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TGA presentation: 7th Annual EyeforPharma Conference, 11 October 2017

Updates from the Therapeutic Goods Administration

31 October 2017


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  • Presented by: Adrian Bootes
  • Presented at: 7th Annual EyeforPharma Conference, Sydney
  • Presentation date: 11 October 2017
  • Presentation summary: Update on initiatives relevant to prescription medicines and expedited pathways and transparency


Updates from the Therapeutic Goods Administration

Adrian Bootes
Assistant Secretary, Prescription Medicines Authorisation Branch
Therapeutic Goods Administration

7th Annual EyeforPharma Conference, 11 October 2017

Slide 1 - Snapshot of TGA medicines regulation

Prescription medicines approvals - 2016-17
Application type Submissions approved
New Chemical Entity 38
New fixed-dose combination 4
Extension of Indication 45
New generic medicine 111
Additional trade name 49
Major variations 46
Minor variations 1361
Changes to PI (requiring evaluation of data) 63

Slide 2 - Expedited pathways

  • To facilitate earlier access to medicines that address unmet clinical needs for Australians, without compromising standards for safety, efficacy and quality.
  • Two new 'expedited' pathways for prescription medicines based on the government response to the recommendations of the MMDR review:
    • Priority Review of a complete data dossier within a reduced timeframe in certain circumstances
      • Implemented 1 July 2017
    • Provisional Approval on the basis of early data on safety and efficacy, where the immediate availability of the medicine outweighs the risk that more data is required
      • Under development, planned for first quarter of 2018

Slide 3 - Designation process

  • New designation step for Priority Review and Provisional Approval
  • Entry ticket to expedited pathways
  • To use the pathways, designation must be obtained before lodging a submission
  • Formal process to assess against the eligibility criteria
  • Validity of the designation will lapse after six months (however sponsors may apply for a six month extension of provisional designation only)
  • Positive designation decisions only will be published online (including the designated indication)
  • Ineligible designation decisions are appealable by the sponsor only

The first three approved Priority Review designation applications have been published on the TGA website.

Slide 4 - Pre-submission and submission

  • Pre-submission meeting
  • Designation application
    • Priority Review
    • Provisional Approval*
    • Orphan Drugs
  • Submission for registration
    • Standard
    • Standard plus Orphan
    • Priority
    • Priority plus Orphan
    • Provisional*
    • Provisional* plus Orphan

Note: reforms are being developed separately to allow use of reports from comparable overseas regulators in the registration process

* The Provisional Approval pathway will be available from the first quarter of 2018 pending the passage of necessary legislative amendments

Slide 5 - Pre-submission meetings and submission phase

  • We strongly recommend discussion at a pre-submission meeting before applying for Priority Review or Provisional Approval designation
    • This will be important for any application with less than a full dossier, as it may be encouraged to consider the Provisional Approval pathway (however, the standard pathway may be suggested in some circumstances)
    • We can answer questions, but we cannot pre-suppose the designation outcome
  • Whilst introducing more flexible pathways for sponsors, the standard pathway will be clearly defined as requiring a full data dossier, unless in exceptional circumstances

Slide 6 - Eligibility criteria

  • New prescription medicine or new indication
  • High level eligibility criteria for Priority Review and Provisional Approval
    • Serious condition; and
    • Positive comparison against existing therapeutic goods; and
    • Major therapeutic advance
  • Priority Review based on 'substantial evidence'

    Provisional Approval based on 'preliminary clinical data'

  • Sponsors may apply for the Orphan designation prior to or simultaneously with a Priority Review or Provisional Approval designation application

Slide 7 - Priority review

  • Introduced 1 July 2017 through legislative change
  • TGA's Chief Medical Adviser to make designation decision within 20 working days
  • Flexible business processes to reduce the registration timeframes
  • Target total 150 working days consistent with international regulators
  • 'Partnership' with applicant and standard timeframes will apply if requirements for Priority Review are not met
  • Full registration in the Australian Register of Therapeutic Goods (ARTG)

Slide 8 - Approved priority designations

Active Ingredient(s) Designation Indication Approval date
Alectinib (ALCENSA) Priority review For the treatment of patients with anaplastic lymphoma kinase (ALK)-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC). 21/08/17
Emicizumab (HEMLIBRA) Priority review For routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes in patients with haemophilia A (congenital factor Vlll deficiency) with factor Vlll inhibitors. HEMLIBRA can be used in all age groups 29/08/17
Nivolumab (OPDIVO) Priority review For the adjuvant treatment of Stage III/IV melanoma in patients who have undergone complete resection. 28/09/17

