Rotorua cystic fibrosis sufferer OJ Daniels holding a box of Trikafta. Photo / Andrew Warner
Trikafta is one step closer to being publicly funded after Pharmac received a clinical recommendation it should be funded for people aged six and older.
Pharmac has already re-ranked Trikafta on its Options for Investment list following updated advice from its Pharmacology and Therapeutics Advisory Committee.
However, the public funding of Trikafta is not a done deal just yet and Pharmac is still in negotiations with Vertex on the price.
Today, Pharmac published the latest record of advice from its Pharmacology and Therapeutics Advisory Committee and a summary of how Pharmac has assessed Trikafta.
In a Pharmac media statement, Pharmac director of operations Lisa Williams said Trikafta was a medicine it would like to fund and it had two proposals for it on its Options for Investment list - one for those aged 6 and over and one for those aged 12 and over.
"We now have recommendations from both our Respiratory Advisory Committee and from Pharmacology and Therapeutics Advisory Committee that the medicine should be funded for people six years and over," Williams said.
Trikafta was last considered by the Pharmacology and Therapeutics Advisory Committee in November 2021.
Since then, Pharmac had received more evidence from the supplier Vertex and information from consumer groups and healthcare practitioners.
In April 2022, the Respiratory Advisory Committee reviewed the evidence and information and reiterated its recommendation to fund Trikafta for people 6 years and over with "high priority".
In May 2022, the Pharmacology and Therapeutics Advisory Committee who had previously deferred making a recommendation for people 6 and older, had now recommended funding for them with medium priority following consideration of the new evidence and information.
Pharmac used recommendations from clinical experts, evidence from the supplier, and other interested people and groups to feed into its assessment and work to determine where the application was ranked against other medicines.
Today, Pharmac also released its Technology Assessment Report for Trikafta - a health economic analysis used to determine the cost-effectiveness of a medicine.
"Our assessment is that, if Trikafta were funded, there would be some savings to the health system through the reduction of the need for hospitalisations, lung transplants and supportive care," Williams said.
Pharmac also estimated Trikafta could give people with cystic fibrosis aged 6 and older a "longer and improved quality of life" - an equivalent of 27 more years at "full health" when compared to current funded treatments.
Williams said Pharmac assessed the health benefits to the person taking the medicine, the benefits to the healthcare system and how much the medicine cost.
"It is very important we hear and listen to the voices of people directly affected when assessing a medicine like Trikafta.
"Hearing from them, alongside clinical evidence, helps us to understand the impact funding Trikafta would have. As we've seen with the positive recommendation from the Pharmacology and Therapeutics Advisory Committee, it is encouraging and reinforces that it continues to be a medicine we want to fund."
The statement said Pharmac had already re-ranked Trikafta on its Options for Investment List having considered the updated advice from its clinical experts.
It is continuing commercial discussions with the supplier to try to reach an agreement that would enable the funding of Trikafta.
Williams said there were currently 118 proposals on its Options for Investment List for 75 different treatments.
"Pharmac operates on a fixed budget, so we need to make difficult decisions about how to spend any available funding."
Pharmac would use factors for consideration to rank and compare treatments on the list. The four factors for consideration are need, health benefits, costs and savings, and suitability. Each factor had three aspects - the individual, the family, whānau and society, and the health system.
"The Government announced a significant budget uplift to the pharmaceutical budget for this year and next, so we're doing our very best to fund as many items on the Options for Investment list that we can," Williams said.
Trikafta: Summary of assessment
Cystic fibrosis is a condition affecting around 500 New Zealanders. It arises when someone is born with two faulty genes which disturb the function of a specific protein, and causes the body to create abnormally thick mucus.
The mucus blocks the tubes of organs, including the lungs, pancreas, liver, intestine and reproductive system. The mucus stops organs working well and results in infection.
Someone with cystic fibrosis has an estimated life expectancy of 37 years - the average life expectancy for the general population is 82.
There is no cure for cystic fibrosis. Most people used oral medicines, inhalers, nebulisers and chest physiotherapy to help improve their quality of life.
Current treatments for most people with cystic fibrosis took up a lot of time with many healthcare appointments and sometimes hospital admissions.
This impacts people and their caregivers' ability to do day-to-day activities such as going to school and work. Supporting someone with cystic fibrosis could be a "difficult experience" and caregivers may need to change jobs or take time off work.
As the disease progresses, some people may need both lungs transplanted - a complex procedure which comes with risk.
Trikafta is a treatment for cystic fibrosis and works by helping the faulty protein make the mucus thinner. This stops the mucus blocking the body's organs.
Trikafta significantly improves lung function, patient weight, and reduces the likelihood of being admitted to hospital.
"We expect that these benefits would allow people with cystic fibrosis to live healthier, longer lives," the summary said.
Trikafta is a tablet that is taken twice a day, which would be much easier for people to take than supportive care. We expect it would also reduce the need for other medicines and therapies, the summary said.
Costs and savings:
If Trikafta were funded for people with cystic fibrosis aged 6 and over, Pharmac estimates there would be 332 eligible people in the first year of funding, increasing to 358 in the fifth year. Pharmac understands this would make a significant financial difference to people with cystic fibrosis, their whānau and their communities.
Trikafta has a market price of about $330,000 per person per year.
"We know that funding Trikafta would reduce the spend on other medicines and other health sector costs, such as hospitalisations and lung transplants," the summary said.