Health Committee on June 3rd, 2010

[The Vice-Chair, Liberal, Joyce Murray]

Thank you.

Dr. Carrie, you have a comment?

[Colin Carrie, Conservative, Oshawa, ON]

Yes.

Maureen, what is the reason they're not available in Canada? Has anybody applied through the special access program or anything along those lines? What's the rationale you've been given?

[Maureen Coleman]

They do apply through the special access program, though some people completely circumvent it. People with money just go out of the country. There's a high incidence of people with money going out of the country. They go through the special access program, but then I'm not sure how well educated physicians are on the special access program. And when they do apply, it's quite lengthy.

[Colin Carrie, Conservative, Oshawa, ON]

Seriously? What are we talking about?

[Maureen Coleman]

It can be lengthy. It could be months, and when somebody's quite sick, that's a long time.

[Colin Carrie, Conservative, Oshawa, ON]

It would be, yes.

[Maureen Coleman]

As important as that is, they can't afford to pay the airfare, so the special--

[Colin Carrie, Conservative, Oshawa, ON]

So the special access program...my understanding is it should be available in Canada.

[Maureen Coleman]

No, not in Canada.

[Colin Carrie, Conservative, Oshawa, ON]

So they apply for the special access program and they are getting denied?

[Maureen Coleman]

Some people will be. They get accepted to go out of the country. They don't get accepted for Canada, and that means they can't afford to go. So they can't take advantage of it because it's not regulated here.

[The Vice-Chair, Liberal, Joyce Murray]

Dr. Urbain.

[Dr. Jean-Luc Urbain]

Colin, this is the key question. That's a very good question.

Let me try to frame this. You know we have been dealing with a major isotope crisis of molybdenum and technetium; they're 80% of the isotopes we use in nuclear medicine. Rare disorders need rare isotopes. It's very difficult to determine regulatory process, to have access to import into Canada what I'm going to call those rare isotopes. The way we're trying to cope with the system is we're trying every single path we can.

I'm going to give you a specific example. Three weeks ago, I got a phone call from Montreal, then from Halifax, and yesterday from Vancouver, to let me know that the special access program is now closing the importation of yttrium, which Maureen mentioned, just because the companies have not submitted a proper dossier to Health Canada to import those isotopes. The issue is that those companies cannot submit a proper dossier based on clinical trials in Canada. It would take 15 to 20 years. In other words, the processes with Health Canada are antiquated. What we have found is that it really depends on the person you're dealing with in a country of overzealous bureaucratic processes.

So in the case of neuroendocrine tumours, for example, that's something that we have had as a special access program for 15 years, and for 15 years we've been told we have to do the proper study, while, as Maureen said, the European Union has proper access to those drugs. There's a major disconnect between patient needs, Canadian needs, and the health care regulatory process.

One of the reasons why I really wanted to come here--and again, thank you for the invitation--is to plead with this committee to make sure that we put in place with Health Canada the proper processes for Canadians to have access to rare drugs or rare isotopes for rare disorders, besides the big picture, which is a totally different problem.

[Colin Carrie, Conservative, Oshawa, ON]

So you're saying Health Canada is demanding a Canadian study. It would be too small a study. Committee members would take 10 years to have that done. Is that what you're saying? They're not recognized in international studies or anything like that?

[Dr. Jean-Luc Urbain]

Yes. That's exactly the point. When you deal with cardiovascular disease or cancer, you can generate data very quickly. When you deal with rare disorders--and Durhane mentioned those numbers are in your information—it's virtually impossible. At the same time, we feel very bad for our patients, because we constantly feel it's a tennis table between the federal and provincial agencies. Federal agencies have regulations; provincial is basically reinforcement. Patients are trapped in between. At the end of the day, they don't get the care they need in the western world.

[The Vice-Chair, Liberal, Joyce Murray]

Mr. Brenders.

[Peter Brenders]

Thank you.

I think Maureen's and Dr. Urbain's stories are great examples of a symptom of a challenge that Canada presents and that other countries don't have, which Durhane mentioned. If you think about it, Canadian patients are at a disadvantage. Researchers are at a disadvantage. Our health system is at a disadvantage, because we don't have the structure in the system. So what happens elsewhere in the world? It started with the United States in 1983 when they introduced an Orphan Drug Act, and it was called “orphan” because these diseases were deemed to be orphans. No one was looking after them. Rare disorders were basically neglected.

In the 10 years prior to 1983, there were only 10 drugs available in the whole world to treat a very limited number of orphan diseases. After the act came into place, you saw a whole upswing of research and development. Today there are over almost 400 products on the market to treat rare disorders and there are over 2,000 products in research and development. I mentioned earlier there are 21 Canadian companies that are doing it. But a lot of that research is all done outside of our country. It's done in these jurisdictions, like in the U.S., like in Europe and Japan, every developed nation except us, and we're in line with I think it's South Africa, Saudi Arabia, and India right now in not really having a defined structure to deal with orphan products to allow for the research, the development, the introduction, and the treatment. We're behind the times.

As I heard Durhane say, there are only two things that we need. One is we just need a regulatory structure to recognize these and create that support, because these are different from the sorts of common treatments that people see out there. Then we need a framework to allow for that coordination of diagnosis, treatment, and support of these patients. We need some federal leadership, which can be a regulatory change, a non-cost activity for us as a country to be able to put this in place and give that leadership down to the provinces that we're treating everyone the same. It's not new to us. We've talked about this for a number of years.

This committee has had some tremendous leadership in that area. Parliament has had it as well. Yet we continue to be stuck, and we're a little frustrated by that, especially as companies trying to introduce new treatments and wanting to make sure that Canadians can get early access in the trials, early access in the support, and that all that money we've been spending on primary research in the early days ultimately turns into products for Canadians.

