Spotlight on Prescription Medicines Reforms Priority Review, - - PowerPoint PPT Presentation

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

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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.

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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

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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

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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)

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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

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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

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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

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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

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Implementation

June

• Amendments to the Therapeutic Goods Act changes passed on 19 June 2017

July

• 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)
• 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

Aug/ Sep

• First designation decisions under the Priority Review designation process are possible
• Earliest opportunity to lodge Pre-submission Planning Form (PPF) with valid Priority Review designation
• TGA starts to accept submissions for registration with valid Priority Review designation for priority evaluation

Dec

• Ongoing monitoring of the number of submitted designation applications and decision outcomes and the time

from designation application lodgment to decision July 2018

• The impact of changes will be reviewed considering designation application numbers, designation outcomes and

stakeholder feedback. The guidance material will be reviewed and updated if required

10

More information on our website

https://www.tga.gov.au/priority-review-pathway-prescription- medicines

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Provisional Approval

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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

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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

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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

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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
• Designation validity
• Transition period
• Two pathways
• Documentation
• 1. To ensure that the correct medicines are

being facilitated to be assessed free-of-charge

• 2. To ensure a more consistent proportion of

medicines meet the Orphan criteria in the face

• f an increased ‘personalisation’ of medicines

Applies to the Priority Review, Provisional Approval and standard pathways

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• 1. Standard Pathway

Orphan Drug Program Criteria Incentive Designation validity Eligible application types Previous

• ≤ 2,000 Australians OR not financially

viable

• No refusal to approve overseas for safety
• 100% fee

waiver for registration

• Indefinite
• A, B, C, D, F

New

• < 5/10,000 Australians OR not financially

viable

• No refusal to approve overseas for safety
• Life threatening or seriously debilitating

condition

• Comparison against existing goods
• Medical plausibility
• 100% fee

waiver for registration

• 6 months

+ 6 months possible extension

• A, B, C, F*

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• 2. New dosage form medicine pathway

An orphan designation for a medicine that is a new dosage form:

• Life-threatening or seriously debilitating condition
• Not financially viable
• No refusal to approve overseas
• Comparison against existing goods

Definition of new dosage form:

• has the same chemical, biological or radiopharmaceutical active ingredient (or fixed combination of

such ingredients) as another medicine that is included in the Register

• has an indication in common with that other medicine
• does not have the same dosage form as that other medicine

Benefits small patient populations, e.g. paediatrics

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Criteria comparison

Priority review Standard orphan New dosage form medicine orphan Life threatening or seriously debilitating    Comparison against existing therapeutic goods    Major therapeutic advance  Prevalence threshold OR Not financially viable    Medical plausibility 

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Where we have landed – selected items

Paediatric indications

• Standard orphan drug pathway- paediatric indications will be considered eligible subsets where:

– prevalence is met in relation to the whole of the disease – where the disease is different in, or specific to the paediatric subgroup

• New dosage form medicines pathway- designed to benefit small patient populations, such as paediatrics

Validity of designations

• Priority review designations remain in force for a period of 6 months, OR

when an effective section 23 application is lodged until the application is finally determined

• Orphan drug designation will remain in force for a period of 6 months, OR

When a 6 month extension of designation is approved for a period of 12 months

• All existing Orphan designations prior to 1 July 2017 will expire on 1 July 2018.

Impact of orphan designation on the PBAC process All enquiries regarding applications to the Pharmaceutical Benefits Advisory Committee should be directed to PBAC@health.gov.au

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Orphan Drug Reform Implementation Plan

July 2017

• The new orphan drug regulation came into effect 1 July 2017
• Orphan Drug guidance published
• New designation application e-form available
• Applications must be lodged using the new e-form
• Applications required to be in the correct format and address new criteria and guidance

Aug 2017

• First orphan drug designation decisions are possible under the new orphan drug regulation

Dec 2017

• Ongoing monitoring of the number of applications, outcomes and timeliness of process

Jul 2018

• Review impact of changes considering designation application numbers, outcomes and

stakeholder feedback

• Guidance material reviewed and updated if required
• The validity of all orphan designations lodged prior to 1 July 2017 lapses

