National Health (Highly Specialised Drugs Program) Special Arrangement Amendment (May Update) Instrument 2023 (Cth)

PB 37 of 2023

National Health (Highly Specialised Drugs Program) Special Arrangement Amendment (May Update) Instrument 2023

National Health Act 1953

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

Date                 27 April 2023                                        

NIKOLAI TSYGANOV

Assistant Secretary

Pricing and PBS Policy 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 2023.

(2)This instrument may also be cited as PB 37 of 2023.

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 May 2023	1 May 2023

Note:          This table relates only to the provisions of this instrument as originally made.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. Schedule 1, entry for Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor

substitute:

Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor	Pack containing 56 tablets elexacaftor 50 mg with tezacaftor 25 mg and with ivacaftor 37.5 mg and 28 tablets ivacaftor 75 mg	Oral	Trikafta	C13932 C13991	See Schedule 2	See Schedule 2
Pack containing 56 tablets elexacaftor 100 mg with tezacaftor 50 mg and with ivacaftor 75 mg and 28 tablets ivacaftor 150 mg	Oral	Trikafta	C13962 C13980	See Schedule 2	See Schedule 2

1. Schedule 1, entry for Ustekinumab

insert in numerical order in the column headed “Circumstances”: C13975 C13976 C14010

1. Schedule 2, entry for Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor

omit from the column headed “Circumstances”: C12962 C12972             substitute: C13932 C13962 C13980 C13991

1. Schedule 2, entry for Ustekinumab

insert in numerical order in the column headed “Circumstances”: C13975 C13976 C14010

1. Schedule 3, entry for Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor

substitute:

Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor	C13932	Cystic fibrosis Initial treatment Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation. Patient must have at least one F508del mutation in the cystic fibrosis transmembrane conductance (CFTR) gene; AND The treatment must be given concomitantly with standard therapy for this condition; AND Patient must have either chronic sinopulmonary disease or gastrointestinal and nutritional abnormalities, prior to initiating treatment with this drug. Patient must be aged between 6 and 11 years inclusive. This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information. The authority application must be in writing and must include: (1) a completed authority prescription; and (2) a completed Cystic Fibrosis Authority Application Supporting Information Form; and (3) details of the pathology report substantiating the patient having at least one F508del mutation - quote each of the: (i) name of the pathology report provider, (ii) date of pathology report, (iii) unique identifying number/code that links the pathology result to the individual patient; and (4) current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.	Compliance with Written Authority Required procedures
C13962	Cystic fibrosis Initial treatment Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation. Patient must have at least one F508del mutation in the cystic fibrosis transmembrane conductance (CFTR) gene; AND The treatment must be given concomitantly with standard therapy for this condition; AND Patient must have either chronic sinopulmonary disease or gastrointestinal and nutritional abnormalities, prior to initiating treatment with this drug. Patient must be at least 6 years of age. This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information. The authority application must be in writing and must include: (1) a completed authority prescription; and (2) a completed Cystic Fibrosis Authority Application Supporting Information Form; and (3) details of the pathology report substantiating the patient having at least one F508del mutation - quote each of the: (i) name of the pathology report provider, (ii) date of pathology report, (iii) unique identifying number/code that links the pathology result to the individual patient; and (4) current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.	Compliance with Written Authority Required procedures
C13980	Cystic fibrosis Continuing treatment Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation. Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND The treatment must be given concomitantly with standard therapy for this condition. Patient must be at least 6 years of age. This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information. The authority application must be in writing and must include: (1) a completed authority prescription; and (2) a completed Cystic Fibrosis Continuing Authority Application Supporting Information Form; and (3) current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.	Compliance with Written Authority Required procedures
C13991	Cystic fibrosis Continuing treatment Must be treated by a specialist respiratory physician with expertise in cystic fibrosis or in consultation with a specialist respiratory physician with expertise in cystic fibrosis if attendance is not possible due to geographic isolation; AND Must be treated in a centre with expertise in cystic fibrosis or in consultation with a centre with expertise in cystic fibrosis if attendance is not possible due to geographic isolation. Patient must have previously received PBS-subsidised treatment with this drug for this condition; AND The treatment must be given concomitantly with standard therapy for this condition. Patient must be aged between 6 and 11 years inclusive. This pharmaceutical benefit is not PBS-subsidised for this condition in a patient who is currently receiving one of the strong CYP3A4 inducers outlined in the Product Information. The authority application must be in writing and must include: (1) a completed authority prescription; and (2) a completed Cystic Fibrosis Continuing Authority Application Supporting Information Form; and (3) current CYP3A4 inhibitors, CYP3A4 inducers and IV antibiotics.	Compliance with Written Authority Required procedures

