National Health (Growth Hormone Program) Special Arrangement Amendment Instrument 2014 (No. 1) - PB 14 of 2014

EXPLANATORY STATEMENT

NATIONAL HEALTH ACT 1953

National Health (Growth Hormone Program) Special Arrangement Amendment Instrument 2014 (No.1)

PB 14 of 2014

Authority

Subsection 100(1) of the National Health Act 1953 (the Act) enables the Minister to make special arrangements for the supply of pharmaceutical benefits. Subsection 100(2) of the Act provides that the Minister may vary or revoke a special arrangement made under subsection 100(1).

Subsection 100(3) of the Act provides that Part VII of the Act, and instruments made for the purposes of Part VII have effect subject to a special arrangement made under subsection 100(1).

Purpose

The purpose of this legislative instrument, made under subsections 100(1) and (2) of the Act, is to amend the National Health (Growth Hormone Program) Special Arrangement 2011 (PB 88 of 2011) (the Special Arrangement) to make changes to the special arrangement relating to the Growth Hormone Program.

The Special Arrangement provides for the supply of pharmaceutical benefits with recombinant growth hormone (Somatropin) to eligible children and adolescents through the Growth Hormone Program. The Special Arrangement also sets out the dose of pharmaceutical benefit that may be approved for eligible patients.

The changes made to the Special Arrangement by this instrument reflect recommendations regarding the supply of Somatropin as a pharmaceutical benefit made by the Pharmaceutical Benefits Advisory Committee and clarify other administrative arrangements.

In summary these changes:

• set out the eligibility of patients who have reached the 10th adult height percentile for sex;
• clarify the data requirements for determining growth velocity;
• update current definitions or insert new definitions as required;
• amend the number of units of the pharmaceutical benefit that may, in one prescription, be directed to be supplied on any one occasion;
• amend the exclusion criteria which apply to persons applying for growth hormone treatment;
• require a person applying for initial treatment in the category of biochemical growth hormone deficiency where the person is diagnosed with hypothalamic-pituitary disease secondary to a structural lesion and associated with other pituitary hormone deficits (i) to provide additional information in the application;(ii) to meet a new treatment criteria requiring 12 months of observation; and (iii) describe the circumstances in which eligibility for this category must be assessed with regards to expert advice; 
• clarify how applications for change of category are assessed;

• provide that the Secretary has the power to determine continuing eligibility for persons with a mature skeleton to access growth hormone treatment under the Special Arrangement;
• remove ‘did not achieve and maintain mid parental height standard deviation score’ from continuing treatment provisions (where relevant), and add ‘achieves and maintains a mid parental height standard deviation score’ to the ‘response criteria’;
• allow twelve months treatment to be provided to patients commencing growth hormone treatment on the maximum dose, even if none of the response criteria are met after six months of treatment;
• remove the radiation dose threshold for the use of expert advice where eligibility is being determined in the category of growth retardation secondary to an intracranial lesion or cranial irradiation for the lesion;
• amend the circumstances in which the Secretary may determine a person is eligible to receive the next available dose level of the pharmaceutical item and a higher dose than the next available dose level of the pharmaceutical item;
• clarify the dose which the Secretary may determine a person who is recommencing treatment is eligible to receive;
• remove the eligibility criterion for initial treatment in the category of Turner Syndrome of a person having less than the 25th growth velocity percentile for bone age;
• amend the criteria for which the Secretary must assess a person’s eligibility using expert advice, for applications for initial treatment for Turner Syndrome; 
• clarifies that applications made for initial treatment in the category of infant at risk of hypoglycaemia secondary to biochemical growth hormone deficiency, may be assessed with advice from an individual member of the GHAC;
• set out the procedure for the review of patients with Prader-Willi Syndrome who have uncontrolled morbid obesity;
• provide that there is an increased starting dose of 6mg/m2/per week (unless otherwise requested by the treating doctor) for an older child receiving treatment in the categories of biochemical growth hormone deficiency, growth retardation secondary to an intercranial lesion or secondary to cranial irradiation for the lesion and growth hormone deficiency and precocious puberty;
• provide for three dose levels instead of four, with 1.5mg increments instead of 1.0mg;
• include other minor drafting amendments as required for interpretation; and
• state the transitional provisions which apply.

