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PB 37 of 2022 Arrangements as made
This instrument amends the National Health (Highly Specialised Drugs Program) Special Arrangement 2021 to add, delete and make changes to drugs, forms, manners of administration, brands, authorised prescribers, maximum quantities and repeats and the circumstances for prescribing various pharmaceutical benefits (including authority requirements).
Administered by: Health
Registered 29 Apr 2022
Table of contents.

Commonwealth Coat of Arms of Australia

 

PB 37 of 2022

 

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

 

National Health Act 1953

 

I, MARIANA CRANK, Assistant Secretary (Acting), 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                 28 April 2022

 

 

 

 

 

 

 

 

 

 

 

MARIANA CRANK

Assistant Secretary (Acting)

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 (May Update) Instrument 2022.

(2)          This instrument may also be cited as PB 37 of 2022.

2      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 May 2022

1 May 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.

3      Authority

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

4      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 1Amendments

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)    elexacaftor with tezacaftor and with ivacaftor, and ivacaftor;

  (j)    eltrombopag;

  (k)   epoprostenol;

  (l)    etanercept;

  (m)  iloprost;

  (n)   infliximab;

  (o)   ivacaftor;

  (p)   lenalidomide;

  (q)   lumacaftor with ivacaftor;

  (r)    macitentan;

  (s)    mepolizumab;

  (t)    midostaurin;

(u)   nusinersen;

(v)   omalizumab;

(w) onasemnogene abeparvovec;

(x)   pasireotide;

(y)               pegvisomant;

(z)   pomalidomide;

(aa)  ravulizumab

(bb) riociguat;

(cc)  risdiplam

(dd) rituximab;

(ee)  romiplostim;

(ff)  sildenafil;

(gg)  tadalafil;

(hh) teduglutide;

(ii)               tezacaftor with ivacaftor and ivacaftor;

(jj)               tocilizumab;

(kk) ustekinumab;

(ll)   vedolizumab.

[2]        Schedule 1, entry for Cabotegravir and rilpivirine

omit from the coumn headed “Form”: Pack containing 1 vial cabotegravir 600 mg in 3 mL and 1 vial rilpivirine 900 mg in 3mL   
substitute: Pack containing 1 vial cabotegravir 600 mg in 3 mL and 1 vial rilpivirine 900 mg in 3 mL

[3]        Schedule 1, entry for Dupilumab in the form Injection 200 mg in 1.14 mL single dose pre-filled syringe

omit from the column headed “Circumstances”: C11925

[4]        Schedule 1, entry for Dupilumab in the form Injection 300 mg in 2 mL single dose pre-filled syringe

omit from the column headed “Circumstances”: C11901

[5]        Schedule 1, entry for Lenalidomide in each of the forms: Capsule 5 mg; Capsule 10 mg; Capsule 15 mg; and Capsule 25 mg

(a)        omit from the column headed “Circumstances”: C10349 C10350

(b)        insert in numerical order in the column headed “Circumstances”: C12689 C12840 C12841

[6]        Schedule 1, entry for Nusinersen

omit from the column headed “Circumstances”: C11058 C12017 C12037             substitute: C12643 C12667 C12672 C12676

[7]        Schedule 1, after entry for Omalizumab in the form Injection 150 mg in 1 mL single dose pre‑filled syringe

insert:

Onasemnogene abeparvovec

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 3 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 4 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 5 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 6 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 7 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 8 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 1 vial solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 3 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 4 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 5 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 6 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 5.5 mL and 7 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 2 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 3 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 4 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 5 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 6 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 7 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 8 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

 

Pack containing 9 vials solution for I.V. infusion 20 trillion vector genomes per mL, 8.3 mL

Injection

Zolgensma

C12639 C12641

 

See Schedule 2

See Schedule 2

[8]        Schedule 1, entry for Pomalidomide in each of the forms: Capsule 3 mg; and Capsule 4 mg

omit from the column headed “Circumstances: C7791 C7952 C12256 C12283              substitute: C12693 C12698 C12893 C12914

[9]        Schedule 1, entry for Risdiplam

omit from the column headed “Circumstances”: C11995 C11996 C12057                             substitute: C12675 C12678 C12679 C12682

[10]      Schedule 1, entry for Selexipag

substitute:

Selexipag

Tablet 200 micrograms

Oral

Uptravi

C11193 C11195 C11261

P11193 P11195

60

5

 

 

 

 

C11193 C11195 C11261

P11261

140

2

 

Tablet 400 micrograms

Oral

Uptravi

C11193 C11195

 

60

5

 

Tablet 600 micrograms

Oral

Uptravi

C11193 C11195

 

60

5

 

Tablet 800 micrograms

Oral

Uptravi

C11193 C11195 C11261

P11261

60

3

 

 

 

 

C11193 C11195 C11261

P11193 P11195

60

5

 

Tablet 1 mg

Oral

Uptravi

C11193 C11195

 

60

5

 

Tablet 1.2 mg

Oral

Uptravi

C11193 C11195

 

60

5

 

Tablet 1.4 mg

Oral

Uptravi

C11193 C11195

 

60

5

 

Tablet 1.6 mg

Oral

Uptravi

C11193 C11195

 

60

5

[11]      Schedule 2, entry for Dupilumab

substitute:

Dupilumab

C11844 C11897 C11926 C11964

1 pack

Sufficient for 32 weeks of treatment

 

C11924

1 pack

Sufficient for 24 weeks of treatment

[12]      Schedule 2, entry for Lenalidomide

substitute:

Lenalidomide

C10452 C10453

14 tablets

3

 

C10429

21 tablets

1

 

C12841

21 tablets

2

 

C4282 C4287 C10428

21 tablets

3

 

C10373 C10427 P12689 P12840

21 tablets

5

 

C10334 C10335

28 tablets

2

[13]      Schedule 2, entry for Nusinersen

substitute:

Nusinersen

C12643

1 dose

0

 

C12667 C12672 C12676

1 dose

3

[14]      Schedule 2, after entry for Omalizumab

insert:

Onasemnogene abeparvovec

C12639 C12641

1 dose

0

[15]      Schedule 2, entry for Pomalidomide

substitute:

Pomalidomide

C12698 C12914

1 pack (21 capsules)

5

 

C12693 C12893

1 pack (14 capsules)

2

[16]      Schedule 2, entry for Risdiplam

substitute:

Risdiplam

C12675 C12678 C12679

1

0

 

