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Biologicals Regulation in Australia
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Biologicals Regulation in Australia


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  • 1. Biologicals regulation in Australia Evolving perspectives Albert Farrugia Senior Principal Research Scientist Therapeutic Goods Administration Presented to the Second Asian Bio Manufacturing Conference, Kuala Lumpur, Malaysia, November 2007
  • 2. Current position
    • TGA Act recognises medicines and medical devices as regulated therapeutic goods (no definition of biologicals)
    • Products such as blood products, biotechnology medicines, vaccines etc are regulated as prescription medicines
    • Products of tissue engineering eg autologous chondrocytes regulated as devices
    • Some products eg fresh blood, banked tissues not subject to pre-market review and ARTG so – only regulated outcome is GMP license
  • 3. Current responsibilities Biologicals
    • DSEB – all registrable biologicals which are medicines eg
        • Biotechnology medicines – monoclonal a/bs, recombinant products etc
        • Vaccines (including cell based vaccines)
        • Plasma derivatives
    • ODBT – Devices section – all registrable biologicals which are devices
          • Recalls section – advice re blood, blood product and tissue recalls
    • MAB – manufacturing license for products subject to the requirement
  • 4. Role of the Blood and Tissues Unit
    • Situated within the ODBT – historical reasons – link to GMP
    • Reviews pre-market data for biotech drugs – plasma products, recombinants etc
    • Reviews technical files for fresh blood components, cord blood
    • Develops policy for blood, tissues and biologicals
    • Provides advice on pathogen safety – viruses, prions
    • Interfaces with government(s) on blood and tissue policy issues
  • 5. International relationships BTU
    • WHO Expert Committee on Biological Standardisation
    • Council of Europe Committee of Experts on Blood transfusion
    • Council of Europe Committee on cells, tissues and organs
    • European Pharmacopeia Group pf Experts 6B – Blood Products
    • WHO Global Collaboration for Blood Safety
    • [World Federation of Hemophilia Medical Advisory Panel]
  • 6. Current structure Blood and Tissues Unit
    • Unit head (SPRS)
      • Cells and tissues EL2
        • Evaluators EL1 (x 3)
      • Blood and Plasma products EL2
        • Evaluators EL1 (x4), APS6
      • Pathogen safety, PMFs EL2
        • Evaluators EL1 (x1), APS6 (x2)
      • Operations Manager EL2
        • EA, APS5
    Medical Advisor ODBT
  • 7. Drivers for change Biologicals regulation in Australia
    • Internationally accepted principle
    • Difficulties in using traditional paradigms eg medicinal GMPs for biologicals
    • High public and political interest
    • Publicly funded activities
  • 8. Regulation of biologicals Proposed approach
    • Scope
    • Blood, blood components and blood products
    • Biotechnology equivalents of blood, blood components and blood products
      • Recombinant clotting factors
      • Haemoglobin solutions
      • Etc
    • Cell and tissue therapies
      • Banked “non-viable” tissue
      • Cellular therapies
      • (under government policy review) solid organs, reproductive tissue
  • 9. Regulation of biologicals Proposed approach
    • Principles
    • All products to be subject to requirements of registration ie pre-market review for safety, quality and efficacy before market entry
    • Level of review linked to classification system
    • Office of Biologicals established as the Regulator within the agency
  • 10. Regulation of biologicals Proposed approach
    • Class 1, 2 & 3 - Basis for classification
    • Risk-benefit ratio
    • Governance arrangements
    • Level of manipulation
    • Intended use
  • 11. Regulation of biologicals Proposed approach
    • Class 1 – goods which are
      • Governed through medical practice – specific donor-patient r’ship
      • Of low risk compared to benefit for individual patient
      • Processed with minimal technology – will be defined
      • Eg solid organs, autologous/directed HPCs in clinical units, autologous/directed blood, hospital based blood manufacture
    • Class 1 will be regulated through Standards established by professional bodies
    • Class 1 standards will be overseen and attested by the regulated entity – declaration of conformance or by facility accreditation (NATA)
    • The requirements are not “lower” than other Classes – different governance
  • 12. Regulation of biologicals Proposed approach
    • Class 2 – goods which are
      • Governed through manufacturing practice – long term banking
      • Of moderate risk compared to benefit – established medical indications, safety profile – for population
      • Processed with moderate technology – will be defined – including banking
      • Have a homologus function ie same function in recipient as in donor
      • Eg banked tissue, banked HPCs detached from medical environment, non-autologous/directed, mainstream blood components
    • Class 2 will be regulated through standards for safety and quality in product and manufacture which will be overseen by the agency
    • Class 2 efficacy requirements will be minimal and based on established knowledge eg literature, no clinical trials will be solicited
  • 13. Regulation of biologicals Proposed approach
    • Class 3 – goods which are
      • Governed through manufacturing practice
      • Of potentially high risk compared to benefit, for population and indvidual
      • Processed with industrial technology – will be defined
      • Eg plasma derivatives, biotechnology medicines, cellular therapies
    • Class 3 cell and tissue products will be further subdivided into 2 types
      • Depending on whether function is homologous or not
      • Different requirements for clinical trials/efficacy assessment
    • Class 3 will be regulated through standards for safety, quality and efficacy in product and manufacture which will be overseen by the agency
    • Class 3 efficacy requirements will be based on clinical trials which will be staged on risk based principles
  • 14. Regulatory model for biologicals
    • Class 1 (many still under policy review)
      • Haematopoietic progenitor cells
      • Autologous blood transfusions
      • Solid organs
    • Class 2
      • Mainstream blood components
      • Banked cord blood – haematopoietic reconstitution
      • Banked tissues
    • Class 3
      • Plasma products
      • Biotech equivalents of blood products eg rVIII etc
      • Cell based vaccines
  • 15.
    • Class 1
    • Unbanked/unprocessed
        • Medical practice governance
    • Class 2
    • B anked/unprocessed
        • Manufacturing practice governance
        • Homologous function
    • Class 3
    • B anked/processed
        • Manufacturing practice governance
    Organs Blood Progenitor (stem) cells Hospital based manufacture Banked blood components Banked tissues Processed cells and tissues Manufactured blood products “ Class III” – homologous function “ Class IV” – non-homologous function Plasma products Recombinant plasma products Processed blood components eg virally inactivated platelets Currently exempt Currently regulated through GMP – no ARTG Currently regulated as medicines or therapeutic devices “ listable” Biologicals – no efficacy requirements “ registrable” Biologicals –efficacy requirements Proposed regulatory model for Biologicals
  • 16. Clinical trial framework Proposed approach
    • Two levels
      • CTX – detailed safety data reviewed by regulator
      • CTN – less detailed data requirements
    • Role of HRECs
    • For biologicals –
      • Class 3 Cell therapies are CTX
      • Gene therapy when vehicled through cell therapies is CTX
      • Xenotransplantation is CTX
      • New substances may be CTN if overseen by HREC – NHMRC new system
  • 17.
    • - National minimum dataset of reportable adverse events
    • - Agreed definitions of these events, with standards for their investigation
    • - Collection within institution or blood establishment
    • - Notification to the competent authority (TGA) of any serious adverse events
    • and serious adverse reactions
    Haemovigilance Rapid Report generated by hospital Adverse event in public or private hospital and/or pathology service Health Department Regulator TGA Supplier Rapid validation process