Decision to fund elexacaftor with tezacaftor and ivacaftor (Trikafta) for people with cystic fibrosis

7 March 2023

Medicines Decision

What we’re doing

We're pleased to announce that elexacaftor with tezacaftor and ivacaftor (brand name Trikafta) will be funded for people aged 6 years and above with cystic fibrosis, subject to eligibility criteria, from 1 April 2023.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 4 December 2022. We received lots of consultation feedback supporting the proposal. We are really grateful to those who took the time to provide feedback, in particular those who courageously shared their personal stories with us.

We have not made any changes to the original proposal following feedback. A summary of the feedback we received and our responses is detailed below.

Who we think will be most 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

Detail about this decision

Elexacaftor with tezacaftor, ivacaftor and ivacaftor (Trikafta) will be listed in Section B (as PCT only – Specialist pharmaceutical) and Part II of Section H of the Pharmaceutical Schedule from 1 April 2023 at the following price and subsidy (as applicable) (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 will apply to Trikafta that will reduce the net price.

Elexacaftor with tezacaftor, ivacaftor and ivacaftor (Trikafta) will 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:
1. Patient has two cystic fibrosis-causing mutations in the cystic fibrosis transmembrane regulator (CFTR) gene (one from each parental allele); or
2. Patient has a sweat chloride value of at least 60 mmol/L by quantitative pilocarpine iontophoresis or by Macroduct sweat collection system; and
Either:
1. Patient has a heterozygous or homozygous F508del mutation; or
2. 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) will be listed as a ‘PCT only – Specialist’ pharmaceutical in Section B of the Pharmaceutical Schedule. This means that funded treatment can only be dispensed from a Te Whatu Ora inpatient hospital pharmacy.

We are not proposing that Trikafta be dispensed through community pharmacies at this time. This includes retail or outpatient pharmacies within Te Whatu Ora hospitals. This is due to the high cost of this medicine. We will monitor access and feedback about accessing this medicine and intend to work with our health sector partners to develop equitable, patient-centred distribution models.

How can people access Trikafta?

Prescribers will need to apply for a Special Authority on behalf of people living with cystic fibrosis. Prescribers will need to confirm that the person meets the access criteria detailed above.

Once a Special Authority application has been approved, people will need to go to any Te Whatu Ora inpatient hospital pharmacy to collect Trikafta. People should talk to their prescriber about which Te Whatu Ora hospital they would like to collect their medicine from. People will be able to choose the Te Whatu ora hospital that is most convenient for them – for example the hospital where they attend clinic or the hospital nearest to their home/school.

Cystic fibrosis caused by rare mutations

We are aware that some people may have rare mutations which evidence shows respond to Trikafta but are not covered by the access criteria. Prescribers can apply for a Named Patient Pharmaceutical Assessment (NPPA) for people with these rare mutations. We would require evidence to demonstrate that Trikafta works for the person’s specific mutation(s). Information on NPPA applications and how to apply can be found at Make a NPPA application.

Our response to what you told us

We are grateful for the time people took to respond to this consultation. A summary of the main themes raised in feedback and our responses to the feedback received are available below.

