Douglas Rice and Minister for Health

[2015] AATA 312

[2015] AATA 312  

Division	GENERAL ADMINISTRATIVE DIVISION
File Number	2014/5097
Re	Douglas Rice
APPLICANT
And	Minister for Health
RESPONDENT

DECISION

Tribunal	Deputy President P E Hack SC
Date	8 May 2015
Place	Brisbane

The decision under review is affirmed.

......................[Sgd]..................................................

Deputy President P E Hack SC

CATCHWORDS

HEALTH AND AGEING – therapeutic goods – access to unapproved therapeutic goods – approval for human growth hormone – whether clinical justification for use of product – whether efficacy and safety of product established – weighed against seriousness of condition – off-label prescription – no evidence of therapeutic benefit – no objective clinical justification – efficacy and safety of product unknown – decision under review affirmed.

LEGISLATION

Therapeutic Goods Act 1989 (Cth), ss 4, 9A, 19

SECONDARY MATERIALS

Therapeutic Goods Administration, Access to unapproved therapeutic goods via the Special Access Scheme (Department of Health and Ageing, Canberra, 2009)

Saugy M, et al, ‘Human Growth Hormone Doping in Sport’ (2006) 40 British Journal of Sports Medicine Suppl 1.1 i35-9

Ho K, ‘Consensus Guidelines for the Diagnosis and Treatment of Adults with GH Deficiency II: A Statement of the GH Research Society in association with the European Society for Pediatric Endocrinology, Lawson Wilkins Society, European Society of Endocrinology, Japan Endocrine Society, and Endocrine Society of Australia’ (2007) 157 European Journal of Endocrinology 695-700

REASONS FOR DECISION

Deputy President P E Hack SC

12 May 2015

Introduction

1. The applicant, Mr Douglas Rice, suffers from a number of debilitating medical conditions. He and his general practitioner, Dr Mark Laws, are convinced that the use of a therapeutic dose of human growth hormones (hGH) has aided in abating the symptoms of those conditions and improved Mr Rice’s general well-being.

2. In June 2014 Dr Laws made application to the Therapeutic Goods Administration for approval to import a particular variety of hGH, somatropin hGH (Jintropin). That product was not listed on the Australian Register of Therapeutic Goods, hence, the approval of the Therapeutic Goods Administration was required. It was not given.  On 1 July 2014, Mr Rice sought internal review of the decision by the respondent, the Minister for Health, to refuse approval.  On 3 September 2014, a delegate of the Minister confirmed the initial decision.

3. Mr Rice seeks a review of the decision made by the Minister’s delegate. 

   The legislative background

4. The matter is governed by the Therapeutic Goods Act 1989 (Cth). The objects of that Act are set out in s 4 in these terms:

   (1)The objects of this Act are to do the following, so far as the Constitution permits:

   (a)provide for the establishment and maintenance of a national system of controls relating to the quality, safety, efficacy and timely availability of therapeutic goods that are:

   (i)used in Australia, whether produced in Australia or elsewhere; or

   (ii)exported from Australia;

   (b)to provide a framework for the States and Territories to adopt a uniform approach to control the availability and accessibility, and ensure the safe handling, of poisons in Australia.

5. The Act, by s 9A, established the Australian Register of Therapeutic Goods “for the purpose of compiling information in relation to, and providing for evaluation of, therapeutic goods for use in humans”. It will suffice for present purposes to say that therapeutic goods includes goods represented to be, or likely to be taken to be, for therapeutic use. There is no doubt that the substance Mr Rice seeks to have approved comes within the definition of therapeutic goods.

6. Part 3-2 of the Act establishes a mechanism for the evaluation of therapeutic goods and, if the Secretary is satisfied the goods meet the proper standards, for the inclusion of the therapeutic goods in the Register. The Act contains prohibitions on the importation or supply of therapeutic goods that are not included on the Register and are not otherwise exempt. The present case concerns such an exemption, that provided for by s 19 of the Act. It reads:

   (1)The Secretary may, by notice in writing, grant an approval to a person for the importation into, or the exportation from, Australia or the supply in Australia of specified therapeutic goods that are not registered goods, listed goods or exempt goods:

   (a)for use in the treatment of another person; or

   (b)for use solely for experimental purposes in humans;

   and such an approval may be given subject to such conditions as are specified in the notice of approval.

7. In November 2009 the Therapeutic Goods Administration promulgated a policy document, “Access to unapproved therapeutic goods via the Special Access Scheme”. That Scheme allows for the importation of unapproved therapeutic goods for a single patient on a case-by-case basis. The document specifies three criteria for determining whether approval should be given – the patient, the product and the prescriber. It says this about those criteria:[1]

   [1]It is not necessary to reproduce the text concerning Criterion 3, The prescriber.

