Australia: Clinical Trials Under the Microscope

Last Updated: 29 November 2005
Article by Amanda Turnill

Originally published July 2005

Key Point

  • The Therapeutic Goods Administration ("TGA") and the National Health and Medical Research Council ("NH&MRC") handed down their report on the Review of Access to Unapproved Therapeutic Goods in Australia in late February this year. The Report evaluates existing regulatory controls for clinical trials and access to unapproved therapeutic goods in Australia and New Zealand. It makes 49 recommendations, many of which concern the role of the future trans-Tasman agency.

The report says the trans-Tasman agency should actively monitor clinical trial activity, and in particular:

  • advise the Human Research Ethics Committees ("HREC") and the NH&MRC of regulatory requirements for clinical trials;
  • assist HRECs in determining how specific trials should be evaluated if requested;
  • review adverse drug reactions that are both serious and unexpected; and
  • conduct a trial inspection program.

The report also suggests that the clinical trial system be harmonised by having either the regulator or an ethics committee with substantial membership and scope for review assess certain types of trials. Finally, it also recommends the establishment of a mandatory, comprehensive register including all clinical trials conducted with medicinal products in Australia and New Zealand.

We will summarise these recommendations and focus on those that present the biggest changes to the pharmaceutical industry.

Legislative overview of access to unapproved therapeutic products

Under the Therapeutic Goods Act 1989 (Cth) ("The Act") and its associated Regulations, therapeutic goods for human use, which are imported into Australia, manufactured in Australia, supplied by a corporation, supplied interstate or to the Commonwealth, or exported from Australia must be included in the Australian Register of Therapeutic Goods ("ARTG") unless specifically exempted from that requirement.

The legislation permits the supply of products not included in the ARTG (so called "unapproved therapeutic goods"), in the following limited situations:

  • clinical trials;
  • provision under the Special Access Scheme ("SAS");
  • provision by Authorised Prescribers; and
  • importation for personal use.

In regulating the supply of unapproved therapeutic goods, the TGA is obliged to balance two competing considerations. On the one hand there is a broad community interest that therapeutic products available in Australia have acceptable quality, safety and efficacy standards. Unapproved therapeutic goods would have undergone no evaluation by the TGA. Use of any such goods, therefore, carries with it some risks that have not been defined in the Australian context. On the other hand, the need for timely access to potentially lifesaving and enhancing treatments is an important consideration. It is arguable that patients who have serious illness with reduced life expectancy should be granted access to certain unapproved therapeutic goods.

Clinical Trials

Current regulatory framework

Clinical Trials in Australia are currently regulated by the TGA under two schemes - the Clinical Trial Notification ("CTN") Scheme and the Clinical Trial Exemption ("CTX") Scheme.

The major difference between the schemes is the TGA's level of involvement in reviewing data about the therapeutic good involved in the trial before the trial begins.

Under the CTN Scheme, the TGA does not review any of the data before the trial begins. All material relating to the trial is submitted directly to the HREC of the institution where the trial will be conducted. Approval for the conduct of the trial is given by this institution on the advice of its HREC after review of the scientific and ethical validity of the trial. The trial can commence once the TGA has been notified of these approvals by the sponsor of the trial. The HREC is responsible for monitoring the conduct of the trial at its institution. Of note, almost all trials in Australia are currently being conducted under the CTN Scheme.

Under the CTX Scheme, applications to conduct clinical trials must be submitted to the TGA for evaluation and comment. The TGA then reviews summary scientific data about the safety of the product and decides whether or not to object to its proposed usage. Clinical trials cannot proceed until any TGA objections have been overcome. If no objection is raised by the TGA, the sponsor may conduct any number of clinical trials of the product under that particular CTX approval without further assessment by the TGA, provided such use falls within usage guidelines approved by the TGA. However, approval to conduct individual trials under the CTX Scheme must also be given by the HREC of the organisation carrying out the trial, which is responsible for review and approval of the trial protocol and the ethical approval for the study.


The Review essentially endorsed the current system of access to clinical trials for unapproved therapeutic goods, approved the CTN trial process and made the following recommendations:

  • giving HREC guidance in determining how best to undertake scientific reviews;
  • establishing a limited number of Nationally Accredited Human Research Ethics Committees. Their purpose would be to provide ethical review for large-scale, multi-site clinical trials;
  • that there be an increase in the auditing of clinical trials by the TGA. After comparing the audit rate of Australian trials with global figures, Australia was noted to be "significantly behind the rest of the world"; and
  • that a proposed trans-Tasman agency should develop an inspection program based on compliance with internationally accepted Good Clinical Practice requirements and should aim to audit between one percent and three percent of trial sites or HRECs within three years.

Clinical trials register

The Review discussed the advantages and disadvantages of having a register of all clinical trials conducted with medicinal products in Australia and New Zealand. It noted that the overarching concern of the pharmaceutical industry appeared to be that commercial-in-confidence information should not be disclosed about their research.