1. Schedule 3, entry for Ustekinumab

insert in numerical order after existing text:

C13975	Moderate to severe ulcerative colitis Initial treatment - Initial 1 (new patient) Must be treated by a gastroenterologist (code 87); OR Must be treated by a consultant physician [internal medicine specialising in gastroenterology (code 81)]; OR Must be treated by a consultant physician [general medicine specialising in gastroenterology (code 82)]. Patient must have failed to achieve an adequate response to a 5-aminosalicylate oral preparation in a standard dose for induction of remission for 3 or more consecutive months or have intolerance necessitating permanent treatment withdrawal; AND Patient must have failed to achieve an adequate response to azathioprine at a dose of at least 2 mg per kg daily for 3 or more consecutive months or have intolerance necessitating permanent treatment withdrawal; OR Patient must have failed to achieve an adequate response to 6-mercaptopurine at a dose of at least 1 mg per kg daily for 3 or more consecutive months or have intolerance necessitating permanent treatment withdrawal; OR Patient must have failed to achieve an adequate response to a tapered course of oral steroids, starting at a dose of at least 40 mg prednisolone (or equivalent), over a 6 week period or have intolerance necessitating permanent treatment withdrawal, and followed by a failure to achieve an adequate response to 3 or more consecutive months of treatment of an appropriately dosed thiopurine agent; AND Patient must have a Mayo clinic score greater than or equal to 6; OR Patient must have a partial Mayo clinic score greater than or equal to 6, provided the rectal bleeding and stool frequency subscores are both greater than or equal to 2 (endoscopy subscore is not required for a partial Mayo clinic score); AND The treatment must not exceed a single dose to be administered at week 0 under this restriction. Patient must be at least 18 years of age. 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), which includes: (i) the completed current Mayo clinic or partial Mayo clinic calculation sheet including the date of assessment of the patient's condition; and (ii) details of prior systemic drug therapy [dosage, date of commencement and duration of therapy]. All tests and assessments should be performed preferably whilst still on treatment, but no longer than 4 weeks following cessation of the most recent prior conventional treatment. The most recent Mayo clinic or partial Mayo clinic score must be no more than 4 weeks old at the time of application. An assessment of a patient's response to this initial course of treatment must be conducted between 8 and 16 weeks of therapy. Where a response assessment is not conducted within the required timeframe, the patient will be deemed to have failed to respond to treatment with this drug, unless the patient has experienced a serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment. 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 this treatment cycle. Serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as a treatment failure. If treatment with any of the above-mentioned drugs is contraindicated according to the relevant TGA-approved Product Information, details must be provided at the time of application. If intolerance to treatment develops during the relevant period of use, which is of a severity necessitating permanent treatment withdrawal, details of this toxicity must be provided at the time of application. A maximum of 16 weeks of treatment with this drug will be approved under this criterion. Two completed authority prescriptions should be submitted with every initial application for this drug. One prescription should be written under S100 (Highly Specialised Drugs) for a weight-based loading dose, containing a quantity of up to 4 vials of 130 mg and no repeats. The second prescription should be written under S85 (General) for the subsequent first dose, containing a quantity of 1 pre-filled syringe of 90 mg and no repeats.	Compliance with Written Authority Required procedures
C13976	Moderate to severe ulcerative colitis Initial treatment - Initial 2 (change or recommencement of treatment after a break in biological medicine of less than 5 years) Must be treated by a gastroenterologist (code 87); OR Must be treated by a consultant physician [internal medicine specialising in gastroenterology (code 81)]; OR Must be treated by a consultant physician [general medicine specialising in gastroenterology (code 82)]. Patient must have received prior PBS-subsidised treatment with a biological medicine for this condition in this treatment cycle; AND Patient must not have already failed, or ceased to respond to, PBS-subsidised treatment with this drug for this condition during the current treatment cycle; AND The treatment must not exceed a single dose to be administered at week 0 under this restriction. Patient must be at least 18 years of age. 