Certainly. Thank you for the opportunity.
The Canadian Organization for Rare Disorders is the national network for organizations for rare diseases in Canada. We are affiliated with both the National Organization for Rare Disorders in the U.S.A. and with EURORDIS, the European Organization for Rare Diseases.
The issue is that in Canada people with rare disorders are not well served. About 1 in 12 persons in Canada has a rare disorder. This may be surprising, but it affects nearly 3 million Canadians. There are between 6,000 and 7,000 rare disorders in the world, and many are found in Canada. So it is in fact a significant public health problem.
The way health care is set up in Canada, rare disorders receive disproportionately fewer and poorer services. Most people with rare disorders have poor access to diagnosis, poor access to treatment, and poor access to care. Around the world what's been happening, especially in Europe, is that they have recognized this. In the European Union they have introduced a recommendation that all member organizations, all member states, should adopt a national plan for rare disorders. This is what we're asking for in Canada as well, to make sure that we are able to treat patients with rare disorders in the same way as we do patients with more common diseases.
Within that plan, we're looking for three main things. First, we desperately need a definition of rare disorders. We're one of the few developed countries that has no national definition of rare disorders. Most countries define a rare disorder as a disease that affects fewer than 1 in 2,000 people. This definition would allow us to provide comprehensive services to patients right across the country. I think it would open us up to having a regulatory framework.
We are probably the only developed country that does not have a plan for orphan drugs and rare disorders. This means that our patients receive disproportionately less access to new treatments derived from research and development. It means that we have a more difficult time getting clinical trials for patients with rare disorders. It means that, unfortunately, Canadian patients with rare disorders are among the last patients in the developed world to get access to new medicines.
So as they're watching—we have families who are connected internationally—they see patients in other countries getting access to drugs. We have not set up a regulatory framework that would provide incentives for companies to bring their trials and products to Canada. So we are disproportionately served. Our patients are diagnosed later and treated later. Many of them become sick or die, even when treatments are available right outside the country and available to other patients who are suffering from exactly the same diseases. So we need the regulatory framework.
Second, we need a national plan for rare disorders. Countries around the world are establishing plans for providing integrated, comprehensive services for patients with rare disorders in the same way as they would provide services for common diseases, while recognizing that rare disorders are different. It takes more to be able to provide that expertise, but it takes more by way of organization, not necessarily by way of funding. What we know is that we pay for patients with rare disorders already; we just do it poorly, and we do it inefficiently, and we do it in such a way that we oftentimes don't really begin to take care of them until they're much sicker than they need to be. So our care and our treatment is just as costly as it would be if we had a good plan. We just don't service it well; we don't provide the same benefits. I think we also need to make sure that we have a national, integrated, comprehensive framework within which we can make recommendations for the funding of treatments for rare disorders.
We thank the health committee—I think it was two and half years ago—for holding a review on the Canadian Agency for Drugs and Technologies in Health. At the time, we recommended that CADTH come up with an appropriate way of reviewing drugs for rare disorders. What this committee recognized was that the way these drugs were being reviewed was not serving patients well. Patients with rare disorders do not get the access to treatments that they ought to. Drugs are approved, but then there is no program to allow them to be funded. We urge this committee, then, to actually pick up on what you began three years ago and to make sure that that does happen.
I'll leave it at that, but I look forward to the discussion.
And by the way, I love your hair colour and I think what you're doing is perfect.