Nevertheless, the Review recommended establishing a mandatory, comprehensive register disclosing all clinical trials being carried out in Australia. This register is to be maintained and kept up to date by the TGA/ trans-Tasman agency with the cost of establishing and maintaining the register being met by Government. Its purpose would be to foster greater knowledge of trials that are ongoing, as well as completed. The register should be in the public domain, and at a minimum should contain information on the disease being treated, contact details to enable the public to enquire about the trial, and the start and completion dates of the trial.

Special Access Scheme

The SAS allows access to unapproved products by individual patients on a case by case basis. The arrangements rely on different sections of the Act depending on the classification of the patient.

Category A patients are those defined by the Regulations as "persons who are seriously ill with a condition from which death is reasonably likely to occur within a matter of months, or from which premature death is reasonably likely to occur in the absence of early treatment". For Category A patients, use of an unregistered medicine or medical device is obtained via a process of authorisation by their medical practitioner, and notification to the TGA. The treating medical practitioner is required to complete an "Authority to Supply" form and send it to the sponsor of the product. This provides the sponsor with the legal authority to supply the product. A copy of this form must then be sent to the TPA. The TGA has no discretion at law to refuse a Category A notification from a registered medical practitioner.

For all other patients ("Category B" patients), access is obtained by approval from a TGA delegate, or an external delegate.

The Review Committee recommended that there be no substantial change to the SAS scheme.

Authorised Prescriber Scheme

Under the Act, the TGA is able to grant medical practitioners the authority to prescribe specified unregistered therapeutic goods or classes of unregistered medical products to specified recipients or classes of recipients. Requirements for authorisation are contained in Regulation 12B. Upon authorisation, the practitioner (or "Authorised Prescriber") may continue to prescribe such goods to their patients without the need for further authorisation. This authorisation may be revoked by the TGA.

The Review Committee recommended that there be no substantial change to the Authorised Prescriber Scheme.

Personal Importation

The legislation allows for a person to import a limited quantity of most medical products for themselves or their immediate family. The limitation on this is that goods containing a substance which is a prohibited import and injections containing material of human or animal origin are prohibited from being imported. The Review Committee made no recommendation about personal importation.


Overall, the Review endorses the current regulatory framework of clinical trials in Australia. It is supportive of the low level of involvement by the TGA in many aspects of the clinical trials process. Where changes have been recommended, it is important to bear in mind that they must be negotiated with representatives from both Australia and New Zealand due to the proposed establishment of the trans-Tasman agency.

Public comment on the report has now closed. We will monitor future developments in Insights.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

To print this article, all you need is to be registered on

Click to Login as an existing user or Register so you can print this article.

Some comments from our readers…
“The articles are extremely timely and highly applicable”
“I often find critical information not available elsewhere”
“As in-house counsel, Mondaq’s service is of great value”

Mondaq Advice Centre (MACs)
Up-coming Events Search
Font Size:
Mondaq on Twitter
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
Email Address
Company Name
Confirm Password
Mondaq Topics -- Select your Interests
 Law Performance
 Law Practice
 Media & IT
 Real Estate
 Wealth Mgt
Asia Pacific
European Union
Latin America
Middle East
United States
Worldwide Updates
Check to state you have read and
agree to our Terms and Conditions

Terms & Conditions and Privacy Statement (the Website) is owned and managed by Mondaq Ltd and as a user you are granted a non-exclusive, revocable license to access the Website under its terms and conditions of use. Your use of the Website constitutes your agreement to the following terms and conditions of use. Mondaq Ltd may terminate your use of the Website if you are in breach of these terms and conditions or if Mondaq Ltd decides to terminate your license of use for whatever reason.

Use of

You may use the Website but are required to register as a user if you wish to read the full text of the content and articles available (the Content). You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these terms & conditions or with the prior written consent of Mondaq Ltd. You may not use electronic or other means to extract details or information about’s content, users or contributors in order to offer them any services or products which compete directly or indirectly with Mondaq Ltd’s services and products.


Mondaq Ltd and/or its respective suppliers make no representations about the suitability of the information contained in the documents and related graphics published on this server for any purpose. All such documents and related graphics are provided "as is" without warranty of any kind. Mondaq Ltd and/or its respective suppliers hereby disclaim all warranties and conditions with regard to this information, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. In no event shall Mondaq Ltd and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use or performance of information available from this server.

The documents and related graphics published on this server could include technical inaccuracies or typographical errors. Changes are periodically added to the information herein. Mondaq Ltd and/or its respective suppliers may make improvements and/or changes in the product(s) and/or the program(s) described herein at any time.


Mondaq Ltd requires you to register and provide information that personally identifies you, including what sort of information you are interested in, for three primary purposes:

  • To allow you to personalize the Mondaq websites you are visiting.
  • To enable features such as password reminder, newsletter alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our information providers who provide information free for your use.

