Years of lobbying appear to have paid off in the fight for funding of a revolutionary drug to treat cystic fibrosis.
Pharmac has today announced it’s reached a provisional agreement with drug company Vertex to fund ivacaftor, better
known as Kalydeco. If feedback from 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.
“This is a monumental day for the CF community and marks the culmination of many years of lobbying for access to
innovative medicines,” says Cystic Fibrosis NZ Chief Executive Jane Bollard.
“I want to thank everyone who has been a part of this campaign for their persistence and determination to keep on
fighting” says Bollard. “A special thanks to Eddie and Emma Porter of the ‘Kalydeco for Kiwis’ advocacy group for their
work on the campaign”.
The Porters are parents to Otis, aged two and a half, who was born with the inherited, life-threatening lung disorder
that effects 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. Pharmac agreed in principal to fund Kalydeco last February, but only as a low priority,
meaning the chances of it ever being funded were slim and unclear. This agreement now gives patients far more certainty.
“This is so exciting for the future of not only our own little boy but dozens of other CF patients in New Zealand with
the G551D mutation who are in desperate need of Kalydeco” says Eddie Porter. “This will completely change Otis’ future
and give him a life expectancy well beyond the current CF average of 37 years. 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”.
.Around 530 New Zealanders live with CF. 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.
Hailed as a ‘miracle drug’, Kalydeco is the first medication to fix the underlying cystic fibrosis defect, as opposed to
current medicines which only treat the symptoms. It drastically improves life expectancy and reduces life-threatening
symptoms. 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.
“While we are hugely excited Kalydeco has been funded, we look forward to this being the first of many more modern
drugs Pharmac considers funding to treat many more of the 530 New Zealanders living with the disease, such as Orkambi,
Symdeko and Trikafta” says Jane Bollard. “The fight for these patients continues”.