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

Progressive familial intrahepatic cholestasis (PFIC)

Initial treatment (PFIC diagnosed prior to turning 18 years of age)

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

Patient must have a clinical diagnosis of progressive familial intrahepatic cholestasis (PFIC); AND

Patient must have elevated serum bile acids at treatment initiation with this drug; AND

Patient must be experiencing itch at treatment initiation with this drug, with other factors causing pruritus excluded through both: (i) physical examination, (ii) patient history; AND

Patient must have had an average pruritus score of at least 2 on the PrucisionTM ObsRO Pruritus Scale assessed on three occasions over a one-week period prior to treatment initiation with this drug; AND

Must be treated by a specialist experienced in the management of progressive familial intrahepatic cholestasis (PFIC), who is either a: (i) gastroenterologist, (ii) hepatologist; AND

Patient must be aged between 6 months and 17 years inclusive; or

Patient must be/have been diagnosed prior to turning 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 pruritus score using the Prucision^TM ObsRO Pruritus scale;

(2) details of serum bile acids (date and micromol/L).

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).

Prescriber must exclude any other causes of pruritus which include any of the following:

(i) drug related (e.g., opioid-related pruritus);

(ii) drug hypersensitivity or adverse effect; contact dermatitis; allergy;

(iii) differential diagnoses (e.g., xerosis; infestations; iron deficiency; chronic kidney disease; polycythaemia vera/leukaemia/lymphoma; hypothyroidism; uncontrolled diabetes, eczema, scabies).

At the time of the authority application, the prescriber should request the appropriate strength(s) and quantity based on the patient's weight, according to the dosing schedule in the TGA-approved Product Information to provide sufficient drug for one month's supply with 2 repeats. A separate authority approval is required for each strength requested.

An application for continuing treatment must occur following an assessment of response conducted up to 3 months of therapy. This will enable ongoing treatment for those who meet the continuing restriction for PBS-subsidised treatment.

An application for dose modification treatment must occur following an assessment of response conducted from 1 month of therapy up to 3 months of therapy. This will enable ongoing treatment for those who meet the dose modification restriction criteria for PBS-subsidised treatment.

If a patient fails to demonstrate a response to treatment with this drug after 1 month, they will be eligible to receive PBS-subsidised treatment at a higher dose with this drug for this condition under the dose modification treatment phase for a maximum of 5 months; annotate any remaining repeat prescriptions with the word 'cancelled' where this occurs within this treatment phase.

All diagnostic reports/tools must be documented in the patient's medical records.

An adequate clinical response to initial treatment is defined as an average of at least 1 grade improvement in pruritus score on the Prucision^TM ObsRO Pruritus Scale, assessed on 3 occasions over a one-week period prior to assessment of response.

Eligible patients must not be recommencing treatment directly through this treatment phase.