Off-label use in Australia and relevant considerations

Mabel Tsui

Academic at the Queensland University of Technology, Brisbane

With the growth of off-label use there is now increasing enthusiasm in how pharmaceutical companies will promote their products to health providers for use which has not been authorised by the relevant national drug authority. In this article, Mabel Tsui will consider the practice of off-label prescription by medical practitioners and hospitals in Australia, as well as some lessons from the US in terms of potential implications pharmaceutical companies may face during the course of promotion.

The Therapeutic Goods Act and “off-label” prescription

The Therapeutic Goods Act 1989 (Cth) is the national legislative scheme under which pharmaceuticals are regulated in Australia. The Act is administered by the Therapeutic Goods Administration, a branch of the Commonwealth Department of Health and Ageing. Section 4 lists the objectives of the Act, one of which is to provide “for the establishment and maintenance of a national system of controls relating to the quality, safety, efficacy and timely availability of therapeutic goods” which are produced or used in Australia or exported from Australia.

“…off-label prescription of medicines in Australia is a growing phenomenon…”

“Off-label” use of medicines is not expressly defined in the Act, and as such the term is used loosely in Australia. This article adopts the New South Wales Therapeutic Advisory Group (NSW TAG) which refers to such use as “prescriptions for registered medicines for use in a manner not listed in the approved prescribing guidelines”.1 The closest reference to such use in the Act is section 19, entitled Exemptions for Special and Experimental Uses. Essentially, this section sets out limited circumstances under which off-label use is allowed, including for “experimental purposes in humans”. In addition, section 19(5) expressly provides that a “specified medical practitioner” may be authorised to supply drugs for off-label use, with the relevant approval authority able to impose any necessary conditions to this supply. The legislative intention behind this section is undoubtedly to promote research and development, while ensuring compliance with safety and research ethics.

As detailed as the Act may appear, professional bodies such as the Australian and New Zealand College of Anaesthetists (ANZCA) have pointed out that section 19 only refers to off-label use in very limited circumstances.2 In addition, research conducted by the author found cases from Australian courts which considered only the import / export aspect of section 19 or the section overall as a secondary issue. There is therefore very little judicial guidance on the operation of section 19 in terms of prescription by a specified medical practitioner.

“…some literature suggests that failure by a health provider to recommend off-label use of certain medications in some cases constitutes a form of malpractice.”

However, in light of the general acceptance of off-label use and prescription in Australia, and in fact world-wide, theory-based operations of the legislative provisions regulating such use is to a large extent moot. To prove this point, the literature in fact provides statistics which demonstrate that off-label prescription of medicines in Australia is a growing phenomenon and some literature suggests that failure by a health provider to recommend off-label use of certain medications in some cases constitutes a form of malpractice.

Encouraged with appropriate guidelines

What may be the clearest demonstration of an unspoken endorsement of off-label promotion by medical practitioners and hospitals is that of government endorsement – specifically from the New South Wales (NSW) state government. In September 2003, the NSW TAG, with funding support from the state government’s Department of Health, released a working paper which expressly recognised that off-label use of medicines “has long been recognised as an important issue in teaching hospitals” and the objective of the paper was to “…provide guidance … in formulating local policy for the use of medicines used off-label and unregistered medicines imported for personal use and administered in hospitals.” In November 2006, the Group’s recommendations were published in the Medical Journal of Australia and emphasised the importance of evaluating evidence of appropriateness (which reflects the accepted practice world-wide) and patient knowledge that the prescription is on an off-label basis, consistent with the philosophy of the Australian government objective of Quality Use of Medicines (QUM) .

The Act and off-label promotion – potential lessons from the United States

In contrast to the provisions regarding off-label use, the Australian legislative scheme is quite strict on pharmaceutical companies and their advertising and promotional activities. The Act and the associated Therapeutic Goods Regulations 1990 as well as the Therapeutic Goods Association Advertising Code have the combined effect that certain marketing activities in Australia are either fully prohibited or restricted. However, the Act makes a very clear concession again to the possibility of off-label use as section 42AA expressly provides that prohibitions do not apply to advertisements which are directed exclusively to “medical practitioners, psychologists, dentists, pharmacists…” and a list of other members of the health profession.

“…the court concluded that FDA attempts to restrict off-label promotion and information was in fact unlawful.”

There has not been much scope for analysis or discussion in case law, and thus a general consideration of similar laws in other countries is required. In this regard, the US has been quite helpful in providing insights into the possible implications relating to off-label promotion to the public or the medical profession. It must be noted that in the US, only pharmaceutical companies or sponsors are targeted for unlawful off-label promotional activities while it has been acknowledged that medical professions and off-label prescriptions are out of the jurisdiction of the US Food and Drug Administration (FDA).

In 1998 a significant case, Washington Legal Foundation v Friedman was handed down in the District of Columbia court. This case considered whether restrictions on the content contained in off-label promotion material imposed by the FDA constituted a violation of the First Amendment, specifically whether it abridged the freedom of (scientific and academic) speech. After making a number of findings, including that off-label use of drugs was a common practice and that marketing activities was a form of commercial speech, the court concluded that FDA attempts to restrict off-label promotion and information was in fact unlawful. While this case is interesting from a constitutional law point of view, it holds less weight to Australian parties and companies simply because the Australian Constitution does not have an equivalent free speech guarantee. However, the analysis by the court of pharmaceutical and off-label activities in the US may hold some persuasive value to Australian courts should an opportunity to litigate this issue ever arise.


1. NSW TAG report:


About the author:

Mabel Tsui is an academic at the Queensland University of Technology Faculty of Law. Her research interests are in pharmaceutical regulation and consumer protection.

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