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Jared Wayland uses a lift to ascend to the deck of his St. Catharines, Ont., home to see his wife, Sydney, and 8-month-old son, Llewelyn on Sept. 8. Jared has type 3 spinal muscular atrophy (SMA) and has spent tens of thousands of dollars on an accessible van and accessibility updates to his home as his condition continues to worsen.Aaron Lynett/The Globe and Mail

When Desirée Parisien, a 41-year old mother of one in Saskatoon, started treatment for her type 3 spinal muscular atrophy (SMA) in 2019, it wasn’t just the progression of her neuromuscular disorder that halted. Expenses related to her disability, which had been mounting as her health deteriorated, also stopped growing.

Ms. Parisien credits Spinraza – a gene therapy sold by Biogen whose cost is a prohibitive $708,000 in the first year of treatment and $354,000 for every year thereafter – for retaining and improving her ability to perform daily activities like driving and cooking. She also believes the treatment, which Health Canada approved in 2017, is the reason she hasn’t needed to ramp up the professional, in-home caregiving she relies on, something she estimates saved her family up to $5,000 a month in extra costs.

But Ms. Parisien’s experience receiving quick provincial approval for funding for her treatment isn’t common in many other provinces, some advocates say. And such uneven access to drugs isn’t limited to SMA. Treatment for rare diseases can have serious knock-on repercussions on patients’ personal finances.

In St. Catharines, Ont., Jared Wayland, 34, who also has type 3 SMA and became a father earlier this year, says he has yet to hear back about his 2019 request to receive Spinraza. In the meantime, he has been watching his household expenses soar as his condition, which affects the ability to use voluntary muscles, robbed him of the ability to push himself in a manual wheelchair and sit behind the wheel of his car.

As more and more ultra expensive drugs for rare diseases hit the market, decisions about who should receive public funding for treatments remain inevitably high-stakes and often fraught. Regulators in different provinces at times reach different conclusions based on the same evidence. And patients in provinces with virtually identical eligibility rules often have vastly different experiences with the official review process for their cases, depending on where they live, some advocates and doctors say.

When it comes to SMA, “the clients that we support with system navigation of access to treatments and therapies have not been denied in Nova Scotia or Saskatchewan,” said Stacey Lintern, CEO of Muscular Dystrophy Canada, speaking about SMA patients who are 18 or older and usually require a case-by-case review for treatment eligibility.

But others, in many other provinces, “have expressed their frustration with the denials or no decision to date,” Ms. Lintern added.

And in Canada’s patchy system of financial supports for people with disabilities, some patients with rare and progressive diseases are left to wonder whether their ability to receive treatment will affect not only their health and chance of survival but also their ability to continue to make ends meet.

“It’s pretty damn depressing,” Mr. Wayland said of the amount he and his wife recently spent on accessibility upgrades to their home and a 2014 Dodge Grand Caravan with a ramp and restraints that can accommodate his power wheelchair.

The minivan alone cost around $28,000, he said. “It’s just wild.”

Jared and Sydney play with Llewelyn in their St. Catharines, Ont., home.Aaron Lynett/The Globe and Mail

Mr. Wayland, who is a graphic designer, said he’s also increasingly struggling to perform parts of his job. Anything that requires a very steady hand, he said, has become hard to do.

“There are times where you have to outsource work because it’s just physically beyond my ability,” he said.

And with his health needs growing, Mr. Wayland also worries about whether his wife, who has until now been his primary caregiver, will be able to return to work full-time after her parental leave.

Households with disabilities typically face higher living costs, with routine expenses for everything from prescription drugs, health care services and assistive devices, said Michael Prince, a professor of Social Policy at the University of Victoria.

Some expenses can be prohibitive for families with modest incomes. A power wheelchair, for example, can cost more than $10,000, and requires regular servicing and tire changes. A personal support worker costs between $28 and $35 per hour in Ontario, according to Closing the Gap Healthcare, which provides home care and clinic-based health care services in Ontario and Nova Scotia.

Provincial welfare programs cover some of those needs but have extremely low income thresholds for eligibility. Other sources of provincial funding are typically available through a system that’s patchy, limited and often hard to navigate, Mr. Prince said.

Tax breaks like the medical expense tax credit can help defray some of those costs, but many Canadians with disabilities and their families are still left with significant out-of-pocket expenses, he added.

It doesn’t help that, of the people with disabilities who are able to work, many are employed in small and medium-sized companies, which often don’t offer group insurance benefits, he added.

In a recent survey of Canadians with neuromuscular disorders, including SMA, found that families were spending $10,000 a year out-of-pocket on assistive devices and $22,000 a year on paid caregivers on average, according to Ms. Lintern.

The estimated cost in terms of loss of productivity for an adult with the disorder was $36,000 a year on average, while for family caregivers it amounted to $50,000 a year, according to the survey. And costs often increase as neuromuscular disorders progress. (The study received some financial support from Biogen Canada and Hoffman La-Roche, among other pharmaceutical companies.)

It’s why Mr. Wayland is asking himself what access to treatment could mean, not only for his health but for his family’s ability to cope financially.

The trouble with SMA treatments is that, while there’s solid evidence of their benefits in babies and children, there are no large, randomized clinical trials on adults. The Canadian Agency for Drugs and Technologies in Health (CADTH), which advises all provinces except Quebec on drug funding, recently recommended that Spinraza costs not be reimbursed for SMA patients over the age of 18.

Currently all provinces except Quebec have strict age caps limiting access to treatments for SMA, though Canadians who don’t meet the eligibility requirements can often apply to be considered on a case-by-case basis.

For another and more recently introduced SMA treatment, known as Evrysdi, from Roche, which was approved by Health Canada last year, major clinical trials only involved patients up to the age of 25. The treatment is expected to cost between $335,000 and $354,000 a year in patients older than age 2, according to CADTH.

While some SMA specialists are highly skeptical of treating adults, others point to a growing number of smaller trials that have shown meaningful benefits even for older patients.

“There is mounting evidence that adult patients with SMA can have improvement with nusinersen,” said Jodi Warman Chardon, director of the Neuromuscular Centre at the Ottawa Hospital, using the generic name for Spinraza.

Ms. Parisien, who received approval for treatment with Spinraza within months, describes it as life-changing.

“I wasn’t able to lay down and even raise my head up so that’s how weak I was,” she said, recalling that she had already filed paperwork for provincial funding to pay for four to six hours of at-home caregiving, up from the current half hour of service she receives to help her get ready in the morning.

With treatment, Ms. Parisien said, “I went from that position to then being able to roll over independently to then be able to get onto my elbows and be able to lift my head up fully.” She is now considering purchasing a standing frame that will allow her to stand upright for periods of time.

Mr. Wayland, on the other hand, said he has yet to hear back about his 2019 application to receive Spinraza. He has also applied for Evrysdi.

To date, he said, “I have not heard a peep.”