Cystic fibrosis (CF) is a progressive genetic condition that causes persistent lung infections and limits the ability to breathe over time. There is no cure and one in 25 New Zealanders carry the faulty gene that causes CF.
It took eight years for New Zealanders living with CF to access the first of four cutting-edge medications, called Kalydeco, from Vertex Pharmaceuticals. These medications effectively ‘switch off’ the genetic condition by temporarily correcting the effects of the faulty gene.
A combination of high price tags for the CF medicines, of which Vertex leads the market, and Pharmac’s conservative investment model, has meant that Kiwis with CF are disadvantaged with a very real and observable human cost.
“Cystic Fibrosis NZ campaigned for several years to secure funding for Kalydeco. Getting access to Kalydeco in Aotearoa was a huge victory, but New Zealand is still so far behind with access to precision treatments for CF,” says Chief Executive Jane Bollard.
Trikafta is the latest of these precision medications, also known as CFTR modulators, and received FDA approval in late 2019. It is available in the US, and in recent weeks England, Wales and Northern Ireland secured a deal with Vertex for Trikafta, in anticipation of its approval in Europe.
The breakthrough treatment has been widely heralded as having the potential to turn CF from a life-threatening condition, to a manageable condition and has the potential to work for up to 90% of people with CF.
“Cystic Fibrosis NZ continues to work with Vertex, Pharmac and other stakeholders to achieve funding for Trikafta in the most timely way possible,” Jane continues. “But as with the campaign for Kalydeco, getting Trikafta and other precision medicines is made infinitely more difficult due to the Pharmac funding model. The model means New Zealand ranks amongst the worst in the OECD for access to modern medicines, and this has to change.
“We are thrilled to hear that Sir Bob Elliott has paid for a member of the New Zealand CF community to privately receive Trikafta. For Bella and her family this is an incredible gift. But it only exemplifies the inequality of medicine funding in New Zealand, whereby individuals are forced to privately fund modern treatments,” says Jane. “We must improve access to modern medications for all New Zealanders who could benefit”.
Cystic Fibrosis NZ is working closely with Vertex who are developing a proactive strategy for drug access in New Zealand. We urge Vertex to submit an application to Pharmac for the public funding of Trikafta as soon as possible. Kiwis with CF can’t wait any longer.
Cystic Fibrosis NZ strongly encourages New Zealanders to help call on the Government to reform the Pharmac funding model by signing the “Reform Pharmac and Double the Pharmac Budget” petition, which currently sits at almost 28,000 signatures.
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Notes to editors
For interviews please contact:
Jane Bollard, Chief Executive, Cystic Fibrosis NZ - 021 283 0051
For further information or imagery, please contact:
Laura Huet| +64 021 195 5787 | laura@cfnz.org.nz
About Cystic Fibrosis NZ
Cystic Fibrosis NZ is the only charity dedicated to supporting and improving quality of life for people with CF and their families. Established in 1968 as a volunteer support group for parents with a newly diagnosed child, we dedicate ourselves to shaping a brighter future for everyone with CF. We do this through local family and individual support by our team of social workers, by covering the cost of essential medical equipment, providing organ transplant assistance, information packs, welfare assistance and many other means of support. We also fund CF research and advocate on behalf of the community for better access to services and care.
About cystic fibrosis
Cystic fibrosis (CF) is a serious illness that affects lungs and digestion. It is the most common life-threatening genetic disorder affecting Kiwis. It is caused by a faulty gene that has been passed down from a baby's mum and dad and is usually diagnosed soon after birth. One in 25 people carry the faulty gene that causes it, often without even knowing.
There are over 540 Kiwis with CF, and it’s usually diagnosed through the heel prick test as part of new-born screening. CF affects everyone differently, but for many it involves a rigorous daily treatment regime including chest physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. CF can cause diabetes, asthma, liver disease, and permanently reduced lung function which, in many cases, requires a transplant
People with CF secrete thick, sticky mucus in their bodies. The mucus in their lungs traps bacteria which can results in chronic infections, meaning that people with CF struggle with reduced lung function, progressive lung damage and possible respiratory failure. In the digestive system CF reduces the amount of insulin produced and stops the digestive enzymes that aid digestion, leading to poor growth and physical weakness.
One of the most striking features of CF is cross infection. Two people with the condition should never be in close contact as their lungs harbour specific bacteria that are a risk to someone else with the condition. This means no group support and the added stress of having to physically avoid others with CF.
For further information please visit: www.cfnz.org.nz
