If we look at what's happening internationally—and I think we all agree that we have to use an international framework, especially with rare disorders—we can see exactly what you're suggesting. That is, the Europeans and the U.S. actually work quite collaboratively, including the Japanese and the Australians, with regard to international review bodies agreeing on what constitutes an appropriate clinical trial. So I certainly think that with rare disorders we need to make sure we are linked internationally.
I think it's happening, as you can see, with the progressive licensing framework, where there's harmonization with the international review bodies so that we're not reinventing or redoing it. Even more so with rare disorders, we don't have enough patients. When you have a disorder affecting two, five, or even thirty patients in Canada, we cannot actually do a separate process. On the other hand, we do want to make sure the process is internationally linked.
So what we're suggesting then is a process that certainly has a place within what is happening in terms of reviews, both regulatory and in health technology assessment, in Canada, but also then has a place within the international framework.
While it may seem untidy to you to say, well, we're going to have a lot of separate bodies here, what we're really suggesting is something that actually makes a lot more sense, in terms of having exactly what you're talking about: agreement around what constitutes appropriate evidence; agreement around what constitutes appropriate costing; agreement around what constitutes long-term monitoring; and the necessity then of collecting that information to determine ongoing safety and effectiveness. I don't think we can do it in Canada separately.
So if you're going to have this, then the important thing is that every European country we looked at—and also the European Union—all have bodies that have now been designed specifically for rare disorders. The models are there. The U.K. has a whole program around it. The European Union has a collaborative framework that includes patients and researchers and clinicians. The French have one within their own country. The Dutch have one. So I think if we looked internationally, we would see there has been strong agreement that these diseases need to be considered separately.
It is unfortunate that Canada sits as the only developed country I know of that does not have an orphan drug program. And it puts us at a severe disadvantage in terms of working internationally, to make sure that we do have the right information to provide the drugs and also to provide the long-term safety and efficacy you're talking about.