December 2023

Modulator Therapies

Since funding started on 1 April 2023, many in the CF community have experienced the benefits of Trikafta, as well as coping with the changes and challenges that this life-changing medicine can bring. Together with Kalydeco, more than 360 people with CF in New Zealand are now able to access medicines that treat the cause of their CF.

But not everyone is yet able to benefit. Some people cannot tolerate Trikafta while, for others, modulator therapies simply don't work for the genetic mutations they carry. Some are not yet old enough to access Trikafta while others fall outside the eligibility criteria for the already funded Kayldeco. CFNZ's goal is for everyone to have access to a therapy that treats the cause of their CF.

Kalydeco

The immediate focus of CFNZ's advocacy is widening access to Kalydeco for those who currently fall outside Pharmac's current eligibility criteria. We have made clear to Pharmac that people waiting for access to Kalydeco must be treated fairly and given equal opportunity, consisten with those who can access a funded modulator therapy.

In Australia, the Pharmaceutical Benefits Advisory Committee (PBAC) announced on 15 December 2023 that it has recommended that access to Kalydeco be widened, to include those from 4 months of age and additional rare mutations.[1]

Trikafta

Approval has recently been given for Trikafta to be made available to children aged 2 to 5 years old in the UK and Europe. In Australia, PBAC will consider an application to make Trikafta available to this age group at its March 2024 meeting.

Vertex announced on 24 November 2023 that it intended to seek regulartory approval for this in several countries including New Zealand .  [2] No date for the application in New Zealand has yet been confirmed. Vertex also advised that it intends to seek approval from the US Food and Drug Administration (FDA) for additional rare mutations responsive to Trikafta.

Access to Medicines in New Zealand

The coalition agreements signed by the new Government contain a range of initiatives that suggest there could be positive change for access to medicines and the opertation of Pharmac - New Zealand's medicines funding agency.

These initiatives include:

• Update Pharmac's decision-making model to ensure it appropriately takes into account the patient voice.
• Reform the funding model to account for the positive fiscal impacts of funding more medicines.
• Require the Ministry of Health to publish a Medicines Strategy every three years.
• Require Medsafe to approve new pharmaceuticals within 30 days of them being approved by two overseas regulatory authorities recognised by New Zealand.
• Increase funding for Pharmac every year. [3]

We look forward to positive changes in funding, decision-making, and access to medicines in New Zealand.

[1] pbac-web-outcomes-11-2023.pdf (pbs.gov.au)

[2] https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-european-medicines-agency-validation-marketing

[3] https://assets.nationbuilder.com/nationalparty/pages/18466/attachments/original/1700778592/National_ACT_Agreement.pdf?1700778592

https://assets.nationbuilder.com/nationalparty/pages/18466/attachments/original/1700778597/NZFirst_Agreement_2.pdf?1700778597

4 December 2022, Pharmac released a proposal to fund elexacaftor with tezacaftor and ivacaftor (Trikafta) from 1 April 2023 for people aged 6 years and over with cystic fibrosis (CF) in New Zealand, subject to eligibility criteria. Pharmac is now seeking public feedback on this proposal.