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

Short stature associated with biochemical growth hormone deficiency

Initial treatment

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 have a current height at or below the 1st percentile for age and sex; or

Patient must have a current height above the 1st and at or below the 25th percentiles for age and sex and a growth velocity below the 25th percentile for bone age and sex measured over a 12 month interval (or a 6 month interval for an older child); or

Patient must have a current height above the 1st and at or below the 25th percentiles for age and sex and an annual growth velocity of 14 cm per year or less if the patient has a chronological age of 2 years or less; or

Patient must have a current height above the 1st and at or below the 25th percentiles for age and sex and an annual growth velocity of 8 cm per year or less if the patient has a bone or chronological age of 2.5 years or less; 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 not have previously received treatment under the PBS S100 Growth Hormone Program; 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.

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 recent growth data (height and weight measurements) or a minimum of 6 months of recent growth data for an older child. The most recent data must not be more than three months old at the time of application; or

(b) Height and weight measurements, not more than three months old at the time of application, for a patient whose current height is at or below the 1^st percentile for age and sex.

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

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

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 associated with biochemical growth hormone deficiency

Recommencement of treatment

Patient must be undergoing recommencing treatment following a temporary treatment break (i.e. a lapse) from this drug for the stated indication above - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) reclassify the PBS indication; AND

Patient must have had a lapse in growth hormone treatment; AND

The treatment must not be for the purposes of resuming 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 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.

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. Recent growth data (height and weight, not older than three months).

2. 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 associated with biochemical growth hormone deficiency

Recommencement of treatment as a reclassified patient

Patient must be undergoing treatment that is simultaneously: (a) recommencing treatment following a temporary break in treatment (i.e. a lapse), plus (b) reclassifying the PBS indication whilst continuing with the same growth hormone; subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) reclassify the PBS indication where the most recent authority approval was for a different growth hormone; AND

Patient must have had a lapse in growth hormone treatment; 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

Initial treatment

Patient must have a current height at or below the 1st percentile for age and sex; AND

Patient must have a growth velocity below the 25th percentile for bone age and sex measured over a 12 month interval (or a 6 month interval for an older child); or

Patient must have an annual growth velocity of 8 cm per year or less if the patient has a bone or chronological age of 2.5 years or less; 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 not have previously received treatment under the PBS S100 Growth Hormone Program; 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 167.7 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 maturational or constitutional delay in combination with an estimated mature height equal to or above 160.1 cm; or

Patient must be female and must not have maturational or constitutional delay in combination with an estimated mature height equal to or above 148.0 cm; 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.

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 recent growth data (height and weight measurements) or a minimum of 6 months of recent growth data for an older child. The most recent data must not be more than three months old at the time of application.

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

3. Confirmation of the patient's maturational or constitutional delay status.

4. If the patient has maturational or constitutional delay, confirmation that the patient has an estimated mature height below the 1^st adult height percentile.

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

Recommencement of treatment

Patient must be undergoing recommencing treatment following a temporary treatment break (i.e. a lapse) from this drug for the stated indication above - subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) reclassify the PBS indication; AND

Patient must have had a lapse in growth hormone treatment; AND

The treatment must not be for the purposes of resuming 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 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.

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. Recent growth data (height and weight, not older than three months).

2. 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

Recommencement of treatment as a reclassified patient

Patient must be undergoing treatment that is simultaneously: (a) recommencing treatment following a temporary break in treatment (i.e. a lapse), plus (b) reclassifying the PBS indication whilst continuing with the same growth hormone; subsidy through this treatment phase must not: (i) initiate treatment, (ii) change the prescribed drug, (iii) reclassify the PBS indication where the most recent authority approval was for a different growth hormone; AND

Patient must have had a lapse in growth hormone treatment; 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.

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. Recent growth data (height and weight, not 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.