I'm happy to take questions on that.

[The Vice-Chair, Liberal, Joyce Murray]

Carolyn Bennett.

[Carolyn Bennett, Liberal, St. Paul's, ON]

I think what we're hearing is that there's a huge opportunity here, that it is about rare.... The fact that it's rare means you're not going to get classical double blind trials in Canada. We've got to change the rules. I believe that if we can get it right for the rare diseases, we actually would get a better regulatory system for all diseases, meaning that we have been a bit blinkered in Canada with this idea that you have to have Canadian trials in order to go forward.

Maybe you could share with me what other countries are doing, because I thought the FDA had begun a system where you can have a committee of patients and health care providers looking at the international evidence and saying, this is good enough for me, for us, and this gets fast-tracked into Canada, or saying we don't like the way this trial was done, it doesn't look independent, it looks whatever, and that goes into the regular system. But it seems that we need to look at what you would seriously recommend we do from other countries so that we could quickly fix this. There isn't the expertise within that little office.

The idea that the difference between a drug and a radiopharmaceutical...the people in that office only know about drugs. They don't know about radiopharmaceuticals. It's now a real block that we as a committee I think could just suggest that it get done immediately with no money. With Fabry's disease, I think the feds, the provinces.... I was at the meeting where all the health ministers came together, knowing that the pharmaceutical companies were prepared to go a third, a third, a third and help pay for this. It was unevenly placed across Canada, Nova Scotia, and Alberta. All the health ministers said we should help with this, because it's not really fair and because there was almost an epidemic of diagnosis and a very expensive treatment.

I think what we want today is some advice. If we were to write a letter to the minister, if we were to be able to help do this, what would you want in that letter, and would you suggest a structure that has worked in other places? How did Cuba, Poland, Serbia, get around this such that their patients are getting access--on the neuroendocrine, but then also on the pharmaceutical as well?

[The Vice-Chair, Liberal, Joyce Murray]

Ms. Leslie.

[Megan Leslie, NDP, Halifax, NS]

Thank you, Chair.

Actually, I think it makes the most sense to have a response to Dr. Bennett's question.

Perhaps you can come back to me.

[The Vice-Chair, Liberal, Joyce Murray]

Do any of our guests want to respond to that?

I think Ms. Wong-Rieger does and Mr. Urbain.

[Dr. Jean-Luc Urbain]

Carolyn, I think you're absolutely right. This is a unique opportunity for Canada. The way I see it, the unique opportunity is coming from the fact, as Gail said, that they call them genetic diseases.

We are in the era of the personalized human genome project and personalized medicine. These diseases--and don't take this the wrong way--are the perfect templates to look for a solution for common diseases in the future at limited cost. So they could be wonderful approaches to unroll personalized medicine in Canada, since we know for most diseases the genetic origin and mechanism, and the industry can develop drugs for diagnosis and treatment for those orphan diseases.

[The Vice-Chair, Liberal, Joyce Murray]

Is there another answer to the question Dr. Bennett posed?

[Dr. Durhane Wong-Rieger]

I think Dr. Bennett is absolutely right. We don't need to re-invent the wheel on this.

Gail and I were recently at the European organization for rare disorders conference. There were 3,000 persons there and every country was represented.

People are putting together national plans to do this, and there is every opportunity for Canada to be a partner at that table, to actually follow the models that are being put together for national plans and look at the pillars they've already identified.

They've put out guidelines in the European Union for how countries should structure their plans. There are a dozen different models, so there's no one-size-fits-all. But there are blueprints to help you identify them.

First and foremost is what Peter talked about, and that is that we need that regulatory framework. It speaks to what Dr. Brenders and Dr. Bennett are saying. We have to modernize our Canada Health Act and the way in which Health Canada works so that we can take advantage of what is being put in place in other countries to deal with modern medicine.

The Canada Health Act is 50 years old, and in many respects, even the regulators can't do what they want to do. This is a tragedy because it results in all the kinds of inefficiencies we're talking about, unnecessary suffering, and people not getting treatment. As you say, it's difficult for companies to come to Canada to set up shop, do their clinical trials, or make these therapies available to people.

So you're absolutely right. First and foremost, we have to do the regulatory framework. I would say that in Canada we're a hair's breadth away from getting it. There are some draft regulatory pieces that have been put together. We have provided input on them to Health Canada. We know we're very close. It really needs the parliamentarians to say, “Yes, do it.” It needs this health committee to turn to the minister and the rest of Parliament and say we need to do this.

We believe that patients with rare disorders are different; therefore, we have to approach the problem differently. We have the models for how to do it. At the end of the day, it doesn't cost any more. As Maureen says very clearly, it can cost us a whole lot less and make it so much better for people if we do it right. The good news in being so far behind the rest of the world is that we can learn not only from other people's experiences, but we can take the best of the plans. I think we've got a lot of good blueprints.

As Dr. Bennett said, in many other countries, patients, researchers, clinicians, and the regulators are at the table to provide input into design of the clinical trials, the review of that information, and even on the next step in terms of how to make them available and accessible to patients.

Dr. Bennett said the solution that we had for Fabry's disease--that you had for Fabry's disease--was not the only model, but it was a very good model of how the feds, the provinces, and the territories can work together. I will wager that the feds played a leading role in making that happen, and I think we can.

So the blueprints are there. All of the things we need to do are there, and what you're hearing as to one specific disorder is exactly the case. This is the problem in many other aspects we get with all of the rare disorders, and we believe it as well. If we can fix this problem for rare disorders, we can do a heck of a lot better for many common disorders. We can get ourselves into a position of being one of the leading countries, because we're very close. We have lots of good stuff going on in the background. It's up to the committee and Parliament to make it happen.