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

Progressive familial intrahepatic cholestasis (PFIC)

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

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

Patient must have demonstrated an adequate clinical response to the most recent course of treatment; or

Patient must be undergoing dose escalation to 120 micrograms/kg/day if they were unable to maintain a previously demonstrated adequate clinical response at 40 micrograms/kg/day in continuing treatment; AND

Patient must not be undergoing treatment with this drug for this condition, where an adequate clinical response was unable to be maintained at 120 micrograms/kg/day; 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; or

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

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 continuing treatment must be provided with the authority application and documented in the patient's medical records (include assessment of adequate clinical response to preceding supply).

A patient may be eligible for treatment at the higher dose (120 micrograms/kg/day), if a loss of response occurred, or if an adequate response was unable to be maintained at the lower dose (40 micrograms/kg/day) during continuing treatment. To be eligible for continuing treatment at the higher dose (120 micrograms/kg/day), the patient must demonstrate and maintain an adequate clinical response at the higher dose.