C12682

1

5

[17]      Schedule 3, entry for Dupilumab

(a)        omit:

 

C11901

 

Uncontrolled severe asthma
Grandfather treatment
Must be treated by a respiratory physician, clinical immunologist, allergist or general physician experienced in the management of patients with severe asthma.
Patient must have received non-PBS-subsidised treatment with this biological medicine for this condition prior to 1 April 2021; AND
Patient must have demonstrated or sustained an adequate response to treatment with this biological medicine if the patient has received at least the week 28 dose of this biological medicine; AND
Patient must be receiving treatment with this drug for this condition at the time of application; AND
Patient must be under the care of the same physician for at least 6 months; OR
Patient must have been diagnosed by a multidisciplinary severe asthma clinic team; AND
Patient must have a diagnosis of asthma confirmed and documented by a respiratory physician, clinical immunologist, allergist or general physician experienced in the management of patients with severe asthma, defined by the following standard clinical features: (i) forced expiratory volume (FEV1) reversibility greater than or equal to 12% and greater than or equal to 200 mL at baseline within 30 minutes after administration of salbutamol (200 to 400 micrograms), or (ii) airway hyperresponsiveness defined as a greater than 20% decline in FEV1 during a direct bronchial provocation test or greater than 15% decline during an indirect bronchial provocation test, or (iii) peak expiratory flow (PEF) variability of greater than 15% between the two highest and two lowest peak expiratory flow rates during 14 days; OR
Patient must have a diagnosis of asthma from at least two physicians experienced in the management of patients with severe asthma; AND
Patient must have had a documented blood eosinophil count greater than or equal to 150 cells per microlitre while receiving treatment with oral corticosteroids prior to initiating a biological medicine for this condition; OR
Patient must have had a documented total serum human immunoglobulin E greater than or equal to 30 IU/mL measured no more than 12 months prior to initiating a biological medicine, with past or current evidence of atopy, documented by skin prick testing or an in vitro measure of specific IgE no more than 12 months prior to initiating a biological medicine for this condition; AND
Patient must have received regular maintenance oral corticosteroids (OCS) in the last 6 months with a stable daily OCS dose of 5 to 35 mg/day of prednisolone or equivalent over the 4 weeks prior to commencement of a biological medicine treatment for severe asthma; AND
Patient must have had a duration of asthma of at least 1 year prior to commencement of this biological medicine; AND
Patient must have documented a failure to achieve adequate control with optimised asthma therapy, despite formal assessment of and adherence to correct inhaler technique, prior to initiating a biological medicine for this condition; AND
Patient must not receive more than 24 weeks of treatment under this restriction; AND
The treatment must not be used in combination with and within 4 weeks of another PBS-subsidised biological medicine prescribed for severe asthma.
Patient must be aged 12 years or older.
Optimised asthma therapy includes:
(i) Adherence to maximal inhaled therapy, including high dose inhaled corticosteroid (ICS) plus long-acting beta-2 agonist (LABA) therapy for at least 12 months, unless contraindicated or not tolerated;
AND
(ii) treatment with oral corticosteroids as outlined in the clinical criteria.
If the requirement for treatment with optimised asthma therapy cannot be met because of contraindications according to the relevant TGA-approved Product Information and/or intolerances of a severity necessitating permanent treatment withdrawal, details of the contraindication and/or intolerance must be provided in the Authority application.
The following initiation criteria indicate failure to achieve adequate control with optimised asthma therapy and must be declared to have been met at the time of the application:
(a) an Asthma Control Questionnaire (ACQ-5) score of at least 2.0 prior to commencement with a biological medicine for severe asthma; AND
(b) while receiving optimised asthma therapy in the 12 months prior to commencing treatment with a biological medicine for severe asthma, experienced at least 1 admission to hospital for a severe asthma exacerbation, OR 1 severe asthma exacerbation, requiring documented use of systemic corticosteroids (oral corticosteroids initiated or increased for at least 3 days, or parenteral corticosteroids) prescribed/supervised by a physician.
An Asthma Control Questionnaire (5 item version) assessment and/or an assessment of a reduction in the patient's maintenance oral corticosteroid dose to determine whether the patient has achieved or sustained an adequate response to non-PBS-subsidised treatment, must be conducted immediately (no later than 4 weeks after the last dose of non-PBS-subsidised treatment) prior to this application if the treatment duration has been 28 weeks or greater.
If a patient fails to demonstrate a response to treatment with this drug they will not be eligible to receive further PBS-subsidised treatment with this drug for this condition within the same treatment cycle.
A treatment break in PBS-subsidised biological medicine therapy of at least 12 months must be observed in a patient who has either failed to achieve or sustain a response to treatment with 4 biological medicines within the same treatment cycle.
The length of the break in therapy is measured from the date the most recent treatment with a PBS-subsidised biological medicine was administered until the date of the first application for recommencement of treatment with a biological medicine under the new treatment cycle.
There is no limit to the number of treatment cycles that a patient may undertake in their lifetime.
A multidisciplinary severe asthma clinic team comprises of:
A respiratory physician; and
A pharmacist, nurse or asthma educator.
An adequate response to this biological medicine is defined as:
(a) a reduction in the Asthma Control Questionnaire (ACQ-5) score of at least 0.5 from baseline,
OR
(b) maintenance oral corticosteroid dose reduced by at least 25% from baseline, and no deterioration in ACQ-5 score from baseline or an increase in ACQ-5 score from baseline less than or equal to 0.5.
A Grandfathered patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a Grandfathered patient must qualify under the continuing treatment criteria.
The authority application must be made in writing and must include:
(a) a completed authority prescription form; and
(b) a completed Uncontrolled severe asthma dupilumab initial grandfather PBS authority application form which
seeks details of the following (if not already provided):
(i) prior optimised asthma drug therapy (date of commencement and duration of therapy); and
(ii) a eosinophil pathology report (eosinophil counts and dates) prior to initiating a biological medicine for this condition; or
(iii) IgE results prior to initiating a biological medicine for this condition; and
(iv) ACQ-5 scores including the date of assessment of the patient's symptoms, or details of the maintenance oral corticosteroid dose; and
(v) Date of commencing non-PBS-subsidised treatment with this drug.

