An Ontario woman is urging the government to reconsider the terms of insurance for a drug that could save her life but cost hundreds of thousands of dollars.
Numa Hamash, 39, a Toronto resident, 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.
“This is a slow death,” Hamas told CTV News Toronto during a telephone interview.
SMA is a rare genetic disease that affects the body’s motor neurons – nerve cells that control the voluntary movement of muscles. Over time, muscles weaken and contract.
“SMA causes progressive weakness in the arms and legs, progressive respiratory muscle weakness known as respiratory failure, and also causes swallowing problems in many patients,” said Dr. Aaron Isenberg, a neurologist and SMA specialist at Sunnybrook Hospital in Toronto. in front of CTV News Toronto.
In 2016, Hamash moved to Canada. She now works at Spinal Cord Injury Ontario as a mediator and advocates for other patients facing similar situations.
However, after Hamash’s arrival in Canada, her condition worsened and she has not yet found a successful treatment plan.
“It simply came to our notice then [get] in and out of bed, but now I can’t, ”she explained. “I managed to turn in bed, now I can never do that.”
Hamma says physical weakness is not the only loss she mourns.
“There are so many ways in which I continue to lose my strength and thus lose my independence,” she said.
“We are not just talking about physical death – when the spirit leaves the body – but also about the death of [an] independent living. ”
In June 2017, Hamash felt she had almost found a solution when Health Canada approved Spinraza for Canadian patients with SMA. Spinraza, a trademark of the drug nusinersen, is used to treat spinal muscular atrophy and slows the progression of the disease.
However, her dreams soon shattered when she realized she would not have access to coverage for the expensive drug. Without insurance, reimbursement or government coverage, the value of Spinraza for the first year is about $ 700,000.
“I have never received a single dose,” Hamash said. When asked why, she explained, “because I’m an adult.”
Ontario currently offers Spinraza coverage only to patients under the age of 18 due to a lack of controlled studies and data in older groups. This eligibility was slightly expanded in 2019 to include some adults on a case-by-case basis, but Hammash still does not meet the criteria.
“In general, for nusinersen, there are only data from randomized controlled trials up to 12 years of age,” said Isenberg. “Furthermore, there is still data, but it has simply not been studied in … placebo-controlled randomized controlled trials.
Although there is nothing to stop these attempts, Isenberge said they were “logistically challenging and expensive”. Because Health Canada seeks evidence-based research when a drug is only required by a small population, this can complicate and extend the approval process.
“You need a huge number of patients for a long 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 is simply impossible.”
In a recent interview with CTV News Toronto, Beth Vanstone, a mother from Ontario whose child suffers from cystic fibrosis (CF), says conditions such as SMA and CF are the reason Canada needs a national framework for rare diseases and new drugs when come out.
“[Cystic fibrosis] it is not the only disease that has these challenges – all rare diseases have many challenges – especially the rare diseases that only a few patients in the country or in the world have. ”
She says Canadian health and insurance policies are out of step with science.
“Science has moved forward,” she said. “Unfortunately, our system just doesn’t last.”
Isenberg, meanwhile, said he hoped research into the drug would continue.
“Ultimately, what would be amazing is if we get super strong data that will show unequivocal benefits,” he said, although he acknowledged that he had no idea if or when it would happen.
However, for patients with SMA, time is of the essence and many of them have been waiting for years.
“What will happen in five years if I can’t work and if I can’t touch my nose and drink my water – how will I do it?” Hamash asked.
Hamash says if she has to wait years for clinical data to assess her need for Spinraza, it may be too late.
“I think I may already be dead.”
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