‘A slow death’: Ontario woman with rare disease pleads with province for life-saving drugs costing thousands

An Ontario woman urges the government to reconsider insurance for a drug that could save her life but cost hundreds of thousands of dollars a year.

Toronto resident Nouma Hammash, 39, grew up in the United Arab Emirates. She was diagnosed with spinal muscular atrophy (SMA) at a young age and has lived most of her life losing the ability to use her muscles.

“It’s a slow death,” Hammash told CTV News Toronto during a phone interview.

SMA is a rare genetic disease that affects the body’s motor neurons — nerve cells that control voluntary muscle movements. Over time, the muscles weaken and shrink.

“SMA causes progressive weakness in the arms and legs, progressive weakness of the respiratory muscles known as respiratory failure, and in many patients it also causes difficulty 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 believed had more opportunities for the disabled.

“In Canada there is more respect, more opportunity, more equality for 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 some point I could [get] in and out of bed, but now I can’t,” she explained. “I could turn over in bed, now I can never do that again.”

Hammah says that the physical weakness is not the only loss she mourns.

“There are so many ways I keep losing my power and in doing so I lose my independence,” she said.

“We are not just talking about physical death – when the spirit leaves the body – but about the death of… [an] independent living.”

In June 2017, Hammash felt like she was close to having the solution when Health Canada approved Spinraza for Canadian SMA patients. Spinraza, a brand name of the drug nusinersen, is used to treat spinal muscular atrophy and slow the progression of the disease. It is currently the only approved drug of its kind in Canada.

However, her dreams were soon dashed when she learned that she would not be able to access coverage for the expensive medication. Without insurance, Spinraza can cost nearly a million dollars the first year.

“I’ve never gotten 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 studies and data in older age groups. That eligibility has expanded somewhat in 2019 to include a few adults per case, but Hammash still doesn’t meet the criteria.

“Basically, for nusinersen, there are only randomized controlled trial data up to age 12,” Izenberg said. “In addition, there is still data, but it just hasn’t been studied in … placebo-controlled randomized control trials.”

While there’s nothing stopping those trials, Izenberge said they’re “logistically challenging and expensive.” Because Health Canada is looking for evidence-based research, it can complicate and lengthen the approval process when a particular drug is needed by only a small population.

“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 problem — and paying out of pocket for these drugs just isn’t 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 why Canada needs a national framework for rare diseases and new drugs like they come true.

†[Cystic fibrosis] is not the only disease that has these challenges – all rare diseases have many challenges – especially rare diseases that only have a few patients in the country or the world.”

She says Canada’s health and insurance policies aren’t keeping up with science.

“Science has progressed,” she said. “Wonderful things are being done — you can take a few pills a day and have your genetic defect corrected.”

“Unfortunately, our system hasn’t kept up with it.”

Meanwhile, Izenberg said he hopes research on the drug will continue.

“What would be great in the end is if we get super strong data that will show an unequivocal advantage,” he said, although he admitted he has no idea if and when this will happen.

However, for SMA patients, time is of the essence and many have been waiting for years.

“What will happen in five years if I am unable to work and if I cannot touch my nose and drink my water – how am I going to do that?” Hammas asked.

“I think I may already be dead.”

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