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

Short stature associated with biochemical growth hormone deficiency

Continuing treatment

Patient must be undergoing continuing PBS-subsidised therapy with this drug - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) recommence treatment, (iv) reclassify the PBS indication; AND

Patient must have achieved the 50th percentile growth velocity for bone age plus sex following the most recent supply; or

Patient must have achieved an increase in height standard deviation score for chronological age plus sex following the most recent supply; or

Patient must have achieved a minimum growth velocity of 4 cm per year following the most recent supply; or

Patient must have achieved a mid-parental height standard deviation score following the most recent supply; or

The treatment must have been administered at a dose that is lower than that recommended in the approved Product Information in the most recent supply; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

2. A bone age result performed within the last 12 months where the patient has a chronological age greater than 2.5 years.

3. The final adult height (in cm) of the patient's mother and father (where available).

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature associated with biochemical growth hormone deficiency

Change of drug

Patient must be undergoing existing PBS-subsidised growth hormone treatment where the prescribed drug is changing within the same PBS indication - subsidy through this treatment phase must not: (i) initiate treatment, (ii) recommence treatment, (iii) reclassify the PBS indication; AND

Patient must have been treated with PBS-subsidised growth hormone for less than 32 weeks; or

Patient must have been treated with PBS-subsidised growth hormone for at least 32 weeks, with an adequate response to treatment (as defined further below) having been demonstrated; or

Patient must have been treated with PBS-subsidised growth hormone for at least 32 weeks, with an adequate response to treatment (as defined further below) not demonstrated due to at least one of: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance to treatment arising from social/family problems, (v) sub-optimal dosing (i.e. the dose was less than the permitted upper dose range); AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Must be treated by a specialist or consultant physician in paediatric endocrinology; or

Must be treated by a specialist or consultant physician in general paediatrics in consultation with a nominated specialist or consultant physician in paediatric endocrinology; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

Definition:

An adequate response to the preceding supply of growth hormone for which the patient is changing from is one where the patient, for their sex, has achieved at least one of:

(a) the 50^th percentile growth velocity for bone age;

(b) an increase in height standard deviation score for chronological age;

(c) a minimum growth velocity of 4 cm per year;

(d) a mid-parental height standard deviation score.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

2. A bone age result performed within the last 12 months where the patient has a chronological age greater than 2.5 years.

Where growth data has been supplied within 3 months of this authority application, do not resupply this data.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature associated with biochemical growth hormone deficiency

Continuing treatment as a reclassified patient

Patient must be undergoing continuing PBS-subsidised therapy with this drug where the most recent authority approval for this drug was for a different PBS indication to that stated above - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) recommence treatment, (iv) reclassify the PBS indication where the most recent authority approval was for a different growth hormone, (v) reclassify the PBS indication and recommence treatment simultaneously; AND

The treatment must not be for the purposes of continuing treatment that is known to be non-efficacious for the patient - where an inadequate response has been observed for the most recent supply of this drug, it must have been confounded by at least one of the following: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance due to social/family problems, (v) a lower than recommended (as specified by this drug's approved Product Information) dose; AND

Patient must have had a height at or below the 1st percentile for age and sex immediately prior to commencing treatment; or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and a growth velocity below the 25th percentile for bone age and sex measured over the 12 month interval immediately prior to commencement of treatment (or the 6 month interval immediately prior to commencement of treatment if the patient was an older child at commencement of treatment); or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and an annual growth velocity of 14 cm per year or less in the 12 month period immediately prior to commencement of treatment, if the patient had a chronological age of 2 years or less at commencement of treatment; or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and an annual growth velocity of 8 cm per year or less in the 12 month period immediately prior to commencement of treatment, if the patient had a bone or chronological age of 2.5 years or less at commencement of treatment; AND

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 2 pharmacological growth hormone stimulation tests (e.g. arginine, clonidine, glucagon, insulin); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 pharmacological growth hormone stimulation test (e.g. arginine, clonidine, glucagon, insulin) and 1 physiological growth hormone stimulation test (e.g. sleep, exercise); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) with other evidence of growth hormone deficiency, including septo-optic dysplasia (absent corpus callosum and/or septum pellucidum), midline abnormality including optic nerve hypoplasia, cleft lip and palate, midfacial hypoplasia and central incisor, ectopic and/or absent posterior pituitary bright spot, absent empty sella syndrome, hypoplastic anterior pituitary gland and/or pituitary stalk/infundibulum, and genetically proven biochemical growth hormone deficiency either isolated or as part of hypopituitarism in association with pituitary deficits (ACTH, TSH, GnRH or vasopressin/ADH deficiency); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) and low plasma IGF-1 levels; or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) and low plasma IGFBP-3 levels; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in paediatric endocrinology; or

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in general paediatrics; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

An older child is defined as a male with a chronological age of at least 12 years or a bone age of at least 10 years, or a female with a chronological age of at least 10 years or a bone age of at least 8 years.

