Reforms to Support Safe and Timely Access to Medicines Adjunct Professor John Skerritt Deputy Secretary for Health Products Regulation Commonwealth Department of Health #PharmAus2017

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 #PharmAus2017

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 #PharmAus2017

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 #PharmAus2017

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’ #PharmAus2017

Designation Process Applicable to Priority Review, Provisional Approval and Orphan Drugs #PharmAus2017

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 #PharmAus2017

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 #PharmAus2017

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 #PharmAus2017

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) #PharmAus2017

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 #PharmAus2017

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” #PharmAus2017

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 #PharmAus2017

Thank You #PharmAus2017