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

Transthyretin amyloid cardiomyopathy

First PBS-subsidised prescription for this drug

The condition must have documented evidence of transthyretin precursor protein present; AND

Patient must have experienced at least one episode of hospitalisation that was a direct result of heart failure; or

Patient must have clinical evidence of heart failure without hospitalisation that required treatment with a diuretic for improvement; AND

Patient must have/have had New York Heart Association class I heart failure at the time of commencing this drug; or

Patient must have/have had New York Heart Association class II heart failure at the time of commencing this drug; AND

Patient must have an end-diastolic interventricular septal wall thickness of at least 12 mm on imaging; AND

Patient must have an estimated glomerular filtration rate (eGFR) greater than 25 mL/minute/1.73 m2; AND

Must be treated by a medical practitioner who is any of the following: (i) a cardiologist, (ii) a consultant physician with experience in the management of amyloid disorders; this authority application must be sought by the same medical practitioner providing treatment.

Applications for authorisation of initial treatment must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail.

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

(a) details of the proposed prescription; and

(b) 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).

Evidence of clinical findings to establish the diagnosis:

In this authority application, confirm that there is documented evidence of transthyretin precursor protein through either (1) alone, or, both (2) and (3), from the list below:

Confirm the following has been completed:

(1) amyloid expert centre histology findings derived via immunohistochemistry or mass spectrometry; OR

(2) bone scintigraphy with grade 2-3 finding

AND

(3) Confirm that there are negative results for monoclonal protein on each of the following three tests:

(a) serum immunofixation (also known as protein electrophoresis)

(b) urine immunofixation

(c) serum free light chains blood test

State which of (1) to (3) above has been completed, as well as the:

(i) date of the finding,

(ii) imaging/pathology report number/code that links the finding to the patient,

(iii) name of the amyloid expert centre in this authority application (if applicable).

For end-diastolic interventricular septal wall thickness (at least 12 mm), confirm that:

(i) imaging (echocardiogram or magnetic resonance imaging) has been undertaken; and

(ii) that the imaging report is stored in the patient's medical records.

State the date that the imaging was performed and the thickness (in mm) in this authority application.

Where this authority application is to transition a patient from non-PBS-subsidised to PBS-subsidised supply (i.e. a 'grandfathered' patient), confirm the following:

(i) the patient's heart failure has not worsened to persistent New York Heart Association Class III/IV heart failure while taking this drug.

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

Transthyretin amyloid cardiomyopathy

Second and subsequent PBS-subsidised prescriptions for this drug

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

Patient must have an estimated glomerular filtration rate (eGFR) greater than 25 mL/minute/1.73 m2; AND

The treatment must be ceased where the patient's heart failure has worsened to persistent New York Heart Association (NYHA) Class III/IV heart failure; AND

The treatment must be ceased where the patient has received any of: (i) a heart transplant, (ii) a liver transplant, (iii) an implanted ventricular assist device; AND

Must be treated by a medical practitioner who is any of the following: (i) a cardiologist, (ii) a consultant physician with experience in the management of amyloid disorders; this authority application must be sought by the same medical practitioner providing treatment.

Confirm whether heart failure has worsened to NYHA Class III/IV since the last authority application (yes/no).

If 'no', continued PBS subsidy is available.

If 'yes', continued PBS subsidy is available, but the prescriber must undertake a review of the patient within 3 months to determine whether the worsening heart failure was transient or persistent.

Where this subsequent clinical review finds that the heart failure persists as NYHA Class III/IV heart failure despite active treatment with this drug, then PBS subsidy is not available.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 2

If heart failure has worsened to NYHA Class III/IV since the last authority application, no more than 2 repeat prescriptions must be prescribed.