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Research & Innovation

Horizon 2020


European perspectives in healthcare sciences and implementation

Horizon 2020 Information Day12 September 2013

Brussels

From omics to health promotion – novel approaches and collaborations


Disclaimer

Pending formal adoption by the EU of the Horizon 2020 legal texts, the content of these slides is not definitive and so should not be taken as such, nor used or referred to as such in any way or for any purpose

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Workshop agenda14:00-14:45 Using omics data and personalised medicine in healthcare

• Health promotion and diseases prevention, translating omics into stratified approaches

• Piloting personalised medicine in health and care systems

• Questions and Answers

14:45-15:30 Reaching out to other sectors

• Health promotion and disease prevention: improved inter-sector co-operation for environment and health based interventions

• Foresight for health policy development and regulation


15:30 – 16:00 Coffee break

16:00-17:00 Looking for efficiency gains

• Evaluating existing screening and prevention programmes

• Developing and comparing new models for safe and efficient, prevention oriented, health and care systems



"From 'omics' to health promotion - novel approaches and collaborations"


WORK PROGRAMME

"Impacts, challenges and

limitations of EU-funded public

health research - Priorities for

future research" Report of an

independent Expert Group

Consultation workshop on

future of public health

research, January 31, 2013

Health 2020: the European

policy for health and well-being

"Use of '-omics' technologies in

the development of personalised

medicine" – Staff working

document

BACKGROUND

NOVEL APPROACHES COLLABORATIONS

COMMISSION SERVICES MEMBER STATES


EnvironmentalDeterminants

CLIMATE CHANGE

&DISASTERS:

• Heatwaves• Fires• Drought• Floods• Mudslides• Chemical Spills

• Food Security• Changes in • Infectious

disease habitat

Social & Economic

Determinants

AGEING

ECONOMIC DECLINE

INEQUALITIES

URBANISATION

MIGRATION

TECHNOLOGY

Health Outcomes

NCDs

LONGERLIFE

EXPECTANCY

Health SystemImplications

INCREASED:DEMAND & COSTS

DECREASEDWORKFORCE

Solutions

SUSTAINABLEPREVENTION& PROMOTION

PRIMARYHEALTH

CARE & Health systems

tranformation

PROTECTIONEmergency

preparedness

INNOVATIVEWIN – WIN-WIN

APPROACHESE.g. Personalised

medicine

Risks & Projections (Foresight) RESEARCH IMPLICATIONS Innovative solutions

Public Health Challenges and possible solutions for 2050*

*Courtesy: Division of Health Systems and Public Health, WHO Regional Office for Europe

Novel approaches collaborations


Novel approaches & collaborations

• Learning from the past:

• Clear EU added value

− Research that clearly demonstrate a potential for translation and policy uptake to benefit the health of all EU citizens

− Research that would not necessarily be supported by national funding agencies.

• Importance of knowledge translation & capacity building for effective uptake


Novel approaches and collaborations

• Looking towards the future

• More action oriented research

• Multi stakeholder, multi-disciplinary research

• Change of mind set


Using omics data and personalised medicine in healthcare


Personalised medicine – towards a definition

"Personalised medicine refers to a medical model using molecular profiling for tailoring the right therapeutic strategy for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely and targeted prevention"


• Non-communicable diseases worldwide will cause an output loss of €35 trillion over the next two decades

• Current medical treatments

• Increasing pressure on European healthcare systems

• The EU is not closing the persistent gap with global innovation leaders

• Biomedical companies are finding drug development in Europe economically challenging

Personalised medicine to address significant challenges…


…and benefit from opportunities

• Stratified and subsequently personalised medicine can deliver better outcomes for patients and potential cost savings

• Recent studies suggest cost savings of 37% for breast cancer and 46% for CVD when a stratified approach is taken

• Europe can lead implementation of personalised medicine thanks to favourable conditions


Healthcare opportunities: making medical treatments more personalised

• Avalanche of new –omics and molecular information following the sequencing of the human genome

• Translation of – omics from basic to clinical research can bring us better understanding of health and disease

• Need innovative approaches for changing the focus from

treatment and cure to prediction and prevention

Clinical definition of disease

Molecular definition of disease

Molecular definition of health


Personalised Medicine Workshops & conference

• Vision for Europe 2020: identify bottlenecks for PM implementation into clinical practise

• 2010: Preparatory workshops (-omics, biomarkers, clinical trials/regulatory, uptake)

