Drug Approval and Development

Describe the processes by which new drugs are approved for research and clinical use in Australia, and to outline the phases of human drug trials (Phase I-IV)

Drug Approval

The Therapeutic Goods Administration (TGA) approves medicine for both research and clinical use in Australia.


Drug trials are approved for research purposes under two schemes:

  1. Clinical Trials Exemption
    Drugs must be evaluated by an expert committee to evaluate all aspects of pharmacology, toxicology, mutagenicity, teratogenicity, organ dysfunction, and other side-effects.
  2. Clinical Trials Notification
    A drug which has been approved in another nation with similarly stringent requirements (New Zealand, Netherlands, UK, Sweden, US) may be used in a trial with oversight by a local ethics committee.

Clinical Use

The TGA classifies medicines into:

  • Registered Medicines
    Assessed by the TGA for quality, safety, and efficacy.
    • All prescription (high-risk) medicines. Assessed on:
      • Quality
        • Composition of drug substance
        • Batch consistency
        • Stability data
        • Sterility data (if applicable)
        • Impurities
      • Non-clinical
        • Pharmacology data
        • Toxicology data
      • Clinical
        • Efficacy: results of clinical trials
    • Most OTC (low-risk) medicines
    • Some complementary medicines
  • Listed Medicines
    Assessed by the TGA for quality, safety, but not efficacy.
    • Some OTC medicines
    • Most complementary medicines

Phases of Drug Development

  • "Phase 0"
    • Pre-clinical R&D
    • In vitro and animal testing
  • Phase I
    • First administration in humans
    • Basic pharmacokinetic and toxicology data
    • 20 - 100 human subjects
  • Phase II
    • Administration to select patient groups
    • Aim to establish dose-response curve
    • Evidence of efficacy
  • Phase III
    • Full-scale evaluation of benefits, potential risks and costs analysis
    • 2000-3000 patients, usually treated in groups of several hundred for relatively short durations (3-6 months), regardless of the length of time the drug will be used in practice3
    • May not reveal uncommon or long-term risks
  • Phase IV
    • Post-marketing surveillance


  1. PS Myles, T Gin. Statistical methods for anaesthesia and intensive care. 1st ed. Oxford: Butterworth-Heinemann, 2001.
  2. Medicines and TGA classifications. Therapeutic Goods Administration. Available at: https://www.tga.gov.au/medicines-and-tga-classifications
  3. Chris Anderson. Pharmaceutical Aspects and Drug Development. ICU Primary Prep.
Last updated 2020-07-26

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