Mondaq (and its affiliate sites) do not sell or provide your details to third parties other than information providers. The reason we provide our information providers with this information is so that they can measure the response their articles are receiving and provide you with information about their products and services.

If you do not want us to provide your name and email address you may opt out by clicking here .

If you do not wish to receive any future announcements of products and services offered by Mondaq by clicking here .

Information Collection and Use

We require site users to register with Mondaq (and its affiliate sites) to view the free information on the site. We also collect information from our users at several different points on the websites: this is so that we can customise the sites according to individual usage, provide 'session-aware' functionality, and ensure that content is acquired and developed appropriately. This gives us an overall picture of our user profiles, which in turn shows to our Editorial Contributors the type of person they are reaching by posting articles on Mondaq (and its affiliate sites) – meaning more free content for registered users.

We are only able to provide the material on the Mondaq (and its affiliate sites) site free to site visitors because we can pass on information about the pages that users are viewing and the personal information users provide to us (e.g. email addresses) to reputable contributing firms such as law firms who author those pages. We do not sell or rent information to anyone else other than the authors of those pages, who may change from time to time. Should you wish us not to disclose your details to any of these parties, please tick the box above or tick the box marked "Opt out of Registration Information Disclosure" on the Your Profile page. We and our author organisations may only contact you via email or other means if you allow us to do so. Users can opt out of contact when they register on the site, or send an email to with “no disclosure” in the subject heading

Mondaq News Alerts

In order to receive Mondaq News Alerts, users have to complete a separate registration form. This is a personalised service where users choose regions and topics of interest and we send it only to those users who have requested it. Users can stop receiving these Alerts by going to the Mondaq News Alerts page and deselecting all interest areas. In the same way users can amend their personal preferences to add or remove subject areas.


A cookie is a small text file written to a user’s hard drive that contains an identifying user number. The cookies do not contain any personal information about users. We use the cookie so users do not have to log in every time they use the service and the cookie will automatically expire if you do not visit the Mondaq website (or its affiliate sites) for 12 months. We also use the cookie to personalise a user's experience of the site (for example to show information specific to a user's region). As the Mondaq sites are fully personalised and cookies are essential to its core technology the site will function unpredictably with browsers that do not support cookies - or where cookies are disabled (in these circumstances we advise you to attempt to locate the information you require elsewhere on the web). However if you are concerned about the presence of a Mondaq cookie on your machine you can also choose to expire the cookie immediately (remove it) by selecting the 'Log Off' menu option as the last thing you do when you use the site.

Some of our business partners may use cookies on our site (for example, advertisers). However, we have no access to or control over these cookies and we are not aware of any at present that do so.

Log Files

We use IP addresses to analyse trends, administer the site, track movement, and gather broad demographic information for aggregate use. IP addresses are not linked to personally identifiable information.


This web site contains links to other sites. Please be aware that Mondaq (or its affiliate sites) are not responsible for the privacy practices of such other sites. We encourage our users to be aware when they leave our site and to read the privacy statements of these third party sites. This privacy statement applies solely to information collected by this Web site.

Surveys & Contests

From time-to-time our site requests information from users via surveys or contests. Participation in these surveys or contests is completely voluntary and the user therefore has a choice whether or not to disclose any information requested. Information requested may include contact information (such as name and delivery address), and demographic information (such as postcode, age level). Contact information will be used to notify the winners and award prizes. Survey information will be used for purposes of monitoring or improving the functionality of the site.


If a user elects to use our referral service for informing a friend about our site, we ask them for the friend’s name and email address. Mondaq stores this information and may contact the friend to invite them to register with Mondaq, but they will not be contacted more than once. The friend may contact Mondaq to request the removal of this information from our database.


This website takes every reasonable precaution to protect our users’ information. When users submit sensitive information via the website, your information is protected using firewalls and other security technology. If you have any questions about the security at our website, you can send an email to

Correcting/Updating Personal Information

If a user’s personally identifiable information changes (such as postcode), or if a user no longer desires our service, we will endeavour to provide a way to correct, update or remove that user’s personal data provided to us. This can usually be done at the “Your Profile” page or by sending an email to

Notification of Changes

If we decide to change our Terms & Conditions or Privacy Policy, we will post those changes on our site so our users are always aware of what information we collect, how we use it, and under what circumstances, if any, we disclose it. If at any point we decide to use personally identifiable information in a manner different from that stated at the time it was collected, we will notify users by way of an email. Users will have a choice as to whether or not we use their information in this different manner. We will use information in accordance with the privacy policy under which the information was collected.

How to contact Mondaq

You can contact us with comments or queries at

If for some reason you believe Mondaq Ltd. has not adhered to these principles, please notify us by e-mail at and we will use commercially reasonable efforts to determine and correct the problem promptly.