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), which includes: (i) the completed current Mayo clinic or partial Mayo clinic calculation sheet including the date of assessment of the patient's condition; and (ii) the details of prior biological medicine treatment including the details of date and duration of treatment. An application for a patient who has received PBS-subsidised biological medicine treatment for this condition who wishes to change or recommence therapy with this drug, must be accompanied by evidence of a response to the patient's most recent course of PBS-subsidised biological medicine treatment, within the timeframes specified below. An assessment of a patient's response to this initial course of treatment must be conducted between 8 and 16 weeks of therapy. Where a response assessment is not conducted within the required timeframe, the patient will be deemed to have failed to respond to treatment with this drug, unless the patient has experienced a serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment. 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 this treatment cycle. Serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as a treatment failure. A patient who fails to demonstrate a response to treatment with this drug under this restriction will not be eligible to receive further PBS-subsidised treatment with this drug in this treatment cycle. A patient may re-trial this drug after a minimum of 5 years have elapsed between the date the last prescription for a PBS-subsidised biological medicine was approved in this cycle and the date of the first application under a new cycle under the initial 3 treatment restriction. A maximum of 16 weeks of treatment with this drug will be approved under this criterion. Two completed authority prescriptions should be submitted with every initial application for this drug. One prescription should be written under S100 (Highly Specialised Drugs) for a weight-based loading dose, containing a quantity of up to 4 vials of 130 mg and no repeats. The second prescription should be written under S85 (General) for the subsequent first dose, containing a quantity of 1 pre-filled syringe of 90 mg and no repeats. Details of the accepted toxicities including severity can be found on the Services Australia website.	Compliance with Written Authority Required procedures
C14010	Moderate to severe ulcerative colitis Initial treatment - Initial 3 (recommencement of treatment after a break in biological medicine of more than 5 years) Must be treated by a gastroenterologist (code 87); OR Must be treated by a consultant physician [internal medicine specialising in gastroenterology (code 81)]; OR Must be treated by a consultant physician [general medicine specialising in gastroenterology (code 82)]. Patient must have previously received PBS-subsidised treatment with a biological medicine for this condition; AND Patient must have had a break in treatment of 5 years or more from the most recently approved PBS-subsidised biological medicine for this condition; AND Patient must have a Mayo clinic score greater than or equal to 6; OR Patient must have a partial Mayo clinic score greater than or equal to 6, provided the rectal bleeding and stool frequency subscores are both greater than or equal to 2 (endoscopy subscore is not required for a partial Mayo clinic score); AND The treatment must not exceed a single dose to be administered at week 0 under this restriction. Patient must be at least 18 years of age. 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), which includes: (i) the completed current Mayo clinic or partial Mayo clinic calculation sheet including the date of assessment of the patient's condition; and (ii) the details of prior biological medicine treatment including the details of date and duration of treatment. All tests and assessments should be performed preferably whilst still on treatment, but no longer than 4 weeks following cessation of the most recent prior conventional treatment. The most recent Mayo clinic or partial Mayo clinic score must be no more than 4 weeks old at the time of application. An application for a patient who has received PBS-subsidised biological medicine treatment for this condition who wishes to recommence therapy with this drug, must be accompanied by evidence of a response to the patient's most recent course of PBS-subsidised biological medicine treatment, within the timeframes specified below. An assessment of a patient's response to this initial course of treatment must be conducted between 8 and 16 weeks of therapy. Where a response assessment is not conducted within the required timeframe, the patient will be deemed to have failed to respond to treatment with this drug, unless the patient has experienced a serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment. 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 this treatment cycle. Serious adverse reaction of a severity resulting in the necessity for permanent withdrawal of treatment is not considered as a treatment failure. A maximum of 16 weeks of treatment with this drug will be approved under this criterion. Two completed authority prescriptions should be submitted with every initial application for this drug. One prescription should be written under S100 (Highly Specialised Drugs) for a weight-based loading dose, containing a quantity of up to 4 vials of 130 mg and no repeats. The second prescription should be written under S85 (General) for the subsequent first dose, containing a quantity of 1 pre-filled syringe of 90 mg and no repeats. Details of the accepted toxicities including severity can be found on the Services Australia website.	Compliance with Written Authority Required procedures