The story of neuroendocrine tumours remains a story, and there are many like that in Canada. Unfortunately, it is often dealt with case by case. Fabry disease is another case that was dealt with to some extent. Often, the case was handled because parents or adults went to the media and managed to obtain a positive answer. But it is unacceptable that some people, in addition to being sick, have to go to the media to demand access to good medical care and treatments. There are many similar examples. Inequities happen all across Canada because each province offers different solutions.
For example, myosin is used to treat Crohn's disease. In our province, adults don't have access to it, but in our neighbouring province, in Ontario, adults can at least try this drug to see if it is effective, and can have an assessment done.
I could list a number of inequities like that. That is why we need a regulatory framework to standardize what happens in Canada with orphan diseases. The regulatory framework should address two issues: the development of drugs and access to them.
We know that drug benefits are under provincial jurisdiction. But, in terms of assessment, why redo the assessment of a rare drug, an orphan drug, in each province, when it is already something difficult to do? There must be a special way to review it. And that is what is going on at the moment.
We know that Quebec has excluded itself from drug review in general, but for orphan drugs, I believe in the need for a framework so that resources can be used better. We could do standardized assessments. We know that each province decides on the reimbursement, but at least the assessment would be properly done for orphan drugs, which are not the same as drugs for common diseases.
There is also the issue of developing drugs. I would like to give a positive example of an initiative that we could take more often in our country. In Quebec, a patient organization was able to have a company from California come to do a clinical study. Those people succeeded in turning the Montreal Children's Hospital into a site for international study. It is not an easy thing to do, but they managed because of those few patients with Morquio syndrome, who are actually more numerous in Quebec. In fact, that allows Canada to have expertise in a disease. That hospital could become a specialized clinic for that syndrome. It could become a site for phase 3 clinical trials for that drug. That is very beneficial for the patients, obviously, but also for drug research and development.
But, even if that drug has phase 3 trials in Montreal, we are afraid that we might be facing the same obstacles in terms of approval and access in Canada. There is also the question of quid pro quo in the research. Patients give their time and they travel to participate in the study. So, if they can't even have access to the drugs after that, it would be a real shame.