Thank you very much, Maureen.
Let me continue by talking about our regulatory process and how it could be improved to facilitate access to orphan drugs.
I think Maureen's predicament stems from the fact that Canada is still the only developed country without an orphan drug policy. The biggest downside of that is that companies are not motivated to conduct clinical trials here in Canada. The U.S. passed an orphan drug act back in 1983, the European Union followed suit in about 2000, and other countries as well.
In reality, only about 50% of orphan drugs get authorized for use here in Canada. That may not be a problem, unless you're a patient or a parent who's waiting for a drug and has a debilitating disorder and are trying desperately to get something before it's too late.
To address these barriers and to facilitate patient access, we urge the federal government to implement as quickly as possible the draft federal orphan drug regulatory framework that has already been developed by Health Canada. Within this framework was a definition of rare disease as a condition that affects fewer than 1 in 2,000 people.
Even in cases where treatments are approved in Canada to treat rare diseases, patients experience important difficulties in accessing them. We know that most Canadians experience gaps in drug coverage, but for patients with rare diseases it's exacerbated. Our survey in 2015 showed that 1 in 3 patients with rare diseases could not access an appropriate drug treatment that was, in fact, in Canada.
Every country is struggling with how to address rare diseases and orphan drugs, but sadly, I think Canada is doing worse than most other developed countries to provide equitable and sustainable access.
Very briefly, the experience of patients who have Fabry's disease illustrates the delays in accessing orphan drugs. As you may know, in 2004 Health Canada approved two products to treat this disease, which is a debilitating condition that leads to organ failure and early death. Unfortunately, because they were using a very traditional cost-effectiveness assessment, the Canadian Agency for Drugs and Technologies in Health recommended that these drugs not be covered by the public drug plans. As a consequence, patients with private drug insurance were all able to access the medicines, but the patients covered by the public plans could not. It took about two years for the governments to set up a managed access program to fund the drugs. Very recently, another report confirmed the clinical effectiveness of these therapies.
What did we learn from this example and many others like it? Access to drugs for rare disease in this country is not effectively serving the patients. The problem is that the health system isn't saving any money by doing it this way, because the gaps in patient access to the right drugs lead to increased morbidity and poorer quality of life, and also to increased costs for the families, the health care system and, ultimately, the Canadian economy, as Maureen's story and those of thousands of other patients illustrate.
CORD supports a national pharmacare program, but we feel it should be guided by a set of core principles, including equity, quality of care and treatments, patient involvement, the incentivization of development and marketing of new therapies, and collaboration and coordination. We don't really care whether the program is administered federally or is a pan-Canadian federal-provincial-territorial approach, but we do believe that it has to have the following key characteristics: first, access to medicines according to a single set of eligibility criteria; second, flexibility and exceptional adjudication measures to ensure that the unique circumstances of each patient can be addressed, and addressed fairly; and third, a national pooling across all drug plans to ensure equitable and affordable access across the country.
I would be remiss not to address the prices of orphan drugs. We think that drugs for highly specialized diseases for small patient populations will, no doubt about it, cost more per patient than common drugs. There are some obvious factors involved, as you can imagine, including the higher cost for research and development of these drugs, as well as the smaller size of the patient population. To put that in perspective, rare diseases are more costly to diagnose, they're more costly to treat in hospitals, and they're more costly to follow by family physicians and pediatricians. They are simply more costly because they're rare, and they're severe and debilitating. Does that mean they're less worthy of treating, just because they're costly?
I think Pope Francis said it very well when he called for the globalization of empathy, which should be used to value all human lives, including people with rare diseases.
I want to conclude by saying that patients with rare diseases are really wanting to work collaboratively with all stakeholders. We want to make sure that the drugs are used appropriately and responsibly, and we also want to make sure that the prices in Canada are fair and—very importantly—sustainable.
We know that patients with rare diseases have extraordinary, unnecessary barriers to getting the appropriate care and treatment. We really encourage the federal government to do its part in helping address these challenges by implementing the orphan drug regulatory framework, and by working with the provinces to put in place a coordinated, national approach or program to ensure timely care and appropriate and equitable access to rare disease therapies. We think all Canadians with rare diseases deserve the same quality of care available to patients in other countries and patients with common conditions.