Londoner worries about fate of 'life-changing' cystic fibrosis drug

Diagnosed with cystic fibrosis (CF) at six months old, Sylvie van Geel never expected to live past 19: the same age her big sister was when she passed away from the condition.

Instead, the 51-year-old Londoner is alive and says she's feeling better than ever, thanks to the cystic fibrosis drug Orkambi, which she began taking February last year.

"I can breathe a lot better, especially with our humidity in the summer that we get," said van Geel, adding that the humid weather usually keeps her indoors.

"This summer, here I was out golfing in the 30 C weather that we were having."

The drug is out of reach for many CF patients, thanks to its hefty price tag: approximately $250,000 per year.

Van Geel, whose husband's insurance pays for her Orkambi prescription, says she wants the drug to be paid for by public drug programs as well — something that's looking less and less likely.

Orkambi is approved by Health Canada, but its coverage in various public drug benefit plans — like OHIP+ or ODSP — is based in part on advice by the Canadian Agency for Drugs and Technologies in Health (CADTH).

CADTH had previously recommended that Orkambi not be reimbursed, but this year the agency began re-reviewing the drug after the company that manufactures it submitted a new application.

And this fall, CADTH came back with the same recommendation: that Orkambi not be reimbursed.

In clinical tests, the drug review body said Orkambi's positive effect on lung function was "smaller than the minimum amount of improvement that is generally accepted as reflecting a clinically meaningful change." In an email, a spokesperson for CADTH added that clinical testing found no consistent improvements in "other" important clinical outcomes, either​.

For van Geel, the improvement brought about by Orkambi wasn't small — it was "life-changing." And she said she worries that if public benefit plans won't cover the drug, people like her great-niece — also born with CF — will suffer unnecessarily.

"She's going to be suffering for how many years, and she can just get this pill and it would be so much better for her," said van Geel.

There are about 4,200 people in the country living with CF, about half of whom could benefit from Orkambi, according to Kim Steele, director of government and community relations for Cystic Fibrosis Canada.

Steele added that many private drug plans already restrict Orkambi prescriptions to those with very specific clinical criteria, and thinks it would be reasonable for public plans to do the same.

"It's not like 2,100 people are going to rush to be on this drug," said Steele.

"[Patients] have to have certain clinical markers to access this drug if they're going through private insurance, and we would expect the same kind of criteria to be wrapped around any public insurance," she said.

"That would limit how many people can get the drug, and it would also ensure that the people who really need the drug can get it."

Going forward, CF Canada says it is urging both the manufacturer of Orkambi and Canada's provincial governments to negotiate a fair price for Orkambi.

Meanwhile, Sylvie van Geel is enjoying the long walks and golf games that are made possible by the drug. But she's added another activity to her retirement: writing letters to her MP about how important it is to people like her.

"I'm hoping they will reconsider and look into the matter a little more, because I think there are a lot of people that are benefiting from it."