This document discusses new expedited pathways being introduced by the Therapeutic Goods Administration (TGA) to provide earlier access to medicines and medical technologies in Australia. It outlines priority review pathways for prescription medicines and expedited review for novel medical devices. The priority review pathway for medicines allows provisional approval based on early clinical data showing promising safety and efficacy. The expedited pathway for devices provides front-of-queue assessment for breakthrough technologies that address unmet needs. Both pathways aim to make therapies available sooner while maintaining standards of safety, quality and effectiveness through post-market monitoring and follow-up requirements.

1. Earlier access to medicines and
medical technologies and the MMDR
Dr John Skerritt
Health Products Regulation Group
Commonwealth Department of Health
ARCS Conference, Sydney
24 August 2017

2. Overarching principles for regulation as endorsed
by Government
• The Australian Government retains responsibility for approving the
inclusion of therapeutic goods in the ARTG
– however need to make much greater use of overseas evaluations
• Need to introduce greater flexibility in approval pathways
– to enable earlier access to therapeutics without compromising safety,
efficacy and quality
• TGA could more appropriately align level regulation with the actual
risk posed by the products in certain areas

3. Prescription Medicines
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4. Expedited pathways for prescription medicines
• Priority Review of a complete data dossier in certain circumstances
• Provisional Approval on the basis of early data on safety and efficacy,
where the immediate availability of the medicine outweighs the risk that
additional data is still required
• Some of eligibility criteria for Priority Review and Provisional Approval similar:
– serious condition
– major therapeutic advance
– comparison against existing therapeutic goods
• Others are quite different - eligibility for:
– Priority Review is based on ‘substantial evidence’
– Provisional Approval will be based on ‘promising evidence from early clinical data’
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5. Designation process
Applicable to Priority Review, Provisional Approval and Orphan drugs
Pre-
designation
notification/
meeting
Designation
application
Submission
and acceptance
Designation
decision
0
weeks
2
weeks
12
weeks
6-7
months
Assessment
of designation
application
-Against
relevant
eligibility criteria
11
weeks
6
weeks
Dossier
submission
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6. Priority Review – now in place
• New and flexible business processes for NCEs/ EOIs
– First and second round assessment will be condensed across 90 days and
no first round assessment report
– Delegates overview, expert advice, decision planned for the last 60 days
• Designations lapse if a submission is not provided within six months -
sponsors will be able to seek internal review of the designation decision
• We will publish priority designations on our website – two designations
already granted
• Target of 150 working days consistent internationally
• Pathway provides full registration in the ARTG 6

7. Provisional pathway designation
• To be implemented from Q1 2018, subject to passage of legislation
• Provisional review pathway designations will be published by TGA
– Negative designation decisions will be appealable by sponsor (only)
• Early clinical data may be based on surrogate endpoints rather than
Phase III trials
– Rolling submissions of clinical data during review would be
negotiated on a case-by-case basis
• Review timeframes will be standard 255 working days maximum but
every effort will be made to complete reviews faster
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8. Provisional Registration
• Registration granted for a specified period
– proposed to be 2 years with maximum of 2 extensions
• The registration decision will take into account whether:
– there is sufficient evidence that the benefit-risk balance is positive
– there is promising evidence that early availability of the medicine will
provide a significant benefit to Australian patients with unmet clinical needs
– sponsor has demonstrated capacity to submit comprehensive clinical data
on efficacy and safety within the timeframes of provisional registration
• Sponsors may be able to obtain full registration when enough
confirmatory data provided
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9. Overview of Provisional Approval pathway
1.
Designation
process
2.
Pre-market
registration
process
3.
Provisional
registration
period
4.
Transition to
fully registered
if data allows
3-6 months pre-dossier
As quickly as possible
(max. 255 working days)
2 years (with the possibility of 2
extensions)
As quickly as possible
(max. 255 working days)
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10. Medical Devices
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11. The regulatory system
Device Classification determines minimum
Conformity Assessment requirements
Risk based classification system
Conformity Assessment (CA) by EU body or TGA
Essential Principles, Manufacturing requirements
Some applications subject to audit if underwent EU CA
Inclusion in the Australian Register of Therapeutic Goods

12. Expedited review for certain ‘novel’ devices
• We have consulted on criteria for expedited evaluation
• Will designate particular devices for priority review in under 6 weeks
– if eligible, the sponsor must submit application within 3 months
• Our criteria are similar, but broader than FDA criteria
– TGA proposes to include devices that can prevent diseases and conditions
– include “seriously debilitating” not just “irreversibly debilitating” conditions
• Rejection by a foreign regulator and failure to continue meeting
eligibility criteria can result in loss of status
• Impacts on other assessments will need to be managed

13. Expedited review for certain ‘novel’ devices
• A ‘front-of-queue’ approach, with normal assessment requirements:
– must still comply with Essential Principles etc.
– compliance with Clinical Evidence Guidelines is critical
• Criteria focused on health outcomes
– device prevents, diagnoses or treats a life threatening or seriously
debilitating disease or condition
– addresses an unmet clinical need in Australian patients
– “breakthrough” technologies
• Implementation scheduled for January 2018
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14. Clinical evidence requirements
• Evidence requirements for expedited pathway same as for other devices
• Clinical data can be generated in Australia or overseas
• Detailed guidance document developed for manufacturers on what constitutes
clinical evidence and the process of clinical data generation and evaluation
• Specific guidance on
– Total and partial joint prostheses
– Cardiovascular devices (patency or functional flow)
– Implantable pulse generators
– Heart valve prostheses
– Supportive meshes, patches and tissue adhesives
– Implantable medical devices in the MRI environment
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15. Enhanced postmarket monitoring
• Better integration and timely analysis of datasets
– Enhanced safety signal detection
– Prescription Sequence Symmetry analysis
– Linking datasets – e.g. PBS, MBS and hospitals, 45 & Up
– Analytics regarding unapproved goods notifications
• Adverse Event Management IT System
– to receive adverse event reports online
– Disproportionality Analysis Report and an Electronic Data
Interchange
• Enhanced collaboration with overseas Regulatory
Authorities on safety information
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16. Specific initiatives for medicines
• Black triangle” scheme to alert practitioners and consumers
– that a drug is newly registered and to encourage reporting of adverse events
• Pharmacovigilance inspection scheme
– Following on from a pilot scheme to audit sponsor obligations
• RMP Compliance Monitoring Program
– Better tools to monitor sponsor’s compliance with Risk Management Plans
– RMP requirements published on the TGA website
• Revision of Product Information (PI) templates
– to put information relevant to health practitioners front and centre
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17. Medical device postmarket monitoring
For higher risk
devices: an AIMD,
Class III or
implantable Class IIb
device
Three consecutive
annual reports to the
TGA required
Adverse event
reporting
Environmental
scanning
(confidential advice
from other regulators
and review of medical
literature, regulatory
news, media and
other sources)
Options for
establishment of
registries for
high-risk
implantable
devices still to
be considered
by Government

18. Relationship to reimbursement ?
• Reimbursement was outside the terms of reference of the MMDR
– We are well aware that for high cost medicines and devices, “early access”
involves government reimbursement decisions
– Public Hospital availability of products determined separately
• TGA is working closely with the Health Benefits Group
– On better process alignment for applications, greater use of common
information, sharing of clinical expertise
– To expand parallel processing and parallel TGA/PBAC review of certain
new prescription medicines submissions
– My colleague, Andrew Stuart can speak to other policy work
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