9 September 2021

Trikafta Managed Access Programme

Earlier this year, Vertex put in place a Managed Access Programme for people with CF in New Zealand to access Trikafta.  The Managed Access Programme is managed by Vertex and the criteria for access are set globally. Since the announcement of the New Zealand Managed Access Programme, CFNZ has become aware that there is uncertainty within the CF community as to how the Managed Access Programme works, what the criteria for access are, and who may be eligible to apply.  

Below we provide a brief explanation of the key aspects of the Managed Access Programme to try to address this uncertainty.  However, this information does not replace a discussion with your CF clinician. Your local CF team is the point of contact for any person with CF to discuss their specific situation and potential eligibility for the Managed Access Programme.

Process for Enrolment

The Managed Access Programme enables CF clinicians to apply to Vertex for Trikafta to be provided free of charge to those people with CF who are in critical need. People with CF and their families cannot apply direct to Vertex for access to the Programme.  Vertex has specified the criteria to determine critical need, and CF clinicians must use these criteria when identifying those people with CF who may be eligible, and for whom they will make an application. All CF teams in New Zealand are bound by confidentiality, so the criteria for access cannot be made public.

Local CF teams will assess those people with CF in their area who may meet the criteria for the Managed Access Programme. CF clinicians will then submit an application after discussing it with the person with CF and gaining the required consent. Vertex will then respond by either accepting or declining the individual’s application. Applications are anonymous.

The process for consideration of some people with CF may differ for New Zealand because NZ does not have access to all of Vertex’s other CF modulators. It may therefore take longer for some people with CF to receive confirmation of whether or not they have been enrolled on the Programme. Those people with CF who are accepted for the Managed Access Programme will be subject to close clinical monitoring.   

CFNZ understands that the Managed Access Programme will remain open for 12 months from the registration of Trikafta in New Zealand, during which time people with CF who meet the required criteria can continue to be enrolled. Medsafe is currently working through Vertex’s application for registration of Trikafta and has granted the application priority status.

Ongoing Supply of Trikafta

Once a person is enrolled in the Managed Access Programme, they will continue to receive Trikafta free of charge until the drug is funded by Pharmac or until Vertex decides to cancel the Programme. The contracts between Vertex and DHBs do enable Vertex to cancel the Managed Access Programme and terminate the supply of Trikafta to those who are enrolled. However, while there is no guarantee of continued supply of Trikafta to enrolled people, CFNZ understands the risk of Vertex terminating the Programme and ceasing supply is considered to be low. However, it is important to be aware that DHBs would not fund Trikafta if Vertex were to cease supply.

The Managed Access Programme provides a lifeline for those people with CF who are in critical need. However, the process of working through whether or not you may be eligible, waiting for a response once an application has been made, being accepted or being declined, are challenging to cope with. If you need support or someone to talk to, please contact your local CFNZ fieldworker. And CFNZ asks that everyone in the CF community is mindful of the stress on those people with CF and their families who are going through this process, and that we respect their right to privacy.

If anyone has concerns, issues or queries about the Managed Access Programme, it is essential that they seek advice direct from their local CF team. It is CF clinicians who are managing the arrangements with Vertex and who can provide appropriate advice and information.