A leading rare disease advocate is criticizing Canada’s national strategy for rare disorders as “underwhelming,” “unequal,” and even “suicidal.” The advocate warns that the federal government is failing patients by not adopting global best practices or leading national coordination efforts.
In an exclusive interview with True North, John Adams, co-author of a recent Macdonald-Laurier Institute report on the state of Canada’s rare disease policy, said federal initiatives lack substance, leadership, and vision.
“Canada has been intransigently digging its head in the sand like an ostrich,” Adams said. “We’ve been freeloading on U.S. innovation for decades, while Europe, Japan, and even Russia have adopted orphan drug acts. We haven’t.”
Adams, who is also a member of Health Canada’s implementation advisory group on rare diseases, said the group has yet to meet once in 2025.
“The meetings are virtual. We’ve had no engagement this year. It’s underwhelming,” he said.
Canada’s National Strategy for Drugs for Rare Diseases was launched in 2023 with a three-year $1.5 billion funding envelope. Seven drugs have been publicly named under the program, with provinces allowed to select which to include in bilateral funding agreements. Adams said this has worsened Canada’s long-standing “postal code lottery” problem.
“When the strategy was announced, we were told it would reduce inequities,” he said. “Instead, federal dollars are going to provinces covering only three or four drugs, while others are covering six or seven. There’s no consistency. It’s actually baking inequality into the system.”
Adams pointed to Ontario and Quebec as stark examples of disparity.
“A baby born today in Ottawa is screened for 35 rare conditions,” he said. “Cross the river into Gatineau, and that number drops to 16. There’s no national coordination on newborn screening, and that’s a federal failure.”
Canada has no orphan drug law, despite the U.S. enacting its Orphan Drug Act in 1983 and seeing over 5,000 drug approvals since.
“Canada charges developers fees to submit orphan drugs,” Adams said. “The U.S. waives those fees. That’s one of many barriers that make small companies think twice before entering Canada.”
He noted that rare disease therapies, which often treat small patient populations, require tailored policies, flexible evidence standards, and faster pathways.
“We have five layers of gatekeepers in Canada for drug approvals,” said Adams. “No other country has that. We are proud to be slow.”
He added that the use of outdated health economic metrics is also blocking access to life-altering therapies.
“Our system still uses a $50,000 per QALY [quality-adjusted life-year] threshold based on 1990s thinking. It hasn’t been adjusted for inflation, severity, or rarity,” Adams said. “That’s why patients get denied access to approved drugs.”
The father of a son with PKU, Adams, shared a personal example: a drug approved by the FDA in 2007 and by Health Canada in 2010 is still not covered in many provinces, despite his son benefitting from it through private insurance.
“Thank God for employer plans,” he said. “But we shouldn’t rely on private coverage to fill in the gaps of a broken public system.”
Canada spends over $370 billion on healthcare annually.
“Spending $500 million a year on rare diseases is a drop in the bucket,” said Adams. “It’s not even a down payment on a house of better health for rare disease patients. It’s just a deposit.”
Despite calling the national strategy flawed, Adams remained optimistic that reforms are still possible.
“We don’t need to take over provincial jurisdiction,” he said. “We just need federal leadership to harmonize newborn screening, to facilitate collaboration, to stop pretending inequality is acceptable.”
When asked why Health Canada avoids the term “orphan drug,” Adams didn’t mince words: “It’s anti-Americanism. Even when something works, we resist it if it comes from the U.S. Meanwhile, patients suffer.”
Canada currently recognizes over 11,000 rare diseases, yet fewer than five per cent are treatable.
Adams said the cost of doing nothing is high economically, morally, and medically.
“We’re too comfortable with average,” he said. “But in this case, average has become mediocre. And mediocrity, when it comes to rare diseases, is deadly.”