5 Nov

Precision medications still out of reach for New Zealanders with cystic fibrosis

5 November 2019

Over the past few weeks, international cystic fibrosis organisations have celebrated more milestones in their mission to improve the lives of people with cystic fibrosis (CF). At the same time, New Zealanders with CF have become even more disadvantaged.

On 21 October, the US Food and Drug Administration (FDA) approved the use of the triple combination CFTR modulator Trikafta (elexacaftor/ivacaftor/tezacaftor) for people with cystic fibrosis aged 12 and over who have at least one F508del mutation in the CFTR gene.

Clinical trials of Trikafta have shown dramatic improvements in key measures of CF, and in time more than 90% of people with CF could benefit from this highly effective therapy.

Australia also announced increased access to Orkambi (ivacaftor/lumacaftor) and access to Symdeko (tezacaftor/ivacaftor) for people with CF aged 12 and older who have two copies of the F508del mutation or who have one of 26 specified mutations, even if they don’t have a copy of the F508del mutation.

And also last month, the National Health Service (NHS) England announced a two-year managed access agreement with Vertex for Orkambi and Symdeko, with agreement that Trikafta would be submitted to the National Institute of Health Care Excellence (NICE) for appraisal. This follows the announcement of a similar five-year deal in Scotland.

While Cystic Fibrosis NZ is delighted by the success overseas, it once again highlights just how far behind New Zealand is with securing these vital medications for people with CF in New Zealand.

“Our CF community continues to be left behind, with no access to these life changing medicines, whose impact on improving the quality of life of people with CF has been proved time and again,” Jane says.

“I have written to Vertex to congratulate them on their success and to urge them to engage with PHARMAC and we will continue to put pressure on the Government to ensure our voice is heard. New Zealand cannot continue to be left behind,” she says.

“I encourage everyone to sign the two petitions below, calling for Kalydeco to be publicly funded and to double the PHARMAC budget. Please share with your networks to show the Government how important access to these life-saving medications is for us.”

Sign the petitions

Publicly fund Kalydeco medication for New Zealanders with G551D cystic fibrosis petition – closes on 20 November 2019

Reform PHARMAC and double the PHARMAC budget petition – closes on 26 March 2020