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Presentation: The patient will see you now! A regulator, policy maker and patient perspective

TGA presentations given by the TGA at the 2019 ARCS Annual Conference, Sydney, 6-8 August 2019

10 September 2019


These presentation papers are provided on the TGA's website solely for the purpose of indicating or suggesting what TGA representatives spoke about to the various conferences and seminars to which it relates. The papers are not legislative in nature and should not be taken to be statements of any law or policy in any way.

The Australian Government Department of Health (of which the TGA is a part) advises that (a) the presentation papers should not be relied upon in any way as representing a comprehensive description of regulatory requirements, and (b) cannot guarantee, and assumes no legal liability or responsibility for, the accuracy, currency or completeness of the information contained in the presentation paper.


  • Presented by: Dr Jane Cook
  • Presented at: 2019 ARCS Annual Conference
  • Presentation date: 6 August 2019


Opening plenary session - The patient will see you now!

A regulator, policy maker and patient perspective

Dr Jane Cook
First Assistant Secretary - Medicines Regulation Division
Health Products Regulation Group (TGA), Department of Health

ARCS Annual Conference, 6 August 2019

Slide 1 - Overview

  • Background
  • Technological challenges and the Therapeutic Goods Act
  • Patient focus -access to new promising therapies
  • New technologies with challenges for patients and regulators
  • Involving consumers in regulation and patient reported outcomes
  • What is patient centred care?
  • Future role

Slide 2 - "The patient will see you now" Eric Topol

  • "bottom-up medicine,"
  • "digitally empowered" patients take charge of their own health care.
  • smart phones and social media democratise medicine
  • massive open online medicine (MOOM)

Slide 3 - Problems or not??

  • Information asymmetry
  • Market failure
  • Health literacy - Dr Google
  • Security
  • Informed choice:
    • Better understanding of risks and benefits Improved compliance and better outcomes
  • Targeted therapies

Slide 4 - Role of the Regulator

  • Ensure therapeutic products (devices, medicines, biologicals, blood and tissue products) marketed in Australia are safe, of good quality, efficacious or fit for purpose for the public.
  • We do this through evaluating data submitted by sponsors (safe and effective/fit for purpose), undertake GMP inspections (quality) and post market monitoring

Slide 5 - Challenges - massive technological changes since Therapeutic Goods Act introduced in 1989 - does it remain fit for purpose?

It can accommodate:

  • New clinical trial designs
  • Biosimilars
  • Personalised medicine
  • Self testing, including genetic DTC
  • Cell and tissue therapies
  • CART - T cells
  • Medical apps
  • 3D printing

Slide 6 - How we evaluate therapeutic goods is not specified in any detail in the legislation

Therapeutic Goods Act 1989

Section 25 Evaluation of therapeutic goods

... the Secretary must evaluate the goods for registration having regard to whether the quality, safety and efficacy of the goods for the purposes for which they are to be used have been satisfactorily established

Slide 7 - Patient focus?

Vision: Better health and wellbeing for all Australians through regulatory excellence

  • New pathways - earlier access to promising new therapies
  • Improved information for consumers and health care professionals:
    • Product Information and Consumer Medicine Information reformatting
    • Information Hubs - Breast implants, urogynaecological mesh, codeine, advertising
    • Public facing databases - DAEN, PI/CMI (MedSearch app), medicine shortages
  • Supported by modification to schemes and then guidance to industry as therapies change:
    • Faecal Microbial transplantation
    • Software as a medical device
    • 3D printing

Slide 8 - New pathways - earlier access

  • Promising medicines:
    • Priority pathway (full dossier) reduced assessment time
    • Provisional pathway (early data on safety and efficacy, immediate availability outweighs uncertainty
  • Other medicines:
    • Comparable Overseas Regulator (COR A and B) pathways
    • Work-sharing - Health Canada, Swiss medic and Singapore
  • Role of Patient reported outcomes

Slide 9 - Gene therapy

  • Will see increasing number for approval
  • CRISPR has made gene modification easier, cheaper and more accurate
  • Use genetically modified viral vectors or genetically modified cells
  • Tisagenleucel KMYRIAH - lymphoma/leukemia
  • Not without some risk:
    • Cytokine-release Syndrome or Cytokine storm
    • Permanent or not?
    • Targets wrong cells
    • Results in unwanted mutations
    • Unknown or unintended long term consequences?

Slide 10 - Faecal Microbial Transplantation

  • Promising new therapy
  • Treatment of Clostridium difficile - good evidence
  • Other uses:
    • Gastro-intestinal such as Cohn's Ds and Irritable Bowel
    • Non-bowel - obesity, insulin resistance, fatty liver?
    • Depression and other psychiatric conditions?
  • Do it yourself FMT?