• 2011: European Perspectives in PM conference

Available on: http://ec.europa.eu/research/health

http://ec.europa.eu/research/health

http://ec.europa.eu/research/health


“Omics”

Technologies

Data

Samples

Statistics

R&Dthe basics

R&D stratifying

tools

R&Dtest in human

In patients

Uptake in healthcare

Towards the market

Clinical trials MethodologiesEthics

Patient -recruitment

Diagnostics & Therapies

Approval processes

Regulatory aspects

Pricing &Reimbursement

Health economy

HTA

Novel models of healthcare organisation

Prediction - Prevention – Treatment - Cure

Availability & usability in the clinic

Patient perspective

Equal treatment

Training of professionals

Personalised Medicine: a novel approach with many challenges along the way

BiomarkersIdentificationQualificationValidation

Data modelling tools

Technical aspects & challenges


EU Health Research Programme: Enabling personalised medicine 2007-2013

• large scale data gathering and "-omics"

• technology development

• diagnostics

• biomarkers

• pre-clinical and clinical research

• public health research

• IMI projects with pharma industry

Over 1 billion EUR to top research


Overview 2007-2013 Large-scale data gathering

Resources

Genomics & other -omics

Genetic epidemiology

International cooperation

Model organisms

Structural biologyand Cell biology

Basic knowledge

genetic/genomic epidemiology genetic epidemiology of disease

(hypertension, infections, pre-eclampsia...)

proteomicslipidomics

epigenomicscancer genomics

metagenomicsproteomics standardisation

receptors, channels & transporters, signalling proteins

Stem cells

zebrafish, fly, mouse (IKMC), rat & dog as models for human disease

35 projects€ 351 million


International K.O. Mouse Consortium

Contributing to international cooperation

International Human Microbiome Consortium

International Human Epigenome Consortium

International Rare Diseases Research

Consortium

International Initiative for Traumatic Brain

Injury Research

© rtg

uest/Fo

tolia

.com

Global Alliance for Chronic Diseases

Global Research Collaboration for Infectious

Disease Preparedness


International Cancer Genomics Consortium


• Structuring research areas within EU and beyond

• Setting standards of high quality data collection, integration and analysis

• Open access data repositories

Main achievements

Project example: BLUEPRINT

• BLUEPRINT preliminary results enable discovery of causative epigenetic factor for rare blood disease (TAR syndrome)



Staff Working Document onUse of '-omics' technologies in the development of personalised medicine

• the potential for, and issues with, the use of '-omics' technologies in personalised medicine, and the related EU research funding

• recent developments in EU legislation for placing medicinal products and devices on the market

• factors affecting the uptake of personalised medicine in health care systemsAvailable on: http://ec.europa.eu/health/files/latest_news/2013-10_personalised_medicine_en.pdf

http://ec.europa.eu/health/files/latest_news/2013-10_personalised_medicine_en.pdf




Use of '-omics' technologies in the development of personalised medicine Main Conclusions

• Horizon 2020 to address research challenges

• Efficient regulatory framework for medicines and in vitro diagnostics

• EU cooperation in HTA

-> essential responses from the EU to the current challenges

-> continued fruitful dialogue with all stakeholders


Identified Key research challenges

Translating knowledge to medical applications

disease taxonomy, biomarker validation, clinical trials

Understanding the value & economic aspects health care pilots, HTA, comparative effectiveness research, value chain

Breaking barriers & speaking the same language

"cross-disciplinarity", capacity building, education & training

Generating knowledge & developing the right tools

standards, clinical bioinformatics, adaptation of tools


Health promotion and diseases prevention: translating omics into

stratified approaches

(2014 topic)


• ‘Omics’ research (including but not limited to genomics, epi-genomics, meta-genomics and proteomics) is moving at a breath-taking pace.

• A major challenge for the next decade is to determine when and how this wealth of ‘omics’ information can be usefully applied by both the public and private sectors for the development of personalised /stratified approaches in health promotion and disease prevention.

Translating omics into stratified approaches for health promotion and disease prevention: Specific Challenge


proteomics

epi-genomics

genomics

transcriptomicsmeta-genomics

…. - omics

"-omics"

economics


• Develop and assess a personalised / stratified health promotion or disease prevention programme, taking into account the ‘omics’ characteristics of individuals, complemented by environmental and/or lifestyle factors

• Include the development of tools and methods for the use of 'omics' data in such programmes

Translating omics into stratified approaches for health promotion and disease prevention: Scope


• Include a multi-disciplinary approach to assess the validity and utility of ‘omics’ data in preventive medicine or in prevention programmes targeting specific population groups. This should include:

• The assessment of the predictive value of such programmes in identifying at-risk groups throughout their lives, as compared with conventional methods;

• The assessment of the usefulness of ‘omics’ data for improving the health of individuals or populations;

• The assessment should take into account age and gender aspects where appropriate.

