For 21 years, a Whitby woman and her family have been in and out of hospitals, fundraising and hoping for a breakthrough that would help her in her fight against a rare disease.
The road has been long and it’s been exhausting for Tori Lacey, who suffers from Spinal Muscular Atrophy (SMA) Type 2, but finally the miracle is here in the form of a drug called Spinraza.
“It’s frustrating because I’m in university and I’m trying to focus on my studies, I’m trying to focus on my career, and this is not something I have time and energy to fight for,” Tori said. “It would mean that I would be able to plan my future, I would just have a sense of knowing what’s going to happen in my future, because I know I that won’t get any weaker.”
However, she found out that her type of SMA doesn’t allow for her to be covered by health insurance in Ontario. The cost of a vial of Spinraza is approximately $118,000 and Tori would need to take six doses in the first year and four moving forward after that.
Spinraza was approved by Health Canada in 2017 and in October 2018, provinces announced they would cover the cost of the treatment for those with Type 1 SMA.
As for those with Type 2 and 3, it is up to each province to decide if it wants to cover the drug under its own provincial health plan. In December 2018, the Quebec government announced it would cover Spinraza, but as of right now, Ontario does not cover the drug.
WATCH: Miracle drug access denied for SMA patient. Tom Hayes reports.
Spinal muscular atrophy is a devastating degenerative disease that affects one’s nervous system, specifically the part that controls muscle movement.
“We’re actually there and we have a treatment available. To me it makes it that much more frustrating that we can’t access it,” said Tori’s mom, Tracy Lacey.
Ontario Health Minister Christine Elliot told Global News that more proof of effectiveness is needed for the province to make the decision as to cover it or not.
“I know it’s very difficult for many families, I have heard from many people about this,” Elliot said. “This is such a new drug, Spinraza, that we hope that it will be effective for everyone.
“But it’s just part of the process that we need to follow in order to make sure that if the government is going to fund certain drugs for certain exceptional circumstances that it’s going to be effective.”
Elliot said that while she hopes funding for those suffering from SMA Type 2 and 3 will be dealt with soon, Tori and her family should apply to the Trillium program for assistance in the meantime.
But for Tori, who has seen friends from other provinces and countries improve while on Spinraza, she said she wants Elliot to know that her life is “worth it” too.
“It really hurts me to know that there’s a dollar value placed on my life because in the grand scheme of things, that’s what it comes down to, is that it’s expensive.”
As soon as Tori, who relies on a wheelchair and caregiver, was first diagnosed with SMA, her family went to work. By the time she was two years old, Tori said her parents had started a charity that has raised $1.2 million for SMA research.
“It hurts not only me but it hurts to see my family, that they’ve put so much time and energy into helping this drug come into fruition and now I can’t access it,” Tori said.
Contacting the drug company directly
Tracy said that when the drug was approved in 2017, she naively assumed it would be only a matter of weeks until Tori would get access to it. When she realized that wasn’t the case, she said she contacted the manufacturer, Biogen, directly to see about a compassionate program or how to get private funding.
“Because time is just not on our side with our daughter,” she said. “She’s 21 years old and in the last two years alone she’s lost about 20 per cent of her function.”
The family was told that Biogen did have a compassionate care program which they said were told would be a 50 per cent discount off the drug, so the family would need to pay $60 thousand instead. Tracy said they were in contact with the manufacturer on a weekly basis after they were accepted into the program in 2017.
The Lacey’s put a mortgage on their home and sold some investment properties they had.
“We spent the last year and a half getting in a position to make sure that we could not only afford the drug at $60 thousand, that we had a sustainable plan in place to make sure that we could afford it for years to come,” Tracy said.
The first dose was set to take place a few weeks after the end of September 2018 when Tracy said she received an email from Biogen in October 2018 stating they were no longer willing to cover the cost.
“With respect to our previous discussions with you, we wish to highlight to you that an out of pocket option is not a sustainable option for patients to remain on long-term treatment for this debilitating disease,” the email read in part. “As a result SMA 360 is focusing on supporting patients to navigate the public and private reimbursement system in Canada to get sustainable access to nusnersen and cannot be supporting an out of pocket option.”
For the family, Tracy said it was like the rug was pulled out from under them.
“All of our 20 years of efforts actually finally paid off,” she said. “And then to spend the next two years working towards getting the injections for her and being literally two to three weeks away from getting it, and then having them rip it right out from under you is completely unethical.”
When contacted by Global News, a Biogen spokesperson said they “empathize” with families and patients who struggle with access to SMA treatment and that they are working with the provinces across Canada to provide broader access to Spinraza. To date, Biogen said they have reached an agreement to provide access for all Type 1 patients across Canada.
“In the meantime, some families are considering funding access to treatment out of their own pocket. No Canadian citizen should have to pay out of pocket to fund a life-changing treatment for a severe, debilitating disease,” the statement said.
“Biogen cannot support or encourage out of pocket access to Spinraza for individual patients as this is not a sustainable way to fund a lifelong treatment.”
When pressed about why the seemingly sudden change of heart for the Lacey family, the company provided another statement, saying a similar sentiment.
“Biogen empathizes with families and SMA patients that need access to treatment. Since the approval of SPINRAZA in Canada, we have provided programs to help patients access treatment including the largest compassionate use program while Provinces continue to deliberate covering Spinraza. No Canadian citizen should have to pay out of pocket to fund a life-changing treatment for a severe, debilitating disease. Biogen is committed to pursuing access to SPINRAZA for all patients with SMA and continues to work with the provinces to provide all Canadians that need it equal opportunity for treatment.”
The Lacey family said their hope is that Biogen changes its mind and honours what they told the family for the past two and a half years. But until then, the family said they will do what they can to cover the hefty price tag for their daughter who is set to take her first dose Wednesday.
“We’re basically at this point, going to have to take it one dose at a time and see what happens,” Tori said. “Just hope and pray that at some point they come to terms with doing the right thing.”
—With files from Tom Hayes and Felicia Parrillo
© 2019 Global News, a division of Corus Entertainment Inc.