Compliance with Written Authority Required procedures

(b)        omit:

 

C11925

 

Uncontrolled severe asthma
Grandfather treatment
Must be treated by a respiratory physician, clinical immunologist, allergist or general physician experienced in the management of patients with severe asthma.
Patient must have received non-PBS-subsidised treatment with this biological medicine for this condition prior to 1 April 2021; AND
Patient must have demonstrated or sustained an adequate response to treatment with this biological medicine if the patient has received at least the week 28 dose of this biological medicine; AND
Patient must be receiving treatment with this drug for this condition at the time of application; AND
Patient must be under the care of the same physician for at least 6 months; OR
Patient must have been diagnosed by a multidisciplinary severe asthma clinic team; AND
Patient must have a diagnosis of asthma confirmed and documented by a respiratory physician, clinical immunologist, allergist or general physician experienced in the management of patients with severe asthma, defined by the following standard clinical features: (i) forced expiratory volume (FEV1) reversibility greater than or equal to 12% and greater than or equal to 200 mL at baseline within 30 minutes after administration of salbutamol (200 to 400 micrograms), or (ii) airway hyperresponsiveness defined as a greater than 20% decline in FEV1 during a direct bronchial provocation test or greater than 15% decline during an indirect bronchial provocation test, or (iii) peak expiratory flow (PEF) variability of greater than 15% between the two highest and two lowest peak expiratory flow rates during 14 days; OR
Patient must have a diagnosis of asthma from at least two physicians experienced in the management of patients with severe asthma; AND
Patient must have had a documented blood eosinophil count greater than or equal to 300 cells per microlitre measured no more than 12 months prior to initiating a biological medicine for this condition; OR
Patient must have had a documented blood eosinophil count greater than or equal to 150 cells per microlitre while receiving treatment with oral corticosteroids, measured no more than 12 months prior to initiating a biological medicine for this condition; OR
Patient must have had a documented total serum human immunoglobulin E greater than or equal to 30 IU/mL measured no more than 12 months prior to initiating a biological medicine, with past or current evidence of atopy, documented by skin prick testing or an in vitro measure of specific IgE no more than 12 months prior to initiating a biological medicine for this condition; AND
Patient must have had a duration of asthma of at least 1 year prior to commencement of this biological medicine; AND
Patient must have documented a failure to achieve adequate control with optimised asthma therapy, despite formal assessment of and adherence to correct inhaler technique, prior to initiating a biological medicine for this condition; AND
Patient must not receive more than 24 weeks of treatment under this restriction; AND
The treatment must not be used in combination with and within 4 weeks of another PBS-subsidised biological medicine prescribed for severe asthma.
Patient must be aged 12 years or older.
Optimised asthma therapy includes:
(i) Adherence to maximal inhaled therapy, including high dose inhaled corticosteroid (ICS) plus long-acting beta-2 agonist (LABA) therapy for at least 12 months, unless contraindicated or not tolerated;
AND
(ii) treatment with oral corticosteroids, either daily oral corticosteroids for at least 6 weeks, OR a cumulative dose of oral corticosteroids of at least 500 mg prednisolone equivalent in the previous 12 months, unless contraindicated or not tolerated.
If the requirement for treatment with optimised asthma therapy cannot be met because of contraindications according to the relevant TGA-approved Product Information and/or intolerances of a severity necessitating permanent treatment withdrawal, details of the contraindication and/or intolerance must be provided in the Authority application.
The following initiation criteria indicate failure to achieve adequate control with optimised asthma therapy and must be declared to have been met at the time of the application:
(a) an Asthma Control Questionnaire (ACQ-5) score of at least 2.0 prior to commencement with a biological medicine for severe asthma; AND
(b) while receiving optimised asthma therapy in the 12 months prior to commencing treatment with a biological medicine for severe asthma, experienced at least 1 admission to hospital for a severe asthma exacerbation, OR 1 severe asthma exacerbation, requiring documented use of systemic corticosteroids (oral corticosteroids initiated or increased for at least 3 days, or parenteral corticosteroids) prescribed/supervised by a physician.
An Asthma Control Questionnaire (5 item version) assessment and/or an assessment of a reduction in the patient's maintenance oral corticosteroid dose to determine whether the patient has achieved or sustained an adequate response to non-PBS-subsidised treatment, must be conducted immediately (no later than 4 weeks after the last dose of non-PBS-subsidised treatment) prior to this application if the treatment duration has been 28 weeks or greater.
If a patient fails to demonstrate a response to treatment with this drug they will not be eligible to receive further PBS-subsidised treatment with this drug for this condition within the same treatment cycle.
A treatment break in PBS-subsidised biological medicine therapy of at least 12 months must be observed in a patient who has either failed to achieve or sustain a response to treatment with 4 biological medicines within the same treatment cycle.
The length of the break in therapy is measured from the date the most recent treatment with a PBS-subsidised biological medicine was administered until the date of the first application for recommencement of treatment with a biological medicine under the new treatment cycle.
There is no limit to the number of treatment cycles that a patient may undertake in their lifetime.
A multidisciplinary severe asthma clinic team comprises of:
A respiratory physician; and
A pharmacist, nurse or asthma educator.
An adequate response to this biological medicine is defined as:
(a) a reduction in the Asthma Control Questionnaire (ACQ-5) score of at least 0.5 from baseline,
OR
(b) maintenance oral corticosteroid dose reduced by at least 25% from baseline, and no deterioration in ACQ-5 score from baseline or an increase in ACQ-5 score from baseline less than or equal to 0.5.
A Grandfathered patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a Grandfathered patient must qualify under the continuing treatment criteria.
The authority application must be made in writing and must include:
(a) a completed authority prescription form; and
(b) a completed Uncontrolled severe asthma dupilumab initial grandfather PBS authority application form which
seeks details of the following (if not already provided):
(i) prior optimised asthma drug therapy (date of commencement and duration of therapy); and
(ii) a eosinophil pathology report (eosinophil counts and dates) prior to initiating a biological medicine for this condition; or
(iii) IgE results prior to initiating a biological medicine for this condition; and
(iv) ACQ-5 scores including the date of assessment of the patient's symptoms, or details of the maintenance oral corticosteroid dose; and
(v) Date of commencing non-PBS-subsidised treatment with this drug.

