Trikafta® (elexacaftor/tezacaftor/ivacaftor) is a new is a triple combination therapy that may benefit many people living with CF. Trikafta® is on the agenda for the March 2021 PBAC meeting for the treatment of CF in patients aged 12 years or older who have at least one F508del gene change.
What is Trikafta®?
Elexacaftor/tezacaftor/ivacaftor, or Trikafta®, is a type of medication known as a CFTR modulator. CFTR modulators work differently to other medications for CF. They work by correcting the malfunctioning protein that causes CF. In doing so, these medications directly address the cause of CF, rather than just the symptoms. They are not a cure, but they help the body’s cells to function more normally.
Trikafta® is a triple combination therapy. It has three active ingredients, elexacaftor, tezacaftor and ivacaftor. Elexacaftor and tezacaftor help the F508del-CFTR protein change to the correct shape, move to the surface of the cell and stay there longer. Ivacaftor then helps to open the channel so that chloride can flow in and out of the cells.
What will happen at the March PBAC meeting?
The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent body of doctors, health professionals, health economists and consumer representatives. The PBAC’s main role is to recommend new medicines for listing on the Pharmaceutical Benefits Scheme (PBS). No new medicine can be listed unless the Committee makes a positive recommendation.
Trikafta® is on the agenda for the March Pharmaceutical Benefits Advisory Committee (PBAC) meeting for the treatment of CF in patients aged 12 years or older who have at least one F508del gene change. This means that at their March 2021 meeting, the PBAC will review Trikafta® and consider:
- medical conditions for which the medicine was registered for use in Australia (in this case CF)
- its clinical effectiveness
- safety and cost-effectiveness (‘value for money’) compared with other treatments.
There are three outcomes from the PBAC meeting. They can:
- Recommend Trikafta® be listed on the PBS
- Defer their decision and consider it again at a later meeting
- Reject the application for Trikafta® to be listed on the PBS
It is expected that the outcome of the March 2021 PBAC meeting will be announced on 23 April 2021.
We are hoping that the PBAC will recommend Trikafta® be listed on the PBS. However, if they defer or reject the application we will continue to advocate for it to be recommended for listing.
If PBAC recommend Trikafta® be listed on the PBS, it generally takes approximately 6 months to negotiate the final agreement between Vertex and the Australian Government.
How you can help?
The PBAC have a website where people can provide comments from a personal, or group, perspective that will be considered when they are reviewing Trikafta®.
When making comments through the website (portal), think about:
- How CF affects your quality of life?
- What would you most like to see from this treatment or expect will happen if you do take it?
- If you are taking Trikafta® , what was your experience of any benefits or side effects?
Go to the PBAC consumer comments portal
The consumer comments portal is open for comments until the 27 January 2021.