Prince of Wales clinical school and Lowy cancer research centre

Decision making – square pegs in round

holes: the Australian HTA experience

Professor Robyn Ward

Director POW Cancer centre, Clinical Associate Dean UNSW

Chair of Medical Services Advisory Committee and Member PBAC

Pharmaceuticals

$8 billion pa

Non-

pharmaceuticals

$16 billion pa

Reimbursement of health care through

the public purse

PBAC MSAC

“In theory there is no

difference between

theory and practice HTA of drugs and other

health interventions

In practice there is”

Yogi Berra

Overview of talk

The MSAC process

• review the steps

• coordination with TGA, PBAC, PLAC

Three case studies

• Prepare for the future

• Follow the money

• Exit strategy

The future

The MSAC process

• review the steps

• coordination with TGA, PBAC, PLAC

Overview of the reimbursement process

Positioning and dialogue

Preparing the submission

Appraisal of evidence

Implementation

Submission

lodgement

• Complete short application form

• Protocol advisory committee

Define the question for public subsidy Define the bellwether issue – evidence, economics, data Consultation – with HESP, public, competitors and allies

PASC is not compulsory but its functions are

Positioning and dialogue

Preparing the submission

• Contracted assessment

OR

• Prepare your own

Evidence should be fit for purpose

• RCT

• Observational studies

• MBS data

for utilisation

for patterns of care

• Grey literature

• Consumer voice

• TGA submissions

• PLAC evaluations

• Submissions to other HTA bodies

“Any belief that the controlled trial is the only

way … to generate evidence would mean

not that the pendulum had swung too far but

that it had come right off the hook”

Bradford Hill the architect of the RCT

Lodgement of the submission

• External critique of submission

• Review by evaluation subcommittee

• Policy input

• Opportunities for rebuttal

• MSAC consideration and

recommendations, outlining way forward

• Public summary documents

Appraisal of the evidence

Implementation - departmental work, government decision and

release to the wild

• Potential time savings because

policy input and MBS costing now

part of the MSAC process

The MSAC process

• review the steps

• coordination with TGA, PBAC, PLAC

Prepare application

Evidence appraisal

Government decision

Health system

Diagnostic

& pharmaceutical company

PBAC and MSAC

PBD & MBD

Opportunities for coordination co-dependent technologies

Coordination is possible

test and drugs

• Melanoma - BRAF testing and dabrafenib

• Colorectal cancer - KRAS and anti-EGFR

antibodies

• Lung cancer – TKIs and EGFR testing

Egs

• Botox for bladder dysfunction

• Sculptra for lipoatrophy

Coordination is possible

drugs and services

Success factors

• Role (responsibility) delineation

• Taking genetic testing seriously

• Champions in government

• Commitment to making the system work

Case studies

1. Prepare for the future

esp. for co-dependent test/drug

2. Follow the money

3. Exit strategy

Case 1 – prepare for the future

The idea of a single target - single

treatment model (for cancer) is a

conceptual fallacy

Case 1 - Pre-existing state

EGFR antibodies and KRAS testing reimbursed for

patients with metastatic colorectal cancer

History

2004 - FDA approved companion diagnostic in

DakoCytomation EGFR pharmDx immunostaining

(detects overexpression of EGFR)

2008 – KRAS mutations a negative predicator of

response to anti-EGFR antibodies

Case 1 - The catalyst for change: Testing for KRAS status is not good enough

Other activating RAS mutations may

also be negative predictive biomarkers

for anti-EGFR therapy.

September 2013

Case 1 – Consequences, lessons learnt

• Yet again reinforces the conceptual fallacy of single target -

single treatment model

• Yet again shows that the net benefits of a co-dependent test

and drug are negated if incorrect test assignment exposes

patients to inferior treatments

• Yet again reinforces the importance of providing preclinical

data on drug/target interactions (biological plausibility)

• Yet again highlights the importance of including a plan for

updating evidence – esp. emerging molecular data and impact

on risk/benefit of treatment

Case 2 – follow the money

Case 2 – Pre-existing state

Unloved MBS item number (42740) for eye

injections - $295.15 - rarely used

Case 2 – The catalyst for change

• Ranibizumab (LUCENTIS®) listed on the

Pharmaceutical Benefits Scheme (PBS) on 1 August

2007 for the treatment of age related macular

degeneration.

BUT

• Costs of administration of Lucentis (using

previously unloved MBS item number 42740) was

not evaluated.

Case 2 - Consequences

0

10,000

20,000

30,000

40,000

50,000

60,000

70,000

80,000

Jan-03 Jan-04 Jan-05 Jan-06 Jan-07 Jan-08 Jan-09 Jan-10 Jan-11 Jan-12 Jan-13

Number of eye injection services - 2003 to 2013

LUCENTIS® listed on the PBS

Rebates for

Avastin®

restricted

Serv

ices p

er

quart

er

Benefits paid for out-of-hospital eye injection

services ($Million)

0.0

10.0

20.0

30.0

40.0

50.0

60.0

70.0

EMSN

Rebates

Response from government

• To address excessive fee charging by a

small number of providers

• Extended medicare safety net benefit

capped at equal to 80% of the MBS fee –

introduced on 1st November 2012 (2012-

2013 budget)

Average fees charged fell

$0

$200

$400

$600

$800

$1,000

$1,200

$1,400

Jan-Mar 2011 Jul-Sept 2011 Jan-Mar 2012 Jul-Sept 2012 Jan-Mar 2013

Average fees charged for eye injection services in postcodes with the highest average fees prior to

capping

Postcode A

Postcode B

Postcode C

Capping introduced (1 November 2012)

Avera

ge f

ee c

harg

ed (

$)

Case 2 – Consequences for

submissions

• Consider out of pocket patient expenses

• Consider incentives for health

practitioners in terms of MBS billing

• Consider the knock-on effects on the

health system of new technologies

Case 3 – Removal of an item from the schedule

Case 3 - Pre-existing state

Hyperbaric oxygen therapy funded

for non-diabetic wounds on the

MBS since 2003 on an “interim

basis”

Case 3 - The catalyst for change

Scheduled interim funding review in 2011

MSAC reviewed the evidence

MSAC advised the minister that it did not

support continuation of interim funding

(scientific judgement)

Minister discontinued public funding in 2012

Case 3 - Consequences

• Political interventions

• Multiple reconsiderations by MSAC

• Commonwealth Senate hearing

• Items not reinstated on schedule

The future

Lay bare the evidence gaps and describe if

and how they can be addressed

Live with the consequences of a changing

evidence base and disinvest if

drug/test/technology can’t be shown to work

Communicate intentions & progress to all

is it possible…..