Pharmac has reached a provisional agreement with medicine supplier Vertex to fund the long-campaigned for drug Kalydeco for New Zealanders with cystic fibrosis.
The announcement was made today and - if feedback from a a consultation process with health professionals is supportive, and the proposal approved - patients could get access to the drug from as early as March 1st.
Kalydeco is the first medication that fixes the underlying cystic fibrosis defect, essentially turning off cystic fibrosis for patients with the G551D mutation. While current medications only treat the symptoms, Kalydeco has been hailed as a breakthrough in cystic fibrosis research.
Until now, New Zealand has been the only OECD country to not fund Kalydeco, with many patients considering a shift to Australia to gain access to it.
Health Minister Dr David Clark says he is delighted Pharmac has made the step.
“Pharmac’s funding decision on this medicine was based on strong new evidence of its effectiveness. I also understand the Government’s $60m boost to Pharmac’s funding last year on top of the $40m increase in Budget 2019 was also a significant factor in securing this deal with Kalydeco’s supplier," he says.
“I’m particularly happy that Pharmac is taking steps to ensure this medicine is available to children under six."
Cystic Fibrosis NZ chief executive, Jane Bollard says it's a "monumental day for the cystic fibrosis community".
She said it "marks the culmination of many years of lobbying for access to innovative medicines”.
“I want to thank everyone who has been a part of this campaign for their persistence and determination to keep on fighting,” Ms Bollard says.
The announcement is welcome news for the Porter family.
Sixteen-month-old Otis Porter's parents are pleading for Kalydeco to be funded, to help the toddler live.
Eddie and Emma Porter are parents to Otis, aged two and a half, who was born with the inherited, life-threatening lung disorder that affects the lungs and digestive system.
The couple established the ‘Kalydeco for Kiwis’ campaign in 2018, a year after Otis’ birth, culminating in the delivery of an 11,000-strong petition to Parliament last November calling for the drug to be funded immediately.
“This will completely change Otis’ future and give him a life expectancy well beyond the current cystic fibrosis average of 37 years," says Mr Porter.
"He now realistically faces less sickness and hospitalisation from debilitating lung infections in the future and the chance of living a far more normal life, like other Kiwi boys his age."
Julian and Camilla Cox are inspired by their 19-year-old daughter, who has cystic fibrosis.
Earlier this month, parents of another cystic fibroisis patient - Dunedin couple Julian and Camilla Cox - set off on a tandem bike ride to Parliament with a coffin and petition in tow. They called on the Goverrnment to double Pharmac's current drug-buying budget on behalf of their 19-year-old daughter.
Around 530 New Zealanders live with the condition.
If given the final green light, Kalydeco will treat an estimated 30 New Zealand adults and children living with the G551D mutation of the disease.
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