A change made by this instrument also reflects a change to the National Health (Listing of Pharmaceutical Benefits) Instrument 2010, which commences on the same day. This instrument amends Schedule 1 of the Special Arrangement to remove one pharmaceutical benefit with the listed drug ‘Somatropin’ and brand ‘Genotropin’.

Further information regarding the changes in each item is set out in the Attachment.

Consultation

These amendments have been made having regard to advice provided by the Pharmaceutical Benefits Advisory Committee (PBAC) and the Growth Hormone Advisory Committee (GHAC).

The PBAC, is an independent, expert advisory body comprising doctors, other health professionals and a consumer representative, which makes recommendations to the Australian Government about PBS listings.   PBAC members are selected from consumers, health economists, practising community pharmacists, general practitioners, clinical pharmacologists and specialists, with at least one member selected from each of those interests or professions.  Remaining members are persons whom the Minister is satisfied have qualifications and experience in a field relevant to the functions of the PBAC, and that would enable them to contribute meaningfully to the deliberations of the PBAC.

The GHAC provides assistance to the Australian Government Department of Health in its administration of the Pharmaceutical Benefits Scheme (PBS) Growth Hormone Program. The GHAC consists of five members, the Chair, Deputy Chair and three other members. Each member is appointed on the basis of their individual qualifications, knowledge and expertise in the field of paediatric endocrinology and holds their appointment at the discretion of the First Assistant Secretary, Pharmaceutical Benefits Division, Department of Health.

In addition, all affected clinicians were provided an opportunity to comment on the changes to the Growth Hormone Program prior to implementation.

This Instrument commences on 1 March 2014.

This instrument is a legislative instrument for the purposes of the Legislative Instruments Act 2003.

ATTACHMENT

PROVISION BY PROVISION DESCRIPTION OF THE NATIONAL HEALTH (GROWTH HORMONE PROGRAM) SPECIAL ARRANGEMENT AMENDMENT INSTRUMENT 2014 (No. 1)

Section 1  Name of Instrument

This section provides that this Instrument is the National Health (Growth Hormone Program) Special Arrangement Amendment Instrument 2014 (No. 1) and that it may also be cited as PB 14 of 2014.

Section 2  Commencement

This section provides that this Instrument commences on 1 March 2014.

Section 3   Amendments to PB 88 of 2011

This section provides that Schedule 1 amends the National Health (Growth Hormone Program) Special Arrangement 2011 (PB 88 of 2011) (the Special Arrangement).

Schedule 1

Item 1 inserts a definition of 1st adult height percentile.

Item 2 indicates that there is a new definition of 25th percentile for age and sex.

Item 3 inserts a definition of Departmental medical adviser to clarify this refers to those persons employed as Medical Officers within the Department.

Item 4 amends the definition of growth velocity so as to implement a reduced data requirement for a patient who has been identified as an older child and clarifies that where the person is not an older child, growth velocity is calculated over a year unless the Special Arrangement specifically provides otherwise.

Item 5 amends the definition of maturational or constitutional delay to omit the criterion relating to the estimated final height of the person so as to correct an inconsistency with the use of the term in eligibility and exclusion criteria.

Item 6 amends the definition of older child, reducing the qualifying chronological and bone age by 2.5 years. This item also indicates that there is a new definition of non-mature skeleton in subsection 3(2A).

Item 7 inserts a definition of Turner Syndrome – Ranke growth velocity chart.

Item 8 clarifies the requirements for a person to have a non-mature skeleton.

Item 9 amends the definition of percentile for height in relation to a person’s age and sex to exclude the 1st adult height percentile and the 25th percentile for age and sex.

Item 10 removes the word European to correctly cite the name of the Journal for Paediatrics.

Item 11 amends the definition of 1st percentile for age and sex so that this is correctly determined for persons 24 months and younger by reference to the document 1st percentile length for age values - children ages 0-3 years and for persons between 2 and 21 by reference to the document 1st percentile stature for age values – children ages 2-20 years. This amendment also clarifies that the titles of these documents have been updated in 2014 to correctly refer to the age of the persons.

This amendment also includes a new definition of 25th percentile for age and sex, indicating that for persons 24 months and younger this will be determined by reference to the document 25th percentile length for age values – children ages 0-3 years, and for persons older than 2 years and younger than 21, is determined with reference to the document 25th percentile stature for age values - children ages 2-20 years, published by the Department in 2014.