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. (a) A minimum of 12 months of growth data (height and weight measurements) from immediately prior to commencement of treatment, or a minimum of 6 months of growth data from immediately prior to commencement of treatment if the patient was an older child at commencement of treatment; and the result of a bone age assessment performed within the 12 months immediately prior to commencement of treatment where a patient had a chronological age greater than 2.5 years at commencement of treatment); OR

(b) Height and weight measurements from within three months prior to commencement of treatment for a patient whose height was at or below the 1^st percentile for age plus sex immediately prior to commencing treatment.

2. Evidence of biochemical growth hormone deficiency, including the type of tests performed and peak growth hormone concentrations.

3. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

4. A bone age result performed within the last 12 months where a patient has a chronological age greater than 2.5 years.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

Biochemical growth hormone deficiency should not be secondary to an intracranial lesion or cranial irradiation for applications under this category.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature associated with biochemical growth hormone deficiency

Transitioning from non-PBS to PBS-subsidised supply - Grandfather arrangements

Patient must be undergoing privately funded treatment (e.g. through a clinical trial, a sponsor compassionate access program, supply from an overseas jurisdiction) with this drug at the time of this authority application - subsidy through this treatment phase must only occur once per lifetime; AND

The treatment must not be for the purposes of continuing treatment that is known to be non-efficacious for the patient - where an inadequate response has been observed for the most recent supply of this drug, it must have been confounded by at least one of the following: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance due to social/family problems, (v) a lower than recommended (as specified by this drug's approved Product Information) dose; AND

Patient must have had a height at or below the 1st percentile for age and sex immediately prior to commencing treatment; or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and a growth velocity below the 25th percentile for bone age and sex measured over the 12 month interval immediately prior to commencement of treatment (or the 6 month interval immediately prior to commencement of treatment if the patient was an older child at commencement of treatment); or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and an annual growth velocity of 14 cm per year or less in the 12 month period immediately prior to commencement of treatment, if the patient had a chronological age of 2 years or less at commencement of treatment; or

Patient must have had both a height above the 1st and at or below the 25th percentiles for age and sex immediately prior to commencing treatment and an annual growth velocity of 8 cm per year or less in the 12 month period immediately prior to commencement of treatment, if the patient had a bone or chronological age of 2.5 years or less at commencement of treatment; AND

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 2 pharmacological growth hormone stimulation tests (e.g. arginine, clonidine, glucagon, insulin); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 pharmacological growth hormone stimulation test (e.g. arginine, clonidine, glucagon, insulin) and 1 physiological growth hormone stimulation test (e.g. sleep, exercise); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) with other evidence of growth hormone deficiency, including septo-optic dysplasia (absent corpus callosum and/or septum pellucidum), midline abnormality including optic nerve hypoplasia, cleft lip and palate, midfacial hypoplasia and central incisor, ectopic and/or absent posterior pituitary bright spot, absent empty sella syndrome, hypoplastic anterior pituitary gland and/or pituitary stalk/infundibulum, and genetically proven biochemical growth hormone deficiency either isolated or as part of hypopituitarism in association with pituitary deficits (ACTH, TSH, GnRH or vasopressin/ADH deficiency); or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) and low plasma IGF-1 levels; or

Patient must have evidence of biochemical growth hormone deficiency, with a peak serum growth hormone concentration less than 10 mU/L or less than or equal to 3.3 micrograms per litre in response to 1 growth hormone stimulation test (pharmacological or physiological e.g. arginine, clonidine, glucagon, insulin, sleep, exercise) and low plasma IGFBP-3 levels; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in paediatric endocrinology; or

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in general paediatrics; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

An older child is defined as a male with a chronological age of at least 12 years or a bone age of at least 10 years, or a female with a chronological age of at least 10 years or a bone age of at least 8 years.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. (a) A minimum of 12 months of growth data (height and weight measurements) from immediately prior to commencement of treatment, or a minimum of 6 months of growth data from immediately prior to commencement of treatment if the patient was an older child at commencement of treatment; and the result of a bone age assessment performed within the 12 months immediately prior to commencement of treatment where a patient had a chronological age greater than 2.5 years at commencement of treatment); OR

(b) Height and weight measurements from within three months prior to commencement of treatment for a patient whose height was at or below the 1^st percentile for age plus sex immediately prior to commencing treatment.

