Atypical haemolytic uraemic syndrome (aHUS) Program in Pharmaceutical Benefits Scheme (PBS) 012-18051103

This document outlines details of PBS-subsidised eculizumab and ravulizumab for patients with atypical haemolytic uraemic syndrome (aHUS).

For details on how to process a PBS Authority, see Processing Complex Authority Required Listings.

Restrictions	Authority level and section	PA assessment	Processing system	Prescriber type	Prescriber self-serve
Initial PB119 form	Written Electronic S100: eculizumab ravulizumab	Yes	OPA	Must be treated by, or in consultation with a: nephrologist haematologist	Yes - delayed assessment due to DPMQ (cost)
Grandfather PB359 form	Written Electronic S100: ravulizumab	Yes	OPA	Must be treated by, or in consultation with a: nephrologist haematologist	Yes - delayed assessment due to DPMQ (cost)
Continuing PB125 form	Written Electronic S100: eculizumab ravulizumab	Yes	OPA	Must be treated by, or in consultation with a: nephrologist haematologist	Yes - delayed assessment due to DPMQ (cost)
Recommencement PB176 form	Written Electronic S100: eculizumab ravulizumab	Yes	OPA	Must be treated by, or in consultation with a: nephrologist haematologist	Yes - delayed assessment due to DPMQ (cost)
Balance of supply	Written Electronic S100: eculizumab ravulizumab	Yes	OPA	Must be treated by, or in consultation with a: nephrologist haematologist	Yes - delayed assessment due to DPMQ (cost)

