© ASHM 2012© ASHM 2012

Presented by: Anna Roberts

Deputy CEO, Division Manager Policy and Education

Pre-Exposure Prophylaxis

(PrEP):

Regulatory Issues

© ASHM 2012

© ASHM 2012© ASHM 2012

• ASHM CEO sits on the Gilead research scholarship

review committee. Honoraria proceeds are donated

to the ASHM Gift Fund

• ASHM receives unrestricted educational grants and

sponsorship from Industry partners for conferences

and educational events. All contracts are governed

by the ASHM Partnership Program

Declaration of conflict

© ASHM 2012

Regulatory Framework –Therapeutic Goods Administration

• The TGA conducts pre-market assessments to have a drug/device added onto the Australian Register of Therapeutic Goods.

• For a drug to be available on the Australian market and be considered for subsidisation by the PBAC, it must be TGA approved

• The TGA only considers the efficacy and purpose of the drug, not the price.

• The TGA cannot evaluate a product without a sponsor submission and the TGA cannot invite, encourage or otherwise cause an application to be lodged

• Once a drug is TGA approved, it can be purchased on the Australian market. This does not mean that it will be subsidized

© ASHM 2012

TGA Processes Review 2015

Justification for the Review?

• Duplication of processes & burden on Industry

• Lacking the flexibility required to facilitate early access to innovative products

• Burden of regulation not commiserate with risk posed by product

• Complex and poorly understood process

Government focus on efficiency and ‘competitiveness agenda”

‘If a system, service or produce has been approved under a trusted

international standard or risk assessment, Australian regulators should not

impose any additional requirements unless it can be demonstrated that there is

a good reason to do so’

© ASHM 2012

What does the Review hope to achieve?

Policy Objectives of the Review

1. Timely access to the medicines that Australians need at a cost individuals and the

community can afford

2. Medicines meet appropriate standards of quality, safety and efficacy

3. Maintenance of a responsible and viable medicines industry

Principles of the review

1. The role of regulation is to manage risk in order to protect public health and safety

2. Level of regulation should be commensurate with the risk posed by the product

3. A risk-benefits approach to regulation is appropriate

4. Regulation should take a whole of lifecycle approach (data, review, development,

withdrawal)

5. Regulation should remain with the Commonwealth

© ASHM 2012

How long does a TGA application take?

• Median approval time for new medicines in Australia

was 350 days in 2013

• Europe: 478 days in Europe

• Japan: 342 days

• USA: 304 days

© ASHM 2012

Regulatory Framework –Pharmaceutical Benefits Advisory Committee

• The PBAC considers whether drug should be reimbursed through Medicare

• Not all successful applications to TGA will go to the expense of a PBAC application

• Like the TGA, the PBAC cannot evaluate a product without a sponsor and cannot invite a submission

• Minister cannot act without a positive recommendation

• Public Interests submissions can be placed by non-commercial sponsors with no financial interest in the application. The manufacturer/supplier must agree to negotiate with the PBAC around price if the application is successful

© ASHM 2012

© ASHM 2012

PBAC Regulatory Steps to Approval

1. Pre-lodgement meeting- Scheduled at the sponsor’s convenience

2. Lodgement- Set times throughout the year

3. Initial consideration by PBAC

4. Drug Utilisation Sub-Committee (DUSC)/ Sponsor Rebuttal

5. Economic Sub-Committee (ESC)/ Sponsor Rebuttal

6. Public/community submissions

7. Final Consideration by PBAC

8. Publication of the PBAC Minutes (recommendation to Minister)

9. Government process, negotiating price and wording of listing- No timeframe, yellow book is update

© ASHM 2012

© ASHM 2012

PBAC: A collaborative approach

• The PBAC recognizes the expense and difficulty

associated with applications and seeks to reduce the

number of non-meritorious applications they receive

• There is a mandatory pre-lodgement meeting to

discuss the nature of the application

• The PBAC provides pre-lodgement advice to assist

sponsors in determining the merit and most

advantageous framing for their application

© ASHM 2012

PBAC and ‘Public Interest’

• The PBAC is required to charge application sponsors a fee to

cover the costs of evaluation

• There is an option to apply to have the fee waived where the

sponsor will not financially gain from the application and there is

little likelihood of being able to otherwise recover the fee from a

sponsor

• All fee waiver applications are evaluated by the PBAC delegate

and must then be approved by the Minister for Health.

• Any organisation that would benefit financially from a successful

PBAC listing must be wholly excluded from all public interests

submissions

© ASHM 2012

Parallel applications?

• For convenience, commercial agencies will often

prepare TGA & PBAC applications at the same time

• TGA applications are seen as a lower bar to meet as

the drug must only meet quality criteria. PBAC is more

difficult because of the cost effectiveness requirement

• PBAC can not consider an application until there has

been a positive recommendation in respect to a TGA

application

© ASHM 2012

Where is PrEP?

• Truvada has been TGA listed in Australia since February 2006 for

use a treatment for HIV infection

• Using Truvada for PrEP is not a part of the current TGA listing

and therefore a new TGA application is require to cover this

change in use

• Gilead has lodged a TGA application to have Truvada separately

licensed for HIV prevention

• Should the TGA application be successful, Truvada would be

available for PrEP through unsubsidized private purchase

© ASHM 2012

Where is PrEP?

• Gilead has indicated that they are not interested

submitting a PBAC application until they have a

positive outcome from the TGA.

• Truvada off patent in July 2017 or 2021 (Tenofavir

2017 & Emtricitabine 2021)

© ASHM 2012

Tea Reading

Will Gilead TGA application be successful?

• Highly likely

Would a PBAC submission be successful?

• Could be a drawn out process.

• The issues of concern might be:

• Political; will it be seen as subsidising sex

• Difficulty in cost effectiveness modelling; we don’t

know how long the drugs will be used on average

• A change in the leadership at PBAC may pull

back from consideration of prevention in decision

making

• Cabinet could block if estimated cost greater than

$10 mil

© ASHM 2012

What needs to happen for a successful

submission• Modelling of real life, Australian-specific risk scenarios to

determine cost effectiveness

• Scientific understanding of the chemical supremacy of PrEP over PEP

• Drawing parallels with contraception and treatment of STIs

• Epidemiological understanding of the contribution of very early infection to onward transmission of HIV

• Would we be having these discussions if Truvada cost $1 per day?