they hope a national rare disease strategy will eliminate. She says parts of the lottery issue will be addressed in the country's pharamcare plan.
"It shouldn't spend on where you live whether you get access or not," said Beth.
Sometimes it can be years before drugs get covered by provinces after being approved by Health Canada.
"We need something that works for all of Canada," she said.
On the Canada4Rare website, Canadians will find a petition they can sign on change.org, as well as easy steps they can quickly take to let their elected representatives know their support to “Save Rare Lives” and to share the initiative through their social media platforms.
"I really think that it's important that Canadians become aware and expect more from our government so when something happens, they are looked after, too," she said. "It's just a matter of putting patients first."
