That's an excellent question.
The future of pediatric drugs is going to be something we all have to grapple with. It's in the name. These are expensive drugs for rare diseases.
By and large, these are drugs for a very small population. It is relatively difficult to get high-quality evidence in the way we used to do it, through randomized controlled trials and that sort of thing. We often have imperfect evidence as part of our development strategy, and that also impacts reimbursement. Should we be paying $100,000, $1 million or $2 million for medication for a child with a significant rare disease?
It's important to balance lots of different things. The magnitude of the disease matters. If it is a child whose life is threatened, I think it makes sense to have more focused and specific end points that may be about that child. It might be that we cover a drug and then we monitor that child's symptoms. We need to develop the evidence infrastructure on how to follow evidence for a very small population.
That also impacts reimbursement decisions, again. Our current systems, CADTH and INESSS, account for patient perspectives and for the magnitude of diseases. That needs to be further fleshed out.
At a federal level and at a governmental level, there are lots of dollars involved. What amount of funding should we be supporting this with? I would say this is something that should be commensurate with the disease burden.
All those things should be part of a rare disease strategy. I would encourage everyone to look up CORD and other national advocacy organizations that have very fleshed-out positions on this.