Slide 11 - Precision medicine/ personalised medicine

  • Diagnosing/preventing genetic disease
  • Cancer diagnosis and treatment
  • Determining patient suitability for particular medicines
  • Chronic multi-genic conditions - diabetes, heart disease
  • Targeted therapies designed after identification of a biomarker associated with the disease/condition
  • companion diagnostic is essential for the safe and effective use of the therapeutic - to identify patients who will respond, and to not inflict the wrong treatment on unresponsive patients
  • Requires coordinated commercial development and regulatory review of the therapeutic and diagnostic
  • Regulators will need to identify and assess whether tests are true companion diagnostics for targeted therapeutics are needed

Slide 12 - Software as a medical device

  • Smart phone - a powerful computer you carry around capable of monitoring, diagnosing and treating
  • Software easily developed and available in the app store or google play - being downloaded and used in large numbers through "personal importation"
  • Uses machine learning and AI - robustness of the algorithms used?
  • If a medical device does it meet the essential principles - is it safe, of good quality and fit for its intended use?

Slide 13 - Self testing - IVDs

  • Already some - glucose, pregnancy
  • Direct-to-Consumer (DTC) advertising on internet
  • Not on ARTG
  • Include:
    • infections - influenza, chlamydia, HIV
    • Genetic testing - curly hair to specific disease markers
    • Tests for serious disorders such as heart attacks and cancer
  • Current thought is there needs to be medical supervision

Slide 14 - New technologies bring new uncertainties - regulators must adjust risk and safety thresholds for different populations

The intended population is critical - patients consistently will accept more risk than regulators

Slide 15 - Consumers and regulation

Regulators involve patients in Committees e.g. ACM, ACMD

Guidelines used such as:

  • ICH Harmonized Guideline: Revision of M4E Guideline on Enhancing the Format and structure of Benefit-Risk Information
  • Patient-Focused Drug Development framework
  • Guidance for Industry (FDA)
  • Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man - the use of patient-reported outcome (PRO) measures in oncology studies (EMA)

Slide 16 - Patient report outcomes

  • Capture patients perspectives, complement other clinical outcome measures
  • Ensure the clinical outcomes are relevant valued, preferred by the patient
  • Include single and multidimensional measures of symptoms, HRQL, health status, adherence to treatment and satisfaction with treatment
  • Outcome measures such as Overall Survival, Progression Free Survival, biomarker measures and adverse events do not capture the impact of treatment on the individual

Slide 17 - Do we have the right tools?

  • Growing area in clinical trial design but becoming standard practice in CTs
  • Can measure primary or secondary outcomes in clinical trials evaluating new medications and treatments
  • PROMs and PREMs
  • Personal impact of illness and treatment, including physical functioning, ability to maintain daily activities, and emotional wellbeing
  • Can be general eg HRQoL
  • Evidence that condition specific PROs are more sensitive to change following intervention
  • These measures have also been shown to facilitate patient-provider communication and share decision-making

Slide 18 - Do we have the right tools?

  • SPIRIT-PRO: Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols
  • Examples:
    • Quality of life e.g. EQ-5D, AQoL
    • Symptoms e.g. pain (NPRS), fatigue (FSS)
    • Distress e.g. depression (K10, PHQ2), anxiety (GAD7)
    • Functional ability e.g. WHODAS 2.0, OD
    • Self-reported health status e.g. SF-36
    • Self-efficacy e.g. GSE
  • Collected by:
    • Paper
    • Social media
    • Website - Smart phone/tablet/computer

Slide 19 - Patient-centred care - isn't this what we want?

Picker's Eight Principles of Patient Centred Care

  • Respect for patients' preferences
  • Coordination and integration of care
  • Information and education
  • Physical comfort
  • Emotional support
  • Involvement of family and friends
  • Continuity and transition
  • Access to care

Picker Institute and Harvard Medical School

Slide 20 - Regulators role in patient centred care

  • Some of key principles fall outside of the purview of a regulator
  • The following apply:
    • Respect for patient values, preferences and expressed needs
    • Information and education
    • Access to care
  • More than just involvement in drug development, evaluation and benefit risk assessment
  • Importance of patient-based evidence.

Slide 21 - The future

  • Consumers have access to information and products - internet, social media
  • Is the role moving to that of needing to provide information that helps consumers make the right decision for them based on scientific data
  • Improving health literacy
  • Greater consumer collaboration in regulation

Slide 22 - Thank you

Slide 23 - END SLIDE

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