Christine Perrins: Give Kiwis with cystic fibrosis a future to look forward to

Volunteers such as Yvonne Rooney have rattled buckets for years to help those with cystic fibrosis. Campaigners believe Pharmac can fund a breakthrough treatment. Photo / George Novak, File

Opinion

OPINION

In the coming weeks, Pharmac will confirm how highly it will rank Trikafta, a life-changing medicine for cystic fibrosis, against the other medicines it wants to fund and whether it will go on to fund it.

For many of the 540+ people with cystic fibrosis in New Zealand, these decisions will be the most important Pharmac has ever made. For them, it will be the difference between living an almost normal life and having hope for the future or struggling with declining health and facing the prospect of premature death.

Cystic fibrosis – CF for short - is an inherited genetic condition that makes the body produce thick, sticky mucus that causes serious clinical consequences for the lungs, pancreas, liver, intestines, reproductive system, and other organs.

People with CF endure a life-long, demanding, and time-consuming daily regime of lung clearance and medication, medical check-ups, frequent hospitalisations and, for some, lung transplantation. The daily treatment burden for CF is high – up to three to four hours per day - and increases with age and severity of the condition.

Having CF shortens your life significantly. Only half of those with CF reach 31 years of age, compared with 80 years for New Zealanders as a whole.

CF also has a major negative impact on the emotional and mental well-being of those with the condition, their families and whānau, due to the stress and anxiety of living with a serious illness and facing premature death. This burden is compounded for the 50+ families in New Zealand with more than one person with CF.

Current treatments for most Kiwis with CF treat only their symptoms, aiming to slow decline and maintain quality of life. But, for the first time, there is a medicine that treats the underlying cause of CF for around 90 per cent of those with the condition - Vertex's triple combination therapy, Trikafta.

Pharmac and its experts have assessed Trikafta and concluded that it's a paradigm-shifting treatment, providing people with CF with substantial improvements in quality of life and life expectancy, and allowing them to live almost normal lives.

These benefits have been clearly demonstrated by the experience of New Zealanders who have been able to access Trikafta and seen life-changing improvements in their health, well-being, and ability to fully participate in life.

I've seen the effects first-hand. Our 26-year-old daughter was enrolled in Vertex's Managed Access Programme earlier this year.

Christine Perrins. Photo / Supplied

Before Trikafta, she was spending hours each day doing treatments, struggling to maintain weight, experiencing frequent bouts of uncontrollable coughing, unable to walk very far without becoming breathless and fatigued, and facing the prospect of a lung transplant.

The benefits were immediate: within a week her cough had almost disappeared, and she had started gaining weight; within four weeks she could walk 5km without fatigue; and three months on she's playing tennis again. And on just three pills a day.

The mental and emotional burden of CF has been lifted from her and our whole family. For the first time in 26 years, we aren't living in fear of what's going to happen next.

Trikafta would also benefit New Zealand's health system. Pharmac's experts concluded Trikafta could reduce lung transplants and current treatment by 80 per cent over time, as well as substantially reduce hospitalisations for lung infections, and the use of antibiotics and other medicines.

Recently, Pharmac's experts gave Trikafta a high priority recommendation.

Following this, Pharmac completed its economic assessment and confirmed that it wants to fund Trikafta.

Pharmac has confirmed what the CF community has known all along – there is an overwhelming case for funding Trikafta.

Investing in Trikafta would benefit nearly 400 eligible Kiwis with CF by enabling them to live an almost normal life. It would also deliver substantial benefits to the NZ health system. And what better time to fund a medicine that substantially reduces the burden on hospital respiratory services than in the midst of a Covid-19 pandemic?

Pharmac has made much of how expensive Trikafta is. Cystic Fibrosis NZ acknowledges the difficult decisions Pharmac has to make when deciding which medicines to fund from its limited budget, however price needs to be put into perspective.

Pharmac prides itself on achieving some of the best commercial prices for pharmaceuticals in the world and we know that it will not pay the full retail price for Trikafta.

Vertex has reached agreements to supply Trikafta in 22 other countries and already has an agreement with Pharmac to supply Kalydeco, another CF medicine - clear evidence that they can reach a commercial agreement if they want to.

So, Pharmac and Vertex, please progress your commercial negotiations as quickly as possible to agree a fair and reasonable price for Trikafta – and make sure that Kiwis with CF have a future to look forward to.

• Retiring in late 2019 after a long career in transport, Christine Perrins began volunteering as the advocacy adviser for Cystic Fibrosis NZ in early 2020. Christine leads Cystic Fibrosis NZ's campaign to have Trikafta funded in New Zealand. She has a daughter with cystic fibrosis.