'What’s the price for sight?': N.S. family can’t access treatment to save daughter’s vision
At five years old, Nora Francis definitely has an eye for art. The young girl loves to draw and will spend hours with her paper and markers at the family’s kitchen table.
But every day, Nora’s eyes are losing sight.
At two years old, she was diagnosed with Leber congenital amaurosis, or LCA, a rare inherited vision disease resulting from mutations in a specific gene. It causes the death of cells in the retina.
"It is a degenerative disease," says her mother, Sitka Polk. "So eventually most, by the time they are teenagers, have gone blind."
The disease means Nora has no night vision. She needs bright lights to see indoors even in daylight and sometimes to play outside when the sun starts to set.
Her vision is worsening with time.
"For any parent who has a child with any disability to know there's a treatment out there for it,” says Polk, “you want to get it done so that you don't have to see any more decline in their vision."
The only treatment available for Nora is gene therapy, approved in Canada last October.
Known as Luxturna, it involves injecting healthy genes into each retina.
Nora's pediatric ophthalmologist at the IWK Health Centre says it can preserve sight for at least a decade, maybe more.
"It specifically corrects the defective gene," says Dr. Johane Robitaille.
But the cost of the drug in Canada hasn't been worked out by the group representing provincial governments, the pan-Canadian Pharmaceutical Alliance.
Until then, patients can't access the treatment.
"For people with LCA that's a huge problem," says Dr. Robitaille. "Because their cells are dying off every month that is delayed."
In the United States, where the treatment was approved in 2017, the cost per patient was set at USD $850,000.
Fighting Blindness Canada, a non-profit group, is pushing provincial governments to work with the drug's supplier to set the Canadian price, so patients can start treatment as soon as possible.
The organization’s president and CEO says the cost of illness for inherited retinal diseases is $1.6 billion, according to IRD Counts Canada figures released in October 2020. That cost is most often borne by families.
Doug Earle says an economic analysis on Luxturna shows it's possible to reach a fair price for the treatment, given it's benefit.
"We've been calling on provincial governments to negotiate," says Earle.
"What's the price for sight? A few million dollars to treat these 42 Canadians that are on the waiting list like Nora, that are losing ten thousand photoreceptors a day," he adds.
When asked, the Province of Nova Scotia would only say Luxturna is "under consideration for negotiation" to reach what it called "an appropriate price."
As Nora shows off her latest artworks, her mom hopes for treatment soon, so the budding artist can keep doing what she loves.