Compliance with Written Authority Required procedures

[18]      Schedule 3, entry for Lenalidomide

(a)     omit:

 

C10349

 

Multiple myeloma
Continuing treatment as monotherapy or dual combination therapy with dexamethasone following initial treatment for progressive disease
Patient must have previously received PBS‑subsidised treatment with this drug for relapsed or refractory multiple myeloma; AND
The treatment must be as monotherapy; OR
The treatment must be in combination with dexamethasone; AND
Patient must not be receiving concomitant PBS‑subsidised bortezomib, carfilzomib or thalidomide or its analogues.
Patients receiving lenalidomide under the PBS listing must be registered in the i‑access risk management program.

Compliance with Authority Required procedures

 

C10350

 

Multiple myeloma
Initial treatment as monotherapy or dual combination therapy with dexamethasone for progressive disease
The condition must be confirmed by a histological diagnosis; AND
The treatment must be as monotherapy; OR
The treatment must be in combination with dexamethasone; AND
Patient must have progressive disease after at least one prior therapy; AND
Patient must have undergone or be ineligible for a primary stem cell transplant; AND
Patient must not be receiving concomitant PBS‑subsidised bortezomib, carfilzomib or thalidomide or its analogues.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24‑hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo‑secretory and non‑secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Oligo‑secretory and non‑secretory patients are defined as having active disease with less than 10 g per L serum M protein.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) a completed Multiple Myeloma lenalidomide Authority Application ‑ Supporting Information Form, which includes details of the histological diagnosis of multiple myeloma, prior treatments including name(s) of drug(s) and date of most recent treatment cycle and record of prior stem cell transplant or ineligibility for prior stem cell transplant; details of the basis of the diagnosis of progressive disease or failure to respond; and nomination of which disease activity parameters will be used to assess response; and
(3) a signed patient acknowledgment.
To enable confirmation of eligibility for treatment, current diagnostic reports of at least one of the following must be provided:
(a) the level of serum monoclonal protein; or
(b) Bence‑Jones proteinuria ‑ the results of 24‑hour urinary light chain M protein excretion; or
(c) the serum level of free kappa and lambda light chains; or
(d) bone marrow aspirate or trephine; or
(e) if present, the size and location of lytic bone lesions (not including compression fractures); or
(f) if present, the size and location of all soft tissue plasmacytomas by clinical or radiographic examination i.e. MRI or CT‑scan; or
(g) if present, the level of hypercalcaemia, corrected for albumin concentration.
As these parameters will be used to determine response, results for either (a) or (b) or (c) should be provided for all patients. Where the patient has oligo‑secretory or non‑secretory multiple myeloma, either (c) or (d) or if relevant (e), (f) or (g) should be provided. Where the prescriber plans to assess response in patients with oligo‑secretory or non‑secretory multiple myeloma with free light chain assays, evidence of the oligo‑secretory or non‑secretory nature of the multiple myeloma (current serum M protein less than 10 g per L) must be provided.
Patients receiving lenalidomide under the PBS listing must be registered in the i‑access risk management program.

Compliance with Written Authority Required procedures

(b)    insert in numerical order after existing text:

 

C12689

 

Multiple myeloma
Initial treatment as monotherapy or dual combination therapy with dexamethasone for progressive disease
The condition must be confirmed by a histological diagnosis; AND
The treatment must be as monotherapy; OR
The treatment must be in combination with dexamethasone; AND
Patient must have progressive disease after at least one prior therapy; AND
Patient must have undergone or be ineligible for a primary stem cell transplant.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24-hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo-secretory and non-secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Oligo-secretory and non-secretory patients are defined as having active disease with less than 10 g per L serum M protein.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) a completed Multiple Myeloma lenalidomide Authority Application - Supporting Information Form, which includes details of the histological diagnosis of multiple myeloma, prior treatments including name(s) of drug(s) and date of most recent treatment cycle and record of prior stem cell transplant or ineligibility for prior stem cell transplant; details of the basis of the diagnosis of progressive disease or failure to respond; and nomination of which disease activity parameters will be used to assess response; and
(3) a signed patient acknowledgment.
To enable confirmation of eligibility for treatment, current diagnostic reports of at least one of the following must be provided:
(a) the level of serum monoclonal protein; or
(b) Bence-Jones proteinuria - the results of 24-hour urinary light chain M protein excretion; or
(c) the serum level of free kappa and lambda light chains; or
(d) bone marrow aspirate or trephine; or
(e) if present, the size and location of lytic bone lesions (not including compression fractures); or
(f) if present, the size and location of all soft tissue plasmacytomas by clinical or radiographic examination i.e. MRI or CT-scan; or
(g) if present, the level of hypercalcaemia, corrected for albumin concentration.
As these parameters will be used to determine response, results for either (a) or (b) or (c) should be provided for all patients. Where the patient has oligo-secretory or non-secretory multiple myeloma, either (c) or (d) or if relevant (e), (f) or (g) should be provided. Where the prescriber plans to assess response in patients with oligo-secretory or non-secretory multiple myeloma with free light chain assays, evidence of the oligo-secretory or non-secretory nature of the multiple myeloma (current serum M protein less than 10 g per L) must be provided.
Patients receiving lenalidomide under the PBS listing must be registered in the i-access risk management program.

Compliance with Written Authority Required procedures

 

C12840

 

Multiple myeloma
Continuing treatment as monotherapy or dual combination therapy with dexamethasone following initial treatment for progressive disease
Patient must have previously received PBS-subsidised treatment with this drug for relapsed or refractory multiple myeloma; AND
The treatment must be as monotherapy; OR
The treatment must be in combination with dexamethasone.
Patients receiving lenalidomide under the PBS listing must be registered in the i-access risk management program.

Compliance with Authority Required procedures

 

C12841

 

Relapsed and/or refractory multiple myeloma
Triple combination therapy consisting of elotuzumab, lenalidomide and dexamethasone
Patient must be undergoing concurrent treatment with elotuzumab obtained through the PBS; AND
Patient must not be undergoing simultaneous treatment with this drug obtained under another PBS listing.
Patients receiving this drug under the PBS listing must be registered in the i-access risk management program.

Compliance with Authority Required procedures

[19]      Schedule 3, entry for Nusinersen

substitute:

Nusinersen

C12643

 

Spinal muscular atrophy (SMA)
Continuing/maintenance treatment of either symptomatic Type I, II or IIIa SMA, or of a patient commenced on this drug under the pre-symptomatic SMA listing
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or initiated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; AND
Patient must not be undergoing treatment through this 'Continuing treatment' listing where the most recent PBS authority approval for this PBS-indication has been for gene therapy.
Patient must have previously received PBS-subsidised treatment with this drug for this condition; OR
Patient must be eligible for continuing PBS-subsidised treatment with risdiplam for this condition; AND
The treatment must not be in combination with PBS-subsidised treatment with risdiplam for this condition; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must be ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug.
Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day.
In a patient who wishes to switch from PBS-subsidised risdiplam to PBS-subsidised nusinersen for this condition a wash out period may be required.