Slide 9 - Priority review implementation


  • Amendments to the Therapeutic Goods Act changes passed on 19 June 2017


  • TGA started to accept notifications of the intent to file/lodge for Priority Review designation applications or requests for pre-designation meetings
  • The new Priority Review regulation came into effect (1 July)
  • TGA published Priority Review guidance prior to 1 July 2017
  • TGA provides a new designation application e-form
  • TGA starts to accept Priority Review designation applications


  • First designation decisions under the Priority Review designation process are completed (August)
  • Earliest opportunity to lodge Pre-submission Planning Form (PPF) with valid Priority Review designation
  • TGA starts to accept submissions for registration with valid Priority Review designation for priority evaluation


  • Ongoing monitoring of the number of submitted designation applications and decision outcomes and the time from designation application lodgment to decision

July 2018

  • The impact of changes will be reviewed considering designation application numbers, designation outcomes and stakeholder feedback. The guidance material will be reviewed and updated if required

Slide 10 - More information on our website

Priority Review: Priority review pathway: prescription medicines

Slide 11 - Provisional approval

  • Provisional Approval implementation scheduled for first quarter of 2018 (subject to legislative amendments)
  • The Provisional Approval pathway will provide earlier access to promising new medicines that do not have a full dossier of clinical data where there is the potential for a substantial benefit to Australian patients with unmet clinical needs Medicines could come to the market up to two years sooner than under the current framework
  • Provisional registration will be granted for specified time periods (2 years + up to 2 extensions of 1-2 years each)

Slide 12 - Provisional approval (cont.)

  • Intended to strike a balance between making decisions on the basis of promising data for the benefit of Australian patients, knowing that we need to proactively manage the risks of more unknowns about the efficacy and safety of these medicines
  • Closely linked to reforms of the post-market monitoring framework for medicines
  • Given provisional registration will be time-limited, it is intended only for those medicines where further confirmatory data is still being gathered.
  • Pathway will be subject to the provision of clear advice to consumers and healthcare professionals and any other conditions imposed by the TGA

Slide 13 - Legislative amendments for provisional approval

  • Implementation of the Provisional Approval pathway will require amendments to the Therapeutic Goods Act 1989 and Therapeutic Goods Regulations 1990
  • More details have been included in the Bill for Provisional Approval, when compared to the approach for Priority Review
  • This takes into account feedback from the Senate Community Affairs Legislation Committee inquiry into the 2016 Amendment Bill
  • Public consultation on an exposure draft of the Bill was held in early September
  • The Bill was introduced into Parliament on 14 September

Slide 14 - Next steps

  • Recently published the outcomes of public consultation on implementation arrangements
    • Now available on the TGA website
    • Feedback informed proposed legislative amendments and business processes
  • Further targeted consultation with industry on draft guidance later in 2017
  • Working closely with consumer and healthcare professional representatives to ensure messaging about the provisional status of medicines is appropriate
  • We will commence pre-submission discussions for potential Provisional Approval applications from the fourth quarter of 2017

Slide 15 - Other considerations

Industry may wish to consider an agreed approach for how the Priority and Provisional pathways are promoted (e.g. through the Medicines Australia Code of Conduct)

Slide 16 - Orphan drug program

Aims to provide an incentive to sponsors to bring medicines for a small population to market and make medicines available to patients who would not otherwise be able to access them.

Slide 17 - Why reform the Orphan Drug Program?

  • 78% increase in Orphan designations in Australia from an average of 14 (program start) to a current average of 23 designations per annum
  • A shift in indications has been observed over time:
    • from broader indications and 'whole' diseases
    • to narrow indications (e.g. molecularly-defined subset of a disease, or limited to very specific stages of a disease)
  • Given TGA operates on a full cost recovery basis, such an increase is a risk to the viability of the program

The Orphan Drug reforms aim to create a fair Australian orphan drug program which is fit-for-purpose and sustainable into the future

Slide 18 - What's changing?

Change Benefits
Modification of the orphan prevalence threshold
  • More diseases may qualify as orphan
  • Change to a ratio takes population growth into account
Introduction of additional orphan criteria
  • Targets the orphan program incentive to the greatest unmet need, where it will have the biggest impact
  • Closer alignment with the orphan criteria applied by the European Medicines Agency
Changes to the designation process
  • The program incentive (fee waiver) will be granted on the basis of current information to facilitate designation for eligible medicines

Slide 19 - 1. Standard pathway

Orphan Drug Program Criteria Incentive Designation validity Eligible application types
  • ≤ 2,000 Australians OR not financially viable
  • No refusal to approve overseas for safety
100% fee waiver for registration Indefinite NCEs, new combinations, EOIs, generics, major variations
  • < 5/10,000 Australians OR not financially viable
  • No refusal to approve overseas for safety
  • Life threatening or seriously debilitating condition
  • Comparison against existing goods
  • Medical plausibility
100% fee waiver for registration 6 months + 6 months possible extension NCEs, new combinations, EOIs, major variations*

