This is incredibly disturbing when you consider how many people in Canada have a rare disease. Approximately 1 in 12 people have a rare disorder, which means around 3 million Canadians are being told to grin and bear it…while our tax dollars are being sent to a system that fully supports the treatment of other diseases. (Yes, I know there’s sometimes a waitlist, but in comparison that is nothing to complain about.)
However (and luckily), we have the Canadian Organization of Rare Disorders (CORD). Thanks to this organization, people with rare diseases can mobilize together in the quest for more equal treatment.
On November 19 and 20, 2015, CORD hosted the Expert Patient Advocates & 21st Century Therapies Forum, a conference designed to teach members of the Canadian rare disease community about the healthcare system and their role in ensuring said system doesn’t continue to overlook rare diseases. Over the two-day forum, attendees were given a crash course on drug development, regulatory guidelines, treatment access and patient submissions. (Given the amount and the subject of the content, I’m pretty sure they’d have made a killing if they’d had a cash bar open at the end of the day.)
As there was a lot of important information and it would take me a really long time to craft the perfect transitioning sentences, we’re just going to lay out the data in bullets.
• Canada needs (and is working on) a rare disease registry, which would list rare diseases found in Canada, their symptoms and etc. This registry would be a huge asset for doctors and researchers when they are looking for patients and evidence, which is important as Canada is pretty far behind in terms of drug development (excluding the lovely Clementia, of course) and needs to bring the research and researchers here.
• Health Technology Assessment (HTA): This is the system that looks at the benefits and risks of new drugs and treatments in order to determine whether it is worth offering them to Canadians. One of the major problems facing rare diseases in Canada is the fact that there is one standard assessment set in place for ALL diseases, even though other diseases have treatments that are meant to attack the symptoms while rare diseases need gene therapy. Also, because of the high cost of the rare drugs, the HTA is less likely to take a chance on (or recommend) them.
• Then, once the drug or treatment has been approved by the HTA, it is up to each province/drug plan to decide whether or not they are going to pay for the drug, under advisement from the Common Drug Review (CDR) run by the Canadian Agency for Drugs and Technologies in Health (CADTH). The CDR looks at things like how does the new drug compare with alternative (i.e. pre-existing) treatments? Note that CDR says the drugs/treatment have to cost less than treatment in a hospital would, and it can take two to three years to actually get reimbursed for the drug fees. Now, if you’re thinking, “That’s okay, I have company health insurance, and that will pay for it,” a lot of employers are removing biologic drugs from the list of things they’ll cover in their drug plans. If you find yourself in this situation, try to stress the importance of the drug to your quality of life—fewer absences, able to work longer, etc.—in order to convince them that the benefits outweigh the costs and they should agree to pay.
• To be slightly less depressing, I suppose a small glimmer of hope is the fact that Patient Evidence Submissions are (becoming) a very important part of the CDR, which is important considering most rare diseases can’t get the large amount of data major diseases can to include in their proposals. (Compare the number of patients in the Palovarotene trial to any AIDS drug trial.) However, these Patient Submissions need to be done carefully. Any quality of life findings have to match the results of the drug or the Canadian Expert Drug Advisory Committee (CDEC) will reject it. These submissions are made by the patient group (which in our case is CFOPN) and should start being collected around six months before the proposal to CDR is made. If you are wondering what these submissions are, you can find them online here.
• I had a lovely lady explain the dangers of National Pharmacare to me (well, me and a few other people). Because it’s a system designed for “everyone”, it will (once again) blatantly ignore the rare. After all, if you only have a certain amount of money to spread around an entire populace, why would you agree to pay an obscene amount for a handful of people? Even now, despite the fact that 1 in 12 Canadians have a rare disease, rare disease funding is at around 1% of overall drug costs/coverage, so you can imagine how much access we’d get in a system that’s designed to meet “everyone’s” needs.
• One of the most important things you will learn today is the existence of the Special (Early) Access Program. This is a system set up so people can (try to) get access to drugs that haven’t made it through the long regulation process in Canada. So once/if Palovarotene is approved everywhere but Canada (which I’m pretty much expecting is what will happen), your doctor can put in a request to get you access to the drug. Obviously it’s not a guarantee, but it is a chance to get the drug when you need it, as opposed to when the government graciously allows you to have access to it…50 years from now.
Does your head hurt yet? This is actually a very condensed version of my notes. But it is the important stuff. Here’s the most important point:
• One of the major themes running through the event was how important advocacy was and is. The fact of the matter is that over 3 million Canadians have a rare disease. Add in their friends and family and, well, that’s a lot of noise we can make if we band together. On their website CORD has a template letter to send to your MP, strongly suggesting that the government set up a federal orphan drug regulatory framework. Get everyone you know to send it to their MP. (Note: If you copy and paste, just make sure you correct the misspelling of Canadians in the last paragraph.)
Okay. You've made it to the end. Now get ready, because the fight is far from over. In fact, it’s barely started. Let’s make noise and ensure that when we get the FOP treatment we so desperately need, it’s not kept out of our grasp by bureaucracy.