2. Evidence of biochemical growth hormone deficiency, including the type of tests performed and peak growth hormone concentrations.

3. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

4. A bone age result performed within the last 12 months where a patient has a chronological age greater than 2.5 years.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

Biochemical growth hormone deficiency should not be secondary to an intracranial lesion or cranial irradiation for applications under this category.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature and slow growth

Continuing treatment

Patient must be undergoing continuing PBS-subsidised therapy with this drug - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) recommence treatment, (iv) reclassify the PBS indication; AND

Patient must have previously received treatment under the PBS S100 Growth Hormone Program under the short stature and slow growth category; AND

Patient must have achieved the 50th percentile growth velocity for bone age plus sex following the most recent supply; or

Patient must have achieved an increase in height standard deviation score for chronological age plus sex following the most recent supply; or

Patient must have achieved a minimum growth velocity of 4 cm per year following the most recent supply; or

Patient must have achieved a mid-parental height standard deviation score following the most recent supply; or

The treatment must have been administered at a dose that is lower than that recommended in the approved Product Information in the most recent supply; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Patient must be male and must not have a height greater than or equal to 167.7cm; or

Patient must be female and must not have a height greater than or equal to 155.0cm; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

2. A bone age result performed within the last 12 months where the patient has a chronological age greater than 2.5 years.

3. The final adult height (in cm) of the patient's mother and father (where available).

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature and slow growth

Change of drug

Patient must be undergoing existing PBS-subsidised growth hormone treatment where the prescribed drug is changing within the same PBS indication - subsidy through this treatment phase must not: (i) initiate treatment, (ii) recommence treatment, (iii) reclassify the PBS indication; AND

Patient must have been treated with PBS-subsidised growth hormone for less than 32 weeks; or

Patient must have been treated with PBS-subsidised growth hormone for at least 32 weeks, with an adequate response to treatment (as defined further below) having been demonstrated; or

Patient must have been treated with PBS-subsidised growth hormone for at least 32 weeks, with an adequate response to treatment (as defined further below) not demonstrated due to at least one of: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance to treatment arising from social/family problems, (v) sub-optimal dosing (i.e. the dose was less than the permitted upper dose range); AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Patient must be male and must not have a height greater than or equal to 167.7cm; or

Patient must be female and must not have a height greater than or equal to 155.0cm; AND

Must be treated by a specialist or consultant physician in paediatric endocrinology; or

Must be treated by a specialist or consultant physician in general paediatrics in consultation with a nominated specialist or consultant physician in paediatric endocrinology; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

Definition:

An adequate response to the preceding supply of growth hormone for which the patient is changing from is one where the patient, for their sex, has achieved at least one of:

(a) the 50^th percentile growth velocity for bone age;

(b) an increase in height standard deviation score for chronological age;

(c) a minimum growth velocity of 4 cm per year;

(d) a mid-parental height standard deviation score.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

2. A bone age result performed within the last 12 months where the patient has a chronological age greater than 2.5 years.

Where growth data has been supplied within 3 months of this authority application, do not resupply this data.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature and slow growth

Continuing treatment as a reclassified patient

Patient must be undergoing continuing PBS-subsidised therapy with this drug where the most recent authority approval for this drug was for a different PBS indication to that stated above - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) recommence treatment, (iv) reclassify the PBS indication where the most recent authority approval was for a different growth hormone, (v) reclassify the PBS indication and recommence treatment simultaneously; AND

The treatment must not be for the purposes of continuing treatment that is known to be non-efficacious for the patient - where an inadequate response has been observed for the most recent supply of this drug, it must have been confounded by at least one of the following: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance due to social/family problems, (v) a lower than recommended (as specified by this drug's approved Product Information) dose; AND

Patient must have had a height no higher than the 1st percentile for age plus sex at the time treatment first commenced; AND

Patient must have had a growth velocity below the 25th percentile for bone age plus sex measured over a 12 month interval (or a 6 month interval for an older child) prior to having commenced treatment; or

