Elexacaftor/Tezacaftor/IvacaftorTrikafta
Treatment of cystic fibrosis in patients aged 6 to 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Decisions on record
- Meeting Dec 2021 Recommended Indication not stated
- Meeting Jul 2021 Recommended Cystic fibrosis aged 12+ with F508del mutation
- Meeting Mar 2021 Deferred Cystic fibrosis; F508del mutation; aged 12+ years
Access path
- TGA registered · Trikafta
TGA label narrower than the PBS population
- Mar 2021Deferred
vs tezacaftor/ivacaftor (F/F and F/RF populations), ivacaftor…
- May 2021Deferred
- Jul 2021Recommended · restricted
Comparator changed: tezacaftor/ivacaftor (F/F and F/RF populations), ivacaftor (F/G…
- Dec 2021Recommended · restricted
- Mar 2023Recommended · restricted
Evidence: RCT → Single-arm
- PBS listing · Authority Required
From the public summary
The PBAC recommended elexacaftor/ tezacaftor/ ivacaftor (ELZ/TEZ/IVA) for the treatment of cystic fibrosis in patients who are aged 6 to 11 years and who have at 35PSD · Nov 2022
The PBAC noted the strong consumer support for extending the availability of ELX/TEZ/IVA to CF patients aged 6 to 11 years.PSD · Nov 2022
The submission presented a modelled economic evaluation comparing ELX/TEZ/IVA with LUM/IVA for the F/F population aged 6 to 11 years and comparing ELX/TEZ/IVA with BSC for the F/MF population aged 6 to 11 years. The model structure was similar to that presented in previous CFTR submissions to the PBAC (e.g. LUM/IVA PSD, July 2019, ELX/TEZ/IVA PSD, March 2021).PSD · Nov 2022
Table 13: Key components of the economic evaluation Component Description F/F, F/MF: Cost-utility analysis Type of analysis Combined population with weight of 62% for F/F and 38% for F/MF F/G, F/RF, F/R117: no economic evaluation presentedPSD · Nov 2022
The submission described ELX/TEZ/IVA as superior in terms of effectiveness and comparable in terms of safety compared with the nominated comparators (LUM/IVA for F/F population, IVA for F/G population and BSC for F/MF, F/RF and F/R117H population).PSD · Nov 2022
• The reliability of the evidence from a single arm study (Study 106) to inform the evidence for the ELX/TEZ/IVA in the F/F population, which may be impacted by the large proportion of patients that missed their clinical assessment visits from Week 12 onwards (>50%).PSD · Nov 2022
The PBAC noted and welcomed the input from individuals (273), health care professionals (4) and organisations (3) via the Consumer Comments facility on the PBS website. Many comments were received from parents and caregivers outlining the potential benefits of ELX/TEZ/IVA treatment in individuals with CF, including improved life expectancy, a reduction in the need for lung transplants and hospital visits (including associated travel costs), improved …PSD · Nov 2022
The PBAC noted the input received from health professionals outlined the improvement in symptoms in CF patients undergoing ELX/TEZ/IVA treatment. One comment described how the medicine appeared to protect those with milder disease from rapid progression of their disease, but outlined the need for monitoring patients for liver dysfunction and side effects in the eyes in children.PSD · Nov 2022
Cost-effectiveness
No ICER stated in the document. Economic model based on cost-effectiveness and cost-comparison analyses, but numeric ICER values not published in this public summary.
The PBAC deferred making a recommendation in order to allow engagement with the sponsor to align the proposed listing of ELX/TEZ/IVA, and associated costs and financial implications with the MAP and risk share arrangements (RSA) of the currently listed CFTR modulators, with the cost-effectiveness link with these comparators having not satisfactorily been established by the submission. PBAC · 2021
Decision context
PopulationChildren aged 6 to 11 years with cystic fibrosis who have at least one F508del mutation in the CFTR gene, across six genotype subpopulations (F/F, F/RF, F/G, F/MF, F/R117H, F/not yet characterised).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2023 | 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 · Other |
| Dec 2021 | Recommended · restricted | — | — | RCT · Other |
| Jul 2021 | Recommended · restricted | TEZ/IVA (F/F and F/RF populations), IVA (F/G population), best supportive care (F/MF and F/not yet characterised populat | — | RCT · ppFEV1 (percent predicted forced expiratory volume in one second) |
| May 2021 | Deferred | tezacaftor/ivacaftor (F/F and F/RF populations), ivacaftor (F/G population), best supportive care (F/MF population) | — | RCT · ppFEV1 |
| Mar 2021 | Deferred | tezacaftor/ivacaftor (F/F and F/RF populations), ivacaftor (F/G population), best supportive care (F/MF population) | — | RCT · ppFEV1 |
Consumer voice
Consumer input from 273 individuals, 4 health professionals, and 3 organisations strongly supported ELX/TEZ/IVA access for cystic fibrosis patients, particularly younger populations, citing improved life expectancy, reduced hospitalisations, quality of life benefits, and prevention of organ damage. Health professionals noted symptom improvements and disease progression prevention but flagged monit
Many comments were received from parents and caregivers outlining the potential benefits of ELX/TEZ/IVA treatment in individuals with CF, including improved life expectancy, a reduction in the need for lung transplants and hospital visits (including associated travel costs), improved quality of life, and allowing an overall sense of normalcy. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted subsidy to ages 6–11 years; TGA label includes all ages 6 years and older.