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Woman living with rare disease fighting for access to miracle drug

Last Updated Feb 11, 2019 at 4:49 pm EST

It is called a miracle drug that can halt, even reverse, a rare disease that slowly destroys all the muscles in the body — but it is also one of the most expensive medications in the world.

Now, people with Spinal Muscular Atrophy (SMA) fear a pending decision in Canada means they won’t be able to afford access to the life-changing drug.

Victoria Lacey was diagnosed with SMA on her first birthday.

Victoria Lacey was diagnosed with SMA when she was a one year old.

“My parents were told by Sick Kids that there was no hope, no treatment, no cure.”

Victoria is now 21 and has been studying at Ryerson University for the last four years.

“The biggest challenge with SMA is knowing each day you’re going to be weaker than the last and not knowing what the future holds for you.”

But for the first time in her life, Victoria, who relies on a wheelchair and a round-the-clock caregiver, was given hope after hearing about Spinraza, a drug that has shown astonishing success in treating SMA.

Victoria Lacey, now 21, speaks to Cynthia Mulligan in her apartment about access to the life-saving drug, Spinraza. CITYNEWS

The drug, which is also the only treatment available for SMA, was approved by Health Canada almost two years ago.

Sofia Gall, who was also diagnosed with SMA just after she turned one, received Sprinaza when she was three years old. Just a week after her first shot, she was able to ride a bike for the first time

Despite doctors saying she would be in a wheelchair by the time she was four, her parents now say, at five years old, her decline has stopped and she is growing stronger every day.

Sofia is one of the lucky ones. The cost of Spinraza is crushing — US$750,000 for the first year and US$375,000 every year after that for life. Thankfully, her father’s insurance covers it.

But, Victoria is still waiting for her miracle and her hope is diminishing.

Despite Health Canada’s approval, the Canadian Agency for Drugs and Technologies in Health (CADTH), an independent body that determines if provinces should cover the costs of new drugs, has recommended only patients with the most severe form of SMA, type 1, should be able to have it covered, citing cost and the need for more evidence.

Victoria has type 2. She said the fact that there is a drug that can help her but is out-of-reach is devastating for her.

“Before Spinraza was FDA approved and Health Canada approved, I had almost come to terms that this was the life I was faced with because there was no hope there was nothing that could help me,” Victoria said. “But now that there is and the fact that I can’t access it is way more emotionally draining even before there was a treatment because now I know there is something out there that can help me and I just can’t access that”

More than 40 countries have approved coverage for Spinraza for all types of SMA and all ages.

Quebec recently made its own decision to approve Spinraza for unrestricted coverage for all patients as well.

Biogen, the manufacturer of the drug, said through negotiations the cost for the drug has come down significantly and it has appealed the CADTH recommendation.

A final decision is expected March 1.

But time is crucial for Victoria. “My progression has been quite rapid,” she notes. “In my first year of university, I lost the ability to type on my laptop.”

“With Spinraza, what I have now will never get worse,” Victoria said. “It’s scary not knowing what your future holds without treatment.”

Biogen estimates there are approximately 500 people in Canada with SMA.

There are at least two other SMA treatments that are in the testing stages. However, it could be years before they are approved and available.

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An NDP government will provide free prescription drugs for all….

February 11, 2019 at 6:46 pm

Lucky for some that insurance covers the drug…. for many others, I’m quite sure the insurance companies / employers would balk at such a cost.

I understand developing new drugs involves significant cost and risk, however this seems a little over the top.

Sadly, there will be a lot of dashed hopes…

February 11, 2019 at 7:18 pm