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At an appointment at Holland Bloorview Kids Rehabilitation Hospital, 15-year-old Burton Goicoechea, seen here on Feb. 27, 2019, is helped by his mother Jan Genga and a physical therapist as he's lifted from his powered wheelchair to a bed. A new report says provincial governments should expand public funding for Spinraza – one of the world’s most expensive medications – to cover more children with spinal muscular atrophy, a rare genetic neuromuscular disorder, but they should not pay for the drug for teenagers and adults.Melissa Tait/The Globe and Mail

Provincial governments should expand public funding for one of the world’s most expensive medications to cover more children with a rare neuromuscular disorder, but they should not pay for the drug for teenagers and adults, a new report says.

The expert committee that advises most provinces on whether to pay for new drugs concluded that the scientific evidence for Spinraza was too weak and inconclusive to justify public funding for anyone over the age of 12 with spinal muscular atrophy (SMA), a genetic disease that is fatal in some cases and debilitating in others.

The sticker price for Spinraza works out to $708,000 in the first year and $354,000 every year thereafter.

The much-anticipated new recommendation from the Canadian Agency for Drugs and Technologies in Health, or CADTH, is welcome news for some younger patients and their families, but a devastating blow for hundreds of older patients with SMA who had hoped to gain public funding for the only approved treatment for their disease.

“They’ve saved a few more patients, but clearly the majority of patients are still left out of the opportunity for treatment," said Susi Vander Wyk, the executive director of Cure SMA Canada and the mother of Holli, a 22-year-old university student with SMA.

Brent Fraser, CADTH’s vice-president of pharmaceutical reviews, said the experts who examined Spinraza simply did not have enough high-quality evidence to conclude that the drug works in older patients with less severe forms of SMA.

“Really, the emphasis of their recommendation was to identify those who would benefit the most and that’s how they crafted their criteria,” Mr. Fraser said.

SMA is an inherited neurodegenerative disorder that, in the worst cases, kills patients before their second birthdays.

Children with less severe versions of the disease might never crawl or walk. Those who do, still face a frightening prognosis. As they grow up, SMA will almost certainly sap their muscle strength, robbing them, little by little, of the ability to do things as basic as standing, turning over in bed and lifting food to their mouths.

In two randomized double-blinded placebo-controlled trials over the past several years, Spinraza helped enough participants regain muscle strength that the researchers stopped the trials at the halfway mark and gave everyone in the trials Spinraza. However, those trials involved babies and children aged 2 to 12, and there is less high-quality evidence that the drug works as well in older groups.

Right now, provincial governments outside of Quebec cover Spinraza only for babies seven months and younger who have begun to display the severe muscle weakness typical of the disease.

That position was based on a previous CADTH recommendation.

CADTH’s new recommendation, released late Friday, widens the net to include children 12 and under who never gained the strength to walk, and infants who have the genetic mutation that causes SMA, but who have yet to display the disease’s telltale muscle weakness.

Although CADTH’s advice is not binding, provincial public drug plans rarely deviate from it.

The exception is Quebec, which has its own organization for evaluating new drugs for reimbursement, called the Institut national d’excellence en santé et en services sociaux, or INESSS.

Relying on the same evidence but taking a more forgiving approach than CADTH to evaluating drugs for rare diseases, INESSS concluded in December that Spinraza should be funded for SMA patients of all ages, regardless of the severity of their disease.

That discrepancy in coverage has some families considering a move to Quebec, including Jan Genge and her son Burton, 15, who was diagnosed with SMA just before he turned two.

Burton, who uses a motorized wheelchair, still has enough strength in his hands and arms to feed himself and use a smartphone; he hoped that Spinraza would, at the very least, allow him to keep those abilities.

“It’s a bittersweet situation because some people will have that opportunity,” Ms. Genge said. “But if you’re not in that boat, what do you do?”

CADTH also concluded that Spinraza is wildly overpriced: Not even with a 95-per-cent price cut would the drug, made by Biogen, satisfy the agency’s value-for-money test.

“Even if they do reduce the price enormously, it’s still not what we would consider something that is cost-effective based on the amount of money that’s being paid for the health effects that you produce,” said Trevor Richter, the director of CADTH’s Common Drug Review, the part of CADTH that reviews all new drugs that are not for cancer.

Marina Vasiliou, Biogen’s managing director for Canada, said CADTH’s approach to evaluating the value-for-money prospects of drugs such as Spinraza does not work.

“No drug that addresses a rare disease is ever found, at any price, to be cost effective using this method,” she said.

She also pointed out that Biogen already lowered the price for Spinraza through a confidential deal with the negotiating alliance that represents all provinces. However, no party to the deal will reveal the real price, now a common practice in the murky world of prescription-drug pricing.