Lumacaftor/IvacaftorOrkambi
Treatment of cystic fibrosis in patients aged 2 to 5 years who are homozygous for the F508del mutation in the CFTR gene.
Decision on record
- Meeting Jul 2021 Noted Cystic fibrosis
Access path
- TGA registered · Orkambi
TGA label equal than the PBS population
- Jul 2019Recommended · restricted
vs best supportive care (BSC)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the Authority Required listing of a new presentation of lumacaftor with ivacaftor, in the form of granules, for the treatment of CF in patients aged 2 years or over who are homozygous for the F508del mutation in the CFTR gene.PSD · Jul 2019
The PBAC recommendation was made on the basis that lumacaftor/ivacaftor granules should be available only under special arrangements under Section 100 (Highly Specialised Drugs Program). The PBAC considered that the supporting evidence was limited but acknowledged the difficulties in obtaining efficacy data from paediatric patients.PSD · Jul 2019
6.23 The economic evaluation presented was a cost-utility analysis (CUA). The structure of the model was the same as in the previous (re)submissions (March 2016, November 2016, July 2017, July 2018), which presented a CUA of lumacaftor/ivacaftor compared with BSC.PSD · Jul 2019
6.24 The ESC noted that the submission did not present an estimate of the incremental cost effectiveness of starting treatment with lumacaftor/ivacaftor from two years of age (early onset treatment) compared with BSC from 2-5 years, followed by lumacaftor/ivacaftor treatment starting at 6 years of age (as is currently funded via the PBS).PSD · Jul 2019
Lumacaftor/ivacaftor plus BSC is non-inferior in terms of safety compared to BSC alone. Source: Table 1.1.1 of the submission pp26-27.PSD · Jul 2019
1.2 This is the first submission for lumacaftor/ivacaftor granules. Lumacaftor/ivacaftor tablets are currently subsidised on the PBS for the treatment of CF patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene.PSD · Jul 2019
6.2 The PBAC noted and welcomed the input from individuals (419) via the Consumer Comments facility on the PBS website. The comments described the potential for improved health outcomes associated with commencing treatment with lumacaftor/ivacaftor at a younger age.PSD · Jul 2019
6.44 The submission proposed an annual price of $'''''''''''' per patient, intended to be achieved via subsidisation caps implemented through an RSA, consistent with the intended annual price per FTE patient for lumacaftor/ivacaftor (of $'''''''''''''') in patients aged 6 years and older.PSD · Jul 2019
Cost-effectiveness
ICER values are redacted (marked as '''''''''''''''''''''') in the publicly available document; commercially sensitive.
Decision context
PopulationChildren aged 2 to 5 years with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene.
Risk sharingRisk sharing arrangement (RSA) under the current Deed of Agreement proposing a reduction in gross cost via a subsidisation cap, with intent to achieve a price of $'''''''''''' per patient per year (redacted). Similar arrangement to ivacaftor granules discussed.
Why it was knocked back
- Non-comparative open-label trial design with no head-to-head comparison to BSC; small sample size (n=60 Part B); short duration (24 weeks) for a chronic disease; absence of comparator group prevented assessment of comparative efficacy/safety; uncertain comparative effectiveness versus deferring treatment to age 6 years; high risk of bias; significant risk that Government would pay higher amount per FTE patient than intended price.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2019 | Recommended · restricted | best supportive care (BSC) | — | Single-arm · Safety and pharmacokinetics |
Consumer voice
Consumer comments described the potential for improved health outcomes associated with commencing treatment with lumacaftor/ivacaftor at a younger age.
The comments described the potential for improved health outcomes associated with commencing treatment with lumacaftor/ivacaftor at a younger age. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both texts specify identical population: cystic fibrosis patients aged 2–5 years homozygous for F508del mutation.