Elexacaftor With Tezacaftor And With Ivacaftor, And Ivacaftor
Extension of listing for cystic fibrosis (CF) in patients aged 2 years and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to elexacaftor/tezacaftor/ivacaftor based on clinical and/or in vitro assay data.
Decisions on record
- Meeting Mar 2025 Recommended Cystic fibrosis (CF), patients aged 2 years or older with at least one CFTR gene mutation responsive to modulators
- Meeting Mar 2024 Recommended Cystic fibrosis (CF) in children aged 2 to 5 years
- Meeting Mar 2023 Recommended Cystic fibrosis in patients aged 6 to 11 years with at least one F508del mutation on the CFTR gene no PSD
- Meeting Nov 2022 Recommended Cystic fibrosis in patients aged 6 to 11 years with F508del mutation
Access path
- Mar 2024Recommended · restricted
vs lumacaftor/ivacaftor (F/F population), ivacaftor (F/G…
- Mar 2025Recommended · restricted
Comparator changed: lumacaftor/ivacaftor (F/F population), ivacaftor (F/G population)…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended that the listing of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) for the treatment of cystic fibrosis (CF) be extended to include patients who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ELX/TEZ/IVA based on clinical and/or in vitro assay data.PSD · Mar 2025
7.2 The PBAC acknowledged the consumer comments strongly supported the extension of the listing for ELX/TEZ/IVA.PSD · Mar 2025
6.26 The submission did not present an economic analysis. The submission stated that this approach was the same as that considered by the PBAC meeting in Nov 2023, for the submission for patients with one of 92 mutations responsive to IVA potentiation based on clinical or in-vitro data.PSD · Mar 2025
6.27 The submissions requested the price of ELX/TEZ/IVA for the non-F508del ELX/TEZ/IVA responsive patients being the same as the current effective price for the F508del mutation group. 17PSD · Mar 2025
6.21 The submission described ELX/TEZ/IVA as: o superior in terms of effectiveness compared with IVA plus BSC in patients with mutations responsive to both IVA and ELX/TEZ/IVA, 16PSD · Mar 2025
6.22 The evaluation considered this claim was not adequately supported. There was no systematic analysis presented of any comparative data or data for patients switching between treatments to determine whether ELX/TEZ/IVA was superior to either IVA or TEZ/IVA. The data presented suggested that patients who switched to ELX/TEZ/IVA from another CFTRm, in most cases IVA, obtained minimal additional benefit.PSD · Mar 2025
6.3 Cystic Fibrosis Australia noted the available clinical evidence for ELZ/TEZ/IVA supports its efficacy and safety in this population and the importance of providing early access to reduce long term damage. CF Together suggested flexible PBS listing criteria would be appropriate that included responsive and non-categorised mutations and proposed a clinical assessment approach to determine the efficacy of ELX/TEZ/IVA for individuals with rare mutations.PSD · Mar 2025
6.37 The submission proposed that the requested population for ELX/TEZ/IVA in this submission be included in the existing Deed with no change to the subsidisation caps.PSD · Mar 2025
Cost-effectiveness
No economic evaluation was presented in the submission to support the listing requested.
Decision context
PopulationCF patients aged 2 years of age or older with at least one mutation in the CFTR gene that is responsive to elexacaftor/tezacaftor/ivacaftor potentiation based on clinical data or in vitro assay
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | IVA plus BSC (for mutations responsive to both IVA and ELX/TEZ/IVA), TEZ/IVA plus BSC (for TEZ/IVA eligible residual fun | — | RCT · Surrogate |
| Mar 2024 | Recommended · restricted | lumacaftor/ivacaftor (F/F population), ivacaftor (F/G population), best supportive care (F/MF, F/RF, F/R117H, F/not yet | — | Single-arm · Surrogate |
Consumer voice
Consumer input from 359 individuals, 8 healthcare professionals, and one organisation (Cystic Fibrosis Australia) supported listing of ELX/TEZ/IVA in children aged 2-5 years, emphasising the importance of early treatment to prevent irreversible lung damage, improve quality of life, and reduce mortality, with benefits extending to carers and family members.
The comments stressed the importance of starting treatment with CFTR modulators at a young age to support better outcomes, including improved quality of life, for individuals with CF. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both require CFTR mutation responsiveness; PBAC specifies the drug name but describes the same eligible population as TGA label.