Each box costs around $20,000 and contains a monthly dose of what Debbie Simpson considers her son’s miracle drug.
“We call it the golden drug because it’s worth so much money and if we lost it, we’d be in big trouble,” said Simpson, who credits Orkambi with saving her son’s life.
“I thought I was going to lose him, you know. How do you prepare yourself for losing your child? You can’t.”
Liam Simpson was diagnosed with cystic fibrosis at two months old. After years of hospital visits and struggling to keep up with his peers in gym class, Liam joined the original Vertex Pharmaceuticals trial for Orkambi when he was 12 years old.
“After I went off the placebo and went on the real thing, holy crap was that the best moment of my life,” recalled Liam.
Orkambi is described by Cystic Fibrosis Canada as a modifying therapy that treats the basic defect in the disease as opposed to just the symptoms.
Liam Simpson says the drug has become his lifeline. Since he started taking it, his lung function increased from around 45 per cent at one point to 80 per cent— allowing him to transform from a kid who stayed home playing video games to an active teen.
“I was able to keep up with everybody,” Liam told Global News. “I was able to do more sports, be more physically active and that was just the best feeling in the world — mentally and physically.”
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Now 18 years old, Liam Simpson is heading to Langara College in January 2019, where he plans to study criminal justice and chase his dream of becoming a police officer.
His cure doesn’t come cheap, though. Orkambi costs $250,000 per year. Fortunately for Liam Simpson, the expensive drug is covered by his mother’s private insurance — but that’s not the case for everyone who could potentially benefit from it.
Cystic Fibrosis Canada told Global News that it believes Orkambi could change the lives of more than 200 British Columbians if it was covered by PharmaCare.
“We know that this drug works,” said Kim Steele with Cystic Fibrosis Canada.
“What we’re hoping is that our governments will give the people who really need access to this drug the opportunity to try it, so that we can see if it works in them.”
“That’s just heartbreaking, honestly,” said Liam Simpson. “There’s people out there that could benefit from it 100 per cent.”
Rejected twice by Canada’s Common Drug Review for lack of evidence on effectiveness, the province of B.C. is once again reviewing Orkambi.
“We don’t want these to be political decisions,” said B.C. health minister Adrian Dix.
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“We want them to be based on evidence. That’s the process we have in Canada and that’s the process we support.”
Dix told Global News the government is taking another look at the recommendations from a rigorous and independent process led by clinicians.
“Those recommendations were again negative on the clinical value of the drug and also negative in terms of listing it on PharmaCare, and so our job is to consider those recommendations again.”
Simpson and her son Liam believe there is only one decision.
“They will die without it. They have potential to continue living with it, my son’s proof of that,” said Debbie Simpson.
“They’re saying lack of efficacy. Look at me, like, I’m right here,” said Liam Simpson.
“The possibilities are endless with Orkambi.”