Compliance with Written Authority Required procedures

 

C12667

 

Pre-symptomatic spinal muscular atrophy (SMA)
Initial treatment of pre-symptomatic spinal muscular atrophy (SMA) - Loading doses
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
The condition must have genetic confirmation that there are 1 to 2 copies of the survival motor neuron 2 (SMN2) gene; AND
The condition must be pre-symptomatic; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not exceed four loading doses (at days 0, 14, 28 and 63) under this restriction; AND
Patient must be untreated with gene therapy.
Patient must be aged under 36 months prior to commencing treatment.
Application for authorisation of initial treatment must be in writing (lodged via postal service or electronic upload) and must include:
(a) a completed authority prescription form; and
(b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
(i) confirmation of genetic diagnosis of SMA; and
(ii) a copy of the results substantiating the number of SMN2 gene copies determined by quantitative polymerase chain reaction (qPCR) or multiple ligation dependent probe amplification (MLPA)

Compliance with Written Authority Required procedures

 

C12672

 

Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA)
Initial treatment - Loading doses
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA.
The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
Patient must have experienced at least two of the defined signs and symptoms of SMA type I, II or IIIa prior to 3 years of age; AND
The treatment must not be in combination with PBS-subsidised treatment with risdiplam for this condition; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not exceed four loading doses (at days 0, 14, 28 and 63) under this restriction; AND
Patient must be untreated with gene therapy.
Patient must be 18 years of age or under.
Defined signs and symptoms of type I SMA are:
i) Onset before 6 months of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type II SMA are:
i) Onset between 6 and 18 months; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Weakness in trunk righting/derotation; or
v) Hypotonia; or
vi) Absence of deep tendon reflexes; or
vii) Failure to gain weight appropriate for age; or
viii) Any active chronic neurogenic changes; or
ix) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type IIIa SMA are:
i) Onset between 18 months and 3 years of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
Application for authorisation of initial treatment must be in writing and must include:
(a) a completed authority prescription form; and
(b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
i) specification of SMA type (I, II or IIIa); and
(ii) sign(s) and symptom(s) that the patient has experienced; and
(iii) patient's age at the onset of sign(s) and symptom(s).

Compliance with Written Authority Required procedures

 

C12676

 

Spinal muscular atrophy (SMA)
Initial treatment occurring after onasemnogene abeparvovec therapy in a patient with one of: (i) Type 1 SMA, or, (ii) pre-symptomatic SMA
Patient must have experienced a regression in a developmental state listed below (see 'Definition') despite treatment with gene therapy - confirm that this: (i) not due to an acute concomitant illness; (ii) not due to non-compliance to best-supportive care, (iii) apparent for at least 3 months, (iv) verified by another clinician in the treatment team - state the full name of this clinician plus their profession (e.g. medical practitioner, nurse, physiotherapist; this is not an exhaustive list of examples); AND
The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not be in combination with PBS-subsidised treatment with risdiplam for this condition.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; AND
Patient must be undergoing treatment under this Treatment phase listing once only - for continuing treatment beyond this authority application, refer to the drug's relevant 'Continuing treatment' listing for the patient's SMA type.
Patient must have a prior authority approval for any drug PBS-listed for symptomatic Type 1 SMA, with at least one approval having been for gene therapy; OR
Patient must have a prior authority approval for any drug PBS-listed for pre-symptomatic SMA, with at least one approval having been for gene therapy.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) 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).
Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.
Confirm that a previous PBS authority application has been approved for one of the following:
(i) Symptomatic Type 1 SMA; or
(ii) Pre-symptomatic SMA treated with nusinersen.
Definition:
Various childhood developmental states (1 to 9) are listed below, some followed by further observations (a up to d). Where at least one developmental state/observation is no longer present, that developmental state has regressed.
0. Absence of developmental states (1 to 9) listed below:
1. Rolls from side to side on back;
2. Child holds head erect for at least 3 seconds unsupported;
3. Sitting, but with assistance;
4. Sitting without assistance:
(a) Child sits up straight with the head erect for at least 10 seconds;
(b) Child does not use arms or hands to balance body or support position.
5. Hands and knees crawling:
(a) Child alternately moves forward or backwards on hands and knees;
(b) The stomach does not touch the supporting surface;
(c) There are continuous and consecutive movements at least 3 in a row.
6. Standing with assistance:
(a) Child stands in upright position on both feet, holding onto a stable object (e.g. furniture) with both hands and without leaning on object;
(b)The body does not touch the stable object, and the legs support most of the body weight;
(c) Child thus stands with assistance for at least 10 seconds.
7. Standing alone:
(a) Child stands in upright position on both feet (not on the toes) with the back straight;
(b) The leg supports 100% of the child's weight;
(c) There is no contact with a person or object;
(d) Child stands alone for at least 10 seconds.
8. Walking with assistance:
(a) Child is in an upright position with the back straight;
(b) Child makes sideways or forced steps by holding onto a stable object (e.g. furniture) with 1 or both hands;
(c) One leg moves forward while the other supports part of the body weight;
(d) Child takes at least 5 steps in this manner.
9. Walking alone:
(a) Child takes at least 5 steps independently in upright position with the back straight;
(b) One leg moves forward while the other supports most of the body weight;
(c) There is no contact with a person or object.
Confirm which developmental state has regressed by: (i) stating the overall developmental state (1 - 9) the patient was in at the time of gene therapy, or, the best developmental state achieved since gene therapy, and (ii) stating the patient's current overall developmental state (i.e. a number that is lower than stated in (i).
Where the patient has neither regressed from a developmental state nor reached the next developmental state, PBS-subsidy of this benefit is not available.

