Proposal to fund elexacaftor with tezacaftor and ivacaftor (Trikafta) for people with cystic fibrosis
What we’re proposing
We are proposing to fund elexacaftor with tezacaftor and ivacaftor (brand name Trikafta) for people aged 6 years and above with cystic fibrosis subject to eligibility criteria.
Consultation closes at 5 pm, Monday 16 January 2023 and feedback can be emailed to consult@pharmac.govt.nz
What would the effect be?
From 1 April 2023, people with cystic fibrosis would have funded access to Trikafta if they:
- are aged 6 years and above, and
- have specific mutations, and
- meet certain clinical criteria.
About cystic fibrosis – Health Need section of the Trikafta Summary of Assessment
Our expert advice indicates that treatment with Trikafta significantly improves lung function and other symptoms of cystic fibrosis. We estimate that Trikafta could give people with cystic fibrosis up to 27 more years at full health, compared to supportive care. This would significantly reduce the impact of the condition on people with cystic fibrosis, their whānau, and communities.
Our assessment of the benefits of Trikafta – Health Benefit section of the Summary of Assessment
We estimate there are more than 360 people in New Zealand who would be eligible for funded treatment under the proposed criteria.
Romaha ora, respiratory health, is one of Pharmac’s Hauora Arotahi, Māori health focus areas. Our clinical advisors have told us it is likely that Māori experience worse health outcomes than non-Māori for cystic fibrosis.
Individuals would be able to access Trikafta through their prescriber. Prescriptions would be sent to and collected from Te Whatu Ora hospital pharmacies.
Who we think will be interested
- People with cystic fibrosis, their whānau, caregivers, and communities
- People interested in the funding of medicines for cystic fibrosis and rare disorders
- Respiratory physicians, paediatricians, respiratory nurses, and other clinicians and health professionals involved in the management of cystic fibrosis
- Advocacy and support groups for people with cystic fibrosis
- Te Whatu Ora Hospitals
- Pharmaceutical suppliers and wholesalers
About cystic fibrosis
Cystic fibrosis affects around 500 New Zealanders of all ages. The condition occurs when someone is born with two faulty genes, which causes the body to make abnormally thick mucus. The mucus stops organs working well and results in infection. People living with cystic fibrosis have shorter lives because of the condition. Cystic fibrosis can also cause other conditions, including diabetes and diseases of the liver and bone.
Cystic fibrosis is a chronic condition and it affects children – both of which are Government health priorities.
There is no cure for cystic fibrosis. The current treatments for most people with cystic fibrosis (known as supportive care) 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.
About Trikafta
Trikafta is an oral medicine in tablet form taken twice a day. It supports the faulty protein to make thinner mucus. This stops the mucus blocking the body’s organs. Trikafta significantly improves lung function, patient weight, and other symptoms of cystic fibrosis, reducing the likelihood of people being admitted to hospital. We expect that these benefits would allow people with cystic fibrosis to live healthier, longer lives.
Trikafta is available in two fixed dose combinations:
- for people over 30 kilograms: two 100 mg elexacaftor with 50 mg tezacaftor and 75 mg ivacaftor tablets in the morning and one 150 mg ivacaftor tablet in the evening; and
- for people under 30 kilograms: two 50 mg elexacaftor with 25 mg tezacaftor and 37.5 mg ivacaftor tablets in the morning and one 75 mg ivacaftor tablet in the evening.
Trikafta is Medsafe approved for people aged 6 years and older in New Zealand.
Medsafe datasheet for Trikafta(external link)
Why we’re proposing this
The pharmaceutical supplier, Vertex, submitted an application to Te Pātaka Whaioranga – Pharmac 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.
The Respiratory Advisory Committee and the Pharmacology and Therapeutics Advisory Committee have recommended funding Trikafta for people with cystic fibrosis who are aged 6 years and older, subject to certain criteria. They considered:
- evidence and information from the supplier
- communication from Cystic Fibrosis New Zealand
- advice from members of Pharmac’s former Cystic Fibrosis Panel.
We have heard from expert advisors and people with cystic fibrosis, their whānau, and communities about the impact of cystic fibrosis and the benefit Trikafta can have. This has helped our understanding and assessment of this proposal.
Trikafta has been ranked on our Options for Investment list since October 2021. The list includes all treatments that we would fund if the budget allowed it. Following the budget announcement in May 2022 and commercial negotiations with the supplier, Vertex, we are now able to progress this proposal.
Details about our proposal
Elexacaftor with tezacaftor, ivacaftor and ivacaftor (Trikafta) would be listed in Section B (as PCT only) and Part II of Section H of the Pharmaceutical Schedule from 1 April 2023 at the following price (ex-manufacturer, excluding GST):
| Chemical | Formulation | Brand | Pack size | Price and subsidy |
|---|---|---|---|---|
| Elexacaftor with tezacaftor, ivacaftor and ivacaftor | Tab elexacaftor 100 mg with tezacaftor 50 mg, ivacaftor 75 mg and ivacaftor 150 mg | Trikafta | 84 | $27,647.39 |
| Elexacaftor with tezacaftor, ivacaftor and ivacaftor | Tab elexacaftor 50 mg with tezacaftor 25 mg, ivacaftor 37.5 mg and ivacaftor 75 mg | Trikafta | 84 | $27,647.39 |
A confidential rebate would apply to Trikafta that would reduce the net price.
Elexacaftor with tezacaftor, ivacaftor and ivacaftor (Trikafta) would be listed in Section B and Part II of Section H subject to the following eligibility criteria:
Special Authority for Subsidy
Initial application from any relevant practitioner. Approvals valid without further renewal (unless notified) for applications meeting the following criteria:
All of the following:
- Patient has been diagnosed with cystic fibrosis; and
- Patient is 6 years of age or older; and
- Either:
- Patient has two cystic fibrosis-causing mutations in the cystic fibrosis transmembrane regulator (CFTR) gene (one from each parental allele); or
- Patient has a sweat chloride value of at least 60 mmol/L by quantitative pilocarpine iontophoresis or by Macroduct sweat collection system; and
- Either:
- Patient has a heterozygous or homozygous F508del mutation; or
- Patient has a G551D mutation or other mutation responsive in vitro to elexacaftor/tezacaftor/ivacaftor (see note a); and
- The treatment must be the sole funded CFTR modulator therapy for this condition; and
- Treatment with elexacaftor/tezacaftor/ivacaftor must be given concomitantly with standard therapy for this condition.
Notes
- Eligible mutations are listed in the Food and Drug Administration (FDA) Trikafta prescribing information https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/212273s004lbl.pdf(external link).
Elexacaftor with tezacaftor, ivacaftor and ivacaftor (Trikafta) would be listed as a ‘PCT only – Specialist pharmaceutical’ in Section B of the Pharmaceutical Schedule. This means that funded treatment could only be dispensed from a Te Whatu Ora hospital pharmacy.
We are not proposing that Trikafta be dispensed through community pharmacies at this time. This is due to the high cost of this medicine. To reduce the number of times people need to travel to their nearest hospital, Te Whatu Ora Hospitals would be able to dispense three months’ worth of treatment at a time.
To provide feedback
Send us an email: consult@pharmac.govt.nz by 5 pm, Monday 16 January 2023.
All feedback received before the closing date will be considered by Pharmac’s Board (or its delegate) before making a decision on this proposal.
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