Patient must have had an annual growth velocity of no higher than 8 cm per year where the patient had either a bone/chronological age no higher than 2.5 years prior to having commenced treatment; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Patient must be male and must not have a height greater than or equal to 167.7cm; or

Patient must be female and must not have a height greater than or equal to 155.0cm; AND

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in paediatric endocrinology; or

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in general paediatrics; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

An older child is defined as a male with a chronological age of at least 12 years or a bone age of at least 10 years, or a female with a chronological age of at least 10 years or a bone age of at least 8 years.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. A minimum of 12 months of growth data (height and weight measurements) from immediately prior to commencement of treatment, or a minimum of 6 months of growth data from immediately prior to commencement of treatment if the patient was an older child at commencement of treatment; and the result of a bone age assessment performed within the 12 months immediately prior to commencement of treatment where the patient had a chronological age greater than 2.5 years at commencement of treatment.

2. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

3. A bone age result performed within the last 12 months where a patient has a chronological age greater than 2.5 years.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.

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

Short stature and slow growth

Transitioning from non-PBS to PBS-subsidised supply - Grandfather arrangements

Patient must be undergoing privately funded treatment (e.g. through a clinical trial, a sponsor compassionate access program, supply from an overseas jurisdiction) with this drug at the time of this authority application - subsidy through this treatment phase must only occur once per lifetime; AND

The treatment must not be for the purposes of continuing treatment that is known to be non-efficacious for the patient - where an inadequate response has been observed for the most recent supply of this drug, it must have been confounded by at least one of the following: (i) a significant medical illness, (ii) major surgery (e.g. renal transplant), (iii) an adverse reaction to growth hormone, (iv) non-compliance due to social/family problems, (v) a lower than recommended (as specified by this drug's approved Product Information) dose; AND

Patient must have had a height no higher than the 1st percentile for age plus sex at the time treatment first commenced; AND

Patient must have had a growth velocity below the 25th percentile for bone age plus sex measured over a 12 month interval (or a 6 month interval for an older child) prior to having commenced treatment; or

Patient must have had an annual growth velocity of no higher than 8 cm per year where the patient had either a bone/chronological age no higher than 2.5 years prior to having commenced treatment; AND

Patient must not have a condition with a known risk of malignancy including chromosomal abnormalities such as Down and Bloom syndromes; AND

Patient must not have an active tumour or evidence of tumour growth or activity; AND

Patient must be male and must not have a height greater than or equal to 167.7 cm; or

Patient must be female and must not have a height greater than or equal to 155.0 cm; AND

Patient must be male and must not have a bone age of 15.5 years or more; or

Patient must be female and must not have a bone age of 13.5 years or more; AND

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in paediatric endocrinology; or

Must be treated by a medical practitioner in consultation with a nominated specialist or consultant physician in general paediatrics; AND

Patient must be undergoing treatment for the stated indication with only one growth hormone at any given time.

Applications for authorisation under this treatment phase must be made via the Online PBS Authorities System (real time assessment) or in writing via HPOS form upload or mail and must include:

1. (a) A minimum of 12 months of growth data (height and weight measurements) from immediately prior to commencement of treatment, or a minimum of 6 months of growth data from immediately prior to commencement of treatment if the patient was an older child at commencement of treatment; and the result of a bone age assessment performed within the 12 months immediately prior to commencement of treatment where a patient had a chronological age greater than 2.5 years at commencement of treatment; OR

(b) Height and weight measurements from within three months prior to commencement of treatment for a patient whose height was at or below the 1^st percentile for age plus sex immediately prior to commencing treatment.

2. Growth data (height and weight) for the most recent 6 month treatment period, including data at both the start and end of the treatment period. The most recent data must not be older than three months.

3. A bone age result performed within the last 12 months where the patient has chronological age greater than 2.5 years.

If the application is submitted through HPOS form upload or mail, it must include:

(i) details of the proposed prescription; and

(ii) 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).

Prescribe an appropriate amount of drug (maximum quantity in units) outlined within the 'Notes' section of this restriction.

Prescribers must keep a copy of any clinical records relating to the prescription, including such records required to demonstrate that the prescription was written in compliance with any relevant circumstances and/or purposes. These records must be kept for 2 years after the date the prescription to which the records relate is written.

In children with diabetes mellitus prescribers must ascertain that a growth failure is not due to poor diabetes control, diabetes control is adequate, and regular screening occurs for diabetes complications, particularly retinopathy.