Thank you very much, Mr. Chairman and members of the Standing Committee on Health, for letting me come today and talk about my concerns about drug funding. In particular, I'm going to talk about kidney cancer treatments and how recent CDR decisions are really going to get in the way of our Canadian patients being treated with what is now a global standard of care and how these decisions also impact on us as physicians, our primary obligation to provide care and benefit to our patients.
I'm a medical oncologist at Princess Margaret Hospital. I'm also a kidney cancer researcher and specialist, and I'm someone to whom many oncologists refer their patients for opinions, and I do a lot of research. But I can tell you today that I'm not speaking just for myself; I'm speaking for at least 30 kidney cancer specialists across the country who I met with on the weekend and who have signed a statement, which--I apologize for not having it translated--I'll get to you later. These are key thought leaders and kidney cancer specialists who really share my concern. For example, Dr. Martin Gleave, who's head of CUOG, the Canadian Urologic Oncology Group; Dr. Simon Tanguay, who works as a researcher in Montreal and McGill; Dr. Peter Venner, who runs the Cross Cancer Institute in Edmonton, and many others.
Basically, kidney cancer is not one of the big four. It's not breast or lung or colon or prostate cancer, so it doesn't get quite as much attention. It's also been a very difficult cancer to look after for many years because, essentially, nothing worked. It was a terrible disease.
If you have metastatic kidney cancer, which is essentially what most people who present end up having, your average survival is about a year. We did many clinical trials for years, and essentially chemotherapy, radiation, nothing really works. And along came this revolutionary new idea, this antiangiogenesis, which you probably heard about, which really means we've got these therapies now where we can't get at the cancer, but we can prevent the cancer from attracting blood vessels that feed it. It doesn't get rid of the cancer, but it stops it from spreading and metastasizing, which is essentially what kills the cancer.
This was science fiction 10 years ago and now we have real agents. Someone is going to get a Nobel Prize for this. Thankfully, some of the drugs that have come along first in their class, that are what we call these antiangiogenesis inhibitors, actually are pretty much home runs in kidney cancer.
The first drug that came along with evidence of survival was Nexavar, and then soon after that came the drug Sutent. Both of these drugs have recently been reviewed by CDR, and they advised that they not be funded in provinces, based on their understanding of the data and their cost-effectiveness analysis, which I think is very flawed.
I need to talk a little about how clinical trials are done. We strive hard to meet ethical standards, so it has to be reviewed through an ethics board, and patients give informed consent. Then usually, in the cases I'm talking about, the new treatment is compared to a standard of care. So the control arm is what would be your best and then the new drug.
In the case of the first example, Nexavar, what happened was kidney cancer patients who had already tried something and were progressing and in trouble were randomized to this trial. It was blinded, and they either got the Nexavar pill or a placebo. Then toward the end of the accrual of the patients, there's something called a data safety monitoring committee, which is independent, and this was also in dialogue with the FDA in the U.S.
One of the end points we look at is called progression-free survival, and you get information about this before you get information on overall survival. Progression-free survival says how long has this patient lived before their tumour has grown in a significant way, and that in itself is an important end point.
Basically the curves were so different at that point that we were advised to un-blind our patients. I hate to say this so bluntly, but anybody who was still alive on placebo could then have a chance to cross over and get the drug. Something like this doesn't happen very often in medical oncology. I remember when we got the message; the excitement was amazing.
I want to tell you about one of my patients, because it helps. Statistics don't tell the stories very well. I had this 46-year-old man with advanced kidney cancer. He was on the trial, but it turned out he was on the placebo and he was dying. We were setting up his hospice care. He came in to see me, which was probably going to be for the last time, and it just happened that the crossover logistics had worked out so that his wife had been delivered the bottle of the Nexavar pills that day--such irony. She asked, “What should I do?” I said, “Well, if he's well enough to swallow, what's the harm? Try to take it.”
He swallowed these pills and slowly started to get better. He came back to see me at about the eight-week mark, and I said, “You know, you're really looking better.” He said, “My pain is better and I'm feeling more hungry.”
We took a CAT scan, and lo and behold, he had a response, and it was shrinking. He went on probably to have another year, probably 10 months, under complete control by that drug, but then he progressed and died.
If you look at the clinical trial data, he's on the control arm and he's counted as control arm. You can actually see the point in time at which this crossover happened. The curves, which were further apart--so the difference was greater--start to come together. None of us would ever argue that that was the wrong thing to do; that was the ethical thing to do, and the whole world has embraced this as a positive trial.
When the CDR reviewed this data, they basically said we don't like this progression-free survival end point, and because the survival is not so great now, despite this crossover, we just don't have confidence that the drug is that different.
There's all kinds of different statistical analysis you can try to do to get the truth of what the true difference would have been, but there's nobody who treats kidney cancer, no expert in the world, who doesn't believe that drug had a meaningful impact on the survival of those patients. It's just that it's awfully hard to put a number on it. Then when you calculate cost-effectiveness and take the smallest number you can possibly get in terms of the benefit and put a big price tag on it, of course it doesn't look like it's that beneficial.
I have lots of other stories, but I want to mention that I came today for moral support with one of my patients, Mr. Clark, who had metastatic kidney cancer for three years. He knows the average survival is one year. He is looking very well. He got that drug through the mechanism of the clinical trial, and he wouldn't be here today, alive, if he hadn't gotten that drug. So the statistics don't tell all the stories.
The next drug that came along was Sutent. It has a similar mechanism of action, but we don't really know what the differences are between these drugs. It was tested in a slightly different patient population, patients who had never been treated before. Once again there was a remarkable separating of the curves, so much so that it went forward for licensing very quickly. The survival data had not matured yet. Then, of course, patients cross over; they move on to other therapies, and the survival curves come together.
Those are the two drugs that have been recently reviewed. In oncology circles, the way we think about these drugs is that they are home runs. We're not curing these patients with advanced disease, but this is the biggest thing that's happened in 30 years, and it has the potential to really build momentum to understand more and to make further progress.
One of my big concerns is not only with the CDR recommending they not be funded and provinces then following that. It's that, first of all, the patients are not going to get what the world considers to be a standard of care. Our patients are no longer getting good therapy. The second issue is that as investigators, how do we go forward--especially as Canadian investigators--in trying to ask the next questions? How do I build on that? All the next trials are going to be about what you do next.
Our patients won't be eligible for those trials because they didn't get the first line. Some patients are getting it paid through private insurance, but most Canadians don't have that kind of insurance, so many are not getting coverage. It's a very difficult thing as an oncologist to speak with your patient and say, not only do you have a bad disease, which is terminal, but we actually know about some new therapies that could prolong your life, but we're not going to be able to give them to you here in Canada. I'm going to fight for you, but I don't know how it's going to work out.
I'll end just by saying we're very unusual here in Canada in not embracing these drugs for funding very quickly. As far as I know, Nexavar, which is the first one that came along, is being reimbursed in Austria, France, Germany, Greece, Ireland, Italy, the Netherlands, Sweden, Spain, Switzerland, the United Kingdom, and the U.S.
I've always been so proud of our Canadian system and our health care system, and we've been real leaders in the world in clinical research, but if we allow this sort of thing to continue, we're really going to fall off very quickly, and our patients will be really woefully under-cared for.
I'll stop there.