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