1. Reforms to Support Safe and Timely Access to Medicines
Adjunct Professor John Skerritt Deputy Secretary for Health Products Regulation Commonwealth Department of Health
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2. TGA – Part of the Department of Health
HQ in Symonston ACT with 5 satellite offices
710 staff / contractors: scientists, engineers, toxicologists, technicians, medical officers, pharmacists, lawyers, and administrative staff
Focus on safety, quality and efficacy
We do not make decisions based on value for money or make decisions about which products receive Government subsidy
Operations fully cost recovered from industry
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3. The Expert Panel Review of Medicines and Medical Devices Regulation
Commissioned by Government in late 2014 – covered medicines and devices regulation but not cells, tissues and blood products
Review process included discussion papers, submissions and interviews with key stakeholders. Followed by stakeholder workshops and other meetings to get feedback
Department considered stakeholder feedback and advised Minister, who took the preferred position to Cabinet
Government intent released in May 2016 budget, and full response released on 15 September 2016
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4. 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
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5. 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 eligibility criteria for Priority Review and Provisional Approval similar:
serious condition and 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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6. Designation Process
Applicable to Priority Review, Provisional Approval and Orphan Drugs
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7. Priority Review – now in place
New and flexible business processes
- First and second round assessments condensed across 90 days and no first round assessment report
- Delegates overview, expert advice, decision planned for the last 60 days
Designations lapse if submission not made within 6 months
Sponsors can seek review of the designation decision
We will publish priority designations on our website
Target of 150 working days consistent internationally
Pathway provides full registration in the ARTG
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8. 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 only be appealable by sponsor
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 the standard maximum period but every effort will be made to complete reviews faster
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9. Provisional Approval – proposed conditions
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 provisional registration timeframe
Sponsors may be able to obtain full registration when enough data provided
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10. 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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11. Enhanced Postmarket Monitoring
Better integration and timely analysis of datasets
- Enhanced safety signal detection
- Prescription Sequence Symmetry analysis
- Linking datasets – PBS, MBS and hospitals, 45 and 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 international regulatory collaboration
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12. Enhanced Postmarket Monitoring
“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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13. Conclusion The most sweeping changes to regulation in 25 years
Final decisions about the specific changes are up to government
Reform and system design costs will be absorbed but some on-going fees and charges will increase or decrease
Other reforms also implemented e.g. orphan drugs, medicines labeling
Business as usual remains critical
TGA publishes detailed reports on our regulatory performance
Staff, budget and IT constraints make running reform plus BAU challenging
Transitional arrangements, sponsor education, guidelines all important
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14. Thank You
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