23 January 2025
Cystic Fibrosis NZ (CFNZ) welcomes Pharmac’s Respiratory Advisory Committee’s recommendation to fund Trikafta with high priority for children aged two to five years with responsive genetic mutations. This marks a further important milestone in the treatment of cystic fibrosis (CF) in Aotearoa.
Trikafta, a triple-combination therapy, has been shown to significantly improve lung function, reduce hospitalisations, and enhance quality of life for people with CF. The Committee recognised that the disease trajectory for CF is generally set before the age of six and that there is no biological or evidence-based reason to delay treatment until six years of age.
CFNZ Chief Executive Lisa Burns says, “This recommendation offers hope to families and represents the next vital step in ensuring all New Zealanders with CF have fair and equitable access to a life-changing medicine that works for them. This recommendation provides the opportunity to further change the landscape of CF care in New Zealand, and align our treatment options to the more than 20 countries where Trikafta has been available internationally for this age group for more than 18 months.
“We’ve witnessed the profound impact of Trikafta on those aged six and older. There has been a notable drop in hospitalisations seen through our CFNZ Social Worker inpatient visits, which have reduced by over 50% since April 2023. Alongside this there is a drastic reduction in lung transplants from only a few years ago when 22% of all lung transplants were due to CF. Now, our youngest and most vulnerable Kiwis with CF are on the path heading towards the chance to access this medicine at a critical stage of their development,” says Lisa.
The Committee noted that funding Trikafta from two years of age would impact Pharmac’s budget, but it would also expect to reduce the need to manage pulmonary exacerbations, maintenance therapy such as physiotherapy, and treatments for pancreatic insufficiency, and inadequate growth. Investing in medicines such as Trikafta that help prevent long term damage and reduce the burden of ill-health on individuals and society are a core part of this Government’s reset for Pharmac.
CFNZ recognises the persistence of families, clinicians, and advocates who have tirelessly continued to campaign for improved access to medicines in New Zealand including for cystic fibrosis. However, the stresses in delays to funding these life-saving treatments that are a standard of care internationally will continue to impact the health of all Kiwis including those with CF, who cannot access the treatments they need. The Committee acknowledged that parents of young children experience anxiety around exacerbations and preserving lung function until their children turn six.
For families like Sophie’s, this funding recommendation is the news they have been wanting to hear. “Sophie was born at just 33 weeks and had life-saving surgery the day after she was born. She’s already been through more than most do in a lifetime. Right now, we have to be extra careful because she doesn’t have access to Trikafta, but we dream of a future where she can run, play, and experience everything her heart desires. Every parent wants their child to outlive them—this medicine gives Sophie the best chance to have a long, happy, and healthy life,” says her family.
“Having Trikafta from 2 years old (August 2025 for Sophie) would be an absolute game changer. We had a rough year last year, lots of poor health and ED visits, and have felt almost forced to look at moving to other countries to give Sophie the best possible chance to live a long and healthy life. This would be such a huge relief to us and our family.”
Lisa says that while we can celebrate today’s progress, we must collectively continue to push for more timely and equitable access to medicines in New Zealand. “Recognising where there are significant positive outcomes through investment in modern medicines. No one should have to fight for years to receive treatments that are standard of care overseas,” she says.
The key therapeutic intent of Trikafta is to improve overall survival and quality of life by stopping disease progression. The evidence is clear—this treatment leads to improved lung function, fewer pulmonary exacerbations, and a reduced need for hospitalisation and intensive therapies like antibiotics, chest physiotherapy, and lung transplants. It also enhances nutrition, weight management, and overall health-related quality of life. The Respiratory Advisory Committee made it clear that the best way to prevent deterioration and long-term decline is to intervene early – at two years of age.”
CFNZ strongly urges Pharmac to work towards swift implementation of this funding recommendation, allowing young children with CF to start treatment without unnecessary delays.
“The earlier children receive Trikafta, the better their long-term health outcomes. We remain committed to advocating for all people with CF, ensuring they have access to the best treatments that support their quality of life,” says Lisa.
Read the Respiratory Advisory Committee Record here >