Item 12 changes the maximum quantity or number of units of the pharmaceutical benefit that may, in one prescription, be directed to be supplied on any one occasion from an amount sufficient for the first 19 weeks of treatment to an amount sufficient for the first 16 weeks of treatment.

Item 13 changes the maximum quantity or number of units of the pharmaceutical benefit that may, in one prescription, be directed to be supplied on any one occasion when the prescription for the first 16 weeks of treatment has already been written, from an amount sufficient for the remaining 13 weeks of treatment to an amount sufficient for the remaining 16 weeks of treatment.

Item 14 provides that the maximum number of occasions on which the supply of the pharmaceutical benefit may, in a prescription for initial treatment, be directed to be repeated, is one for a 16 week period. This enables a person to receive their entire supply of initial treatment by visiting their treating practitioner once, but requires two attendances to the dispenser of the pharmaceutical benefit.

Item 15 clarifies that the exclusion criteria relating to persons applying for treatment in categories other than Prader-Willi Syndrome who have skeletal maturity or maturational or constitutional delay and an estimated mature height in at least the 1st percentile only apply in relation to applications for initial treatment.

Item 16 clarifies that patients who have reached the 10th adult height percentile are excluded from receiving growth hormone treatment, for some categories.

Item 17 requires that applications for initial treatment for a person in the category of biochemical growth hormone deficiency where the person is diagnosed with hypothalamic-pituitary disease secondary to a structural lesion and associated with other pituitary hormone deficits must include radiological evidence of structural hypothalamic-pituitary disease, details of other pituitary hormone deficits and adequacy of hormone replacement therapy; and biochemical evidence of growth hormone deficiency with stable and adequate replacement of other hormone deficits at the time of testing. This information is required in addition to any other information for applications for initial treatment required under section 15.

Item 18 clarifies that patients applying for reclassification to another category must meet both the eligibility criteria for initial and continuing treatment in the category for which treatment is being sought before they can be approved to receive treatment in that category.

Item 19 changes the maximum quantity that may, in one prescription, be directed to be supplied on any one occasion, where the person has been approved for initial treatment, to an amount sufficient for the first 16 weeks of treatment.

Item 20 provides the Secretary discretion to consider expert advice and other matters and determine that a person may still receive treatment under the Special Arrangement notwithstanding the fact that they would otherwise be ineligible for having skeletal maturity.

This provision applies where a person has submitted an application for continuing treatment which indicates that but for their skeletal maturity they would be eligible for continuing treatment. However, it is not intended that the Secretary will consider expert advice in relation to every application that meets these preconditions, but that this discretion would be exercised where the Secretary deems that it is clinically appropriate to do so, including where there is evidence that the person is continuing to respond to growth hormone treatment.

Item 21 includes an additional response criterion against which a person’s response to growth hormone treatment, for a treatment period, is assessed. The additional criterion requires that the person achieves and maintains mid parental height standard deviation score.

Item 22 removes the word European to correctly cite the name of the relevant Journal.

Items 23, 27, 30, 38 and 40 clarify the reduced data requirement for determining the growth velocity for a patient who has been identified as an older child.

Item 24 clarifies that the criterion in paragraph 27(2)(b) refers to the 1st adult height percentile.

Items 25, 29, 32, 34, , 39, 41 and 42 enable 12 months of treatment to be provided to patients who commence growth hormone treatment on the maximum dose, even if none of the response criteria set out in section 21 are met after 6 months of treatment.

Item 26 inserts an additional criterion for a person applying for initial treatment in the category of biochemical growth hormone deficiency where the person has been diagnosed with hypothalamic-pituitary disease secondary to a structural lesion and associated with other pituitary hormone deficits. The additional criterion requires that there has been 12 months of observation of the person by a medical practitioner after treatment for the lesion has been completed, or if a medical practitioner has advised that it was not safe to treat the lesion, there has been 12 months of observation of the person by a medical practitioner.

Item 28 provides that expert advice and other matters must be considered to determine eligibility for initial treatment for a person applying in the category of biochemical growth hormone deficiency where the person has been diagnosed with hypothalamic-pituitary disease secondary to a structural lesion and associated with other pituitary hormone deficits who has a height above the 1st centile for age and sex or a growth velocity percentile for bone age above the 25th centile, but who demonstrate evidence of  biochemical growth hormone deficiency.

Item 31 removes the radiation dose threshold from the criteria for the Secretary to consider expert advice and other matters for treatment in the category of growth retardation secondary to an intercranial lesion or cranial irradiation where also there is no evidence of biochemical growth hormone deficiency for the person.  

