cystic fibrosis
Rare disease · 2010–2025
36decisions
10medicines
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 CFTR gene that is responsive to… | Jul 2025 | Recommended with restriction | 110 individuals |
| 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… | Mar 2025 | Recommended with restriction | 34 individuals |
| ivacaftor 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… | Mar 2025 | Recommended with restriction | 4 individuals |
| elexacaftor with tezacaftor and with ivacaftor, and ivacaftor Treatment of cystic fibrosis 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 CFTR gene. | 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 CFTR gene. The submission addressed five… | Jul 2021 | Recommended with restriction | input received Cystic Fibrosis Australia |
| lumacaftor and ivacaftor Treatment of cystic fibrosis patients homozygous for the F508del mutation in the CFTR gene, aged 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 in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance… | 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 F508 deletion mutation in the CFTR gene, across multiple age groups (2 years and older). | Dec 2021 | Noted | 5 individuals US Cystic Fibrosis Foundation · US CF Foundation |
| lumacaftor/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 in patients aged 12 years or older who have at least one residual function (RF) mutation in the CFTR gene. | 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 | Recommended with restriction | 3,980 individuals |
| 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 | Not recommended | 3,980 individuals |
| lumacaftor and ivacaftor Treatment of cystic fibrosis in patients aged 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance… | Jul 2017 | Not recommended | 214 individuals |
| mannitol Treatment of cystic fibrosis patients aged 6 years and above who are inadequately responsive to dornase alfa, to enable combination therapy with dornase alfa. | Jul 2017 | Recommended with restriction | 18 individuals |
| lumacaftor with ivacaftor Treatment of 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 |
| ivacaftor Cystic fibrosis in patients aged 2 years and older who have a G551D mutation or other class III gating mutations in the cystic fibrosis transmembrane… | Nov 2016 | Deferred | 186 individuals |
| 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 |
| ivacaftor Cystic fibrosis in patients aged 6 years and older who have a G551D or other gating (class III) mutation in the CFTR gene. | Jul 2015 | Deferred | 2 individuals |
| mannitol Treatment of cystic fibrosis in paediatric and adult populations six years and above, as either add-on therapy to dornase alfa or in patients intolerant to, or… | Jul 2015 | Not recommended | input received Thoracic Society |
| ivacaftor Treatment of cystic fibrosis in patients aged 6 years and older with a G551D or other gating (class III) mutation in the cystic fibrosis transmembrane… | Nov 2015 | Recommended with restriction | 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 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 | Recommended | no section in document |
| mannitol treatment of cystic fibrosis (CF) in both paediatric and adult populations six years and above as either add-on therapy to dornase alfa or in patients… | Mar 2011 | Not recommended | unclear |
| mannitol treatment of cystic fibrosis (CF) in both paediatric and adult populations six years and above who are intolerant or inadequately responsive to dornase alfa,… | Nov 2011 | Not recommended | no section in document |
| mannitol Treatment of cystic fibrosis in patients 6 years and above who are intolerant or inadequately responsive to dornase alfa, including those who have failed… | 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 who meet… | 2010 | Not recommended | no section in document |
Who spoke
Cystic Fibrosis Australia · US Cystic Fibrosis Foundation · US CF Foundation · United States Cystic Fibrosis Foundation · Thoracic Society
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.