Drug Development and Regulation

• Three distinct stages of getting new drugs to market:

  1. Discovery
  2. Development
  3. Regulation
• In silico: computer modelling
• In vitro: cells
• In vivo: in whole animals
• Ex vivo: clinical samples
• Diabetes, endocrine and metabolic research areas are growing
• Roughly 30 new compounds are released as drugs each year
• Pharma companies mainly research cancer and immunology as these are drugs that require constant use and will therefore make the most profit
• High throughput screening is the largest drug design approach
• Chronic diseases always have the greatest global sales
• The rationale of a study must be put forward to the AEC
• All projects need to consider the three Rs:

  1. Replacement of animals with other methods
  2. Reduction in the number of animals used
  3. Refinement of techniques to reduce adverse impact on animals
• Nuremberg Code (1945) outlines 10 points for ethical research in humans
• Declaration of Helsinki (1964) changed requirements of consent to allow those who cannot provide informed consent to be able to participate in trials
• All studies must have Human Research Ethics Committee (HREC) approval
• All pharmaceuticals must be registered before they can be marketed
• TGA = therapeutic goods administration
• ARTG = Australian register of therapeutic goods
• Registered = safe medication
• Listing = approved to be made of safe compounds
• Common technical document (CTD) is an internationally agreed set of specifications for pharmaceutical development
• CTD involves studies on pharmacology, pharmacokinetics, and toxicity in animals
• The PBS provides lists of all the medicines available to be dispensed to patients at a subsidised price
• The PBAC decides which medicines go on the PBS
• If the PBAC considers a medicine cost-effective for significant medical conditions, then it will approve its addition to the PBS
• How long does it typically take to get a new pharmaceutical product to market?

  12-16 years
• How much does it typically cost to bring a new pharmaceutical product to market?

  $1-3 billion
• GLP = good laboratory practice
• GMP = good manufacturing practice
• GCP = good clinical practice
• GVP = good vigilance practice
• GLP, GMP and GCP must occur during pre-clinical and clinical studies
• GVP must occur once a pharmaceutical is approved and marketed to the public
•  Traditional development pathway of pharmaceuticals

  1. Drug discovery, development and pre-clinical studies
  2. Clinical trials
  3. Registration
  4. Marketing approval
  5. Reimbursement
  6. Marketing, pharmacovigilance, further clinical studies
• During the reimbursement stage, the pharmaceutical is funded and put onto the PBS
• Pharmacovigilance requires monitoring adverse events to ensure the safety of the pharmaceutical
• Further clinical studies may be required after a pharmaceutical is on the market:

  1. To extend indications, or
  2. To meet requirements for approval set forth my the regulatory authority
• Why Regulate Pharmaceutical Products?

  • Society has expectation about safety, efficacy and timely access to therapeutic products
• • The National Medicines Policy describes how people involved in the development of therapeutic products should work together to meet the expectations of society
• Historical drug safety tragedies have resulted in regulations being put in place