Like the last CORD conference I attended the days were jam packed with important information. Let’s see what sense I can make of my notes. (The slides are also online here.)
Let’s start with the bad news: Canadians with rare diseases are being forced to contend with the MAD (maximum administrative delay) principle, a situation in which a massive bureaucracy has to be conquered before they can get access to treatment. When access is finally granted, it can take up to three years for this approval to cover all provinces and territories.
Even more depressingly the long-standing mindset of our country and bureaucrats has been that cost trumps everything and it’s cheaper to just let people with rare diseases die. This is shocking and depressing for many reasons and is something that desperately needs fixing.
But (and I made note of this quote because it was fantastic) "We may not have best healthcare system but there is one place in which we have no equal: writing reports on how we can be better."
Yes, the system knows there is a problem and is looking at what can be done. Now we just have to make it follow through when it sets out to fix things.
Enter R2D2. (No, not the robot.) This is the new version of regulation the government is working on to improve drug and treatment access for all—including rare—Canadians. With any luck this new change will reduce (red tape and) waiting time for drug approval.
The other factor that may help finally bring us the solution: Pharmacare.
Yes, this goes against what someone explained at the last conference, but that’s because there are caveats to this. In the system currently under discussion there would be two Pharmacares: one for the general population and one for us genetic weirdos. Talks are undergoing regarding the creation of the Expensive Drugs for Rare Diseases (EDRD) plan. (In fact these talks were going on in another room in the Sheraton while the CORD conference was running.) Let’s hope the whole thing doesn’t get lost or scuttled amid government infighting.
The other thing to really pay attention to is the Special Access Program (SAP)/Managed Access Program (MAP). This is likely the route we’ll have to take to access drugs/treatment for FOP when they become available. When drugs/treatment are available EVERYWHERE ELSE and we’re still waiting (ugh, Canada, you make me twitch and I bet Tommy Douglas is rolling in his grave) your doctor can apply on your behalf to try to get you access. Just make sure you throw a rock at anyone who says we don’t need better access to rare disease drugs because we have this system (as apparently this is a common argument).
What did I bring away with me after two very long days? The fight is so far from over. But (to be fair) I already knew this.
So I guess my advice is to gear up for battle. We’re closer to the point where there will be a drug/treatment for FOP and we will (potentially) be able to access the drug after it becomes available…we just have to make it through a booby-trapped obstacle course first.