So, another line that the Ontario Ministry of Health is disseminating in the press is that special access programs like the Case by Case Review program that I applied to are actually a “back door” that pharmaceutical companies use to get their drugs into Canada without having to enter the regulatory and price negotiation process. It’s meant to paint the drug manufacturers as slimy, deceptive operators that are trying to get out of fair dealing with Canada. It’s certainly possible that drug manufacturers are slimy, deceptive operators, but these special access programs are not an example of it. Still, the “back door” metaphor has captured people’s attention and has helped the Ministry control the narrative about these programs – i.e. first they argue that these programs are stupendously expensive, when they aren’t, by implying that the costs of the program add up to $5.2 billion, which they don’t; then they suggest that the pharmaceutical companies are exploiting these programs as a way to sell their drugs in Canada without negotiating fair and reasonable prices. It’s a seductive line of thinking, no?
Here, for example, is part of an email I received from a knowledgable oncologist: “I think we also need to pressure pharmaceutical companies to do the right thing and put in an application to CDA (Canadian Drug Agency). Maybe if they know we are going to support them through the process then they might be more likely to engage. I think we should discuss how to do that. They are taking advantage of the system by using the back door and it is not fair for all Canadians. I wonder what your thoughts are on that?”
So, here is my reply. I didn’t want to get into an argument about how the “back door” language is seductive and deceptive, so I tried to just stick to the facts as I understand them.
“Thank you for your kind and supportive email. And thank you for encouraging my advocacy. I think it is thanks to the zenocutuzumab that I feel well enough to do it!
I’ve been mulling over your thoughts about encouraging drug manufacturers to apply for approval in Canada. In general, I agree with you wholeheartedly. And yet, the more I’ve learned about Canada’s drug approval process and the complexities of the pharma sector, the more I think this is not at all an easy path. I am hardly an expert in this, and can really only speak to my own case, but I do think my own case is a telling one about 3 substantial obstacles.
(1) Precision oncology, and the ever-increasing number of targeted therapies, is amazing. BUT, for some of these targeted therapies, the mutations they target are so rare that the number of patients who can benefit is really small, and this problem is exacerbated in countries with small populations. So, in the case of NRG1 fusion cholangiocarcinoma, there are probably only a handful of patients across Canada that could benefit from zenocutuzumab. And this would depend on them having access to whole genome sequencing which, as I’m sure you know, is not readily available or covered in much of Canada. In other words, that handful of patients may never know they might benefit from zenocutuzumab because they will never get the testing of their biopsy material that would reveal it. I think it is not surprising that a drug manufacturer will look at that situation and decide it is not worthwhile to apply for approval in Canada. Why would they if only 1 or 2 patients per year can benefit from the drug?
(2) The drug approval process is really, really slow in Canada. The “time to patient” (that is, the time that elapses between when a drug manufacturer submits an application to Health Canada and the time when an oncologist can actually get the drug into the body of a patient) is egregious in my opinion. And I’ve attended two conferences now (the CORD conference in Toronto and the CCF conference in Salt Lake City) where reps from the drug manufacturing sector have said that this is often more of a deterrent to applying in Canada than the small patient population. As an example, ivosidenib was approved by the FDA in approximately 6 months, and by the time it is approved and adopted by provincial formularies in Canada, it will probably have been about 4 years. So again, why would a drug manufacturer bother to apply if it is going to take 4 or more years, and their targeted therapy might be irrelevant by the time it is approved because something better has been invented?
(3) Not all drug manufacturers are the same. I’ve learned there is a big difference between “big pharma” and what I have come to think of as “tiny pharma.” For example, Partner Therapeutics manufactures and sells only 2 drugs, zenocutuzumab and a drug that treats radiation sickness. They keep themselves afloat through US government contracts for the radiation sickness drug. And they didn’t actually invent zenocutuzumab; they bid on it and acquired the rights to it from Merus, the inventing company in the Netherlands. I have no idea how much they paid to acquire it, but I imagine they have set the price for zenocutuzumab so high in order to recoup what they spent. I’m really not sure it’s possible to convince a tiny pharma company to apply for approval in Canada when they stand to make no profit doing so. Big pharma might be convinced because they can withstand the loss of profits on one drug through the large profits of another, and maybe they wouldn’t want to risk reputational damage by failing to bring a drug to the Canadian market (I’m just speculating here). But tiny pharma is in a very different situation.
Sorry to go on at so much length, but I wanted to share my thoughts about how complex the situation is. I also think this language of pharma companies entering the market “through the back door” or something like that is problematic. It’s as if the Ministry of Health thinks they get leverage by denying patients through the Case by Case Review policy and can thereby force manufacturers to apply for approval in Canada. That won’t work with a company like Partner Therapeutics. One patient in the whole country (me) is not leverage. If the goal is truly to get drug manufacturers to apply for approval in Canada, they need to be incentivized, and the process needs to be made less cumbersome and slow. If it were up to me, I would make our process more like the UK’s or Australia’s — no provincial role in evaluating a drug or deciding whether to adopt it for their formulary. I would also allow drug manufacturers to skip step one (applying to Health Canada) if they have already been approved by the FDA or by the EU’s regulatory body. Skip directly to the next steps — federal Health Technology Assessment and price negotiations.“
Unfortunately, I have not heard back from this person. I’m not sure if the silence is an example of this or not, but I have learned that some Canadians really get their back up when you point out the failures and inefficiencies of the health care system here. All I can say to that is that I have loved the Canadian healthcare system for 25 years, and it is only now that I am learning about the areas in which it is failing — making precision oncology treatments quickly available to patients is one of them.
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