Compliance with Written Authority Required procedures

[20]      Schedule 3, after entry for Omalizumab

insert:

Onasemnogene abeparvovec

C12639

 

Spinal muscular atrophy (SMA)
Use in a patient untreated with disease modifying therapies for this condition
The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
Patient must have experienced at least two of the defined signs/symptoms of Type 1 SMA specified below; OR
The condition must be pre-symptomatic SMA, with genetic confirmation that there are 1 to 2 copies of the survival motor neuron 2 (SMN2) gene; AND
The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes; AND
The treatment must be given concomitantly with best supportive care for this condition.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; AND
Must be treated in a treatment centre that is each of: (i) recognised in the management of SMA, (ii) accredited in the use of this gene technology by the relevant authority, (iii) will(has) source(d) this product from an accredited supplier, as specified in the administrative notes to this listing; AND
Patient must be undergoing treatment with this pharmaceutical benefit once only in a lifetime; AND
Patient must not be undergoing treatment with this pharmaceutical benefit through this listing where prior treatment has occurred with any of: (i) nusinersen, (ii) risdiplam.
Patient must be no older than 9 months of age; AND
Patient must have symptomatic Type 1 SMA; OR
Patient must have pre-symptomatic SMA.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) 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).
Prescribing Instructions:
In the relevant PBS Authority Application form, specify the following:
(i) the SMA type being treated: symptomatic Type 1 SMA, or, pre-symptomatic SMA;
(ii) for Type 1 SMA, the signs/symptoms that the patient has experienced, together with the patient's age at the onset of these signs/symptoms.
State the weight of the patient in kilograms and request the appropriate product pack presentation with respect to the mix of 5.5 mL and 8.3 mL vials.
Confirm that genetic testing has been completed to demonstrate the following in support of an SMA diagnosis:
(i) 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; or
(ii) deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variance in the remaining single copy of the SMN1 gene.
If the condition is pre-symptomatic SMA, confirm that there is genetic test finding that substantiates the number of SMN2 gene copies determined by quantitative polymerase chain reaction (qPCR) or multiple ligation dependent probe amplification (MLPA).
Quote the date, pathology provider name and any unique identifying serial number/code that links the genetic test result to the patient.
Defined signs and symptoms of type I SMA are:
i) Onset before 6 months of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.

Compliance with Written Authority Required procedures

 

C12641

 

Spinal muscular atrophy (SMA)
Use occurring after treatment with at least one disease modifying therapy for this condition (i.e. switching from nusinersen/risdiplam to onasemnogene abeparvovec)
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes.
Patient must be undergoing treatment with this pharmaceutical benefit following prior PBS-subsidised treatment with at least one other disease modifying therapy for this condition; AND
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic of a recognised hospital in the management of SMA; AND
Must be treated in a treatment centre that is each of: (i) recognised in the management of SMA, (ii) accredited in the use of this gene technology by the relevant authority, (iii) will(has) source(d) this product from an accredited supplier, as specified in the administrative notes to this listing; AND
Patient must be undergoing treatment with this pharmaceutical benefit once only in a lifetime; AND
Patient must be undergoing treatment with this pharmaceutical benefit with the intent that treatment with the replaced disease modifying agent is/has ceased.
Patient must be no older than 9 months of age; AND
Patient must have symptomatic Type 1 SMA; OR
Patient must have pre-symptomatic SMA.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) 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).
Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.
Confirm that a previous PBS authority application has been approved for one of the following:
(i) Symptomatic Type 1 SMA; or
(ii) Pre-symptomatic SMA treated with nusinersen
State the weight of the patient in kilograms and request the appropriate product pack presentation with respect to the mix of 5.5 mL and 8.3 mL vials.
Adhere to any Product Information or local treatment guidelines with respect to treatment-free ('wash out') periods prior to administering this benefit.

Compliance with Written Authority Required procedures


 

[21]      Schedule 3, entry for Pomalidomide

substitute:

Pomalidomide

C12693

 

Multiple myeloma
Initial treatment with triple therapy (this drug, bortezomib and dexamethasone)
The condition must be confirmed by a histological diagnosis; AND
The treatment must be in combination with bortezomib and dexamethasone; AND
Patient must have progressive disease after at least one prior therapy that is either: (i) lenalidomide monotherapy, (ii) contains lenalidomide; AND
Patient must have undergone or be ineligible for a stem cell transplant.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24-hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo-secretory and non-secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Oligo-secretory and non-secretory patients are defined as having active disease with less than 10 g per L serum M protein.
Patients receiving this drug under the PBS listing must be registered in the i-access risk management program.

Compliance with Authority Required procedures

 

C12698

 

Multiple myeloma
Continuing treatment
Patient must have previously been issued with an authority prescription for this drug; AND
Patient must not have progressive disease; AND
The treatment must be in combination with dexamethasone.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24-hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo-secretory and non-secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Patients receiving this drug under the PBS listing must be registered in the i-access risk management program.

Compliance with Authority Required procedures

 

C12893

 

Multiple myeloma
Continuing treatment with triple therapy (this drug, bortezomib and dexamethasone)
Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND
The treatment must be in combination with bortezomib and dexamethasone; AND
Patient must not have developed disease progression while being treated with this drug for this condition.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24-hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo-secretory and non-secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Oligo-secretory and non-secretory patients are defined as having active disease with less than 10 g per L serum M protein.
Patients receiving this drug under the PBS listing must be registered in the i-access risk management program.

Compliance with Authority Required procedures

 

C12914

 

Multiple myeloma
Initial treatment
The treatment must be in combination with dexamethasone; AND
Patient must have undergone or be ineligible for a primary stem cell transplant; AND
Patient must have experienced treatment failure with lenalidomide, unless contraindicated or not tolerated according to the Therapeutic Goods Administration (TGA) approved Product Information; AND
Patient must have experienced treatment failure with bortezomib, unless contraindicated or not tolerated according to the Therapeutic Goods Administration (TGA) approved Product Information.
Bortezomib treatment failure is the absence of achieving at least a partial response or as progressive disease during treatment or within 6 months of discontinuing treatment with bortezomib. Lenalidomide treatment failure is progressive disease during treatment or within 6 months of discontinuing treatment with lenalidomide.
If treatment with either bortezomib or lenalidomide is contraindicated according to the relevant TGA-approved Product Information, the application must provide details of the contraindication.
If intolerance to either bortezomib or lenolidomide treatment develops during the relevant period of use which is of a severity to necessitate withdrawal of the treatment, the application must provide details of the nature and severity of this intolerance.
Progressive disease is defined as at least 1 of the following:
(a) at least a 25% increase and an absolute increase of at least 5 g per L in serum M protein (monoclonal protein); or
(b) at least a 25% increase in 24-hour urinary light chain M protein excretion, and an absolute increase of at least 200 mg per 24 hours; or
(c) in oligo-secretory and non-secretory myeloma patients only, at least a 50% increase in the difference between involved free light chain and uninvolved free light chain; or
(d) at least a 25% relative increase and at least a 10% absolute increase in plasma cells in a bone marrow aspirate or on biopsy; or
(e) an increase in the size or number of lytic bone lesions (not including compression fractures); or
(f) at least a 25% increase in the size of an existing or the development of a new soft tissue plasmacytoma (determined by clinical examination or diagnostic imaging); or
(g) development of hypercalcaemia (corrected serum calcium greater than 2.65 mmol per L not attributable to any other cause).
Oligo-secretory and non-secretory patients are defined as having active disease with less than 10 g per L serum M protein.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) a completed Multiple Myeloma pomalidomide Authority Application Supporting Information form; and
(3) reports demonstrating the patient has failed treatment with, providing details of the contraindication to or details of the nature and severity of the intolerance to lenalidomide; and
(4) reports demonstrating the patient has failed treatment with, providing details of the contraindication to or details of the nature and severity of the intolerance to bortezomib.
Patients receiving this drug under the PBS listing must be registered in the i-access risk management program.