   Criterion 1 – The patient

   The application should contain adequate clinical justification for the use of the product, including an outline of the seriousness of the patient’s condition being treated.  When making an application, the practitioner will need to supply the following information:

   Patient details               Initials

   Date of birth (or age)

   Sex

   Patient ID or unit record number

   Diagnosis

   If applying for an extension of use under SAS – previous approval number, if available

   Clinical justification      This should include an outline of the seriousness of the patient’s condition and details of past treatment. If other approved treatments are available, the applicant will need to justify the use of the unapproved product in preference to those treatments. It is important for the justification to balance the availability of approved therapies against the seriousness of the patient’s condition and to include an appraisal of the expected benefits from the use of the unapproved product.

   Criterion 2 – The product

   The application should indicate how the product is to be used and include an appraisal of the efficacy and safety of the proposed use of the product. The application should include:

   Product details  For unapproved medicines

   Active ingredient
     Trade name
     Company/supplier (sponsor)
     Dose form

   For unapproved medical devices

   Name of device
     Company/sponsor  

   Administration              Dosage

   and monitoring             Route or method of administration

   regimeDuration of treatment

   Details of the techniques to determine both the outcome of the treatment and the occurrence and severity of any adverse reaction.

   This could be provided in terms of clinical, biochemical, haematological and/or immunological monitoring. Monitoring should occur throughout treatment and in some cases it may be appropriate for monitoring to continue for a period thereafter. In the event that a practitioner requests an extension of use under SAS, outcomes of monitoring, including measures of patient response and safety parameters, are required.

   Efficacy/safety data       Efficacy (or device performance) and safety data sufficient to support the proposed use of the product. A copy of the reference articles from which the data have been obtained should be included. Such references can range from evidence from published randomised controlled trials through evidence from published non-randomised trials and case reports, to consensus opinion. The level of evidence required of the reference articles will depend on the seriousness of the condition (see how to balance the criteria).

8. The Minister also emphasises these passages from the document:[2]

   The efficacy and safety data submitted in support of the application should be weighed against the seriousness of the condition. As a general rule, the less serious the clinical need, the higher the degree of evidence needed to support the use of the product.

   …

   In the event that another product is evaluated by TGA and approved for treatment of an indication, the level of evidence required in support of an application to use an unapproved product instead of the new product for that indication is high. This is particularly so for products with the same active ingredient or with active ingredients in the same therapeutic class.

   …

   The clinical justification should include discussion as to why the newly approved product is not acceptable for the treatment of the individual patient and this should be based on medical reasons and not on grounds of cost or convenience. This requirement is imposed in part because it is the TGA’s responsibility to encourage at all times the availability of approved (fully evaluated) products. To do otherwise would remove the incentive for a sponsor to seek registration of the unapproved product or for other sponsors to seek registration of alternative products for treatment of the indication.

   [2]Exhibit 8, Attachment A, page 17.

   The issues

9. It is not in issue that the policy document is an appropriate guide to the exercise of the discretion. Whilst ultimately the statute governs that exercise, properly formulated policy, not inconsistent with the statute, will generally be taken into account by the Tribunal. I see no reason not to do so in the present case.

10. Two matters were relied upon by the Minister’s delegate to refuse approval – the absence of an acceptable clinical justification for use by Mr Rice, and the absence of information to support the standards of manufacture, quality or safety of the product. They remain the issues.

    Consideration

11. Some further factual background needs to be noticed. The first is that in May 2014, Mr Rice was suffering from numerous ailments. It is unnecessary to detail them and to do so would be needlessly intrusive on Mr Rice’s privacy – they are set out in the various reports provided by Dr Laws as well as the material produced by Mr Rice. Mr Rice undertook considerable research on the internet and appears to have been particularly taken with the work of Dr Elmer Cranton. As best as I am able to understand from the material presented, Dr Cranton’s theory is that human growth therapy can slow down the process of ageing. In an extract from his website provided by Mr Rice to the TGA,[3] Dr Cranton explains:

    Aging is to some extent deficiency state. By replacing those things that decline with age we can turn it back some and slow it down some, but we can’t stop the process.

    [3]Exhibit 1, page 170.

12. Dr Laws described his dealings with Mr Rice about human growth hormones in this way:[4]

    He approached me about Human Growth Hormone therapy. I informed him I had limited practical experience in this field, but was aware of the potential benefits and risks. Mr Rice had read widely and deeply on the subject and proposed a dose regime which was reasonable in my subsequent research. This was started in late April 2014. I have been consulting with Mr Rice on a regular basis since then. In the past twelve  months, there have been no problems or side effects from his use of HGH. The benefits were evident in his appearance and mood. He has lost fat and gained muscle. His strength and flexibility have improved significantly. Subjectively, Mr Rice reports improved mood, energy, strength, libido and reduced back, joint and muscle pain. He has had regular blood tests which have not shown any problems with blood glucose, liver, kidney, blood count and thyroid.  His blood pressure is normal. The IGF level on HGH is at the upper end of the normal scale indicating that the dose is adequate, but not excessive.