Item 33 clarifies that the advice provided by the GHAC to the Secretary regarding whether a person’s hypoglycaemia is secondary to biochemical growth hormone deficiency can be provided by any individual GHAC member, rather than the wholly constituted GHAC. This reflects the fact that the appropriateness and effectiveness of treatment for persons seeking treatment in the Infant at risk of hypoglycaemia secondary to biochemical growth hormone deficiency category is relatively clear compared with other treatment categories where the advice of the wholly constituted GHAC is required.

Items 35 and 36 removes the eligibility criterion ‘that the person has not exhibited significant catch up growth’ for a person applying for initial treatment in the category of Turner Syndrome; and removes this criteria from being a circumstance in which the Secretary must assess a person’s eligibility under section 20 (expert advice etc) where the Secretary cannot determine whether the person meets the criterion.

This amendment also implements a new requirement that the Secretary must consider expert advice and other matters to assess a person’s eligibility for initial treatment in the Turner Syndrome category if the person is exhibiting significant catch up growth which is measured as either growth upwardly crossing height percentiles which is determined for the person with reference to the Turner Syndrome growth curve for girls or growth velocity less than the 50th percentile for bone age and sex.

Item 37 enables 12 months of treatment to be provided to patients who commence growth hormone treatment on the maximum dose; even if none of the response criteria set out in paragraphs 21 (1) (a), (b), or (c) are met after 6 months of treatment. This amendment also clarifies that the Turner Syndrome – Ranke growth velocity chart will be used to determine whether growth velocity for bone age for the person is below the mean height velocity for age for a person who has Turner Syndrome who has not received growth hormone treatment.

Item 43 enables 12 months of treatment to be provided to patients who commence growth hormone treatment in the category of Prader-Willi Syndrome on the maximum dose, even if none of the response criteria set out in the item of the table in subsection 45(1) that applies to the person are met after 6 months of treatment.

Item 44 clarifies the procedure for the review of patients who are receiving treatment in the Prader-Willi Syndrome category and have uncontrolled morbid obesity. If a person has uncontrolled morbid obesity the Secretary must consider expert advice and other matters.

Item 45 clarifies that dose is calculated for a patient in the category of Prader-Willi Syndrome, where the patient has a body mass index of greater than the 85th percentile for age and sex, using ideal weight for height to calculate body surface area, instead of actual weight.

This amendment also inserts a note clarifying that Division 6 of the Instrument is not relevant for a person who is not approved for initial or continuing treatment.

Item 46 restructures the subsection so that when the Secretary is making a determination under subsection 47(1), the Secretary is making a determination regarding the dose level for initial treatment and in making a determining under subsection 47(1A), the Secretary is making a determination regarding the dose level for either continuing treatment, a request to change category or a request to recommence treatment. A determination under subsection 47(1A) could be that, an increase, a decrease, or a continuation of the dose the person was receiving during their most recent treatment period is appropriate treatment for the person. Such a determination is still to be made in accordance with the table in section 47 and Division 6. This amendment will enable the Secretary to ensure that the appropriate level delegate will be responsible for decision-making under these new subsections.

Item 47 replaces the table in section 47 implementing three dose levels instead of four, with 1.5mg increments instead of 1.0mg. This will have the effect of patients on dose levels 2 or 3 having a higher dose than previously.  The reference to subsection (2) in Item 4(b) of the table is also amended to subsection (3) to correct a previous drafting error and Item 4 now includes a dose arrangement (c) to provide for a lower dose as set out in subsection (4).

Item 48 provides that the Secretary must approve a lower dose, for a patient in the category of Prader-Willi Syndrome, who has a mature skeleton, and a body mass index greater than the 85th percentile for age and sex, calculated as 0.04mg by ideal weight for height by week.

Item 49 provides that the Secretary may approve a dose other than dose level 1 where this is permitted under new subsection (4A) as well as under subsections (3) and (4).

Item 50 provides for an increased starting dose of dose level 2 (unless otherwise requested by the treating doctor) for an older child receiving treatment in the category of biochemical growth hormone deficiency, growth retardation secondary to an intercranial lesion or secondary to cranial irradiation for the lesion or growth hormone deficiency and precocious puberty.  

