Research Ethics and Governance Office

Authorised Prescriber Guidelines

1. Introduction
The major legislation dealing with the regulation of therapeutic goods in Australia is the Therapeutic Goods Act 1989 (the Act) and the Therapeutic Goods Regulations 1990 (the Regulations) and the Therapeutic Goods (Medical Devices) Regulations 2002 (the Medical Devices Regulations). One important outcome of this legislation is that most therapeutic goods are required to be approved and included on the Australian Register of Therapeutic Goods (ARTG) before they can be supplied unless there is an exemption. The legislation provides the following mechanisms to allow individuals to gain limited access to therapeutic goods not on the ARTG:

• The Special Access Scheme (categories A and B);
• Clinical Trials (CTN and CTX schemes);
• Authorised Prescribers; and
• Importation for personal use.

2. Authorised Prescriber
Basic principles governing the supply of unapproved products using the authorised prescriber mechanism

a) Patient rights
Right of access
The Authorised Prescriber provision of the legislation allows doctors to supply individual patients with unapproved therapeutic goods under a range of circumstances such as:
• access to products which have been withdrawn from the Australian market for commercial or other reasons;
• access to products provided initially to patients through a clinical trial while a marketing application is being considered; and
• access to products available overseas but not marketed in Australia.

The classes of patients who may access unapproved therapeutic goods prescribed by an Authorised Prescriber are those suffering from a life-threatening or otherwise serious illness or condition (Section 19(6) and 41HC of the Therapeutic Goods Act 1989 and Regulation 12B(2) of the Regulations and Regulation 7.6 of the Medical Devices Regulations).

b) Informed consent
It is a condition of the approval to supply an unapproved therapeutic good for use in Australia that the patient or the patient's legal guardian must be in a position to make an informed decision regarding treatment. Informed consent should be in writing unless there are good reasons to the contrary. Informed consent should be freely given and includes an adequate knowledge of the condition and its consequences, an adequate knowledge of the treatment options, the likelihood of recovery and the long-term prognosis. A patient should be specifically informed of the following:
• that the product is not approved (ie registered or listed) in Australia;
• possible benefits of treatment and any risks and side effects that are known;
• the possibility of unknown risks and late side effects; and
• any alternative treatments using approved products which are available.

c) Responsibilities of the Prescriber
Unapproved therapeutic goods have undergone little or no evaluation of quality, safety or efficacy by the Therapeutic Goods Administration. Therefore, the responsibility for prescribing an unapproved product rests with the prescriber.

The prescriber is best able to determine the needs of the patient and to monitor the outcome of therapy. The prescriber has an added responsibility to ensure the patient has given appropriate informed consent prior to treatment. However, an important corollary to the issue of informed consent is that a medical practitioner has the right not to approve the use of an unapproved product if he/she believes there is either insufficient clinical justification or insufficient efficacy and safety data to support the use of the product.

d) Responsibilities of the TGA
In considering requests to supply unapproved (non-evaluated) therapeutic goods, the TGA has a responsibility to maintain a flexible and efficient means of ensuring individuals are able to gain timely access to important new therapeutic developments without jeopardising the broader community interest in ensuring that products available in Australia are evaluated for quality, safety and efficacy.

In relation to applications from medical practitioners to become Authorised Prescribers, the TGA has a responsibility to determine each application on a case by case basis, taking into account the class of patients for whom treatment is intended, the properties of the product and the expertise of the medical practitioner.

In keeping with its overall charter, the TGA also has a responsibility to encourage at all times the availability of approved (evaluated) products. Thus, the various mechanisms for supply of unapproved products are intended to be temporary measures pending general marketing approval of the product. TGA requires that applications to use unapproved products justify adequately why available approved products are not suitable for use. Unfettered access to unapproved products amounts to de-facto marketing and would remove any incentive for a sponsor to seek registration of the unapproved product or for other sponsors to seek registration of alternative, similar products.

e) Rights and Responsibilities of the Sponsoring Company (Supplier)
A company is under no obligation to supply an unapproved product merely because it has been prescribed by an Authorised Prescriber. Applicants should check with companies that they will agree to supply before making an application and what costs may be involved. The Commonwealth does not subsidise (through the Pharmaceutical Benefits Scheme) the cost of unapproved products.

The sponsor must provide the TGA with six monthly reports detailing the supply of unapproved therapeutic goods to Authorised Prescribers.

If a sponsor anticipates long term supply of their product, they should consider whether to submit a marketing application.

In relation to the supply of products under the Authorised Prescriber mechanism, sponsors have a responsibility to monitor the use of their products continually and record the safety of the medicine and the balance of its benefit and risk. Ideally, the use of an unapproved medicine or medical device should be the subject of treatment protocols issued by the sponsor, with clear requirements for the treating doctor to report any adverse outcomes to the sponsor.

Sponsors of unapproved products are also required to communicate rapidly to the TGA information that has an important bearing on the benefit-risk assessment of the product, particularly any information that may lead changes to the usage of the product by Authorised Prescribers.

f) Conditions of Authorisation
When Authorisation is given by the TGA, the Authorised Prescriber will receive a letter of authorisation. This letter includes an attachment which outlines the general conditions upon which any authorisation is granted and, if applicable, a list of conditions specific to that particular authorisation, such as conditions relating to the treatment or monitoring of the specified group of patients, or the use of the specified product. Failure to fulfil these conditions may lead to revocation of the authorisation. A list of general conditions can be found at Appendix 1 of the TGA’s guideline to Access to unapproved therapeutic goods – Authorised Prescribers.

The Authorisation is restricted in that:
• it applies only to the specified product;
• the product can only be prescribed for patients in the Authorised Prescriber's immediate care;
• the medical practitioner must continue to have an appropriate ethics committee endorsement in order to supply the unapproved product; and
• the Authorisation can be revoked at any time following notice from the TGA.

g) Other
Final responsibility for the use of an unapproved product within an institution always rests with that institution. Medical practitioners working in an institution may also need approval from the institution's Ethics Committee or Drug and Therapeutics Committee prior to using a particular medicine or device. Applicants should discuss the use of the product with the Chief Pharmacist or person fulfilling this role before applying for authorisation