Pharmac has today published the record of advice from the May Pharmacology and Therapeutics Committee (PTAC) meeting to again consider Vertex’s application for funding of Trikafta – a medicine to treat the underlying cause of cystic fibrosis (CF).

Pharmac has also published its Technology Assessment Report (TAR) setting out its economic health analysis of Trikafta, and a summary of its assessment of the application for Trikafta including Factors for Consideration. The TAR assesses the cost-effectiveness of a medicine, while Pharmac’s Factors for Consideration are used as part of assessing and prioritising medicines for funding.

Cystic Fibrosis NZ Chief Executive Lisa Burns says, “While it is disappointing that PTAC has again disregarded the advice of Pharmac’s respiratory experts and recommended only a medium priority for Trikafta, PTAC has now recommended funding for those all those 6 years and over who would benefit. We are pleased to see that Pharmac has again made clear that it wants to fund Trikafta, in the light of all the evidence it has received.”

Pharmac has acknowledged the major benefits of Trikafta, including not only savings to the health system but also substantial increases in life expectancy and quality of life. They estimate that Trikafta could give people with CF who are 6 years and over a longer and improved quality of life, equivalent to 27 more years at full health when compared to current funded treatments.

“A projected increase in life expectancy is significant, given that only half of those with CF in New Zealand currently reach 31 years of age this would almost double their life.” said Lisa.

Independent work commissioned by CFNZ for 2019/20, and shared with Pharmac, calculated the approximate annual social, economic, and financial cost of CF to be $116m.

In contrast, the estimated investment of less than $60m in Trikafta would provide significant economic and financial benefits, keeping people out of hospital, lowering the number of infections requiring intervention, reducing lung transplants, improving quality of life, and a notable reduction in burden on New Zealand’s healthcare system which is already under pressure particularly with respiratory illnesses.

Pharmac has also acknowledged the impact and shortcomings of current treatment, with treatments taking up a lot of time and impacting on the day- to- day activities of those with CF and their caregivers, with the long-term positive impact of current treatment being limited. Pharmac also acknowledged that CF has a big impact on whānau and the community, including on their mental health, something the CF community is sadly very aware of.

“There has now been enough assessment, reassessment, and review – it is time for action and decisions. We know Trikafta is a worthwhile investment, we know it works. There is so much evidence from overseas and from Kiwis whose lives have been completely changed by having access to Trikafta,” says Lisa.

CFNZ are aware of several families who are now medical refugees. With no other options left, they have been pushed to leave New Zealand, making the difficult decision to uproot their lives, leaving family, friends, and careers to keep their loved ones alive. Those who have been able to access CF modulators overseas are now unable to return to NZ because they are not funded here.

It is now a year since Pharmac’s respiratory experts originally recommended a high priority for funding Trikafta for all those aged 6 years and over. These experts reconfirmed this recommendation in April this year. Pharmac says that it has already reranked Trikafta in the light of this information and is continuing commercial discussion with Vertex.

“We urge Pharmac and Vertex to move quickly to agree a fair and reasonable price for Trikafta for all those aged 6 years and over who would benefit – and give people with CF those years of full health to look forward to.”

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