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CATAGCouncil of Australian Therapeutic Advisory Groups
National Medicines Policy Executive
and the Council of Australian
Therapeutic Advisory GroupsMeeting
10 December 2010 3:00pm – 5:00pm
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CATAGCouncil of Australian Therapeutic Advisory Groups
About CATAG• Joint TAGs 2005
• CATAG is an authoritative, expert, consensus-based collaboration of representatives from all Australian State and Territory TAGs or jurisdictional equivalent committees
• CATAG aims to standardise and improve medicines use primarily in the hospital sector across Australia through information sharing, advice and advocacy activities
• Funded by NPS together with CATAG member organisations
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CATAGCouncil of Australian Therapeutic Advisory Groups
CATAG Members
ACT QUM Reference Group
NSW Therapeutic Advisory Group
NTDrugs and Therapeutics Committee
Queensland Health Medicines Advisory Committee
South Australian Medicines Advisory Committee
Tasmanian StatewideTherapeutic Drug Committee
Victorian Therapeutics Advisory Group
Western Australian Therapeutics Advisory Group
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CATAGCouncil of Australian Therapeutic Advisory Groups
Areas of interest/expertise• Access
– Cost and cost-effectiveness– Financing and supply– Consistent framework for funding
• Quality, Safety, Efficacy– Efficacy and suitability – Pre- and post-marketing assessment
• QUM– Judicious, appropriate, safe and effective use – Continuity of effective treatment
• Industry – Productive working relationship with industry
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CATAGCouncil of Australian Therapeutic Advisory Groups
Strategies include:• Information sharing• Working partnerships including
– Health professionals – multidisciplinary – Policy / regulators eg TGA, PBAC, NMP – Standard setting organisations eg ACSQHC– Implementation organisations eg NPS– Industry– Researchers
• Shared guidance / position statements– Product Familiarisation Programs– Use of samples (product starter packs)– Complementary and Alternative Medicines
• Submissions– NHHRC– NMP Partnerships Forum
• Correspondence
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CATAGCouncil of Australian Therapeutic Advisory Groups
The wider healthcare context – NMP • 2000 NMP
– 4 arms • Access to medicines at affordable cost• Standards of quality, safety and efficacy• Quality Use of Medicines • Responsible and viable medicines industry
– NMP Committee/Executive: 5 priorities• Medicines policy → health system reform• Informed & active consumers• Evidence → practice & policy• Monitoring medicines in use• Informing the research agenda
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CATAGCouncil of Australian Therapeutic Advisory Groups
The wider healthcare context -NHHN
• 2010 NHHN– COAG April 2010 – (except WA)– 60% public hospital funding, up to 100% outpatient
funding – No specific mention of medicines– Aims (selected)
• Reduce cost-shifting & ensure efficient & sustainable financing
• Improve health system performance (National Performance Authority)
• More efficient and transparent funding mechanisms• ACSQHC – standard setting
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CATAGCouncil of Australian Therapeutic Advisory Groups
Funding & regulation
• Funding and regulation of hospital medicines is complex and fragmented– In general hospitals have capped medicines budget
(majority from State/Territory funding)– Some PBS reimbursement depending on location of
patient (inpatient, outpatient), illness and jurisdiction – Decisions for formulary listing made locally– Decisions about individual use of expensive and off-
label medicines are made locally• Public Hospital medicines expenditure
– ~$1.4 billion • NATSEM report (IMS data 2007/2008) • Purchasing Index www.pibenchmark.com.au
– ~$600 million – Highly Specialised Drugs Program
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CATAGCouncil of Australian Therapeutic Advisory Groups
CATAG submission to NMP
Informing the research
agenda
Monitoring medicines
in use
Evidence →practice & policy
Informed &active
consumers
Medicines policy →
health system reform
Because of complexity and fragmentation public hospitals are struggling to manage:
Off-Label Use of
Medicines
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Off-label use of medicines
Registered Medicines
On-label useAligned with PI
Off-label useDiffers from PI in terms of:
*Indication *Patient age range*Dose *Route
Inappropriate Appropriate
High quality evidence
Formal research
Exceptional use in individual patient
Off-label use occurs in up to:•40% hospitalised adults•90% hospitalised children•85% cancer patients
Off-Label Use of
Medicines
Gazarian et al MJA 2006;185:544-548Mellor et al APJCO 2009;5:242-246
EG:•Evidence has changed but PI hasn’t eg gentamicin tds vs daily•Cost limits applications for additional indications (eg off-patent, few potential patients)
