cystic fibrosis
Rare disease · 2010–2025
36decisions
9medicines
25carried consumer input
11,200individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| vanzacaftor/tezacaftor/deutivacaftor Treatment of cystic fibrosis in patients aged 6 years and older who have at least one mutation in the cystic fibrosis transmembrane regulator (CFTR) gene that… | Jul 2025 | Deferred | 110 individuals |
| elexacaftor/tezacaftor/ivacaftor Extension of listing for cystic fibrosis in patients aged 2 years and older with at least one mutation in the CFTR gene that is responsive to… | Mar 2025 | Recommended with restriction | 34 individuals |
| ivacaftor Cystic fibrosis in patients aged 1 to less than 4 months with either a G551D mutation or other gating (Class III) mutation in the CFTR gene on at least 1… | Mar 2025 | Recommended with restriction | 4 individuals |
| elexacaftor with tezacaftor and with ivacaftor, and ivacaftor Extension of listing for cystic fibrosis treatment in patients aged 2 to 5 years who have at least one F508del mutation in the CFTR gene. | Mar 2024 | Recommended with restriction | 359 individuals Cystic Fibrosis Australia |
| lumacaftor with ivacaftor Treatment of cystic fibrosis in patients aged 1 to less than 2 years who are homozygous for the F508del mutation in the cystic fibrosis transmembrane… | Jul 2023 | Recommended with restriction | unclear |
| elexacaftor/tezacaftor/ivacaftor 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… | Mar 2023 | Recommended with restriction | 273 individuals Cystic Fibrosis Australia |
| ivacaftor Treatment of cystic fibrosis in patients aged 4 months and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation… | Nov 2023 | Recommended with restriction | 11 individuals Cystic Fibrosis Australia · United States Cystic Fibrosis Foundation |
| elexacaftor/tezacaftor/ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance… | Jul 2021 | Deferred | input received Cystic Fibrosis Australia |
| lumacaftor and ivacaftor Treatment of cystic fibrosis patients homozygous for the F508 deletion mutation in the CFTR gene, across multiple age groups (2 years and older). | Jul 2021 | Noted | 5 individuals US Cystic Fibrosis Foundation · US CF Foundation |
| elexacaftor/tezacaftor/ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance… | May 2021 | Deferred | input received Cystic Fibrosis Australia |
| elexacaftor/tezacaftor/ivacaftor Treatment of cystic fibrosis patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator… | Mar 2021 | Deferred | input received Cystic Fibrosis Australia |
| elexacaftor/tezacaftor/ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance… | Dec 2021 | Recommended with restriction | no section in document |
| lumacaftor and ivacaftor Treatment of cystic fibrosis patients homozygous for the F508del mutation in the CFTR gene, aged 2 years and older. | Dec 2021 | Noted | 5 individuals US Cystic Fibrosis Foundation · US CF Foundation |
| lumacaftor with ivacaftor Treatment of cystic fibrosis in patients aged 2 to 5 years who are homozygous for the F508del mutation in the CFTR gene. | Jul 2019 | Recommended with restriction | 419 individuals |
| ivacaftor Treatment of cystic fibrosis in patients aged 12 to 24 months who have a G551D mutation or other class III gating mutations in the cystic fibrosis… | Mar 2019 | Recommended with restriction | 50 individuals Cystic Fibrosis Australia |
| tezacaftor with ivacaftor Treatment of patients with cystic fibrosis aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. | Mar 2019 | Recommended with restriction | 137 individuals |
| tezacaftor with ivacaftor Treatment of cystic fibrosis patients aged 12 years and older who have at least one residual function (RF) mutation in the CFTR gene. | Mar 2019 | Recommended with restriction | 137 individuals |
| tezacaftor with ivacaftor Treatment of cystic fibrosis patients aged 12 years and older who have at least one residual function (RF) mutation in the CFTR gene. Extension of previous… | Nov 2019 | Recommended with restriction | 175 individuals |
| lumacaftor with ivacaftor Treatment of cystic fibrosis in patients aged 6–11 years who are homozygous for the F508del mutation in the CFTR gene. | Jul 2018 | Deferred | 3,980 individuals Cystic Fibrosis Specialist Interest Group · Cystic Fibrosis Australia |
| lumacaftor with ivacaftor Treatment of cystic fibrosis in patients aged ≥12 years who are homozygous for the F508del mutation in the CFTR gene. | Jul 2018 | Recommended with restriction | 3,980 individuals Cystic Fibrosis Specialist Interest Group · Cystic Fibrosis Australia |
| lumacaftor and ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. | Jul 2017 | Not recommended | 214 individuals Cystic Fibrosis Australia |
| mannitol Cystic fibrosis in patients aged 6 years and over who are inadequately responsive to dornase alfa, for use in combination with dornase alfa or as monotherapy… | Jul 2017 | Recommended with restriction | 18 individuals Cystic Fibrosis Australia |