Theme	Pharmac Response
Support for the proposal to fund elexacaftor with tezacaftor and ivacaftor
All consultation responses received were strongly supportive of the proposal. In particular the responses highlighted: the high unmet health need that elexacaftor with tezacaftor and ivacaftor would address the profound negative impacts of the condition on people living with cystic fibrosis, their whānau, caregivers and communities the positive impact this proposal would have, including allowing people living with cystic fibrosis to have hope and future dreams the benefit to whānau of reducing the support caregivers are required to provide with current treatments, including the ability to re-enter or increase paid and unpaid work this proposal would enable people and their whānau to remain (or return) to Aotearoa New Zealand, retaining their support networks the benefit of allowing Special Authority applications from any relevant practitioner to reduce potential access barriers and support healthcare availability in a primary care setting the benefit of criterion 3, which allows for either two specific mutations or a sweat test (rather than both) the impact of advocacy for access to the treatment, and the courage shown by those who have publicly shared their stories.	We are grateful to all the people who took the time to courageously share their personal stories with us. We are pleased to be funding a treatment with the potential to greatly improve the health outcomes of people living with cystic fibrosis, their whānau and communities. The feedback we received reflects what we have heard from the cystic fibrosis community and clinical advisors when assessing this proposal.
Dispensing mechanism
Responses requested dispensing be allowed from community pharmacies, noting: the potential access barriers that may result from hospital-only dispensings hospital pharmacy dispensings are not visible for health professionals to view across shared software platforms.	We acknowledge the risk of access barriers resulting from restricting dispensing to Te Whatu Ora hospitals. Trikafta will not be dispensed through community pharmacies (or retail / outpatient hospital pharmacies) at this time due to the high cost of this medicine. However, we intend to work with health system partners, including Te Whatu Ora and Te Aka Whai Ora in the potential development of a healthcare system distribution mechanism that could be utilised for high-cost medicines in the long-term. We understand that people starting this medicine would be monitored at a secondary care level, or meet with a specialist regularly. This would allow them to collect prescriptions in line with the proposal. We will communicate with Chief Pharmacists to highlight that Te Whatu Ora hospital pharmacies are encouraged to work with prescribers and people living with cystic fibrosis to support access to elexacaftor with tezacaftor and ivacaftor in a way that minimises the burden on them and their whānau. We intend to monitor this and are open to considering different distribution mechanisms in the future to support access to elexacaftor with tezacaftor and ivacaftor in a way that minimises the burden on people living with cystic fibrosis and their whānau. We would consult on any potential future changes to ensure they can be implemented effectively within the sector and work for people living with cystic fibrosis and their whānau.
Special Authority access criteria
Responses raised concerns about people living with cystic fibrosis who might miss out on funded access due to specific circumstances, specifically: people who are already receiving elexacaftor with tezacaftor with ivacaftor and may not meet the exact criteria (ie having available records of previous sweat chloride levels) people with rare mutations not yet assessed by the US FDA.	We understand that some cases, a person’s circumstances may meet the intent of funding criteria but not the exact wording. We will consider a Special Authority waiver application where a person’s circumstances may meet the intent of the access criteria. Details on how to submit a Special Authority waiver application are available on our website (Special Authority waivers). In vitro responsive mutations were included in the criteria to ensure those with rare mutations would have access to treatment. We note the FDA regularly updates the list of in vitro responsive mutations. We would consider applications for people with any other rare mutations on a case-by-case basis, via our Named Patient Pharmaceutical Assessment pathway. Additional information on this is included in this notification.
Responses raised concern with criterion 5, which requires elexacaftor with tezacaftor and ivacaftor to be the sole funded CFTR therapy and requested clarification on this criterion should a generic or alternative treatment becomes available. Respondents suggested an addition that an alternative therapy must have same or better efficacy.	Criterion 5 ensures elexacaftor with tezacaftor and ivacaftor is the only publicly funded CFTR therapy, as we have not assessed the benefit of receiving more than one CFTR therapy at the same time. We would consider amendments to the criteria as required if an alternative therapy or generic became available. Ongoing treatment availability is an important part of our work and any alternative or generic treatments would be assessed for efficacy prior to any funding decision.
Data to support the use for people 2 – 5 years of age
Responses noted future consideration of data for the use of elexacaftor with tezacaftor and ivacaftor in those below the age of 6 would be needed.	Any widened access to elexacaftor with tezacaftor and ivacaftor, including lowering or removing the age restriction, would be assessed via our medicines funding process as the data becomes available. We would also consider listing of alternative presentations as they become available to ensure all eligible people have a suitable presentation.
Funding of other medicines for cystic fibrosis
Responses requested: widened access to ivacaftor (Kalydeco) funding of tezacaftor with ivacaftor (Symdeko) to ensure everyone living with cystic fibrosis in Aotearoa New Zealand has a suitable funded CFTR modulator treatment option.	A proposal for widened access to ivacaftor is currently under assessment, and will then be ranked on our Options for Investment list (application tracker(external link)). We would welcome a funding application for tezacaftor with ivacaftor (Symdeko). We have shared this feedback with the supplier, Vertex, to highlight the public support for a funding application for Symdeko in New Zealand. We acknowledge the strong desire for a funded CFTR treatment for everyone listing with cystic fibrosis. We are committed to working with suppliers, advocacy groups and the cystic fibrosis community on funding applications for cystic fibrosis medicines.
System constraints
Responses noted the potential health system constraints immediately following funding, given the desire from the cystic fibrosis community for rapid access to treatment.	We understand the cystic fibrosis community is likely to want rapid access to elexacaftor with tezacaftor and ivacaftor. There was approximately four months between Pharmac releasing the public consultation and the proposed funding date, which we anticipated would give the health sector time to prepare for the funding. People who are eligible for elexacaftor with tezacaftor and ivacaftor should discuss with their prescriber about accessing this treatment and any additional tests and/or monitoring that may be required as part of taking this medicine. We intend to work with sector stakeholders to support the implementation of this funding decision.
Responses highlighted that access to clinical psychology services is limited, and that these form an important part of holistic care in the case of potential treatment side effects such as depressed mood and/or anxiety.	Discussion of potential treatment side effects and ongoing monitoring is an important part of prescribing competency, and we would expect any prescriber to discuss these aspects with people eligible for funded medicines and their whānau/caregivers before starting treatment. We acknowledge that there are system constraints in many secondary healthcare services, and we will continue working with other health sector partners to support a system that meets the needs of New Zealanders.
Financial Impact
Responses noted the proposal would save the healthcare system money through a reduction in hospitalisations, other medicines and lung transplants. Responses also noted that the increased specialist oversight when initiating people on treatment would be offset by a future reduction in specialist input for those receiving treatment.	We agree that the funding of elexacaftor with tezacaftor and ivacaftor would likely result in some savings to the healthcare system. However, our assessment shows that the cost of the medicine is greater than the potential estimated savings to the healthcare system. You can read more about our assessment in our Technology Assessment Report on the Pharmac website.
Time taken to fund
Responses noted the length of time it has taken for Pharmac to propose funding elexacaftor with tezacaftor with ivacaftor.	Pharmac first received a funding application for elexacaftor with tezacaftor and ivacaftor in July 2021. Elexacaftor with tezacaftor and ivacaftor has been ranked on our Options for Investment list since October 2021. The list includes all treatments that we would like to fund if the budget allowed it, noting that Pharmac operates within a fixed budget. Following a budget increase announcement in May 2022 and commercial negotiations with the supplier, Vertex, we are now pleased to be in a position to fund Trikafta.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.