aHUS disease phases

Table 2

Treatment phase	Eculizumab	Ravulizumab
Initial and initial switching criteria	4 week loading dose available, 12 vials and nil repeats, once patient has met criteria	2 week loading dose, dose dependent on body weight See Resources page for the aHUS dosage schedule table
Initial treatment: balance of supply	ADAMTS-13 test must be supplied and patients are then eligible for a further 20 weeks of treatment If ADAMTS-13 test is provided when initial paperwork is submitted, approval can be given at the same time as the initial loading dose 20 weeks of treatment available (4 x repeats) can be given under this restriction i.e. 8 vials if > 40kg See Resources page for the aHUS dosage schedule table if weight is < 40kg	ADAMTS-13 test must be supplied and patients are then eligible for a further 24 weeks of treatment (8 weeks and up to 2 repeats) If ADAMTS-13 test is provided when initial paperwork is submitted, approval can be given at the same time as the initial loading dose See Resources page for the aHUS dosage schedule table
Continuing	Available to patients that meet criteria after they have had the initial, or to patients who received eculizumab under the switch from ravulizumab in initial or continuing treatment phase Total of 80 weeks treatment available under this restriction Or Not more than 104 weeks supply of a C5 inhibitor under the initial and continuing treatment restrictions if patient had switched C5 inhibitors during the course of initial and continuing treatment 24 weeks treatment available per course (5 x repeats) under this restriction. 8 vials if over 40kg see Resources page for the aHUS dosage schedule table if weight is < 40kg Patients have 3 options after having a total of 104 weeks of treatment, if the patient demonstrates a response, and also the following: no treatment failure, and has perilous organ damage, or a high risk of aHUS recurrence in the short term without eculizumab, they will be eligible for extended continuing treatment demonstrate a response and flare whilst off medication, they will be eligible for recommencement failed to respond, no further treatment is available	Available to patients that meet criteria after they have had the initial or grandfather, or to patients who received ravulizumab under the switch from eculizumab in continuing treatment phase Total of 72 weeks treatment available under this restriction Or Not more than 104 weeks supply of a C5 inhibitor under the initial and continuing treatment restrictions if patient had switched C5 inhibitors during the course of initial and continuing treatment 24 weeks treatment available per course (8 weeks and 2 repeats) See Resources page for the aHUS dosage schedule table Patients have 3 options after having a total of 104 weeks of treatment, if the patient demonstrates a response, and also the following: no treatment failure, and has perilous organ damage, or a high risk of aHUS recurrence in the short term without ravulizumab, they will be eligible for extended continuing treatment demonstrate a response and flare whilst off medication, they will be eligible for recommencement failed to respond, no further treatment is available
Extended continuing	Available to patients that have demonstrated a response to the medication and the following: had no treatment failures have perilous organ damage, or a high risk of aHUS recurrence in the short term without eculizumab Once patients are in this restriction 'loop', they are eligible for continuing criteria indefinitely if: they demonstrate a response every 24 weeks with no treatment failure, and perilous organ damage, or a high risk of aHUS recurrence in the short term without eculizumab 24 weeks treatment is available (5 x repeats) under this restriction i.e. 8 vials if over 40kg See Resources page for the aHUS dosage schedule table if weight is < 40kg	Available to patients that have demonstrated a response to the medication and the following: had no treatment failures have perilous organ damage, or a high risk of aHUS recurrence in the short term without ravulizumab Once patients are in this restriction 'loop', they are eligible for continuing criteria indefinitely if: they demonstrate a response every 24 weeks with no treatment failure, and perilous organ damage, or a high risk of aHUS recurrence in the short term without ravulizumab 24 weeks treatment is available (8 weeks and up to 2 repeats) under this restriction See Resources page for the aHUS dosage schedule table
Recommencement	Available for patients that have had treatment with eculizumab, demonstrated a response to treatment and flared while off eculizumab or for patients who received eculizumab under the switch from ravulizumab in the recommencement treatment phase Patients may qualify for a reload to the medication at this stage. 12 vials and nil repeats + 8 vials and 4 repeats (total of 24 weeks) 24 weeks treatment is available (5 x repeats) under this restriction. That is, 8 vials if > 40kg See Resources page for the aHUS dosage schedule table if weight is < 40kg	Available for patients that have demonstrated a response to treatment with a PBS-subsidised C5 inhibitor 2 week loading dose, dose dependent on body weight See Resources page for the aHUS dosage schedule table
Recommencement of treatment: balance of supply	Available to patients who have received eculizumab recommencement supply 20 weeks of treatment available (4 x repeats) can be given under this restriction i.e. 8 vials if > 40kg See Resources page for the aHUS dosage schedule table if weight is < 40kg	Available for patients that have demonstrated a response to treatment with a PBS-subsidised C5 inhibitor 24 weeks of treatment available (8 weeks and up to 2 repeats) See Resources page for the aHUS dosage schedule table
Continuing recommencement	Once patients are in this restriction 'loop' they are eligible for continuing criteria indefinitely if they demonstrate a response every 24 weeks with no treatment failure 24 weeks treatment is available (5 x repeats) under this restriction. 8 vials if > 40kg See Resources page for the aHUS dosage schedule table if weight is < 40kg	Once patients are in this restriction 'loop' they are eligible for continuing criteria indefinitely if they demonstrate a response every 24 weeks with no treatment failure 24 weeks treatment is available (8 weeks and 2 repeats) per course under this restriction See Resources page for the aHUS dosage schedule table
Grandfather	Not applicable	8 weeks and up to 2 repeats can be sought to provide sufficient drug for the balance of the current treatment phase See Resources page for the aHUS dosage schedule table

Escalating aHUS requests and queries

Table 3

All aHUS requests and enquiries MUST be escalated to a Pharmaceutical Adviser (PA). These applications are time critical and have complex criteria which must be assessed by a PA.

Note: eculizumab and ravulizumab are also listed for other conditions.

Application or query	Action
aHUS application received by HPOS or mail	Name the application in WLM (PaNDA) with the patient Medicare number and Program reference 'aHUS' Unassign and recategorise the application to the PAs via the Written Authority Escalation folder in PaNDA. Make sure the application is left open Comment in the aHUS Teams chat 'work item XXXXXX escalated into PA folder'
aHUS request received as delayed assessment	Comment in the aHUS Teams chat 'Delayed assessment received - Authority Unique ID(s)' Recategorise the application to 'Delayed_aHUS_Escalation'
Prescriber calls about an aHUS request or eligibility	Escalate all calls in relation to aHUS to a PA If afterhours, complete a callback form

Operational Information

Atypical haemolytic uraemic syndrome (aHUS) Program in Pharmaceutical Benefits Scheme (PBS) 012-18051103

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