EG: Drive for innovation EG:•RCTs/evidence unavailable eg children, pregnant women•Potential benefits perceived to outweigh risks
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CATAGCouncil of Australian Therapeutic Advisory Groups
Off-label – The challenges
• Whilst many patients may benefit from off-label use - best decision making is not guaranteed:– Localised rather than centralised– Not standardised– Decision makers have variable membership and skills
• This results in duplication of effort, inefficiency, inconsistency, inequity and potential patient harm
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
The risks of inconsistent decisions are…
• Poor patient outcomes – no beneficial effect +/- harm – Literature r/v: Off label ...use of medicines in children
does lead to increased incidence and seriousness of ADR’s (EMEA report 2004)
– Case series: Rituximab for autoimmune disorders (n=37) Death, infections ~37.8% (BJCP 2008; 66:320-322)
– Industry funded r/v of RCTs: Off-label Eptacog in adults (35 RCTs) - increased arterial thromboembolicevents esp in elderly (NEJM 2010;363:1791-1800)
• Poor economic outcomes– Wasteful use of scarce resources
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Off-label – Further challenges
• Ethical issues unappreciated compared to trials eg informed consent, independent evaluation of benefit vs risk, systematic evaluation of outcomes
• Good evidence but no industry responsibility/incentive for evidence gathering or application for additional indications or updating PI in accordance with evidence
• No standardised mechanism for monitoring benefits and harms
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
So the issue is...• Inconsistent decision making and duplication of
effort PLUS
• No standard mechanism for monitoring outcomes/harms or responsibility for evidence gathering
MEANS • We do not know if:
– Off-label use results in net benefits or harms– QUM has been achieved– Related expenditure represents value for money
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
CATAG work to date• Recognise a systematic approach is needed with a wide
range of stakeholders• Developing “Guiding principles for off-label use of
medicines”• Assessment of consistency between IPU application
forms – and consideration of value of development of a standard form
• Investigating extent of use of off-label rituximab across Australia
• Discussion with RACP re register for off-label medicines (high-cost)
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Proposed solutions• Adopt a national framework for standardised decision
making– Wide and appropriate consultation– Supported by tailored guidance and education program
• Form centralised agency for:– National evidence-based recommendations re off-label
medicine use (where there is high quality evidence)– Collection and collation of outcomes of off-label medicine use
including benefits and harms. (Especially where poor evidence). Feed back to decision makers and inform the research agenda
• Require evidence gathering for common off-label uses and develop mechanism for registration of additional indications and updating of PI where required– Application for additional indications should be able to be
made by body/agent other than sponsor where possible
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Gazarian et al MJA 2006;185:544-548; Gazarian M. WHO expert consultation on Essential Medicines for Children. July 2007
A framework for decision making
Off-Label Use of
Medicines
AND Prospective evaluation of outcomes (efficacy & safety)
• Used/endorsed by:– WHO expert committee on
selection and use of essential medicines for children
– TG Analgesic 2007; TG Psychotropic 2008
– NSW Health policy directive July 2008
– NSW TAG
• Under consideration by: – CATAG– Children’s Hospitals
Australasia– Royal Australasian College
of Physicians
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CATAGCouncil of Australian Therapeutic Advisory Groups
Benefits• Align hospital practice with NMP goals • Align hospital practice with NHHN goals –
Standardisation and Improved Performance• Reduce hospital workload• Reduce risk of poor health and economic outcomes
Off-Label Use of
Medicines
Policy decisions:
Improving consistency and equity of decision making and reducing duplication of effort
Ethical issues:
Embedding informed consent into practice
Clinical practice:
Facilitating best evidence based prescribing and decision making
Research: Facilitating monitoring of outcomes and appropriate feedback of data to decision makers, regulators, researchers, consumers
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CATAGCouncil of Australian Therapeutic Advisory Groups
Role for NMP• Form a national working group to map out scope
and plan implementation across Australia concurrently with health reform implementation
• Need an appropriate mix of expertise• Need WIN, WIN, WIN for all stakeholders• Key stakeholders to include are:
