‘A slow death’: Ontario woman with rare disease begs province to cover thousands of lifesaving drugs

An Ontario woman is urging the government to reconsider her insurance eligibility for a drug that could save her life but costs hundreds of thousands of dollars a year.

Nouma Hammash, 39, a resident of Toronto, 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 use her muscles.

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

SMA is a rare genetic disease affecting the motor neurons of the body, that is, the 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 in the respiratory muscles, known as respiratory failure, and in many patients it also causes swallowing problems,” said 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 which she felt offered more opportunities for people with disabilities.

“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 an effective treatment plan.

“At one point, I was able [get] in and out of bed, but now I can’t,” she explained. “I was able to roll over in my bed, now I can never do that again.”

Hammah says physical weakness isn’t the only loss she mourns.

“There are so many ways that I keep losing my power and with that 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 life. »

In June 2017, Hammash felt she almost had the answer when Health Canada approved Spinraza for Canadian patients with SMA. Spinraza, a brand name of the drug nusinersen, is used to treat spinal muscular atrophy and slows the progression of the disease. It is currently the only drug of its type approved in Canada.

However, her dreams were quickly dashed when she found out she would not be able to access coverage for expensive drugs. Without insurance, the first year’s worth of Spinraza can cost close to a million dollars.

“I never got a single dose,” Hammash said. When asked why, she explained, “because I’m an adult.”

Currently, 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 doesn’t meet the criteria.

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

While there’s nothing stopping those trials from taking place, Izenberge said they were “logistically difficult and expensive.” Since Health Canada seeks evidence-based research, when a certain drug is only needed by a small population, it can complicate and prolong the approval process.

“You need a lot of patients over a long period of time and I don’t know if that will ever 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 has cystic fibrosis (CF), said conditions like SMA and CF are why Canada needs a national framework for rare diseases and new drugs as they come out.

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

She says Canadian health care and insurance policies are not following the science.

“Science has advanced,” she said. “Miraculous things are happening – you can take a few pills a day and have your genetic defect corrected.”

“Our system, unfortunately, just didn’t keep up.”

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

“At the end of the day, what would be amazing is if we got extremely strong data that will demonstrate an unequivocal advantage,” he said, while admitting he has no idea if or when that will happen.

However, for SMA patients, time is running out and many have already been waiting for a year.

“What will happen in five years if I can’t work and if I can’t touch my nose and drink my water – what am I going to do? Hammash asked.

“I think maybe I’m already dead.”

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