Slide 20 - 2. New dosage form medicine pathway

An orphan designation for a medicine that is a new dosage form:

  • Life-threatening or seriously debilitating condition
  • Not financially viable
  • No refusal to approve overseas
  • Comparison against existing goods

Definition of new dosage form:

  • has the same chemical, biological or radiopharmaceutical active ingredient (or fixed combination of such ingredients) as another medicine that is included in the Register
  • has an indication in common with that other medicine
  • does not have the same dosage form as that other medicine

Benefits small patient populations, e.g. paediatrics

Slide 21 - Criteria comparison

Priority / Provisional Standard orphan New dosage form medicine orphan
Life threatening or seriously debilitating
Comparison against existing therapeutic goods
Major therapeutic advance
Prevalence threshold or
Not financially viable
Medical plausibility

Slide 22 - Where we have landed – selected items

Paediatric indications

  • Standard orphan drug pathway: Paediatric indications will be considered eligible subsets where:
    • the disease is different in, or specific to the paediatric subgroup
    • prevalence is met in relation to the whole of the disease
  • New dosage form medicines pathway: designed to benefit small patient populations

Validity of designations

  • Priority review designations remain in force for a period of 6 months, OR when an effective section 23 application is lodged until the application is finally determined
  • Orphan drug designation will remain in force for a period of 6 months, OR when a 6 month extension of designation is approved for a period of 12 months

Impact of orphan designation on the PBAC process

All inquiries regarding applications to the Pharmaceutical Benefits Advisory Committee should be directed to

Slide 23 - Orphan Drug Reform Implementation Plan

Jul 2017

  • The new orphan drug regulation came into effect 1 July 2017
  • Orphan Drug guidance published
  • New designation application e-form available
  • Applications must be lodged using the new e-form
  • Applications required to be in the correct format and address new criteria and guidance

Aug 2017

  • First orphan drug designation decisions are issued under the new orphan drug regulation

Sep 2017

  • First submissions for registration possible for medicines with designation under the new orphan drug regulation

Dec 2017

  • Ongoing monitoring of the number of applications, outcomes and timeliness of process

Jul 2018

  • Review impact of changes considering designation application numbers, outcomes and stakeholder feedback
  • Guidance material reviewed and updated if required
  • The validity of all orphan designations lodged prior to 1 July 2017 lapses

Slide 24 - More information on our website

Orphan drug program reforms

Slide 25 - Publication and transparency update

Several proposals for enhanced transparency for high profile prescription medicines

Fourth quarter 2017:

  • Industry consultation on transparency initiatives
  • Improvements to the existing AusPAR page (search ability and user friendliness)

From January 2018

  • Registration timelines: proposed to be published within standard AusPARs
  • For registration decisions made on or after 1 January 2018

Slide 26 - Publication and transparency update

From March 2018

  • Proposed publication of Prescription Medicines landing pages:
    • For all new priority, provisional and standard chemical and biological prescription medicines
    • Proposed scope: New Chemical Entities (NCEs) and Extension of Indications (EOIs)
    • Publication will commence at the time of the first publically notifiable regulatory action (starting with designation) and will include regulatory history over time
    • For Provisional Approval, designation decisions are proposed to be published following acceptance of the submission

Slide 27 - Publication and transparency update (cont.)

From March 2018

  • Proposed Registration Decision summaries - Stage 1
    • Proposed publication of registration summaries for all new chemical and biological prescription medicines registered through the standard pathway
  • Scope:
    • NCEs and EOIs,
    • publication of positive and negative decisions made from 1 March 2018,
    • publication within 2 weeks of Decision
  • Proposed publication of submissions under evaluation - for those received on or after 1 March 2018

Note: these transparency initiatives are subject to the outcomes of consultation

Slide 28 - MedSearchTM

The trusted source of medicine information in Australia

  • FREE app available from June 2017
  • Consumer Medicine Information (CMI) or Product Information (PI) document
  • Sources directly from the Australian Register of Therapeutic Goods (ARTG)

Slide 29 - MedSearchTM features

Quickly access prescription medicine information from your phone:

  • Simply search the medicine name to find its CMI or PI
  • Favourites - bookmark medicine information in one place
  • Save medicine info documents and view them anytime
  • Share CMI/PI documents with family and carers

Connects consumers, carers, doctors, nurses and pharmacists to trusted and current information about their prescription medicines

Slide 30 - MedSearchTM - future enhancements

  • We are planning enhancements to the Medsearch app
  • These will be developed with industry following the principles of co-design

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