Family desperate for universal access to spinal muscular atrophy drug
CTV Vancouver Island
Published Tuesday, March 12, 2019 9:41AM PDT
A Victoria family is renewing its efforts to advocate for universal coverage of a costly drug.
Spinraza treats spinal muscular atrophy, a rare and often deadly neuromuscular disease.
Vivienne Damatan, the mother of a toddler with the condition, said her daughter Zoe has seen immense progress with it.
"She can walk for over a kilometre now, whereas before, she would fall after about a fourth of a block," said Damatan.
With the help of private benefits, the spinal muscular atrophy patient has been receiving doses of Spinraza into her spine for a year.
"Spinraza actually stops the progression," said Damatan. "All of it is worth it. All of the struggles, the worries, worries about spinal headaches, the fasting that goes into it."
But a recent national drug review isn't recommending Spinraza for everyone, which is concerning to Zoe's parents since Canadian provinces and territories often lean on it when considering coverage.
"Honestly it kind of blindsided us," said Damatan.
Zoe doesn't qualify under the expanded recommendation, which suggests access for SMA patients 12 years or younger.
Other qualifications include the ability to breathe without a ventilator and be unable to walk independently.
The Canadian Agency for Drugs and Technologies in Health says the expanded access is being suggested for "patients who are most likely to benefit from treatment."
But Damatan says her daughter is evidence that the drug is working for her, reaching milestones like mounting on her tip toes and walking on her knees.
It's a positive step, and now Zoe's parents are renewing their pleas for universal coverage while preparing to reapply for private benefits, hoping their health care provider will see the difference.