IvacaftorKalydeco
Recommended Rare diseaseAuthority Required 💬 consumer voice
Treatment of cystic fibrosis in patients aged 1 to 4 months with either a G551D mutation or other gating (Class III) mutation in the CFTR gene on at least 1 allele, or at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
10
Submissions
8 resub
2013–25
On the record
$60k–80k
ICER range
3 sourced ICERs · 2013–2015
Not modelled
Cost basis
Decisions on record
- Meeting Mar 2025 Recommended Cystic fibrosis (CF), patients aged 1 to 4 months with gating or responsive CFTR mutations
- Meeting Nov 2023 Recommended Cystic fibrosis with CFTR gene mutation (ages 4 months and older)
- Meeting Mar 2019 Recommended Cystic fibrosis (CF)
- Meeting Nov 2016 Recommended Cystic fibrosis
- Meeting Nov 2016 Deferred Cystic fibrosis
- Meeting Nov 2015 Recommended Cystic fibrosis
- Meeting Jul 2015 Deferred Cystic fibrosis
- Meeting Nov 2014 Recommended tablet, 150 mg, Kalydeco® Vertex Pharmaceuticals (Australia) Pty Ltd Change to recommended listing (Minor submission) Cystic fibrosis To request an extension of the PBAC’s previous recommendation for the PBS listing of ivacaftor as a Section 100 (Highly Specialised Drugs Program) benefit for the tre
3 earlier decisions
- Mar 2014 Recommended Kalydeco® Vertex Pharmaceuticals (Australia) Pty Ltd Cystic Fibrosis Re-submission to request Section 100 Highly Specialised Drugs (HSD) listing with or without Rule of Rescue consideration or LSDP listing for the treatment of cystic fibrosis in patients 6 years and older who have confirmed class I
- Nov 2013 Recommended Kalydeco® Vertex Pharmaceuticals Matter arising from the July 2013 PBAC Meeting Minutes Cystic Fibrosis At the July 2013 meeting, the submission requested a Section 100 (Highly Specialised Drugs Program) listing or inclusion on the Life Saving Drugs Program (LSDP) for treatment of cystic fibrosis in
- Jul 2013 Deferred Cystic fibrosis
Access path
- TGA registered · Kalydeco
TGA label narrower than the PBS population
- Jul 2013Deferred
vs best supportive care
- Nov 2013Recommended
Comparator changed: best supportive care → best supportive care (BSC)
- Mar 2014Recommended · restricted
Comparator changed: best supportive care (BSC) → best supportive care
- Nov 2014Recommended · restricted
Comparator changed: best supportive care → placebo (KONNECTION trial); best supportive…
- Jul 2015Deferred
Evidence: RCT → Other
- Nov 2015Recommended · restricted
Evidence: Other → Single-arm
- Nov 2016Deferred
Listing: Restricted → Authority Required
- Mar 2019Recommended · restricted
- Nov 2023Recommended · restricted
Comparator changed: best supportive care → best supportive care (BSC) alone
- Mar 2025Recommended · restricted
Comparator changed: best supportive care (BSC) alone → best supportive care
- PBS listing · Authority Required
RecommendedDeferredNot recommended
From the public summary
PBAC outcome
7.1 The PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of ivacaftor granules 13.4 mg for the treatment of cystic fibrosis (CF) in patients aged 1 month to less than 4 months who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor 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 ivacaftor.PSD · Mar 2025
Economic analysis
6.26 The submission did not present an economic analysis. The submission stated that the request for reimbursement is based on equity and the expected benefit of treatment in these patients.PSD · Mar 2025
Drug/ cost/patient Table 6: Drug cost per patient aged 1-4 months for ivacaftor, as applied in financial estimates Gating population Non-gating population Cost per 28 day pack (effective price) $ $ Compliance 100% 100% Cost per 3 months treatment (3.26 scripts)a $ $ Source: constructed during the evaluation from the information in the submission. a 365.25/28/12*3PSD · Mar 2025
Clinical claim
6.21 The submission described ivacaftor as superior in terms of effectiveness and comparable in terms of safety to best supportive care in infants aged 1-4 months with either a G551D mutation or other gating (Class III) mutation in the CFTR gene on at least one allele, or at least one mutation in the CFTR gene that is responsive to IVA potentiation based on clinical and/or in vitro assay data.PSD · Mar 2025
