National Health (Highly Specialised Drugs Program) Special Arrangement Amendment (March Update) Instrument 2022 (PB 14 of 2022) (Cth)

Case

PB 14 of 2022

National Health (Highly Specialised Drugs Program) Special Arrangement Amendment (March Update) Instrument 2022

National Health Act 1953

I, DAVID LAFFAN, Assistant Secretary, Pharmacy Branch, Technology Assessment and Access Division, Department of Health, delegate of the Minister for Health and Aged Care, make this Instrument under subsection 100(2) of the National Health Act 1953.

Date                 28th February 2022

DAVID LAFFAN

Assistant Secretary

Pharmacy Branch

Technology Assessment and Access Division

Contents

1......... Name............................................................................................................................... 1

2......... Commencement............................................................................................................... 1

3......... Authority......................................................................................................................... 1

4......... Schedules......................................................................................................................... 1

Schedule 1—Amendments  2

National Health (Highly Specialised Drugs Program) Special Arrangement 2021
(PB 27 of 2021)   2

  1. Name

(1)This instrument is the National Health (Highly Specialised Drugs Program) Special Arrangement Amendment (March Update) Instrument 2022.

(2)This instrument may also be cited as PB 14 of 2022.

  1. Commencement

(1)Each provision of this instrument specified in column 1 of the table commences, or is taken to have commenced, in accordance with column 2 of the table. Any other statement in column 2 has effect according to its terms.

Commencement information
Column 1 Column 2 Column 3
Provisions Commencement Date/Details
1.  The whole of this instrument 1 March 2022 1 March 2022

Note:          This table relates only to the provisions of this instrument as originally made. It will not be amended to deal with any later amendments of this instrument.

(2)Any information in column 3 of the table is not part of this instrument. Information may be inserted in this column, or information in it may be edited, in any published version of this instrument.

  1. Authority

This instrument is made under subsection 100(2) of the National Health Act 1953.

  1. Schedules

Each instrument that is specified in a Schedule to this instrument is amended or repealed as set out in the applicable items in the Schedule concerned, and any other item in a Schedule to this instrument has effect according to its terms.

Schedule 1—Amendments

National Health (Highly Specialised Drugs Program) Special Arrangement 2021 (PB 27 of 2021)

  1. Section 6, definition for “CAR drug”

substitute:

CAR drug (short for Complex Authority Required drug) means any of the following highly specialised drugs:

(a)  abatacept;

(b)  adalimumab;

(c)  ambrisentan;

(d)  azacitidine;

(e)  benralizumab;

(f)  bosentan;

(g)  dupilumab;

(h)  eculizumab;

(i)  eltrombopag;

(j)  epoprostenol;

(k)  etanercept;

(l)  iloprost;

(m)  infliximab;

(n)  ivacaftor;

(o)  lenalidomide;

(p)  lumacaftor with ivacaftor;

(q)  macitentan;

(r)  mepolizumab;

(s)  midostaurin;

(t)  nusinersen;

(u)  omalizumab;

(v)  pasireotide;

(w)  pegvisomant;

(x)  pomalidomide;

(y)  ravulizumab;

(z)  riociguat;

(aa)  risdiplam;

(bb)  rituximab;

(cc)  romiplostim;

(dd)  sildenafil;

(ee)  tadalafil;

(ff)  teduglutide;

(gg)  tezacaftor with ivacaftor and ivacaftor;

(hh)  tocilizumab;

(ii)  ustekinumab;

(jj) vedolizumab.

  1. Subsection 8(3)

repeal the subsection (including heading), substitute:

Persons receiving treatment by medical practitioners in public hospitals as admitted patients—HSD pharmaceutical benefits that contain eculizumab for the treatment of atypical haemolytic uraemic syndrome

(3)  A person is an eligible patient for an HSD pharmaceutical benefit that contains eculizumab for the treatment of atypical haemolytic uraemic syndrome if the person:

(a)  is, or is to be treated as, an eligible person; and

(b)  is receiving medical treatment by a medical practitioner in a public hospital; and

(c)  is receiving that treatment as an admitted patient (other than a day admitted patient) of the hospital.