| lumacaftor with ivacaftor Cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. | Mar 2016 | Not recommended | 594 individuals Cystic Fibrosis Australia |
| ivacaftor Treatment of cystic fibrosis in patients aged 2 to 5 years who have a G551D mutation or other class III gating mutations in the cystic fibrosis transmembrane… | Nov 2016 | Deferred | 186 individuals Cystic Fibrosis Australia |
| lumacaftor with ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508 deletion mutation in the cystic fibrosis transmembrane… | Nov 2016 | Not recommended | 507 individuals Cystic Fibrosis Australia |
| ivacaftor Cystic fibrosis in patients aged 6 years and older who have a G551D or other gating (class III) mutation in the CFTR gene, with flexibility in dosing for… | Jul 2015 | Deferred | 2 individuals |
| mannitol Treatment of cystic fibrosis in patients 6 years of age or older with FEV1 >30% predicted, to improve airway clearance and reduce respiratory complications. | Jul 2015 | Recommended with restriction | input received |
| ivacaftor Treatment of cystic fibrosis in patients aged 6 years and older with a G551D or other gating (class III) mutation in the CFTR gene who have severe CF disease… | Nov 2015 | Noted | none received |
| ivacaftor Treatment of cystic fibrosis in patients aged six years and older who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. | Mar 2014 | Recommended with restriction | unclear |
| ivacaftor Treatment of cystic fibrosis in patients aged six years and older who have a G551D or other gating (class III) mutation in the CFTR gene. | Nov 2014 | Recommended with restriction | none received |
| ivacaftor Treatment of cystic fibrosis in patients aged 6 years and older who have a G551D mutation in the CFTR gene. | Jul 2013 | Deferred | no section in document |
| ivacaftor Treatment of cystic fibrosis in patients aged 6 years and older who have a G551D mutation in the CFTR gene. | Nov 2013 | Deferred | no section in document |
| mannitol Treatment of cystic fibrosis in paediatric (six years and above) and adult populations as either add-on therapy to dornase alfa or in patients intolerant to,… | Mar 2011 | Recommended with restriction | unclear |
| mannitol Treatment of cystic fibrosis in patients 6 years of age or older who are intolerant of or inadequately responsive to dornase alfa. | Nov 2011 | Not recommended | no section in document |
| mannitol Treatment of cystic fibrosis in patients 6 years of age or older who are intolerant of or inadequately responsive to dornase alfa. | Nov 2011 | Not recommended | no section in document |
| aztreonam Control of gram-negative bacteria, particularly Pseudomonas aeruginosa, in the respiratory tract of patients with moderate to severe cystic fibrosis. | Nov 2010 | Not recommended | no section in document |
Who spoke
Cystic Fibrosis Australia · Cystic Fibrosis Specialist Interest Group · US Cystic Fibrosis Foundation · US CF Foundation · United States Cystic Fibrosis Foundation
What the PBS pays
$2,889M in government benefit over 2024–25, across 137,179 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| elexacaftor/tezacaftor/ivacaftor | $697.2M |
| tezacaftor with ivacaftor | $697.2M |
| elexacaftor with tezacaftor and with ivacaftor, and ivacaftor | $647.1M |
| vanzacaftor/tezacaftor/deutivacaftor | $647.1M |
| ivacaftor | $50.1M |
| lumacaftor and ivacaftor | $50.1M |
| lumacaftor with ivacaftor | $50.1M |
| lumacaftor/ivacaftor | $50.1M |
| mannitol | $0.5M |
Appraised elsewhere
NICE, in England and Wales, has appraised 3 technologies for this condition, recommending 3 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| vanzacaftor tezacaftor deutivacaftorVanzacaftor-tezacaftor-deutivacaftor for treating cystic fibrosis with 1 or more F508del mutations in the CFTR gene in people 6 years and over | TA1085 | recommended restricted | 2025-07 |
| ivacaftor tezacaftor elexacaftor tezacaftor ivacaftor and lumacaftor ivacaftorIvacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis | TA988 | recommended | 2024-07 |
| mannitol dry powderMannitol dry powder for inhalation for treating cystic fibrosis | TA266 | recommended | 2012-11 |
Registered, not subsidised
Medicines on the ARTG for this condition with no PBAC record at all. Registration and subsidy are separate decisions: a medicine can be legally available in Australia and never have been put to the committee.
| Medicine | On the ARTG since | Registrations |
|---|---|---|
| dornase alfaPULMOZYME Dornase alfa 1mg/mL spray solution | 1994 | 1 |
The ARTG export holds current registrations only, so the year is the earliest entry still on the register rather than a first-registration date. The condition here comes from the registered label, which is usually broader than the population a sponsor asks to have subsidised.
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 25 of 30 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.