Spotlight on Rare Diseases: How Well Do You Really Know Your Health Care System? A Canadian Perspective
Read the introduction to this series for context.
When you get the news that your son has a rare and incurable neurodegenerative illness, the first thing you do, of course, is the one thing the doctor warned you not to. You google it. You see children attending primary school in power wheelchairs with assistive breathing devices. You see memorial posts from parents grieving the loss of their teenagers. You see story after story of families fighting for access to life-changing medication that is always just out of reach.
You sink into despair, but you only let yourself stay there for so long. Treatment innovation is moving fast, and many of these stories are from years ago. What’s more, you live in Canada, with access to a modern universal health care system. There is still reason to hope, but there is a lot that needs to be done, and quickly.
As you dig into the details and try to put together a plan of action, the complexity of your situation becomes clearer and clearer. There is a single effective treatment for this condition, and it has been approved by Health Canada. But, you learn, there’s a big difference between “approved” and “available.”[1]
You, like most Canadians, have a private drug plan through your employer[2]. This specialized and expensive drug is not listed on your private plan, so the first thing you do is do is submit a special authorization request to your insurer.
The Patented Medicine Prices Review Board (PMPRB) has already set a limit on the price of the drug, and the manufacturer has thus listed it for sale in Canada at $200k CAD (a roughly 50% discount on the U.S. price). The pan-Canadian Pharmaceutical Alliance (pCPA) has negotiated a further discount for the provinces, but that’s not available to insurers or to individuals paying out of pocket.
In short order, you get what should be good news. The insurer has approved your request. But, even on your high-tier plan—which covers a larger percentage of a wider range of drugs than most—the rare disease drug program only covers a maximum of $100k per year. It’s almost worse than a no. Bridging that extra $100k every year is just as impossible as paying the whole $200k would have been. You appeal for an exception to the coverage cap, but you are advised not to get your hopes up.
So you turn to the public plan. It slowly dawns on you that where specifically you live in Canada is going to have a big impact on what your story looks like. Each province has its own healthcare system and its own public drug plan, and the disparities are huge[3].
You live in Prince Edward Island (PEI), a small province with a less-developed healthcare system that tends to lag behind other provinces in covering high-cost drugs. Neighbouring provinces do cover the drug, but only for pre-symptomatic children under two. Your son, at 28 months old, wouldn’t qualify, even if you were willing to uproot your whole family[4].
Every day, your son’s symptoms are growing more noticeable. His legs are tremoring when he walks and he’s missing speech milestones. The progressive nature of the disease is relentless and only access to treatment can slow it. You know a wheelchair will soon be a necessity. You start doing every single thing you can think of.
You start a crowdfunding campaign, leaning on every family member, every friend, every distant acquaintance in the hopes that you can raise enough money to fund even a single year of treatment. There is no specific charity that focuses on this disease. It’s just too rare. Nonetheless, you reach out to every charitable organization you can think of. You meet other families fighting similar battles, including a once well-to-do family who bankrupted themselves seeking treatment in the U.S. before the drug received Health Canada approval.
You write letters to your provincial and federal Members of Parliament. You contact the local news. You write to the manufacturer of the drug itself. You submit a request to the province for exceptional access, hoping that the National Strategy for Rare Diseases initiative might help, but the process is murky and without clear timelines.
It is so much work. Your spouse has to quit his part-time job and abandon his small business in order to make time, further straining your finances. You take a short leave from work, but you can’t really consider quitting, not least because the private drug plan remains your only lifeline[5].
You organize a fundraising run in your town, raising a few thousand dollars and getting sympathetic local news coverage. Your crowdfunding campaign hits $20k and then begins to plateau. You receive some one-time grants from national charities totalling another $25k. Putting it all together, you’ve raised nearly half of the $100k you need to bridge the difference with your private insurer for one year of treatment. Without a second thought, you and your spouse decide to drain every penny from your retirement savings, empty your chequing account, and request a referral for treatment.
There is no medical facility in PEI that can administer the treatment, so you’re referred to a hospital in Toronto. The province will eventually reimburse your airfare and a portion of your hotel, but the realities of flying to and from Ontario every month is going to put further strain on your already depleted finances, not to mention your family life. Your spouse is not going to be returning to work anytime soon, and you can’t remember the last time you talked about anything other than healthcare. You’ve had to pull your older daughter from her more expensive extracurriculars, and you’re relying on the charity of the community in both time and money to keep her in hockey, her last remaining outlet.
But your son is finally receiving treatment. For a year, at least. You think, if you remortgage or sell the house, you might be able to stretch it to two.
Your son responds well to the treatment and begins to stabilize. The tremor in his legs never goes away, but it’s no longer getting worse. A few months in, your MPP invites you to speak to the Standing Committee on Health and Social Development, having previously raised your family’s plight during Question Period at the PEI Legislative Assembly. This results in another round of press coverage, and you try to steer the tone away from the immediately heartwarming and towards the looming cloud of uncertainty about your son’s ability to stay on treatment.
Nine months into the treatment process, when you are just beginning to speak to your mortgage broker, you receive a call from a representative of the drug manufacturer. They want to offer your son compassionate access to the drug. This changes everything. For the first time in a year, you can breathe.
You’ve learned in talking to other families that this is a surprisingly common outcome for Canadians living in the limbo of rare disease and limited coverage. The drug manufacturers don’t want to see children dying of diseases their products could treat, but you know your visibility in the media has likely played a role in the offer.
Your incredible sense of relief is darkened by a feeling of guilt that other quieter families have been left behind. You vow to continue fighting for these other families, no matter what happens next. And you will still be fighting for your son as well. Because even though your son now has access to treatment, you have no way of knowing how long that will last. Until the day when this treatment is covered by public plans in all provinces, the reprieve your family has won will always rest on unsteady ground.
[1] After Health Canada approval, there is still a lengthy regulatory and bureaucratic process before drugs become readily available. First, the Patented Medicine Prices Review Board (PMPRB) reviews the cost of the drug and sets a maximum allowable price, which can sometimes scare manufacturers away from offering their products in Canada at all. Next, the Canadian Agency for Drugs and Technologies in Health (CADTH) reviews the drug’s clinical value and cost effectiveness before making a non-binding coverage recommendation to provinces. Then, the pan-Canadian Pharmaceutical Alliance (pCPA) negotiates with the manufacturer on behalf of the provinces to obtain a discount and determine the price the provinces will actually pay. Finally, each provincial Ministry of Health (or territorial health authority) in each individual jurisdiction will decide, based on their own budgetary and political considerations, whether or not to actually cover the drug under the public plan. The whole process can take years.
[2] Although Canada has universal hospital and physician coverage under the Canada Health Act, there is no guaranteed pharmacare.
[3] And in Quebec, the whole process is different, as Quebec does not participate in the CADTH or the pCPA. Quebec is the exception to every rule in Canada.
[4] Moving provinces to access a different public provincial plan is technically an option in Canada, but the costs and disruption can be too steep for many Canadians. And, even after moving, there is usually a waiting period before you can enrol in your new province’s plan.
[5] While you’re trying to get your son access to the drug, your insurance is paying for physiotherapy, and they’ll pay for a wheelchair when it becomes necessary.