Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Cystic fibrosis

Initial treatment

Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND

Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND

Patient must have at least one mutation in the CFTR gene that is considered responsive to elexacaftor/tezacaftor/ivacaftor potentiation based on clinical and/or in vitro assay data; AND

The treatment must be given concomitantly with standard therapy for this condition; AND

Patient must have either chronic sinopulmonary disease or gastrointestinal and nutritional abnormalities, prior to initiating treatment with this drug; AND

The treatment must be the sole PBS-subsidised cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy for this condition; AND

Patient must be 2 to 5 years of age.

For the purposes of this restriction, the list of mutations considered to be responsive to elexacaftor/tezacaftor/ivacaftor is defined in the TGA approved Product Information (PI). Mutations that are not listed in the TGA approved PI but considered to be responsive to elexacaftor/tezacaftor/ivacaftor can be accepted with a confirmation that these patients do not harbour two Class I mutations.

This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information.

The authority application must be via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include:

(1) details of the pathology report substantiating the specific mutation considered to be responsive to elexacaftor/tezacaftor/ivacaftor as listed in the TGA approved PI - quote each of the: (i) specific mutation, and if the specific mutation is not listed in the TGA approved PI, confirmation that the patient does not harbour two Class I mutations, (ii) name of the pathology report provider, (iii) date of pathology report, (iv) unique identifying number/code that links the pathology result to the individual patient; and

(2) current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Cystic fibrosis

Continuing treatment

Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND

Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND

Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND

The treatment must be given concomitantly with standard therapy for this condition; AND

The treatment must be the sole PBS-subsidised cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy for this condition; AND

Patient must be 2 to 5 years of age.

This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information.

The authority application must be via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include: current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).