Compliance with Written Authority Required procedures

[22]      Schedule 3, entry for Risdiplam

substitute:

Risdiplam

C12675

 

Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA)
Transitioning from non-PBS to PBS subsided treatment - Grandfather treatment
The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
Patient must have experienced at least two of the defined signs and symptoms of SMA type I, II or IIIa prior to 3 years of age; AND
Patient must have previously received non-PBS-subsidised treatment with this drug for this condition prior to 1 August 2021; AND
Patient must not be receiving invasive permanent assisted ventilation in the absence of a potentially reversible cause while being treated with this drug; AND
The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
Patient must be responding to non-PBS subsidised risdiplam for this condition at the time of application.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic.
Patient must be untreated with gene therapy.
Patient must have been 18 years of age or under at the time non-PBS subsidised treatment with this drug was initiated for this condition.
Application for authorisation of initial treatment must be in writing and must include:
(a) a completed authority prescription form; and
(b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
i) specification of SMA type (I, II or IIIa); and
(ii) sign(s) and symptom(s) that the patient has experienced; and
(iii) patient's age at the onset of sign(s) and symptom(s).
Defined signs and symptoms of type I SMA are:
i) Onset before 6 months of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type II SMA are:
i) Onset between 6 and 18 months; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Weakness in trunk righting/derotation; or
v) Hypotonia; or
vi) Absence of deep tendon reflexes; or
vii) Failure to gain weight appropriate for age; or
viii) Any active chronic neurogenic changes; or
ix) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type IIIa SMA are:
i) Onset between 18 months and 3 years of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
A patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a Grandfathered patient must qualify under the Continuing treatment criteria.

Compliance with Written Authority Required procedures

 

C12678

 

Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA)
Initial treatment
The condition must have genetic confirmation of 5q homozygous deletion of the survival motor neuron 1 (SMN1) gene; OR
The condition must have genetic confirmation of deletion of one copy of the SMN1 gene in addition to a pathogenic/likely pathogenic variant in the remaining single copy of the SMN1 gene; AND
Patient must have experienced at least two of the defined signs and symptoms of SMA type I, II or IIIa prior to 3 years of age; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition; AND
The treatment must be ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic.
Patient must be untreated with gene therapy.
Patient must be 18 years of age or under.
Defined signs and symptoms of type I SMA are:
i) Onset before 6 months of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type II SMA are:
i) Onset between 6 and 18 months; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Weakness in trunk righting/derotation; or
v) Hypotonia; or
vi) Absence of deep tendon reflexes; or
vii) Failure to gain weight appropriate for age; or
viii) Any active chronic neurogenic changes; or
ix) A compound muscle action potential below normative values for an age-matched child.
Defined signs and symptoms of type IIIa SMA are:
i) Onset between 18 months and 3 years of age; and
ii) Failure to meet or regression in ability to perform age-appropriate motor milestones; or
iii) Proximal weakness; or
iv) Hypotonia; or
v) Absence of deep tendon reflexes; or
vi) Failure to gain weight appropriate for age; or
vii) Any active chronic neurogenic changes; or
viii) A compound muscle action potential below normative values for an age-matched child.
Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day.
Application for authorisation of initial treatment must be in writing and must include:
(a) a completed authority prescription form; and
(b) a completed Spinal muscular atrophy PBS Authority Application Form which includes the following:
i) specification of SMA type (I, II or IIIa); and
(ii) sign(s) and symptom(s) that the patient has experienced; and
(iii) patient's age at the onset of sign(s) and symptom(s).

Compliance with Written Authority Required procedures

 

C12679

 