6.22 With regard to effectiveness, the evaluation considered the claim was not adequately supported. The submitted data, taken as a whole, support a conclusion that ivacaftor increases the activity of the CFTR in infants aged 1-4 months with the G551D or other 11PSD · Mar 2025
Consumer comments
6.2 The PBAC noted and welcomed the input from individuals (4) and organisations (2) via the Consumer Comments facility on the PBS website. The comments from individuals described the benefits of treatment with ivacaftor as life-changing stating early access will extend the lives of children afflicted with CF, prevent irreversible scarring of the lungs and lead to better health outcomes.PSD · Mar 2025
6.3 Cystic Fibrosis Australia strongly supported expanding access for infants with CF aged 1 to less than 4 months as early intervention can significantly improve health outcomes and quality of life. It was noted that initiating ivacaftor therapy in infants can maintain healthier lung function, improve weight gain, and reduce complications.PSD · Mar 2025
Cost-effectiveness
No economic evaluation was presented in the submission; listing was requested on the basis of equity and clinical need.
Listing was requested on the basis of equity and clinical need; no economic evaluation was presented in the submission to support the listing requested in the proposed population. PSD · 2025
ICER uncertain ×3ICER / price too high ×3Economic model disputedPrice cut / RSA needed ×2Indirect comparisonCost-effectiveness accepted
Decision context
PopulationCystic fibrosis patients aged 1 to less than 4 months with either G551D mutation or other gating (Class III) mutation in the CFTR gene on at least 1 allele, or at least one mutation in the CFTR gene responsive to ivacaftor potentiation based on clinical and/or in vitro assay data.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | best supportive care | — | Single-arm · Safety |
| Nov 2023 | Recommended · restricted | best supportive care (BSC) alone | — | RCT · Surrogate |
| Mar 2019 | Recommended · restricted | best supportive care | — | Single-arm · Other |
| Nov 2016 | Deferred | best supportive care | — | Single-arm · Other |
| Nov 2015 | Recommended · restricted | best supportive care | $60k–80k | Single-arm · FEV₁ improvement |
| Jul 2015 | Deferred | — | — | Other |
| Nov 2014 | Recommended · restricted | placebo (KONNECTION trial); best supportive care (prior G551D trials) | — | RCT · FEV1 |
| Mar 2014 | Recommended · restricted | best supportive care | $60k–80k | RCT · Surrogate |
| Nov 2013 | Recommended | best supportive care (BSC) | — | RCT · FEV1 |
| Jul 2013 | Deferred | best supportive care | $200k | RCT · FEV1 |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| STRIVE | Ph 3 | 167 | Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Seco… | completed |
| ENVISION | Ph 3 | 52 | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (F… | completed |
| PERSIST | Ph 3 | 192 | Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (S… | completed |
| KONNECTION | Ph 3 | 39 | Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 S… | completed |
Consumer voice
Four individuals and two organisations (Cystic Fibrosis Australia and CF Together) provided input strongly supporting ivacaftor access for infants with CF aged 1 to less than 4 months, emphasising life-changing benefits, improved health outcomes, and better quality of life through early intervention.
the benefits of treatment with ivacaftor as life-changing stating early access will extend the lives of children afflicted with CF, prevent irreversible scarring of the lungs and lead to better health outcomes Consumer comments · PSD
quality of lifeunmet needsurvival benefittreatment burden preventionearly intervention
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to ages 1–4 months; TGA label includes all ages ≥1 month.