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More information on our website

https://www.tga.gov.au/orphan-drug-program-reforms

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New e-form for minor variations

• New electronic form available through TBS
• Minor variations to prescription medicines

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Reducing regulatory burden

• Need for improvements to processes
• Staged approach to improve the processing of minor variations
• Number of minor variations received in 2016-2017 financial year

Category Number 9D(1) 122 Category 3 1345 MEC 458 SAR 1232 SRR 707 Total 3864

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New electronic form

One-stop shop

• Consolidating six PDF forms into one electronic form
• Reduced time and effort in making applications
• Allows a real-time view of ARTG entries
• More efficient processing of requests

Launch

• 18 sponsors were involved in testing
• Progressive soft launch during July
• Full launch 25 July 2017
• Paper forms will be turned off as an option by legal instrument in the near future.

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Summary of changes and associated fees before you submit

Fees

Variation Group Legislative Fee item Fee* basis

A new good with a new ARTG ID will be generated for the following goods based on the variation being made under this legislative basis:

Correct an ARTG entry 9D(1) 2A(a) $1,625.00 Quality Information 9D(3) Product information (PI) 9D(2) 2CA $5,270.00

*Fees are for the 2016/2017 financial year

Prescription Medicines Reforms

Next steps

• Guidance updated and published - printable version and e-book
• Request: sponsors pro-actively review errors and omissions in

register entries via the form

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Outlook – notifications process

• A new notifications process for non-prescription medicines was

launched in July 2017

• The new minor variations e-form is the first step in delivering a

notification process for prescription medicines

• Additional functionality to allow automatic processing of

notifications will be implemented before the end of 2017

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More information

Prescription Medicines Authorisation Branch Application Entry Team AET.application.entry.team@health.gov.au

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Other activities

• 1. Revised committee structure and responsibilities – from 1 Jan 2017
• 2. Current state and future path of the electronic common technical

document (eCTD)

• 3. Recent launch of the MedSearch app

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Revised committee structure from 1 Jan 2017

Advisory Committee

• n

Medicines (ACM)

Advisory Committee on Prescription Medicines (ACPM) Advisory Committee on Non-Prescription Medicines (ACNM) Advisory Committee on the Safety of Medicines (ACSOM)

Advisory Committee

• n Vaccines

(ACV)

Advisory Committee on Prescription Medicines (ACPM) (Functions related to vaccines) Advisory Committee on the Safety of Vaccines (ASCOV)

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Current state and future path of eCTD

Benefit: allows update of relevant dossier sections across multiple applications

• Increasingly important with multiple sponsor applications in process, across multiple evaluation

sections Current state:

• Encouraged for ALL prescription medicines submissions
• Mandatory for TGA’s ‘PPF-only’ Category 1, Priority Review, Provisional Approval (once

implemented)

• Now mandated by FDA, EMA, Health Canada
• Increasingly used for internal TGA workflows

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Current state and future path of eCTD

Future path:

• Expectation is a steady migration to eCTD (expected for Priority and where previous eCTD

applications)

• Direct upload capability for dossiers getting closer; only available for eCTD dossiers
• An update to the eCTD specification primarily to accommodate Priority Review, Provisional

Approval and allowed combinations of minor variations – Consultation later this year – Expected implementation early next calendar year

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MedSearch™

The trusted source of medicine information in Australia

• FREE app available from June 2017
• Consumer Medicine Information (CMI) or Product

Information (PI) document

• Sources directly from the Australian Register of

Therapeutic Goods (ARTG)

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MedSearch™ features

Quickly access prescription medicine information from your phone:

• Simply search the medicine name to find its CMI or PI
• Favourites – bookmark medicine information in one place
• Save medicine info documents and view them anytime
• Share CMI/PI documents with family and carers

Connects consumers, carers, doctors, nurses and pharmacists to trusted and current information about their prescription medicines

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MedSearch™

Polling question Looking at the Medsearch App and its future:

• The functionality is about right, and communications are at the right

level

• The functionality is about right, and should be rolled out in as many

ways as possible

• Functionality could be expanded, to provide other medicine

communications to health care practitioners and patients/carers

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