Cystic fibrosis: Pharmac receives updated guidance for financing Trikafta

Rotorua Cystic Fibrosis sufferer OJ Daniels holding a box of Trikafta. Photo/Andrew Warner

Trikafta is set to be publicly funded after Pharmac received a clinical recommendation that it should be funded for people aged six and over.

Pharmac has already reclassified Trikafta to its list of investment options following updated advice from its Pharmacology and Therapeutics Advisory Board.

However, public funding for Trikafta is yet to be secured and Pharmac is still in negotiations with Vertex over the price.

Today, Pharmac released the latest minutes of advice from its Pharmacology and Therapeutics Advisory Committee and a summary of how Pharmac assessed Trikafta.

In a statement to Pharmac media, Pharmac COO Lisa Williams said that Trikafta was one drug she would like to fund and that she had two proposals for it on her list of investment options – one for those aged 6 and over and one for those aged 12 and over. more.

“We now have recommendations from our Respiratory Advisory Committee and the Pharmacology and Therapeutics Advisory Committee that the drug should be funded for people six years of age and older,” Williams said.

Trikafta was last reviewed by the Pharmacology and Therapeutics Advisory Committee in November 2021.

Since then, Pharmac had received more evidence from supplier Vertex and information from consumer groups and healthcare professionals.

In April 2022, the Respiratory Advisory Committee reviewed the evidence and information and reiterated its recommendation to fund Trikafta for people 6 years and older with “high priority”.

In May 2022, the Pharmacology and Therapeutics Advisory Committee, which had previously deferred making a recommendation for those 6 years of age and older, had now recommended funding for them with medium priority after review of new evidence and information.

Pharmac used recommendations from clinical experts, evidence from the supplier, and other interested individuals and groups to inform its assessment and work to determine where the application ranked relative to other drugs.

Today, Pharmac also released its Technology Assessment Report for Trikafta – a health economic analysis used to determine the cost-effectiveness of a drug.

“Our assessment is that, if Trikafta were funded, there would be savings to the healthcare system through the reduction in the need for hospitalizations, lung transplants and supportive care,” Williams said.

Pharmac also estimated that Trikafta could give people with cystic fibrosis aged 6 years and older a “longer and improved quality of life” – the equivalent of 27 more years in “full health” compared to currently funded treatments.

Williams said Pharmac weighed the health benefits to the person taking the drug, the benefits to the healthcare system, and the cost of the drug.

“It is very important that we hear and listen to the voices of those directly affected when evaluating a drug like Trikafta.

“Hearing them talk, alongside the clinical evidence, helps us understand the impact funding Trikafta would have. As we have 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 has already reclassified Trikafta to its list of investment options after considering updated advice from its clinical experts.

It is continuing commercial discussions with the supplier to try to find an agreement that would allow Trikafta to be financed.

Williams said there are currently 118 proposals on her list of investment options for 75 different treatments.

“Pharmac operates on a fixed budget, so we have to make tough decisions about how to spend any available funding.”

Pharmac would use factors to consider to rank and compare the treatments on the list. The four factors to consider are need, health benefits, cost and savings, and appropriateness. Each factor had three aspects – the individual, the family, the whānau and society, and the health system.

“The government has announced a significant increase in the pharmaceutical budget for this year and next, so we are doing our best to fund as many items on the investment options list as possible,” Williams said.

Trikafta: Evaluation Summary

Health need:

Cystic fibrosis is a disease that affects approximately 500 New Zealanders. It occurs when a person is born with two faulty genes that disrupt the function of a specific protein and cause the body to create abnormally thick mucus.

Mucus blocks the tubes of organs, including the lungs, pancreas, liver, intestine, and reproductive system. The mucus prevents the organs from working properly and causes an infection.

A person with cystic fibrosis has an estimated life expectancy of 37 years – the average life expectancy for the general population is 82 years.

There is no cure for cystic fibrosis. Most people used oral medications, inhalers, nebulizers, and chest physiotherapy to improve their quality of life.

Current treatments for most people with cystic fibrosis were time consuming with many medical appointments and sometimes hospitalizations.

This impacts the ability of people and their caregivers to perform daily activities like going to school and work. Supporting someone with cystic fibrosis can be a ‘difficult experience’ and carers may need to change jobs or take time off work.

As the disease progresses, some people may need a transplant of both lungs – a complex procedure that carries risks.

Health benefit:
Trikafta is a treatment for cystic fibrosis and works by helping the faulty protein to thin the mucus. This prevents mucus from blocking body organs.

Trikafta significantly improves lung function, patient weight and reduces the likelihood of being admitted to hospital.

“We expect these benefits to lead people with cystic fibrosis to live longer, healthier lives,” the abstract reads.

Relevance:
Trikafta is a twice-daily pill, which would be much easier to take than supportive care. We expect this will also reduce the need for other drugs and therapies, the summary states.

Costs and Savings:
If Trikafta were funded for people with cystic fibrosis aged 6 and over, Pharmac estimates there would be 332 people eligible in the first year of funding, rising 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 around $330,000 per person per year.

“We know that funding Trikafta would reduce spending on other drugs and other health sector costs, such as hospitalizations and lung transplants,” the summary states.

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