17 June 2024

An update on access to CFTR Modulator Therapies

Pharmac is currently considering two applications for modulator therapies – the widening of access to Kalydeco and the extension of access to Trikafta for 2-5 year olds. CFNZ met with Pharmac at the end of April to discuss progress with the two applications.

Widening of Access to Kalydeco

Pharmac is currently considering an application to widen access to Kalydeco from the current 9 eligible genetic mutations to the 97 mutations currently approved by the US Food and Drug Administration.

Pharmac’s Respiratory Advisory Committee recommended a high priority for funding in April 2022. However, the application is still awaiting assessment before it can be ranked and considered for inclusion on Pharmac’s Options for Investment list.

At the meeting with Pharmac, CFNZ again expressed its serious concern over the delay in progressing the application, with more than 2 years elapsing since the high priority recommendation was made. We also reiterated concern over the inequitable treatment of Kalydeco compared with eligibility for Trikafta, where an assessment is not required to include additional genetic mutations.

CFNZ has also asked Rare Disorders NZ to raise concerns with Pharmac over the delay in widening access to Kalydeco as part of its regular discussions with the agency.

Almost all of the 97 mutations proposed for widening access to Kalydeco are also included in access to Trikafta under Pharmac’s current eligibility criteria. However, there remain a small number of people with CF who cannot currently access Trikafta, but would qualify for Kalydeco if access were to be widened.

Eligibility for Trikafta includes the ability for clinicians to apply for Trikafta for rare mutations under the Named Patient Pharmaceutical Assessment (NPPA) scheme where there is “evidence to demonstrate Trikafta works based on reasonable extrapolation.” As a result, there may be some people who are waiting for waiting for access to Kalydeco to be widened who could benefit from Trikafta.

We would therefore encourage anyone with who has been unable to access a modulator therapy to talk their CF clinician to see if an application under the NPPA scheme may be possible. Information on the criteria for access to Trikafta, including the link to the NPPA scheme, can be found here.

Trikafta for 2-5 year olds

Vertex applied to Medsafe in January 2024 for safety approval of Trikafta for 2-5 year olds, and to Pharmac for funding in February 2024.

CFNZ wrote to requesting that Medsafe give the application priority status, which has been granted. The application is currently undergoing initial evaluation.

Pharmac has advised CFNZ that clinical advice is currently being sought and that the application will potentially be considered by its Respiratory Advisory Committee in August or September. This would be followed by assessment and, potentially, ranking in early 2025. However, the process and timing for the application have not yet been confirmed.