Spinal muscular atrophy (SMA)
Initial treatment occurring after onasemnogene abeparvovec therapy in a patient with Type 1 SMA
Patient must have experienced a regression in a developmental state listed below (see 'Definition') despite treatment with gene therapy - confirm that this: (i) not due to an acute concomitant illness; (ii) not due to non-compliance to best-supportive care, (iii) apparent for at least 3 months, (iv) verified by another clinician in the treatment team - state the full name of this clinician plus their profession (e.g. medical practitioner, nurse, physiotherapist; this is not an exhaustive list of examples); AND
The treatment must not be a PBS-subsidised benefit where the condition has progressed to a point where invasive permanent assisted ventilation (i.e. ventilation via tracheostomy tube for at least 16 hours per day) is required in the absence of potentially reversible causes; AND
The treatment must be given concomitantly with best supportive care for this condition; AND
The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic; AND
Patient must be undergoing treatment under this Treatment phase listing once only - for continuing treatment beyond this authority application, refer to the drug's relevant 'Continuing treatment' listing for the patient's SMA type.
Patient must have a prior authority approval for any drug PBS-listed for symptomatic Type 1 SMA, with at least one approval having been for gene therapy.
The authority application must be made in writing and must include:
(1) a completed authority prescription form; and
(2) 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).
Do not resubmit previously submitted documentation concerning the diagnosis and type of SMA.
Confirm that a previous PBS authority application has been approved for symptomatic Type 1 SMA.
Definition:
Various childhood developmental states (1 to 9) are listed below, some followed by further observations (a up to d). Where at least one developmental state/observation is no longer present, that developmental state has regressed.
0. Absence of developmental states (1 to 9) listed below:
1. Rolls from side to side on back;
2. Child holds head erect for at least 3 seconds unsupported;
3. Sitting, but with assistance;
4. Sitting without assistance:
(a) Child sits up straight with the head erect for at least 10 seconds;
(b) Child does not use arms or hands to balance body or support position.
5. Hands and knees crawling:
(a) Child alternately moves forward or backwards on hands and knees;
(b) The stomach does not touch the supporting surface;
(c) There are continuous and consecutive movements at least 3 in a row.
6. Standing with assistance:
(a) Child stands in upright position on both feet, holding onto a stable object (e.g. furniture) with both hands and without leaning on object;
(b)The body does not touch the stable object, and the legs support most of the body weight;
(c) Child thus stands with assistance for at least 10 seconds.
7. Standing alone:
(a) Child stands in upright position on both feet (not on the toes) with the back straight;
(b) The leg supports 100% of the child's weight;
(c) There is no contact with a person or object;
(d) Child stands alone for at least 10 seconds.
8. Walking with assistance:
(a) Child is in an upright position with the back straight;
(b) Child makes sideways or forced steps by holding onto a stable object (e.g. furniture) with 1 or both hands;
(c) One leg moves forward while the other supports part of the body weight;
(d) Child takes at least 5 steps in this manner.
9. Walking alone:
(a) Child takes at least 5 steps independently in upright position with the back straight;
(b) One leg moves forward while the other supports most of the body weight;
(c) There is no contact with a person or object.
Confirm which developmental state has regressed by: (i) stating the overall developmental state (1 - 9) the patient was in at the time of gene therapy, or, the best developmental state achieved since gene therapy, and (ii) stating the patient's current overall developmental state (i.e. a number that is lower than stated in (i).
Where the patient has neither regressed from a developmental state nor reached the next developmental state, PBS-subsidy of this benefit is not available.

Compliance with Written Authority Required procedures

 

C12682

 

Symptomatic Type I, II or IIIa spinal muscular atrophy (SMA)
Continuing treatment
Patient must have previously received PBS-subsidised treatment with this drug for this condition; OR
Patient must be eligible for continuing PBS-subsidised treatment with nusinersen for this condition; AND
The treatment must not be in combination with PBS-subsidised treatment with nusinersen for this condition; AND
The treatment must be ceased when invasive permanent assisted ventilation is required in the absence of a potentially reversible cause while being treated with this drug; AND
The treatment must be given concomitantly with best supportive care for this condition.
Must be treated by a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic, or in consultation with a specialist medical practitioner experienced in the diagnosis and management of SMA associated with a neuromuscular clinic; AND
Patient must not be undergoing treatment through this 'Continuing treatment' listing where the most recent PBS authority approval for this PBS-indication has been for gene therapy.
Invasive permanent assisted ventilation means ventilation via tracheostomy tube for greater than or equal to 16 hours per day.
In a patient who wishes to switch from PBS-subsidised nusinersen to PBS-subsidised risdiplam for this condition a wash out period may be required.

Compliance with Written Authority Required procedures

[23]      Schedule 3, entry for Selexipag

omit:

 

C11241

P11241

Pulmonary arterial hypertension (PAH)
Transitioning from non‑PBS subsidised to PBS‑subsidised supply ‑ 'Grandfather' treatment
Patient must have received non‑PBS subsidised treatment with this drug prior to 1 February 2021; AND
Patient must have failed to achieve/maintain a WHO Functional Class II status with PAH agents (other than this agent) given as dual therapy, prior to treatment initiation with this drug; AND
Patient must have had WHO Functional Class III PAH at treatment initiation with this drug; OR
Patient must have had WHO Functional Class IV PAH at treatment initiation with this drug; AND
Patient must not have developed disease progression while receiving treatment with this drug for this condition; AND
The treatment must form part of triple combination therapy consisting of: (i) one endothelin receptor antagonist, (ii) one phosphodiesterase‑5 inhibitor, (iii) selexipag (referred to as 'triple therapy'); OR
The treatment must form part of dual combination therapy consisting of either: (i) selexipag with one endothelin receptor antagonist, (ii) selexipag with one phosphodiesterase‑5 inhibitor, as triple combination therapy with selexipag‑an endothelin receptor antagonist‑a phoshodiesterase‑5 inhibitor is not possible due to an intolerance/contraindication to the endothelin receptor antagonist class/phosphodiesterase‑5 inhibitor class (referred to as 'dual therapy in lieu of triple therapy'); AND
The treatment must not be as monotherapy.
Must be treated by a physician with expertise in the management of PAH, with this authority application to be completed by the physician with expertise in PAH.
Patient must have had at least one PBS‑subsidised PAH agent prior to this authority application.
A prior PAH agent is any of: ambrisentan, bosentan, macitentan, sildenafil, tadalafil, epoprostenol, iloprost, riociguat.
For the purposes of PBS subsidy, an endothelin receptor antagonist is one of: (a) ambrisentan, (b) bosentan, (c) macitentan; a phosphodiesterase‑5 inhibitor is one of: (d) sildenafil, (e) tadalafil.
For the purposes of administering this restriction, disease progression has developed if at least one of the following has occurred:
(i) Hospitalisation due to worsening PAH;
(ii) Deterioration of aerobic capacity/endurance, consisting of at least a 15% decrease in 6‑Minute Walk Distance from baseline, combined with worsening of WHO functional class status;
(iii) Deterioration of aerobic capacity/endurance, consisting of at least a 15% decrease in 6‑Minute Walk Distance from baseline, combined with the need for additional PAH‑specific therapy;
(iv) Initiation of parenteral prostanoid therapy or long‑term oxygen therapy for worsening of PAH;
(v) Need for lung transplantation or balloon atrial septostomy for worsening of PAH.
PBS‑subsidy does not cover patients with pulmonary hypertension secondary to interstitial lung disease associated with connective tissue disease, where the total lung capacity is less than 70% of predicted.
PAH (WHO Group 1 pulmonary hypertension) is defined as follows:
(i) mean pulmonary artery pressure (mPAP) greater than or equal to 25 mmHg at rest and pulmonary artery wedge pressure (PAWP) less than or equal to 15 mmHg; or
(ii) where a right heart catheter (RHC) cannot be performed on clinical grounds, right ventricular systolic pressure (RVSP), assessed by echocardiography (ECHO), greater than 40 mmHg, with normal left ventricular function.

Compliance with Authority Required procedures