  1. Section 23 (heading)

after “eculizumab”, insert: for the treatment of atypical haemolytic uraemic syndrome

  1. Subsection 23(1)

after “eculizumab”, insert: for the treatment of atypical haemolytic uraemic syndrome

  1. Schedule 1, entry for Eculizumab

insert in numerical order in the column headed “Circumstances”: C12510 C12512 C12515 C12533 C12548 C12560 C12568

  1. Schedule 1, entry for Entecavir in the form Tablet 0.5 mg (as monohydrate)

omit:

Entecavir APOTEX C4993 C5036 60 5

  1. Schedule 1, entry for Entecavir in the form Tablet 1 mg (as monohydrate)

omit:

Entecavir APOTEX C5037 C5044 60 5

  1. Schedule 1, after entry for Etanercept in the form Injection 50 mg in 1 mL single use auto‑injector, 4

insert:

Injection 50 mg in 1 mL single use dose-dispenser cartridges, 4 Injection Enbrel C9384 C9417 C10548 C10578 C10579 C10599 C12357 C12407 See Schedule 2 See Schedule 2

  1. Schedule 1, entry for Methoxsalen

substitute:

Methoxsalen Solution for blood fraction 20 microgram per mL, 10 mL Extracorporeal Circulation Uvadex C10971 C10985 C10988 C10989 C12531 C12546 C12567 C12579 P10988 P10989 1 5
C10971 C10985 C10988 C10989 C12531 C12546 C12567 C12579 P12531 P12567 2 0
C10971 C10985 C10988 C10989 C12531 C12546 C12567 C12579 P10971 P10985 2 6
C10971 C10985 C10988 C10989 C12531 C12546 C12567 C12579 P12546 P12579 12 1

  1. Schedule 1, after entry for Raltegravir in the form Tablet 600 mg (as potassium)

insert:

Ravulizumab Solution concentrate for I.V. infusion 300 mg in 3 mL Injection Ultomiris C12509 C12511 C12517 C12518 C12519 C12575 See Schedule 2 See Schedule 2
Solution concentrate for I.V. infusion 1,100 mg in 11 mL Injection Ultomiris C12509 C12511 C12517 C12518 C12519 C12575 See Schedule 2 See Schedule 2

  1. Schedule 1, entry for Sildenafil

omit:

APO‑Sildenafil PHT C11229 C11319 C11338 C11350 C12430 C12431 C12441 C12443 C12456 See Schedule 2 See Schedule 2

  1. Schedule 1, entry for Valaciclovir

omit:

APO‑Valaciclovir C5975 C9267 500 2

  1. Schedule 2, entry for Eculizumab

substitute:

Eculizumab C6626 1 Sufficient for 4 weeks of treatment
C6642 1 4
C6668 C6686 C6687 C6688 1 5
C6637 1 6
C12510 1 0
C12512 C12515 C12548 C12560 C12568 6 5
C12533 8 0

  1. Schedule 2, after entry for Pomalidomide

insert:

Ravulizumab C12517 C12518 C12519 1 dose 0
C12509 C12511 C12575 1 dose 2

  1. Schedule 3, entry for Eculizumab

insert in numerical order after existing text:

C12510 P12510 Paroxysmal nocturnal haemoglobinuria (PNH)
Balance of Supply (transition from non-PBS-subsidised treatment during induction phase)
Patient must have received non-PBS-subsidised eculizumab for this condition prior to 1 March 2022; AND
Patient must have received insufficient quantity to complete the induction treatment phase; AND
Patient must have a diagnosis of PNH established by flow cytometry prior to commencing treatment with eculizumab; AND
Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to commencing treatment with eculizumab; AND
Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to commencing treatment with eculizumab; AND
Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to commencing treatment with eculizumab; OR
Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; OR
Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to commencing treatment with eculizumab; AND
The treatment must not be in combination with ravulizumab.
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.
At the time of the authority application, medical practitioners should request the appropriate number of vials to complete the induction treatment phase, 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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12512 P12512 Paroxysmal nocturnal haemoglobinuria (PNH)
Grandfather 1 (transition from non-PBS-subsidised treatment) - maintenance phase
Patient must have received non-PBS-subsidised eculizumab for this condition prior to 1 March 2022; AND
Patient must have a diagnosis of PNH established by flow cytometry prior to commencing treatment with eculizumab; AND
Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to commencing treatment with eculizumab; AND
Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to commencing treatment with eculizumab; AND
Patient must have demonstrated clinical improvement or stabilisation of condition; AND
Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to commencing treatment with eculizumab; OR
Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; OR
Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to commencing treatment with eculizumab; AND
The treatment must not be in combination with ravulizumab.
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.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12515 P12515 Paroxysmal nocturnal haemoglobinuria (PNH)
First Continuing Treatment
Patient must have received PBS-subsidised treatment with this drug for this condition under an 'Initial', 'Balance of Supply', or 'Grandfather' treatment criteria; AND
The treatment must not be in combination with ravulizumab.
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.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12533 P12533 Paroxysmal nocturnal haemoglobinuria (PNH)
Initial treatment - Initial 1 (new patient) induction doses
Patient must not have received prior treatment with this drug for this condition; AND
Patient must have a diagnosis of PNH established by flow cytometry; AND
Patient must have a 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 not be in combination with ravulizumab.
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.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12548 P12548 Paroxysmal nocturnal haemoglobinuria (PNH)
Grandfather 2 (transition from LSDP-funded eculizumab)
Patient must have previously received eculizumab for the treatment of this condition funded under the Australian Government's Life Saving Drugs Program (LSDP); AND
Patient must have a diagnosis of PNH established by flow cytometry prior to commencing treatment with eculizumab; AND
Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to commencing treatment with eculizumab; AND
Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to commencing treatment with eculizumab; AND
Patient must have demonstrated clinical improvement or stabilisation of condition; AND
Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to commencing treatment with eculizumab; OR
Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; 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 prior to commencing treatment with eculizumab; OR
Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to commencing treatment with eculizumab; AND
The treatment must not be in combination with ravulizumab.
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.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12560 P12560 Paroxysmal nocturnal haemoglobinuria (PNH)
Initial treatment - Initial 2 (switching from PBS-subsidised ravulizumab for pregnancy)
Patient must be planning pregnancy; OR
Patient must be pregnant; AND
Patient must have received PBS-subsidised treatment with ravulizumab for this condition; AND
The treatment must not be in combination with ravulizumab.
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.
Patient may qualify under this treatment phase more than once. In the event of miscarriage, patient may continue on eculizumab if patient is stable, and/or is planning a subsequent pregnancy. For continuing PBS-subsidised treatment, a 'Switching' patient must proceed under the 'Subsequent Continuing Treatment' criteria. 		Compliance with Written Authority Required procedures
C12568 P12568 Paroxysmal nocturnal haemoglobinuria (PNH)
Subsequent Continuing Treatment
Patient must have previously received PBS-subsidised treatment with this drug for this condition under the 'First Continuing Treatment' or 'Switch' criteria; AND
Patient must have demonstrated clinical improvement or stabilisation of condition; AND
The treatment must not be in combination with ravulizumab.
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. 		Compliance with Authority Required procedures

  1. Schedule 3, entry for Methoxsalen

insert in numerical order after existing text:

C12531 P12531 Chronic graft versus host disease
Continuing treatment
Patient must have received, at anytime prior to this pharmaceutical benefit within the same treatment episode, both: (i) this drug subsidised through the Initial treatment listing, (ii) the extracorporeal photopheresis-MBS benefit for initial treatment; AND
Patient must have demonstrated a response to initial treatment with this drug (administered as part of MBS-subsidised extracorporeal photopheresis treatment) obtained through this drug's 'Initial treatment' PBS-listing for the same treatment episode.
Must be treated by a haematologist; OR
Must be treated by an oncologist with allogeneic bone marrow transplantation experience; OR
Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types; AND
Patient must be undergoing concurrent treatment with extracorporeal photopheresis as described in the Medicare Benefits Schedule for this condition; AND
Patient must not be undergoing re-treatment through this treatment phase immediately following a relapse - see 'Initial treatment' for resuming treatment following relapse. 		Compliance with Authority Required procedures - Streamlined Authority Code 12531
C12546 P12546 Chronic graft versus host disease
Initial treatment in a treatment episode
The condition must be inadequately responsive to systemic corticosteroid treatment at a therapeutic dose, but has never been treated with this drug; OR
The condition must have relapsed within 8 weeks of prior PBS-subsidised treatment with this drug administered via extracorporeal photopheresis; OR
The condition must have relapsed with each of the following conditions being met: (i) prior PBS-subsidised treatment with this drug administered via extracorporeal photopheresis last occurred at least 8 weeks ago, (ii) a subsequent trial of systemic corticosteroids at therapeutic doses has been completed.
Patient must be undergoing treatment with this drug that is being administered within at least one of: (i) the first 12 weeks of a treatment episode, (ii) the first 25 doses (inclusive of the 25thdose) of a treatment episode; AND
Must be treated by a haematologist; OR
Must be treated by an oncologist with allogeneic bone marrow transplantation experience; OR
Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types; AND
Patient must be undergoing treatment with this drug following allogeneic haematopoietic stem cell transplantation; AND
Patient must be undergoing concurrent treatment with extracorporeal photopheresis as described in the Medicare Benefits Schedule for this condition. 		Compliance with Authority Required procedures - Streamlined Authority Code 12546
C12567 P12567 Chronic graft versus host disease
Continuing treatment
Patient must have received, at anytime prior to this pharmaceutical benefit within the same treatment episode, both: (i) this drug subsidised through the Initial treatment listing, (ii) the extracorporeal photopheresis-MBS benefit for initial treatment; AND
Patient must have demonstrated a response to initial treatment with this drug (administered as part of MBS-subsidised extracorporeal photopheresis treatment) obtained through this drug's 'Initial treatment' PBS-listing for the same treatment episode.
Must be treated by a haematologist; OR
Must be treated by an oncologist with allogeneic bone marrow transplantation experience; OR
Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types; AND
Patient must be undergoing concurrent treatment with extracorporeal photopheresis as described in the Medicare Benefits Schedule for this condition; AND
Patient must not be undergoing re-treatment through this treatment phase immediately following a relapse - see 'Initial treatment' for resuming treatment following relapse. 		Compliance with Authority Required procedures - Streamlined Authority Code 12567
C12579 P12579 Chronic graft versus host disease
Initial treatment in a treatment episode
The condition must be inadequately responsive to systemic corticosteroid treatment at a therapeutic dose, but has never been treated with this drug; OR
The condition must have relapsed within 8 weeks of prior PBS-subsidised treatment with this drug administered via extracorporeal photopheresis; OR
The condition must have relapsed with each of the following conditions being met: (i) prior PBS-subsidised treatment with this drug administered via extracorporeal photopheresis last occurred at least 8 weeks ago, (ii) a subsequent trial of systemic corticosteroids at therapeutic doses has been completed.
Patient must be undergoing treatment with this drug that is being administered within at least one of: (i) the first 12 weeks of a treatment episode, (ii) the first 25 doses (inclusive of the 25thdose) of a treatment episode; AND
Must be treated by a haematologist; OR
Must be treated by an oncologist with allogeneic bone marrow transplantation experience; OR
Must be treated by a medical practitioner working under the direct supervision of one of the above mentioned specialist types; AND
Patient must be undergoing treatment with this drug following allogeneic haematopoietic stem cell transplantation; AND
Patient must be undergoing concurrent treatment with extracorporeal photopheresis as described in the Medicare Benefits Schedule for this condition. 		Compliance with Authority Required procedures - Streamlined Authority Code 12579

  1. Schedule 3, after entry for Raltegravir

insert:

Ravulizumab C12509 P12509 Paroxysmal nocturnal haemoglobinuria (PNH)
First Continuing Treatment
Patient must have received PBS-subsidised treatment with this drug for this condition under an 'Initial', 'Balance of Supply', or 'Grandfather' treatment criteria; AND
The treatment must not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a maintenance dose based on the patient's weight, as per the Product Information. A maximum of 2 repeats may be requested.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12511 P12511 Paroxysmal nocturnal haemoglobinuria (PNH)
Grandfather (transition from non-PBS-subsidised treatment)
Patient must have received non-PBS-subsidised treatment with this drug for this condition prior to 1 March 2022; AND
Patient must have a diagnosis of PNH established by flow cytometry prior to commencing treatment with ravulizumab; AND
Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to commencing treatment with ravulizumab; AND
Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to commencing treatment with ravulizumab; AND
Patient must have demonstrated clinical improvement or stabilisation of condition, the details of which must be kept with the patient's record; AND
Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to commencing treatment with ravulizumab; OR
Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to commencing treatment with ravulizumab; 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 prior to commencing treatment with ravulizumab; 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 prior to commencing treatment with ravulizumab; 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 prior to commencing treatment with ravulizumab; 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 prior to commencing treatment with ravulizumab; OR
Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to commencing treatment with ravulizumab; AND
The treatment must not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a maintenance dose based on the patient's weight, as per the Product Information. A maximum of 2 repeats may be requested.
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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12517 P12517 Paroxysmal nocturnal haemoglobinuria (PNH)
Initial treatment - Initial 1 (new patient) induction dose
Patient must not have received prior treatment with this drug for this condition; AND
Patient must have a diagnosis of PNH established by flow cytometry; AND
Patient must have a 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 not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, 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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12518 P12518 Paroxysmal nocturnal haemoglobinuria (PNH)
Initial treatment - Initial 2 (switch from LSDP eculizumab) induction dose
Patient must have previously received eculizumab for the treatment of this condition funded under the Australian Government's Life Saving Drugs Program (LSDP); AND
Patient must have a diagnosis of PNH established by flow cytometry prior to LSDP-funded treatment with eculizumab; AND
Patient must have a PNH granulocyte clone size equal to or greater than 10% prior to LSDP-funded treatment with eculizumab; AND
Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to LSDP-funded treatment with eculizumab; AND
Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to LSDP-funded treatment with eculizumab; OR
Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to LSDP-funded treatment with eculizumab; 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 prior to LSDP-funded treatment with eculizumab; 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 prior to LSDP-funded treatment with eculizumab; 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 prior to LSDP-funded treatment with eculizumab; 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 prior to LSDP-funded treatment with eculizumab; OR
Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to LSDP-funded treatment with eculizumab; AND
The treatment must not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, 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) and the upper limit of normal (ULN) for the reporting laboratory
(viii) Multiple of LDH , ULN 		Compliance with Written Authority Required procedures
C12519 P12519 Paroxysmal nocturnal haemoglobinuria (PNH)
Return from PBS-subsidised eculizumab - induction dose
Patient must have received prior PBS-subsidised treatment with this drug for this condition; AND
Patient must have received prior PBS-subsidised treatment with eculizumab through the 'Initial treatment - Initial 2 (switching from PBS-subsidised ravulizumab for pregnancy)' criteria; AND
The treatment must not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a single loading dose based on the patient's weight, as per the Product Information
Patient may qualify under this treatment phase more than once for the purposes of family planning. Where long-term continuing PBS-subsidised treatment with this drug is planned, a 'Returning' patient may proceed under the 'Subsequent Continuing Treatment' criteria. 		Compliance with Written Authority Required procedures
C12575 P12575 Paroxysmal nocturnal haemoglobinuria (PNH)
Subsequent Continuing Treatment
Patient must have previously received PBS-subsidised treatment with this drug for this condition under the 'First Continuing Treatment' or 'Switch' criteria; AND
Patient must have demonstrated clinical improvement or stabilisation of condition; AND
The treatment must not be in combination with eculizumab.
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.
Patient must be aged 18 years or over.
At the time of the authority application, medical practitioners should request the appropriate number of vials for a maintenance dose based on the patient's weight, as per the Product Information. A maximum of 2 repeats may be requested. 		Compliance with Written Authority Required procedures
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