'A slow death': Ontario woman with rare disease begs province to cover life-saving drug costing thousands
Nouma Hammash can be seen above holding her newborn niece. (Handout provided by Hammash)
Published Saturday, May 14, 2022 3:21PM EDT
An Ontario woman is urging the government to reconsider insurance eligibility for a drug that could save her life but costs hundreds of thousands of dollars a year.
Toronto resident Nouma Hammash, 39, grew up in the United Arab Emirates. Diagnosed with spinal muscular atrophy (SMA) at a young age, she has lived most of her life losing the ability to utilize her muscles.
“It’s a slow death,” Hammash told CTV News Toronto during an interview over the phone.
SMA is a rare genetic disease affecting the body’s motor neurons — nerve cells that control voluntary muscle movement. Over time, the muscles weaken and shrink.
“SMA causes progressive weakness in the arms and legs, progressive weakness of breathing muscles, known as respiratory failure, and, in many patients, it also causes problems with swallowing,” Dr. Aaron Izenberg, a neurologist and SMA specialist at Sunnybrook Hospital in Toronto, told CTV News Toronto.
In 2016, Hammash decided to move to Canada – a country she said she felt had more opportunities for disabled people.
“In Canada, there is more respect, more opportunities, more equality to people with disabilities,” she said.
However, since Hammash’s arrival in Canada, her condition has worsened and she has yet to find a successful treatment plan.
“At one point, I was able to [get] in and out of bed but now I can’t,” she explained. “I was able to turn over in bed, now I can never do that.”
Hammah says the physical weakness isn’t the only loss she’s mourning.
“There's so many ways in which I’m continuing to lose my power and with that, I lose my independence,” she said.
“We’re not only speaking about the physical death – when the spirit leaves the body – but about the death of [an] independent life.”
In June 2017, Hammash felt she nearly had the solution when Health Canada approved Spinraza for Canadian SMA patients. Spinraza, a brand name of the medication nusinersen, is used to treat spinal muscular atrophy and slows the progression of the disease. It’s currently the only approved medication of its kind in Canada.
However, her dreams were soon dashed when she found out she wouldn’t be able to access coverage for the costly medication. Without insurance, the first year’s worth of Spinraza can cost nearly a million dollars.
“I’ve never got a single dose,” Hammash said. When asked why, she explained, “because I’m an adult.”
As of now, Ontario only offers Spinraza coverage to patients under the age of 18, due to a lack of controlled trials and data in older age groups. That eligibility expanded slightly in 2019 to include some adults on a case-by-case basis, but Hammash still does not meet the criteria.
“Basically, for nusinersen, there’s only randomized controlled trial data up until the age of 12,” Izenberg said. “Beyond that, there's still data, but it just hasn't been studied in…placebo-controlled randomized control trials.”
While there’s nothing stopping those trials from taking place, Izenberge said they are “logistically challenging and expensive.” Because Health Canada looks for evidence-based research, when a certain drug is only required by a small population, it can complicate and extend the approval process
“You need huge numbers of patients over a long period of time and I don’t know if that’s ever going to happen,” he said. “That's really the issue – and to pay out of pocket for these medications is just not feasible.”
In a recent interview with CTV News Toronto, Beth Vanstone, an Ontario mother whose child suffers from cystic fibrosis (CF), says conditions like SMA and CF are the reason Canada needs a national framework for rare diseases and new medications as they come out.
“[Cystic fibrosis] is not the only disease that has those challenges – all rare diseases have a lot of challenges – particularly rare diseases that only have a couple of patients in the country or the world.”
She says Canadian health-care and insurance policies aren’t keeping up with the science.
“Science has marched forward,” she said. “Miraculous things are being done – you can take a couple of pills a day and have your genetic defect corrected."
“Our system, sadly, just hasn’t kept up.”
Meanwhile, Izenberg said he hopes research into the drug will continue.
“Ultimately, what would be amazing is if we get super strong data that will demonstrate an unequivocal benefit,” he said, although admitting he has no concept of if or when this will take place.
However, for SMA patients, time is of the essence and many have already been waiting for year.
“What's going to happen in five years if I'm not able to work and if I'm not able to touch my nose and drink my water – how am I going to do it?” Hammash asked.
“I think I might be dead already.”