    [4]Exhibit 3.

13. The human growth hormone prescribed by Dr Laws was one of a number on the Register;[5] however, the products registered have been registered for two categories of indications, paediatric growth hormone deficiency and adult growth hormone deficiency. Mr Rice has not been diagnosed with growth hormone deficiency and there is no evidence that he has any of the clinical signs of the condition. There are three validated tests for the diagnosis of the condition;[6] none has been performed on him. Where, as in Mr Rice’s case, it is prescribed or administered for use other than for the indications specified in the Register, the use is described as “off-label”. Off-label prescription cannot qualify for subsidy under the pharmaceutical benefits scheme and Mr Rice was required to pay full price for the hGH treatment prescribed by Dr Laws. The cost of obtaining the drugs to date has been considerable – he mentioned a figure of $12,000.

    [5]Dr Anthony Hobbs, Principal Medical Adviser at the TGA, says that there are 47 human growth hormone products on the Register.

    [6]Exhibit 6, paragraph [24].

14. It seems to me that cost is a major driver of Mr Rice’s application, lodged on his behalf by Dr Laws in June 2014, to obtain approval to import Jintropin, manufactured by GenSci, a Chinese company.

15. So far as the efficacy of the treatment already provided, and that proposed, there is no objective evidence whatsoever that demonstrates the efficacy of hGH therapy in Mr Rice’s circumstances or that demonstrates the clinical efficacy of Jintropin. I do not doubt that Mr Rice genuinely feels much improved following the therapy to date, however, that improved well-being is not shown by any acceptable evidence to be attributable to the therapy and, as Dr Hobbs pointed out, might be explicable on an entirely different basis.

16. What is demonstrated is that GenSci lists paediatric growth hormone deficiency, severe burns and adult growth hormone deficiency as conditions where Jintropin is indicated. Adult growth hormone deficiency is a recognised medical condition with well-defined clinical signs. As I have said, there are three tests each capable of demonstrating the presence of growth hormone deficiency biochemically. Mr Rice has not been diagnosed with growth hormone deficiency nor undertaken any of the recognised tests.

17. One paper referred to by Dr Hobbs notes that no adverse side-effects have been observed with the therapeutic use of hGH.[7]  The paper goes on to point out that the long-term risks are not well known. However, it could not be said that Mr Rice’s use of hGH is a therapeutic use. He does not suffer from any condition for which the use of them is indicated. Moreover, the consensus of expert medical opinion is against the use of hGH where decline is attributable to the ordinary reduction in growth hormone with the passage of time. Consensus guidelines for the diagnosis and treatment of adults with growth hormone deficiency, published by  leading endocrinologists in 2007, note:

    The age-related declined in the GH-IGF-I status does not warrant GH supplementation; however patients with proved GHD should be treated.[8]

    [7]Saugy M et al, ‘Human Growth Hormone Doping in Sport’ (2006) 40 British Journal of Sports Medicine Suppl 1.1 i35-9.

    [8]           Ho K, ‘Consensus Guidelines for the Diagnosis and Treatment of Adults with GH Deficiency II: A Statement   of the GH Research Society in Association with the European Society for Pediatric Endocrinology, Lawson      Wilkins Society, European Society of Endocrinology, Japan Endocrine Society, and Endocrine Society of        Australia’ (2007) 157 European Journal of Endocrinology 695-700.

18. In the result, there is no objective evidence that the therapy already undertaken has had therapeutic benefit, no research that suggests that the therapy would have therapeutic benefit in the future for Mr Rice and the consensus view of highly qualified medical specialists is against the use of hormone growth therapy in Mr Rice’s circumstances.

19. Added to this is the fact that the manufacturer from whom Mr Rice would source the hGH is unknown except for the fact of having committed seemingly serious breaches of the criminal law of the United States of America resulting in the imposition of significant penalties in 2010 for illegally marketing human growth hormones in that country.

20. In those circumstances it is, in my view, impossible to conclude that the discretion be exercised favourably to Mr Rice.

21. Mr Rice asserts that it is in his right, with his doctor, to have medication that he believes assists in his treatment. This decision does not interfere with his right.  His doctor can, in the exercise of his clinical judgement, prescribe for Mr Rice hGH that is recorded on the Register. If Mr Rice wants to go beyond that, the Parliament has determined that certain safeguards should exist. Mr Rice is well short of satisfying those safeguards.

22. The decision under review was undoubtedly correct. It will be affirmed.

I certify that the preceding 22 (twenty -two) paragraphs are a true copy of the reasons for the decision herein of Deputy President P E Hack SC

..........................[Sgd]..............................................

Associate

Dated 8 May 2015

Date of hearing	16 April 2015
Applicant	In person
Counsel for the Respondent	Mr T Howe QC
Solicitors for the Respondent	Therapeutic Goods Administration, Office of Legal Services