Mr. Chair, committee members, thank you for inviting me to appear before you today.
As Ms. Wong-Rieger said, my name is Maureen Smith, and I am a Canadian with a rare disease. It was diagnosed when I was eight years old, after four years of tests and visits to specialists.
For the past five decades, access to treatment and drugs has played a key role in my life.
Over the past 10 years, as a volunteer with the Canadian Organization for Rare Disorders and many other groups and advisory committees, I have heard from many Canadians with rare diseases. I have also been able to learn about the difficulties faced by other stakeholders.
I am delighted to share with you my perspective as a patient.
I thought long and hard about having this opportunity and giving you one message, if I could. I'm going to give that to you.
When you have a rare disease, you face medical uncertainty, and often there's nothing that can be done about that. In my own case, I was on the first Canadian clinical trial—four children with an ultra-rare disease. I was the only girl. You live with that medical uncertainty your entire life. Often, even with the best interest, there's nothing that can be done about that.
When you add the burden of uncertainty of access to treatments, that just adds another layer to your life. If you're fortunate enough to have a treatment, then you constantly worry about whether you're going to be able to access that treatment. If you do get it, then you worry about it being taken away.
I wanted to share that with you. That is one of the most difficult things for patients to deal with. A lot of medical uncertainty can't be dealt with, but I'm hoping that you will try to lessen that burden on Canadians who have rare diseases, and their families.