Girl with cystic fibrosis frustrated by government inaction to lower cost of life-saving drug

A 12-year-old girl with a rare disease scolded the Ontario government on Tuesday for the slow pace of its efforts to help lower the price for a life-saving drug she relies on that costs $349,000 a year.

“I am very frustrated,” said Madi Vanstone from Beeton, Ont. “I don’t know what to say to make them listen.”

Madi relies on Kalydeco to treat a rare form of cystic fibrosis, but is running out of money raised by friends and family and may soon lose insurance coverage that has been helping pay for the costly drug.

Premier Kathleen Wynne promised Madi during a meeting last month that she would expedite a joint effort by the provinces to deal with the American manufacturer of Kalydeco to try and lower the price so it can be covered by medicare.

Alberta, which is leading the talks on Kalydeco, made three proposals to the drug maker Vertex, but the company rejected each one.

Madi returned to the Ontario legislature Tuesday to say things are taking way too long and little is actually being done.

“They’re not moving forward, and we can’t wait,” she said. “There’s no time, and they’re not doing anything.”

Progressive Conservative house leader Jim Wilson made an impassioned plea in the legislature to get Madi and others who need Kalydeco assurances that it will eventually be covered by provincial health insurance plans.

“The money Madi’s family and friends have fundraised is quickly depleting,” said Wilson. “Right now, her dad’s insurance company that covers half of the cost of the drug is reassessing his claim. Premier, when will you fund the drug Kalydeco?”

Wynne told the legislature that Ontario cannot act alone and must work with the other provinces to try and save money on expensive new pharmaceuticals.

“When I met with my colleagues from across the country a few weeks ago, we made it clear that this is not an issue just for Madi, that there are other children and other people in other parts of the country who need this drug to be covered,” said Wynne. “We need that deal. We are pushing very hard.”

There are only about 118 Canadians who would be eligible for Kalydeco if it were covered by provincial health insurance plans.

Madi’s mother, Beth Vanstone, said she too was frustrated it took Alberta’s health minister almost a month just to send a letter on the issue, and she’s worried patients like her daughter could die before the provinces take any real action.

“If it takes them three weeks to get a letter out, I don’t know how many people are going to be left waiting for this drug at the end of this,” said Vanstone. “They’re telling us they’re going to expedite things, but they’re not.”

Insurance coverage from the company where Madi’s father works provides 50 per cent of the cost of Kalydeco, but is under review and could end in June. The manufacturer funds another 30 per cent of the cost, but only if there is insurance coverage.

“We would lose all funding at that point, which means we would have maybe enough in the account we raised so far for a month of drugs, and then we’d be watching her health go back to where it was,” said Vanstone. “It’s heartbreaking.”

Madi told reporters Kalydeco made a huge difference in her life.

“It’s helped everything,” she said. “I used to be tired and I’m not anymore. I used to get headaches and stomach aches caused by the mucus and everything and that’s gone. I have so much more energy, and I love to go running.”