Thank you for that question.
Obviously, I speak both as a coordinator for Orphanet Canada and a researcher but also as a physician for individuals affected by these rare diseases. As their physician, it's nearly impossible at times to resist the urge to embark on further research to get answers for what are truly devastating disorders. Having said that, we do need to recognize at times there's a limited pool of resources, whether that's research or clinical dollars.
I think a few points can be made. One that I already alluded to previously is that occasionally research into a rare disease can often give us tremendous insight into common disease. This story has played itself out many times over in the fields of hypercholestoralemia, and many rare cancers. Through the research into rare genetic forms of cancer, you find the genes that are also the same genes that are mutated in people with common cancer. One point is that the research is often generalizable.
I think going forward, if we're talking about 3,500 rare diseases, the system will be stretched if we're talking about 3,500 new expensive therapies. What I think we may have to envision is collapsing down various rare diseases into families or groups of diseases where maybe you can have a common therapeutic approach.You may have to pool things, given the limited resources.