Thank you, Durhane.
As a Canadian with a rare disease called congenital panhypopituitarism, I thank you for giving me an opportunity to testify before you today to discuss issues that are important to me.
My health deteriorated for 20 years because no research was being done on my condition, which is extremely rare. However, there was a drug approved by Health Canada that could have helped me. But the cost of the medication could not be refunded through insurance plans in my case, as there were no clinical trials on my specific condition.
During that time, I was monitored by numerous specialists and had to be frequently hospitalized. Finally, after more than 20 years, I was able to participate in a clinical trial and gain access to medications. I am now healthier. Unfortunately, my experience, which has been marked by significant difficulties in accessing treatments, is typical of what other Canadians with rare diseases are going through.
Should Canadians with rare diseases have to wait 20 years to gain access to the appropriate treatments and care people with common diseases take for granted?
I think it is time to implement policies and measures to improve the quality of care and treatments available to Canadians with rare diseases. Dr. Wong-Rieger will have some recommendations for you.