Spotlight on Prescription Medicines Reforms Priority Review, Provisional Approval and other reforms Adrian Bootes Assistant Secretary Prescription Medicine Authorisation Branch Medicines Regulation Division, TGA 2017 ARCS Annual Conference August 2017
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 1 Prescription Medicines Reforms
Designation process • New designation step for Priority Review and Provisional Approval • Entry ticket to expedited pathways • Prior to the dossier submission for registration • Formal process to assess against the eligibility criteria • Validity of the designation will lapse after six months for Priority Review • Positive designation decisions will be published online • Designation decisions are appealable The electronic designation application form is now available and the first designation applications have been received. 2 Prescription Medicines Reforms
Pre-submission meetings and submission phase • We strongly recommend discussion at 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 − We can answer questions, but we cannot pre-suppose the designation outcome • Increased scrutiny during submission phase for all registration applications − It will be important to use the correct ‘form’ • Whilst introducing more flexible pathways for sponsors, the standard pathway will be clearly defined as requiring a full data dossier, unless in exceptional circumstances 3 Prescription Medicines Reforms
Eligibility Criteria • New prescription medicine or new indication • High level eligibility criteria for Priority Review and Provisional Approval Serious condition; and Major therapeutic advance; and Positive comparison against existing therapeutic goods Priority Review based on ‘ substantial evidence ’ Provisional Approval based on ‘ promising evidence from early clinical data ’ • Sponsors may apply for the Orphan designation prior to or simultaneously with a Priority Review or Provisional Approval designation application 4 Prescription Medicines Reforms
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) 5 Prescription Medicines Reforms
Flexible registration process • An eCTD dossier will be required • Registration process will commence after dossier validated (no batching) • First and second round evaluation phases will be condensed • Rolling questions during the first round evaluation • Flexible arrangements for seeking expert advice • More resource intensive process cost recovered through new designation, application and evaluation fees 6 Prescription Medicines Reforms
GMP requirements • Good Manufacturing Practice (GMP) licenses/clearances within the 150 working day target timeframe required • Entry requirements: – At designation Evidence of approved or submitted GMP clearance, certification or license tracking number for all manufacturing sites provided – At registration All fees paid and evidence lodged at dossier acceptance • Exit criteria – Priority Review may transition to the standard pathway if GMP requirements not met 7 Prescription Medicines Reforms
Polling Questions What proportion of new chemical entities and new biological entities do you believe that you will submit via the Priority pathway over the next 12 months? – 0-10% – 10-20% – 20-30% – More than 30% – Not sure 8 Prescription Medicines Reforms
Polling Questions What proportion of new indications do you believe that you will submit via the Priority pathway over the next 12 months? – 0-10% – 10-20% – 20-30% – More than 30% – Not sure 9 Prescription Medicines Reforms
Implementation • Amendments to the Therapeutic Goods Act changes passed on 19 June 2017 June • 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) 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 possible Aug/ • Earliest opportunity to lodge Pre-submission Planning Form (PPF) with valid Priority Review designation Sep • 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 Dec from designation application lodgment to decision • The impact of changes will be reviewed considering designation application numbers, designation outcomes and July stakeholder feedback. The guidance material will be reviewed and updated if required 2018 10
More information on our website https://www.tga.gov.au/priority-review-pathway-prescription- medicines Prescription Medicines Reforms 11
Provisional Approval 10 Prescription Medicines Reforms
Provisional Approval Provisional Approval implementation scheduled for first quarter of 2018 (subject to legislative amendments) • Provisional registration of goods in the initial absence of full clinical data on safety and efficacy • Granted for specified time periods (2 years + up to 2 extensions of 1-2 years each) • Sponsors required to collect and submit further clinical data to demonstrate efficacy and safety for full registration • Enhanced post-market monitoring and surveillance • Subject to the provision of clear advice to consumers and healthcare professionals and any other conditions imposed by the TGA 10 Prescription Medicines Reforms
Polling question For Provisional registration Companies when giving undertakings for a product in the (up to) 6 year period will fulfill: – all commitments – most commitments – all commitments but perhaps will need flexibility as trials develop for data sources – most commitments but some will not be completed due to other priorities 11 Prescription Medicines Reforms
Outlook • Recent public consultation on implementation arrangements – Outcomes published on TGA website shortly – Stakeholder feedback informed proposed legislative amendments and business processes • Further targeted consultation with industry on draft guidance later in 2017 • Work closely with consumer and healthcare professional representatives to ensure messaging about the provisional status of medicines is appropriate 11 Prescription Medicines Reforms
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. What’s changing? Why? • New criteria 1. To ensure that the correct medicines are being facilitated to be assessed free-of-charge • Designation validity 2. To ensure a more consistent proportion of • Transition period medicines meet the Orphan criteria in the face • Two pathways of an increased ‘personalisation’ of medicines • Documentation Applies to the Priority Review, Provisional Approval and standard pathways Prescription Medicines Reforms 16
1. Standard Pathway Orphan Criteria Incentive Designation validity Eligible application Drug types Program • ≤ 2,000 Australians OR not financially • 100% fee • Indefinite • A, B, C, D, F Previous viable waiver for • No refusal to approve overseas for safety registration • < 5/10,000 Australians OR not financially • 100% fee • 6 months • A, B, C, F* New viable waiver for + 6 months possible • No refusal to approve overseas for safety registration extension • Life threatening or seriously debilitating condition • Comparison against existing goods • Medical plausibility 3 Prescription Medicines Reforms
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