BC Children’s Hospital has declined the request to fund ERT for Trey. They argue that there is not enough evidence that Elaprase (the ERT for Trey) will work, and that evidence is especially lacking for children under 5. Well naturally, there haven’t been any trials on children under 5. That’s the thing with a new treatment and a very rare condition! But since MPS is progressive, and the FDA found enough evidence that it works to approve Elaprase (for all ages), it would follow suit that we should start treatment for children with MPS II as young as possible.
Kirsten Harkins, the Executive Director of the Canadian MPS Society and a mother of a child with MPS, has explained that this is the problem with not having an Orphan Drug Policy. Everyone will cite some variation of this response as a reason to not pay for ERT.
So now it’s onto the government for funding. Dr. Lorne Clarke and Dr. Sylvia Stockler have a meeting with Bob Nakagawa, Deputy Minister of Health, on Friday to discuss funding and Dr. Clarke says we can expect an answer in 3-5 weeks. So for now, more waiting. At least we’re one down and onto the next.
