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

Hereditary transthyretin amyloidosis

Initial treatment

Patient must not have previously received PBS-subsidised treatment with this drug for this PBS indication; AND

The condition must be hereditary transthyretin amyloidosis confirmed by genetic testing; AND

Patient must have a Polyneuropathy Disability (PND) score description of either I, II, IIIA, IIIB; or

Patient must have a Familial Amyloid Polyneuropathy (FAP) stage description of 1 or 2; AND

Patient must not have previously undergone a liver transplant; AND

Patient must not exhibit heart failure symptoms (defined as New York Heart Association NYHA class III or IV); AND

Must be treated by a consultant with experience in the management of amyloid disorders or in consultation with a consultant with experience in the management of amyloid disorders; AND

Patient must be undergoing treatment with this drug as a monotherapy (i.e. not in combination with any other disease modifying medicines for amyloidosis disorders); AND

Patient must have either: (i) stage 1 polyneuropathy, (ii) stage 2 polyneuropathy; AND

Patient must be at least 18 years of age.

PND scores in the context of this PBS restriction are:

Stage 0 - No symptoms;

Stage I - Sensory disturbances but preserved walking capability;

Stage II - Impaired walking capacity but able to walk without stick or crutches;

Stage IIIA - Walking with help of one stick or crutch;

Stage IIIB - Walking with help of two sticks or crutches;

Stage IV - Confined to wheelchair or bedridden.

FAP stage in the context of this PBS restriction are:

Stage 0 - No symptoms;

Stage 1 - Unimpaired ambulation;

Stage 2 - Assistance with ambulation required;

Stage 3 - Wheelchair-bound or bedridden.

Applications for authorisation under this treatment phase 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).

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

Hereditary transthyretin amyloidosis

Continuing treatment

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

Patient must continue to demonstrate clinical benefit; AND

Patient must not have previously undergone a liver transplant; AND

Patient must not be permanently bedridden; or

Patient must not be receiving end-of-life care; AND

Must be treated by a consultant with experience in the management of amyloid disorders or in consultation with a consultant with experience in the management of amyloid disorders; AND

Patient must be undergoing treatment with this drug as a monotherapy (i.e. not in combination with any other disease modifying medicines for amyloidosis disorders).

Applications for authorisation under this treatment phase 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).