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
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
Prescription Medicines 3
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 ’ 4
Designation process Applicable to Priority Review, Provisional Approval and Orphan drugs Assessment Pre- Designation of designation designation application application Designation Dossier notification/ Submission -Against decision submission meeting and acceptance relevant eligibility criteria 6-7 0 12 11 6 2 months weeks weeks weeks weeks weeks 5
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
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 7
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 8
Overview of Provisional Approval pathway 2. 3. 4. 1. Pre-market Provisional Transition to Designation registration registration fully registered process process period if data allows As quickly as possible As quickly as possible 2 years (with the possibility of 2 3-6 months pre-dossier (max. 255 working days) (max. 255 working days) extensions) 9
Medical Devices 10
The regulatory system Risk based classification system Device Classification determines minimum Conformity Assessment requirements 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
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
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 13
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 14
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 15
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 16
Medical device postmarket monitoring Options for establishment of For higher risk registries for devices: an AIMD, high-risk Class III or Environmental implantable Adverse event implantable Class IIb scanning device reporting devices still to (confidential advice from other regulators be considered and review of medical by Government Three consecutive literature, regulatory annual reports to the news, media and TGA required other sources)
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 18
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