Trikafta Technology Assessment Report (TAR)

Introduction

The pharmaceutical supplier, Vertex, submitted an application to Te Pātaka Whaioranga – Pharmac to fund a medicine which combines three chemicals: elexacaftor, tezacaftor and ivacaftor (brand name Trikafta) for cystic fibrosis in July 2021. The application asked us to fund Trikafta for people with cystic fibrosis who are aged 6 years and older and who have a specific gene mutation. 

This is a summary of our assessment of the application. It includes evidence from the supplier, clinical advice, and what we have heard from people with cystic fibrosis, their whānau and communities.

We use our Factors for Consideration when considering every application, using the factors New Zealanders have told us are important to consider. The following headings relate to the Factors for Consideration.

Health need

Cystic fibrosis is a condition that affects around 500 New Zealanders. It arises when someone is born with two faulty genes, which disturb the function of a specific protein, causing the body to create an abnormally thick mucus. The thick mucus blocks the tiny 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. This is much younger than the average life expectancy for the general population, which is 82 years. People living with cystic fibrosis have shorter lives because of it and also due to other conditions it can cause. These include diabetes and diseases of the liver and bone. 

Cystic fibrosis is a chronic condition and affects children – both of which are Government health priorities.

Detailed analysis on health need [PDF, 951 KB]

The availability and suitability of existing treatments

There is no cure for cystic fibrosis. Most people with cystic fibrosis use a range of therapies that aim to improve their quality of life by preventing and managing the adverse effects of physical and psychological symptoms (known as supportive care). This can include oral medicines, inhalers, nebulisers, and chest physiotherapy. The long-term positive impact of supportive care is limited. 

The current treatments for most people with cystic fibrosis take 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. As the disease progresses, some people may need both lungs transplanted. This is a complex procedure, which comes with risk. It is only considered for people who have severe lung disease. 

Pharmac funds a medicine called ivacaftor (brand name Kalydeco) which also targets a specific gene mutation that causes cystic fibrosis. The gene mutation that Kalydeco targets is different to Trikafta, so it is only appropriate for supporting 10% of people with the condition. 

Health need of whānau, and wider society

Cystic fibrosis has a big impact on whānau and the wider community. Supporting someone with cystic fibrosis can be a difficult experience and caregivers may need to change jobs or take time off work to support someone with cystic fibrosis. These things can impact caregivers’ mental health and wellbeing. 

Impact for Māori and other populations experiencing health disparities

Romaha ora, respiratory health, is one of Pharmac’s Hauora Arotahi, Māori health focus areas. There is limited published data about the health outcomes for Māori with cystic fibrosis, but we know that Māori experience worse health outcomes than non-Māori for many respiratory illnesses. Our clinical advisors have told us this is likely to be the case for Māori with cystic fibrosis. 

Our advisors have also said that Pacific peoples with cystic fibrosis are likely to experience worse health outcomes than non-Pacific peoples. 

Other factors, like socioeconomic status, can also worsen the health outcomes for people with cystic fibrosis.

Health benefit

Trikafta is a treatment for cystic fibrosis. It works by helping the faulty protein, which makes the mucus thinner. This stops the mucus blocking the body’s organs. 

Trikafta significantly improves lung function, patient weight, and other symptoms of cystic fibrosis, 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. 

Detailed analysis of the health benefit [PDF, 951 KB]

Health benefits to the person and others

We estimate that Trikafta could give people with cystic fibrosis (aged 6 and over) benefits equivalent to 27 more years at full health, when compared to supportive care. Full health represents both a longer life and an increased quality of life for people with cystic fibrosis, such as an improved ability to go to school or work and take part in other activities. 

We also expect that Trikafta would significantly reduce the impact of cystic fibrosis on whānau and communities, improving their quality of life. 

Consequences for the health system

In our assessments, we consider the benefits and costs for the wider health system. Trikafta would benefit the health system through a reduction in:

• supportive care
• hospitalisations
• lung transplants. 

Clinical advice

Pharmac’s Respiratory Advisory Committee and Pharmacology and Therapeutics Advisory Committee (PTAC) have reviewed the funding application for Trikafta. They considered:

• evidence and information from the supplier
• communication from Cystic Fibrosis New Zealand
• advice from members of Pharmac’s former Cystic Fibrosis Panel. 

The Respiratory Advisory Committee recommended that Trikafta be funded with a high priority for eligible people with cystic fibrosis aged 6 years and older. PTAC recommended that Trikafta be funded with a medium priority for eligible people aged 6 years and older or for people 12 years and older. The relevant eligibility criteria are outlined in committee meeting records. 

Respiratory Advisory Committee record April 2022 [PDF, 545 KB]

PTAC record May 2022 [PDF, 832 KB]

Suitability

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. 

Costs and savings

If Trikafta were funded for people with cystic fibrosis aged 6 and over, we estimate there would be 332 eligible people in the first year of funding, increasing to 358 in the fifth year. We understand this would make a significant financial difference to people with cystic fibrosis, their whānau and their communities. 

Unfortunately, Trikafta is very expensive medicine with a market price reported in the media of approximately $330,000 per person per year. Vertex is the only company who can make and sell the medicine as it is patented so there is no competition to drive the price down. 

To support the pricing negotiations for Trikafta we cannot disclose the confidential offer that Vertex has made. As with all other countries, Vertex wants confidentiality to ensure pricing in one country does not influence pricing negotiations in another. 

We know that funding Trikafta would reduce the spend on other medicines and other health sector costs, such as hospitalisations and lung transplants. 

Detailed analysis of the costs and savings [PDF, 951 KB]

When assessing a new medicine, we want to know how much better it works, and how much more it costs, compared to other medicines or treatments that are publicly available. We use a Cost Utility Analysis (CUA) to tell us how many Quality-Adjusted Life Years (QALYs) are gained for the money spent. While cost utility analysis is not one of the Factors for Consideration, it is a method of measuring or quantifying several of the Factors – namely some of the Factors in the Health benefits and Costs and savings areas. 

We are unable to release the results of our cost utility analysis because it uses confidential pricing offers made by Vertex. It provided us with its own assessment of the cost utility of Trikafta. We then made changes to the assessment based on the clinical advice we received. We have now shared our latest assessment with Vertex for it to consider in future proposals. 

We have considered cost utility assessments from other countries. However, the pricing used in those assessments may be different to the confidential pricing offered to Pharmac.

More information on cost utility analysis and quality-adjusted life years [PDF, 3.4 MB]

Next steps

Trikafta is a medicine we would like to fund. It is on our Options for Investment list for both age groups (over 6-year-olds and over 12-year-olds). We are continuing to talk with Vertex, including commercial negotiations on the price.

About the priority lists