Health committee  If I could just jump in here, I think it's such an important question, but also one that, for the rare diseases community, has become disproportionately difficult. I mention this because you get treatment guidelines for the more major diseases, but at the same time, for instance,

Health committee  Certainly cystic fibrosis has been front and centre in the effort to try to get access to Trikafta, which has been considered the A+ therapy for patients with cystic fibrosis. Unfortunately, because of the PMPRB, the company has been very clear that it cannot bring it into Canada

Health committee  I think there is certainly the immediate death that can occur. We definitely have gotten notices around immediate death, where people could not get access to an intervention that would have in fact been life-saving. That definitely has happened. What we're more concerned about—a

Health committee  I don't mean to take up all of your question time, but I have to put in a very strong word about rare diseases. For our patients, because they don't normally have a good plan of care and their specialists are oftentimes isolated far away, we have patients who say, “I can get a me

Health committee  Thank you very much. We have also heard many concerns around dispensing and dispensing fees. As well, as you said, they could get only a small supply of the medicines. Some of them could not get them from their normal pharmacies, and when they went to another pharmacy there were

Health committee  Yes. I'm sorry about that. I forgot. We've heard a lot of those same concerns. Again, as I said, we had 50% of the people say that they could not get their regular medication. In almost every case, it had nothing to do with not having the drugs there, though in some cases it was

Health committee  Yes. Is that any better?

Health committee  CORD applauds the global multisectoral collaborations coming together to combat COVID-19. Is that okay?

Health committee  Okay. We applaud the global multisectoral collaborations coming together to combat COVID-19: to treat it, to find a cure and to prevent future infections. We're especially heartened by the public-private partnerships, which are essential for getting to successful interventions a

Health committee  Thank you very much, Mr. Chair and committee members, for the opportunity to be back before this committee on behalf of the Canadian Organization for Rare Disorders, Canada's national association of over 100 rare disease patient organizations, representing approximately 2.8 milli

Health committee  I'm trying to decide whether I should really speak honestly about what I think or whether I should just give you the politically correct response.

Health committee  Speaking honestly, as we were dealing with this regulatory framework, we kept getting promises that it was going to be submitted, and we kept hearing that the provinces are concerned about it because they're afraid that by having this framework, we're going to increase the number

Health committee  Thank you.

Health committee  Again, I think the way in which the process works penalizes the clinician and the patient, and we don't have to do it that way. I think that if you have patients, as you've rightly said, who are on a lifetime drug, the necessity to reapply every three months because it has not ac

Health committee  Certainly we believe that the modernization of the regulatory frameworks has provided some tools that can make that happen. You need to look at where the different barriers are. In some cases companies don't come, because these drugs have been around for a long time, and there's