– Health professionals eg RACP, CHA, SHPA
– Funders/regulators eg TGA, PBAC, States & Territories
– QUM organisations eg CATAG, NPS
– Medicines information developers eg TG, AMH, NHMRC
– Industry
– Consumers
– Researchers
Off-Label Use of
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
High Cost Medicines• High cost medicines:
– Arbitrary definition: Total annual cost >$26,000 per patient (>$500 per patient/week)
• High volume medicines: – Definition not agreed
– Some jurisdictions define as > given % of total medicine spend
– “Cause pain”
• Criteria for Highly Specialised Drugs program: “High unit cost” – not defined
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
TGA registered and PBAC approved (Section 85)
-Hospital funded for inpatients.-PBS funded on discharge in some jurisdictions-Unknown cost-effectiveness for hospital use
TGA registered and PBAC approved (Section 100)
-Hospital funded for inpatients-PBS funded for outpatients-Assessed for cost-effectiveness
TGA registered and PBAC unapproved – used for registered indication
-Hospital funded -Unknown cost-effectiveness for hospital use
TGA registered used for off-label indication – appropriate use
-Hospital funded-Unknown cost-effectiveness for hospital use
High cost medicines come in different shapes & sizes
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
High cost medicines –The challenges
• Inequity of access to effective medicines– Lack of cost control due to regulatory
arrangements• Capped budget• Differing capacity to pay – differing budgets • No reimbursement mechanism for inpatients
– Inconsistent decision making• Information on cost-effectiveness either unavailable
or unclear if suitable for patient population – Disruption in medicine supply
• Inconsistencies regarding responsibilities for ongoing access after discharge
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
CATAG work to date• Submission to NHHRC
– Subsequently included in NATSEM modelling study
• CATAG/MA High-Cost Drug Working Party – Formed August 2008
– To access cost-effectiveness information whether approved or not to inform hospital decisions – parallel to PBAC process
• Sponsor’s economic case
• Independent evaluation by the ESC of PBAC, and
• Final PBAC thoughts/comments)
– A series of meetings with MA but encountered significant obstacles in developing a parallel process for hospitals
• How to establish and administer?
• How to fund? Who pays?
• Legal issues including commercial in confidence
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Proposed solutions• A number of solutions could help align with
NMP and NHHN goals:– Allow States/Territories access to cost-effectiveness
assessments• Will improve local decision making
– Expand role of PBAC to assess hospital drugs • Will standardise decisions and reduce duplication of
effort
– Single funding model• Will allow for improved equity of access, continuity of
care and remove cost-shifting • Will allow for greater economies of scale• Will allow for utilisation/expenditure monitoring
– Others in NATSEM modelling study
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Any solution should:• Address inequity, inefficiency and duplication,
cost-shifting and continuity of care issues
• Allow for tracking of hospital medicines expenditure/utilisation per individual medicine (as per PBAC/DUSC)
• Integrate with solutions for standardised decision making for off-label medicines use
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Role for NMP• Undertake expanded economic modelling study to
examine all options and select best model for redesigning hospital medicines funding
• Need an appropriate mix of expertise to oversee• Key stakeholders to include are:
– Health professionals eg RACP, CHA, SHPA– Funders/regulators eg TGA, PBAC, States & Territories– QUM organisations eg CATAG, NPS– Medicines information developers eg TG, AMH, NHMRC– Industry– Consumers– Researchers
HighCost
Medicines
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CATAGCouncil of Australian Therapeutic Advisory Groups
Key points • Off-label use of medicines
– Don’t know if net benefits or harms– Need systematic decision making – Need to systematically monitor outcomes– Need system for updating PI/ additional indications /
evidence gathering – Form working group
• High-cost medicines– Funding is fragmented and complex– Need to address inequity, inefficiency and duplication,
cost-shifting and continuity of care issues– Need to track hospital medicines expenditure/utilisation
per individual medicine– Undertake detailed economic modelling
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CATAGCouncil of Australian Therapeutic Advisory Groups
Acknowledgements
• CATAG member organisations and their representatives
• Dr Madlen Gazarian, UNSW & Sydney Children's Hospital
• Ms Terry Melocco, St Vincents Hospital • Prof Gabrielle Cooper, U Canberra• Access & Systems Branch, DOHA
