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

Paroxysmal nocturnal haemoglobinuria (PNH)

Initial treatment - Initial 1 (new untreated patient)

Patient must not have received prior treatment with any of the PBS-subsidised therapies listed for this condition; AND

Patient must have a diagnosis of PNH established by flow cytometry; AND

Patient must have PNH granulocyte clone size equal to or greater than 10%; AND

Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal; AND

Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy; or

Patient must have been transfused with at least 4 units of red blood cells in the last 12 months; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 70 g/L in the absence of anaemia symptoms; or

Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 100 g/L in addition to having anaemia symptoms; or

Patient must have debilitating shortness of breath/chest pain resulting in limitation of normal activity (New York Heart Association Class III) and/or established diagnosis of pulmonary arterial hypertension, where causes other than PNH have been excluded; or

Patient must have a history of renal insufficiency, demonstrated by an eGFR less than or equal to 60 mL/min/1.73m2, where causes other than PNH have been excluded; or

Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details; AND

Patient must be at least 18 years of age.

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

(1) details of the proposed prescription(s); and

(2) 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).

At the time of the authority application, medical practitioners must request the appropriate number of vials for 4 weeks supply per dispensing as per the Product Information.

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place)