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

Progressive familial intrahepatic cholestasis (PFIC)

Dose modification (Dose escalation from initial treatment phase; for PFIC diagnosed after turning 18 years of age)

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

Patient must not have demonstrated an adequate clinical response after at least 1 month and up to 3 months following commencement of treatment at 40 micrograms/kg/day; AND

Patient must be undergoing treatment with this drug for this condition at 120 micrograms/kg/day; AND

Must be treated by a specialist hepatologist experienced in the management of progressive familial intrahepatic cholestasis (PFIC); or

Must be treated by a medical practitioner under the supervision of a specialist experienced in the management of progressive familial intrahepatic cholestasis (PFIC).

At the time of the authority application, the prescriber should request sufficient quantity and repeats to provide for the balance to complete up to 6 months of treatment (inclusive of initial and dose modification treatment), if dose modification is being accessed following initial treatment.

An application for continuing treatment must occur following an assessment of response conducted up to a maximum of 6 months from initiation of therapy. This will enable ongoing treatment for those who meet the continuing restriction for PBS-subsidised treatment.

If a patient fails to demonstrate a response to treatment 6 months after initiating treatment with this drug for this condition (inclusive of initial and dose modification treatment), they will no longer be eligible to receive PBS-subsidised treatment with this drug for this condition.

An adequate clinical response under this treatment phase 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.

Confirmation of eligibility for dose modification treatment must be documented in the patient's medical records (include assessment of response score after at least 1 month and up to 3 months after commencing initial treatment).

Any further authority applications occurring immediately after access through this dose modification listing are not to occur through the initial treatment listing.

Dose modification

Where the drug's Product Information indicates variable dosing regimens based on the individual's response/tolerance, apply under this listing to continue treatment with the new strength. Mark any remaining repeat prescriptions for the discontinued strength with the word 'cancelled'. This treatment phase listing recognises that a patient's optimal dose may not always be immediately apparent at the time of treatment initiation and therefore does not require confirmation of an objective, adequate response to the preceding supply of drug.

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