MONTREAL — The federal government will spend up to $1.5 billion over the next three years to improve access to drugs used to treat rare diseases, Health Minister Jean-Yves Duclos said Wednesday.
Almost all the funding envelope — $1.4 billion — will go to the provinces and territories, Duclos said, which will share the costs of the drugs with Ottawa. Another $33 million will go to Indigenous Services Canada to support eligible First Nations and Inuit patients with rare diseases, he added.
Duclos said he wants to increase coverage for existing drugs and create a list of new and emerging treatments that would be subsidized relatively equally across the country, removing the need for people to move outside their home province for care.
"The national strategy will increase and harmonize coverage for existing drugs; it will add coverage with emerging and new drugs; it will speed up the ability to do important diagnostics, especially for children," he told reporters in Montreal.
While there are no treatments for the majority of rare diseases, those that do exist are often expensive — ranging from $100,000 and $2 million per year — and coverage can vary from province to province, Duclos said.
"Canadians must have better access to affordable prescription drugs; for some, paying for much-needed medication represents an impossible choice between managing their health-care situation and paying everyday bills," Duclos said, adding that he's heard "heartbreaking stories" of families who have considered moving to a different part of the country to be treated.
Duclos said 9,600 people could benefit from the new funding, including 4,500 children. Around one in 12 people has a rare disease, Health Canada said.
Treatments for rare diseases account for around 10 per cent of the total cost of drugs in Canada, Duclos said. "The cost of drugs for rare diseases also increases very quickly. Over the last 10 years, it has increased by an average of 32 per cent. So we see where we're heading," he said.
Gail Ouellette, the president and scientific director of a Quebec group representing people with rare diseases — Regroupement québécois des maladies orphelines — said the announcement will give people hope that they can access treatments regardless of where they live in Canada.
Ouellette told the news conference that people with rare diseases face many obstacles in the health-care system, including the length of time it takes to get diagnosed and the high costs of treatment.
"But one of the worst things for these people is to know that a medicine exists for their condition, but to not have access — to know that it is authorized by Health Canada and that people like them have access elsewhere in the world or in other provinces, but not them," she said.
Among the founders of Ouellette's group were individuals with ataxia, a disease that leads to loss of muscle control; Morquio syndrome, a genetic condition that affects the growth of a child's bones and organs; and Fabry disease, a genetic condition that can lead to heart and kidney problems.
Durhane Wong-Rieger, CEO of the Canadian Organization for Rare Disorders, told the news conference that the announcement recognizes the importance of early access to effective, promising medicines for patients "but also the need for early diagnosis and treatments, preferably before patients are seriously compromised."
The new funding includes $20 million over three years to gather evidence on the safety and effectiveness of drugs, while another $32 million will be used over five years to fund research on rare diseases and to establish a clinical trials network.
Wong-Rieger said she's pleased to see the funding for research because only around five per cent of rare diseases have an effective treatment.
This report by The Canadian Press was first published March 22, 2023.
Jacob Serebrin, The Canadian Press