27-28 May 2013, Munich, Germany

EuroBioForum 2013 2nd Annual Conference

Katherine Payne

The University of Manchester

Personalised Medicine(s)?

• From a hyped theoretical concept to clinical application: now the perfect solution to offer good value for money?

• Targeting of medicines using a biomarker or genetic-based diagnostic to identify the eligible patient population

• The most common current applications involve stratifying cancer patient populations using companion diagnostics

• But other personalised applications in medicine are emerging eg. stratified (risk-based) breast screening programmes

The Context • Poor global economic climate: greater emphasis on the

need to effectively use finite healthcare budgets

• There is substantial diversity in how healthcare systems are provided and funded across European countries

• There is a commonality in the need for decision-makers working within these healthcare systems at local, regional or national levels to think about how best to spend the available healthcare budget effectively

• Decision-makers need/want a sufficiently robust evidence base to reassure them they are spending the resources in the best way possible

Whose perspective? Suppliers Suppliers and User User

Academic community (science & applied

research)

Government (health policy makers)

Society

Manufacturer (pharmaceutical &

diagnostic)

Third party healthcare payers

General public

Private laboratories (molecular & pathology)

Health service commissioners (national,

regional, local)

Patients

Hospital based laboratories

(molecular & pathology)

Reimbursement & drug formulary committees

Hospital based pharmacy services

Clinicians (primary care, general &

specialist hospital)

From market access to patient benefit • Medicines: regulatory and reimbursement hurdles

• Same (general) hurdles are relevant to a companion diagnostic but the specifics (processes and evidentiary requirements) completely different

• Different levels of decision making:

– national (centralised)

– provider (hospital or primary care)

– patient-clinician level

• Different evidence requirements likely to be considered to be useful and sufficient to inform introduction of a personalised medicine

Health Technology Assessment • There is substantial variation across Europe in:

– the process of funding and producing HTAs

– the technical details used in the evaluative methods

– the intended use of the HTA reports

• HTAs can potentially be used to inform clinical guidelines or reimbursement decisions for local, regional or national use

• In some jurisdictions, HTAs have a more formal legal status and are used by national decision makers working for healthcare third party payer organisations

Economic evaluation: A framework for measuring ‘added’ value

INPUTS Process of

health care OUTPUTS

Resources:

Medicines

Managing side effects

Tests and lab

Staff

Monitoring etc

Outcomes:

% cases cancer cured

Life years gained

Quality adjusted life years

1. Pharmacogenetic test to target a treatment for breast cancer

2. Current practice (no test)

Perspective: the healthcare system

Time horizon: lifetime

A Model-Based Economic Evaluation QALYs Costs

For 100 women, costs £148,000 more for new drug A but gain 6 QALYs

Incremental cost per QALY = £24,700 per QALY

Costs for new drug A = (70 x 3,000) + (30 x 3,200) = £306,000 Costs for standard care = (60 x 1,500) + (40 x 1,700) = £158,000

£3,000

£3,200

£1,500

£1,700

70 women have a response to new drug A 60 women have a response to standard care

QALYs for new drug A = (70 x 0.80) + (30 x 0.20) = 62 QALYs for standard care = (60 x 0.80) + (40 x 0.20) = 56

Costs

For 100 women, costs £28,250 less for gene test and gain 2 QALYs

Costs for gene test = £2,037,50 Costs for no gene test = £2,320,00

QALYs for gene test = 61 QALYs for no gene test = 59

Gene test = 68.75 women have a response No Gene test = 65 women have a response

QALYs

£3,100

£3,300

£1,600

£1,800

£3,000

£3,200

£1,500

£1,700

Test costs £100

NICE and Personalised Medicine

Companion diagnostic

‘with’

new medicine

Established treatment

pathway & > 1 test

Technology Appraisal

Is the medicine cost effective?

Diagnostics Assessment

Programme

Is the diagnostic cost effective?

Poor (unfocussed) clinical evidence base 34 studies: 25 patient cohorts No-meta analysis due to heterogeneity: alleles included, patient cohorts, outcomes Not possible to populate an economic model Not currently possible to recommend CYP2D6 testing in women receiving treatment with tamoxifen

Example recommendations: short term

Recommendation Possible Approach To understand the current use of HTA to inform

reimbursement and payer decisions for companion

diagnostic medicines across Europe

Structured document review

supported by a survey of key

stakeholders

To identify and create a database of existing

reimbursement and payer systems for diagnostics

and medicines across Europe

Structured document review

supported by a survey of key

stakeholders

To identify how companion diagnostics are priced

and/or charged for at the provider level across

Europe

Survey of providers in hospitals

and service commissioners

To understand the extent of variation in

technologies and processes for the conduct of

companion diagnostic testing within and across

European countries

Survey of research and clinical

laboratories

Example recommendations: medium term

Recommendation Possible Approach To understand how existing reimbursement and

payer systems for diagnostics and medicines across

Europe must be re-aligned to facilitate market entry

Semi-structured interviews and

survey of key stakeholders

To produce guidance on the technicalities of the

design and conduct of the HTA process specific to

companion diagnostic medicines

Establish a working group of key

HTA bodies

To understand the implications of moving from single

companion diagnostics to multiple/profiles for

evidence requirements and service provision

Semi-structured interviews and

survey of key stakeholders

To describe and quantify gaps in the evidence base

and the added value of future research to reduce

current uncertainties to support the introduction of

companion diagnostics

Economic modelling with formal

value of information methods

Example recommendations: medium/long term

Recommendation Possible Approach To identify incentives to encourage manufacturers to invest

in the production of a robust evidence base to support the

clinical & cost effectiveness of companion diagnostic

medicines

Establish a mixed working

group of key HTA bodies

and manufacturers

To identify and target national funding for research to

understand the added value of technologies

Establish working group of

HTA/ research funding

bodies

To align the use of HTA for the reimbursement of companion

diagnostics in line with existing practice for medicines

Establish a working group

of key HTA bodies

To produce clear and explicit guidance on the evidence

decision makers working at national and local providers

levels would like to support the reimbursement of

companion medicine diagnostics

Establish a mixed working

group of key stakeholders

including HTA bodies,

manufacturers and

hospital providers

Acknowledgments

Reflections on market access for personalized medicine: Recommendations for Europe

Katherine Payne, The University of Manchester Lieven Annemans, Ghent University