Mr. Speaker, I am very pleased to rise today to address and fully support Motion No. 426, a motion to address the lack of a rare disease drug policy in Canada.
Health care is always among the top concerns of residents in my community of Thornhill and, of course, all Canadians. From access to treatment to quality of care, residents in my community and all across Canada want a health care system that is there when they need it and that is inclusive.
I am very proud to speak in support of the motion by my colleague from North Vancouver, which has been very much inspired from his own very difficult, sad family experience and has been used in such a positive fashion to help others in the same predicament. I am very proud to support all of his efforts. We were all very moved today to hear the story that he shared, as well as other members' personal experiences.
His motion would move Canada toward a system where Canadians with rare disorders would receive the same standard of care as patients with more common disorders. As a breast cancer survivor for 16 years, if I had not had the drugs available to me at that time, I cannot imagine what would have happened to me. The same is true for many others. I can well understand all those who need to be part of our system. When we consider that other countries are doing so, it is vital that we move together. I am happy to see there is some consensus.
As a member of the health committee and part of the common drug review, it was very clear from the organizations and advocacy groups that this issue has not been adequately addressed. The time has come to do so now.
A telling example was brought to the committee's attention, and it was particularly disturbing. It revolved around Nexavar, a drug to treat kidney disorders. It was rejected by the CDR essentially because it was too effective. The interim data from clinical trials of Nexavar were producing stellar results and the U.S. Food and Drug Administration said that patients in the control arm of the study should be allowed to enter the treatment arm. It was the obvious ethical choice.
Many patients in the control arm of the study, obviously enthusiastically embraced the opportunity to receive this new, groundbreaking drug, but because most U.S. patients had left the control arm, there was not enough data left to satisfy CDR requirements. The placebo data was determined to be statistically insufficient and the drug was rejected on these grounds. Clearly, the placebo data would not be forthcoming and access to Nexavar, available to those suffering in the U.S. and Europe, was severely hampered in Canada.
There is not much motivation in these kinds of cases for Canadian biotech companies at this time to simply duplicate a clinical trial that has already proven to be successful elsewhere. That is one of the other dilemmas.
Because data from trials conducted in other countries is not accepted by the CDR process presently, patients are often forced to wait years for clinical trials to conclude in Canada while patients in other countries, as I have said, have already had access to the drugs being tested. In Canada, patients with rare disorders are forced to wait for no logical reason and to their detriment, except for the rigid requirements presently, in this case, in the CDR process.
Drugs that are often covered by private drug plans to treat conditions such as gigantism, Fabry disease, Fabrazyme, MPS, Gaucher disease and kidney cancer have been rejected by the CDR and are not accessible to those with public drug plans.
Who is being denied treatment the most often? The sad reality is that in many cases it is Canadian children who have rare childhood disorders. In fact, it is those children and their families who are being denied access to treatment. Even in cases where drugs for rare disorders have been approved through Health Canada's progressive licensing framework, often CDR will stand in isolation and essentially reject the conclusions of Health Canada and numerous international studies.
Motion No. 426 asks the government to consider the establishment, and rightly so, of centres of reference for specific rare disorders that would be comprised of national and international experts who would develop the criteria for treating patients based on scientific evidence and patient impact. They would provide ongoing surveillance into the real world safety and effectiveness of these treatments on individual and group bases so that we could consider supporting internationally accepted standards for the conduct of clinical trials in rare disorders appropriate for the challenges inherent to very small patient populations.
By expanding the CDR and developing a separate process for the consideration of rare disorder drugs, we can even the playing field as so many other countries have already done, be it in the U.K., the U.S. The examples are everywhere. It is time for Canada to follow their lead and embrace the international framework model that has proven to be so successful.
Many countries, such as France and the U.K., and others in the EU have successful models of separate bodies that consider treatments for rare disorders. Canada must take a hard look at the cooperation that is happening in these countries as there is much it can learn from their experiences. The time has come, again with our collective resolve, to do so.
It was suggested to our committee that changing the CDR process to allow for the pooling of limited Canadian data rejected by the CDR with rare disorder patient groups outside Canada would likely set the stage for more approvals of treatments of rare disorders, which is what is necessary.
The testimony the health committee heard on this issue was compelling. When our final report on the issue was tabled, included in it was the recommendation, as referenced today by the government, that the government look at options. We are happy to see the government has accepted that recommendation favourably. Again, we will very much be looking to see this is implemented and moved forward in a very expeditious fashion. Often we hear there is interest by a government, but it does not always moved on it. In this case there is no other acceptable option but to move on it.
Clearly there is consensus, as I have mentioned, including the government and the health committee, that a new approach is required and that the one currently taken by the Canadian Agency for Drugs and Technologies in Health in approving drugs for rare disorders is not yet there.
When the government tabled its response, I was pleased it was received well. I very much look to see that this has everyone's total support and that we will see action in this area, which is so vital. It is predominantly affecting children and other Canadians.
I fully support this, and again commend my colleague for moving on and taking leadership forward. We want to see this happen not in a long period of time, but in the very near future so those suffering currently will feel they are part of our health system in every fashion and that they do not have to look elsewhere.
