Drug availability in Australia

Australia’s drug registration process

Before a new drug or drug product is marketed in Australia it must be approved by the Therapeutic Goods Administration (TGA) in Canberra after assessment of its quality, efficacy and safety. Drugs are classified as therapeutic goods under the Commonwealth Therapeutic Goods Act 1989 and regulations to the Act.

A similar procedure must be undertaken if a pharmaceutical company wishes to introduce a new indication for an already established drug.

Once a drug has been marketed it may be prescribed by medical practitioners although its cost may not be subsidised by the Commonwealth Government.

Pharmaceutical Benefits Scheme

After marketing approval for a drug has been granted, the company may seek to have it listed for reimbursement under the Pharmaceutical Benefits Scheme (PBS), which subsidises the cost of a wide range of drugs. An application is made to the Pharmaceutical Benefits Advisory Committee (PBAC), which makes recommendations to the Commonwealth about new listings or changes to current drug listings. The PBAC considers cost effectiveness of drugs, as well as efficacy and safety issues.

Explanations for the PBAC’s decisions are available in Public Summary Documents at: www.health.gov.au/internet/main/publishing.nsf/Content/public-summary-documents

Economic analyses have been mandatory for drugs submitted to the PBAC for PBS listing since January 1993. Once listed, the economic evaluations are passed to a separate group, the Pharmaceutical Benefits Pricing Authority, which comprises government, industry and consumer representatives. It negotiates the PBS price of the drug with the company on behalf of the government.

There are various forms of restrictions to the use of drugs listed, including drugs requiring authorisation to prescribe (Authority prescription) and the Highly Specialised Drugs Program. For further information, see www.pbs.gov.au/html/healthpro/browseby/section100

Access to drugs outside the PBS

This includes private prescriptions (where the patient bears the cost), supply from public hospitals (where a State government may bear the cost), lifesaving drugs and unapproved drugs.

Lifesaving drugs

Currently funds are specifically made available for 3 drugs to treat rare inherited enzyme deficiencies. For further information, see www.health.gov.au/internet/main/publishing.nsf/Content/health-pbs-general-supply-othersupply

Unapproved drugs

There are 4 mechanisms by which non-marketed (unregistered) drugs may be obtained in Australia:

• the Special Access Scheme (SAS)
• authorised prescribers
• importation for personal use
• participation in a clinical trial.

For further information and advice contact an officer of the Drug Safety and Evaluation Branch, phone 02 6232 8111, fax 02 6232 8112.

Special Access Scheme

This scheme provides for the importation and/or supply of an unapproved drug for a single patient, on a case-by-case basis, as described below.

Category A—medical practitioners can supply unapproved drugs to some terminally and/or seriously ill people with life-threatening conditions without the approval of the TGA as long as the TGA is notified within 28 days.

People in Category A are permitted, in consultation with their doctor, to use any drug except those prohibited by State/Territory law. The prescribing doctor assumes responsibility for use of the drug and is the approving authority; the TGA does not approve use in Category A patients at any time. Informed, written consent from the patient or the patient’s legal representative is required unless there is good reason to the contrary. Relevant documentation must be completed and submitted to the supplier and to the TGA.

Category B—approval of an application to supply an unapproved drug is required from a delegate in the TGA, or an external delegate^a.

Approvals are given normally on a case-by-case basis, although certain prescribers may be given approval to use a specific drug or class of drugs without the need for approval in respect of each patient, see Authorised Prescribers. Informed, written consent is required and patients should be monitored appropriately to determine both efficacy and the occurrence and severity of any adverse drug reactions.

Drugs may be obtained from the sponsor company in Australia if available or otherwise by importation (see Importation for personal use). There is no obligation for the sponsor company to provide the drug simply because the prescriber has made a request under the SAS.

For each of these categories, details of any suspected adverse drug reactions should be reported by the prescriber to the TGA. Serious, unexpected, unusually frequent, or fatal reactions should be notified immediately.

Detailed information is available at www.tga.gov.au/docs/html/sasinfo.htm

Authorised Prescribers

Medical practitioners can apply to be granted authority to prescribe an unapproved drug or class of drugs to specified people or classes of people, identified by their medical condition. Authorised Prescribers can prescribe the drug for the specific indication to individuals.

For further information, see www.tga.gov.au/docs/html/authpres.htm

Importation for personal use

Importation of a drug for personal use (by a patient or the patient’s immediate family) may be possible. The individual may need the written authority of a doctor. Importation is allowed for most substances, but it may be helpful to confirm requirements with the TGA. In addition, although there is no legislative requirement, institutional ethics committee approval or approval from peers (eg a hospital Drug and Therapeutics Committee) may be required in certain circumstances for doctors employed by hospitals and affiliated units.

For further information, see www.tga.gov.au/docs/html/personalimp.htm

Investigational drugs in clinical trials

The Clinical Trial Exemption (CTX) and the Clinical Trial Notification (CTN) schemes may allow access to non-marketed drugs. The TGA reviews trial data provided by the sponsor (usually a pharmaceutical company) before commencement of trials submitted under CTX. Under CTN the major requirements of the sponsor are to notify the TGA before commencing a trial, after the relevant institutional ethics committee has given its approval, and to pay the appropriate notification fee.

All adverse events that occur during a clinical trial must be reported by the investigator to the sponsor; if the trial is discontinued, the sponsor must notify the TGA. Provisions also exist for the TGA to recommend that a clinical trial, for both CTX and CTN schemes, be ceased in the public interest^b.

For further information, see www.tga.gov.au/docs/html/clintrials.htm