Item 51 removes the requirement for a person to achieve and maintain mid parental height standard deviation score or to meet at least one of the response criteria for subsection 50(2) to apply. This reflects that the Secretary only determines a dose under this section where a person has been found eligible for continuing treatment.

Item 52 clarifies that Division 6 is not relevant for a person who does not meet the continuing treatment provisions in the relevant category in Division 5. This is because the Secretary will not be determining the dose of a pharmaceutical benefit under Division 6 unless the person is eligible to receiving continuing treatment in the relevant category.  

Item 53 amends the heading to section 51 to reflect that the response criteria are no longer the relevant factors for determining eligibility for persons to receive either the next available dose level or a dose higher than the next available dose level.

Item 54 enables the Secretary to approve a dose level increase for a person not on maximum dose where:

• the person’s treating practitioner requests a higher dose and the person is an older child;
• the person has a bone age reading of 2.5 years or greater and failed to achieve a growth velocity greater than the 90th percentile for bone age and sex (measured over the most recent six month period) where the person has received treatment for two or less treatment periods; or has failed to achieve a growth velocity greater than the 75th percentile for bone age and sex over the most recent treatment period, where growth hormone treatment has been received for three or more treatment periods;
• the person has a bone age reading of less than 2.5 years and a height standard deviation score that is 0.25 standard deviations or less than the person’s height standard deviation score prior to the most recent treatment period; and
• the person’s treating practitioner has requested the higher dose and GHAC or a Departmental medical advisor advises there are exceptional circumstances that justify the higher dose and the Secretary determines a higher dose is appropriate having regard to the advice of the GHAC or the Departmental medical advisor.

Item 55 amends the circumstances in which the Secretary may determine a dose level increase higher than the next available dose level to include where the person’s treating practitioner requests the higher dose and the person is an older child.

Item 56 amends the heading to section 52 to reflect that this section now deals with the circumstances in which the Secretary can determine the same dose level for a person.

This amendment removes section 52 and replaces this with a new section indicating that unless section 51 applies, the Secretary must approve the person to receive the same dose level of pharmaceutical benefit as the dose level approved for the person’s most recently completed treatment period.

Item 57 clarifies that section 53 applies to applications for both initial and continuing treatment.

Item 58 clarifies that the higher dose the Secretary can approve under subsection 53(3) cannot exceed the maximum dose permitted for that treatment category as set out in the table in section 47.

Item 59 amends a previously incorrect reference by stating that the response criteria for a person in the category of Prader-Willi Syndrome are set out in section 45.

Item 60 amends the paragraph to reflect that the response criteria in section 21 are no longer the relevant factors for determining eligibility for a patient’s dose.

Item 61 adds Note 2 to clarify that the criteria referred to in paragraph 55(1)(b) are those criteria relevant to the person’s most recently completed treatment period as described in sections 49 to 54.

Item 62 removes the distinction between how dose is assessed for a person recommencing treatment in the same category and how dose is assessed for a person recommencing treatment in a different category. This reflects that the new dosage levels reduce confusion in this area.

This section requires the Secretary to approve the dose level of pharmaceutical benefit that had most recently been approved for the person before the person’s most recent treatment ceased, unless the person is eligible for another dose level pursuant to the relevant sections of the Division. This acknowledges that the most recent dose level should be maintained unless the person’s circumstances have changed since the person’s most recent treatment period.

Item 63 changes the ‘run out date’ from the date that the pharmaceutical benefit for treatment of a person for the first 19 weeks of an initial treatment period is expected to run out, to the date that the pharmaceutical benefit for treatment of a person for the first 16 weeks of an initial treatment period is expected to run out. This affects when a nominated dispenser is able to dispense the repeat supply of the pharmaceutical benefit, as this may only occur if there are less than 21 days remaining before the persons’ run out date.

Item 64 sets out the applicable transitional provisions. Any decisions relating to applications received by the Secretary prior to 1 March 2014 will be considered on the basis of the Special Arrangement as at 28 February 2014.

Persons on dose level 2 or 3 immediately before 1 March 2014 who become eligible for an incremental dose increase after 1 March 2014 will be eligible to receive dose level 3. Persons on dose level 4 immediately before 1 March 2014 who are eligible for treatment after 1 March 2014 will be eligible to receive dose level 3.

Item 65 removes the entry in Schedule 1 for Somatropin with Form ‘Injection 5mg (15 i.u.) in 1mL cartridge (with preservative)’ and Brand ‘Genotropin’.