Thank you very much.
I am Durhane Wong-Rieger, the president and CEO of the Canadian Organization for Rare Disorders. I'd also like to acknowledge Maureen Smith, the secretary of the board of directors, who's here with us today as well.
We really appreciate the opportunity to present before this committee on truly a very important measure.
I would first like to acknowledge what Debbie was saying. My father died of Alzheimer's almost 40 years ago. Sad to say, even though quality of life and treatments in terms of symptom management are much better than they were when he was diagnosed, I think we're no closer to a real cure. We strongly endorse everything she's saying there and certainly acknowledge the Alzheimer Society for their amazing work.
I'd like to present a little bit of background on the Canadian Organization for Rare Disorders and put into context what our comments are with regard to CETA.
CORD is the national network of patients and patients organizations for people who are affected by rare diseases. A rare disease is defined in the EU and in the pending regulatory framework in Canada as a condition that affects no more than one in 2,000 persons.
There are probably 7,000 to 8,000 rare diseases. It affects one in twelve persons. So really, there are about 2.7 million Canadians who are directly affected by a rare disease. Even though each disease itself is very small, in aggregate it affects a very large number of people. About 50% of these conditions affect infants and children. Many of them actually die before the age of two. About 80% of them have a genetic origin, either a mutation that's passed on or a spontaneous mutation in the genes.
The good news is that about 3,200 of these disorders can be identified today by a very high-technology process, the most sophisticated of which we actually have in Canada: next-generation exome sequencing. It's not widely available yet, but we believe it will become much more available. It can really lead us on a great step forward in terms of identifying patients who are in fact affected by a rare condition, and even carriers.
The sad news is that fewer than about 400 of these 7,000 to 8,000 conditions actually have treatment. This is one of the reasons why, of course, many of the things we're doing are so very, very important. Many of the treatments we have currently are really only symptom management, mitigating the life-threatening condition, and sometimes avoiding some of these problems with very strenuous things like nutrition control or other kinds of symptom management.
In the decade prior to 1983, that entire decade, there were fewer than ten new drugs for rare diseases. In 1983 the U.S. passed the Orphan Drug Act, and it provided incentives for research and development into rare diseases. In 1999 the EU introduced their legislation to also support research and development into what they call orphan drugs. They're called “orphan” drugs because in many cases the drugs would be developed, but because it isn't very feasible to bring them to fruition, they are abandoned.
With the introduction of legislation in both the EU and the U.S., there have now been more than 425 new therapies for more than about 300 conditions, in fact benefiting worldwide more than 10 million patients. This is amazing. Canada, of course, has not been part of this. Up until now, and even today, we still do not have a definition for rare diseases and we do not have supporting regulations.
This has been a real challenge for us, not only because we don't get the same level of R and D in terms of rare diseases in Canada, but also because it means that drugs are being developed elsewhere. Because we don't have a definition and because we don't have a pathway for the regulatory approval of these drugs, they're very slow to come to Canada. Only about 50% to 60% of the drugs that are available to patients worldwide actually are available to patients in Canada. They don't come to Canada. We often have to call up companies and beg them to come to Canada. They see it as a burden in terms of trying to get their drugs approved and getting them available to patients.
The challenge for us, and the solution of course, is the harmonization of regulations and the ability to make sure that as drugs are being developed elsewhere or as we're developing the drugs in Canada, we're going to be able to have worldwide regulatory approval and worldwide orphan designations.
The lack of access early on in terms of clinical trials is not insignificant, because it means that in many cases, especially for patients with life-threatening conditions, they're not going to be in that first wave of therapies. Sometimes even 50% to 60% come to Canada. Sometimes they don't come to Canada until two, five, or even ten years after they're available elsewhere. This of course is not acceptable to us.
As I say, happily much of this dismal scenario is really primed for change. Over the past three or four years, CORD has been working very closely with Health Canada to develop Canada's first orphan drug regulatory framework. It's been designed really well to be closely aligned with the EMA and the U.S. FDA. The beauty of it is that as drugs are being developed, orphan status can be applied for simultaneously in Canada, the EU, and the U.S. This means we can implement clinical trials with the same conditions being set elsewhere so our patients and our clinicians can get in early. It means that as we're developing research and development in Canada, we can have worldwide application immediately. It makes a huge difference when you're talking about these very small patient populations. The regulatory framework was announced by the Honourable Minister Aglukkaq in October 2012. We were very hopeful that we would have the regulations implemented at that time. We were hopeful they were going to be implemented this year. We are truly counting on them being implemented in the first quarter of next year. We think we're on track for that. We're very optimistic about it.
This leads us to our support for CETA and why it's so very important for us. We are even more optimistic now that we've seen the announcement for the comprehensive economic and trade agreement. It will bring us even more closely into alignment with the EU, especially with patent restoration. We've had companies that have not wanted to bring their drugs to Canada because there was going to be a delay in the clinical trials. They were not going to be able to get them approved in time.
We're also very supportive of the proposed rights of appeal to address duplicate litigation under the patented medicines NOC regulations. After some 30 years of the Orphan Drug Act, we're now seeing the first generics for rare diseases. It is very important that there be a good balance between innovation and generics, which are going to give the best options to patients in Canada.
I would say the one thing we are concerned about is that CETA doesn't really fully harmonize the Canadian and EU intellectual property regimes, first in terms of data protection, and second with respect to a very important provision for orphan drugs, which is market exclusivity. It's seven years in the U.S. and ten years in the EU. That gives drug companies exclusive marketing rights for that period of time. This we believe is very beneficial to ensure that we're going to have that same access that other countries have.
We're very supportive of moving forward with Canadian-based R and D, and we think CETA will really help to get more companies in Canada committed to doing that level of research. We are very confident in the long-term benefits of it. We know there are some concerns about the potential health care costs, but we recognize that in the long term, this is going to be much more beneficial.
In closing, we do support the general provisions of CETA. We are very supportive of the measures to compare data collection, analyses, and practices, and we hope those will actually lead into an application into the regulatory framework for drugs as well.
We are very happy about the way the agreement will actually benefit orphan drugs and ultimately patients in Canada.