While the Canadian Association of Drugs and Technologies in Health (CADTH) recommended that the spinal muscular atrophy treatment Spinraza should be made available to a broader population of patients, it said public funding should not cover patients older than 12 — a determination that the treatment’s developer Biogen called “disappointing.”
CADTH expanded its recommendation to include patients 12 and younger who never walked, and to infants who have the genetic mutation that causes spinal muscular atrophy (SMA) but are asymptomatic. It concluded that scientific evidence was insufficient to justify public funding for patients older than 12, and those who have reached the ability to walk independently (SMA type 3).
While acknowledging the new inclusiveness of CADTH’s recommendation, Biogen Canada is “extremely concerned for those patients with SMA who fall outside of CADTH’s updated, yet limited criteria,” said Marina Vasiliou, the company’s managing director, in a news release.
Susi Vander Wyk, Cure SMA Canada‘s executive director, stated: “We at Cure SMA Canada are happy to hear that more of our patients will have the ability to access treatment. For these patients and their families, there is hope for a better future with improved health and a longer life. We are very disappointed, however, that we have left some of our patients with no treatment options. This was their only hope.”
CADTH assesses the costs and benefits of treatments so that it can advise provinces and territories on whether to fund them. Currently, provinces outside of Quebec cover Spinraza for patients up to seven months who are experiencing muscle weakness. Although the agency’s advice is not binding, the provinces’ healthcare plans rarely deviate from it.
Quebec has its own entity for evaluating new treatments for reimbursement — the Institut national d’excellence en sante et en services sociaux (INESSS). Last December, it found that Spinraza should be funded for a broad population of SMA patients.
Health Canada approved Spinraza, the sole treatment for SMA, in June 2017. That December, CADTH announced that the treatment should be covered, but only for patients who have SMA type 1 — the most common and severest form — and who are diagnosed before they turn seven months. At the time, the agency said it found insufficient evidence that Spinraza was efficient, safe, or cost-effective for treatment beyond that group.
CADTH’s recommendation is concerning for many teenage and adult patients with SMA, especially those with type 3 who are or were able to walk independently at some point, said Lawrence Korngut, MD, a neurology professor and director of the Calgary Neuromuscular Program at University of Calgary.
“In my experience, it is the priority of most patients with SMA, due to the inherent weakness and functional limitations, to preserve their mobility and independence as much as possible,” he said. “It is unfortunate that Spinraza will now only be available to this important group of patients only if they live in Quebec but not elsewhere in Canada.”
Manufactured and marketed by Massachusetts-based Biogen, Spinraza is available in more than 45 countries and jurisdictions.