• The assessment of the behavioural, ethical, legal, regulatory and social implications, as well as of the cost-effectiveness of the programme

Translating omics into stratified approaches for health promotion and disease prevention: Scope continued


• Include risk-benefit communication to various groups involved in such a programme, including individuals, policy makers and regulators

• Preference will be given to proposals focusing on diseases with either high prevalence or which present a high risk to the individual, or a high cost to society

Translating omics into stratified approaches for health promotion and disease prevention: Scope continued


• Evidence on the validity, utility and cost-effectiveness of ‘omics’ based health promotion and disease prevention programmes, allowing informed decisions on the organisation of health and care systems

• Budget: proposed EU contribution of between € 4-6 M (total budget € 24 M)

Translating omics into stratified approaches for health promotion and disease prevention: Expected impact and budget


Examples of relevant on-going projects

Predicting Human Metabolic Responses Using Advanced Genomics

METAPREDICT aims to combat diabetes, obesity and cardiovascular disease through translational discoveries by producing molecular ‘predictors’ for the personalised clinical response to exercise training.

www.metapredict.eu


Examples of relevant on-going projects

Epigenetics for Personalised Care of Female Cancers

EPIFEMCARE aims to develop new methods for screening, diagnosing and personalising treatment of breast and ovarian cancers.

www.epifemcare.eu


Piloting personalised medicine in health and care systems

(2015 topic)


• Personalised medicine has the potential to respond to, amongst others, the increasing burden of chronic disease and the complexity of co-morbidities, and in doing so contribute to the sustainability of health and care systems.

• If this potential is to be realised at a larger scale it must first be demonstrated in terms of sustainable benefits, and as a new model of care organisation. Demonstration is however complicated by the diversity of European Union health systems.

Piloting PM in health and care systemsSpecific Challenge


Piloting PM in health and care systemsScope• Pilots of new models of care, based on the concept

of personalised medicine will be conducted in existing health care environments and should take into account Europe's (national and regional) diversity in health system organisation.

• Proposal should ensure coordination with national, regional or local authorities engaging in health sector reform with the design of new models taking into account the views of other relevant stakeholders, including policy makers and citizens. Behavioural, ethical, legal, social implications as well as the gender dimension should be addressed


• The health, economic and social impact of the implementation of these pilots on individual patients, whole or stratified population groups, and their impact at the level of health care systems should be assessed

• The organisational and resource requirements of the piloted models (data, personnel and financing) should be tracked, providing evidence on methods of implementation and benefits of reform while ensuring safety, equity and cost effectiveness. Appropriate measures for knowledge transfer and capacity building should be put in place

Piloting PM in health and care systemsScope continued


• On the basis of quantitative and qualitative indicators, evidence for a validated model of organisation of care based on the concept of personalised medicine should be produced, to be used by policy makers and decision makers in making improvements to health and care systems

• Budget: proposed EU contribution of between € 12-15 M (Total budget € 30 M)

Piloting PM in health and care systemsExpected impact and budget


Examples from the EuroBioForum's Personalised Medicine Observatory

www.eurobioforum.eu

Personalised medicine in Europe


Personalised medicine in Europe

www.medizin.uni-greifswald.de/GANI_MED

www.cancerresearchuk.org

www.imtm.cz

www.geenivaramu.ee


French National Cancer Institute (INCA)

www.e-cancer.frSource: F. Calvo presentation 13-05-2011

Economic impact of molecular testing

Cancer Drug Biomarker

% patients

with mutation

Nb of tested

patients

Number of spared

prescriptions

Median PFS for

non responder

s

Cost of treatment/

patientSpared cost

Public fundings allocated for

the provision of the test

lung cancer

gefitinib EGFR mut

10,3% 1672215000 8 weeks 4 600 €

€69 M €1.7 Merlotinib 15000 8 weeks 4 600 €

colorectal cancer

cetuximab KRAS

mut36% 17250 6 210

8 months

32 419 € € 201 M

€2.5 M8 weeks 9 263 € €57 MPanitu-mumab

4 weeks 4 390 € €27 M


Time for questions


Thank you

www.ec.europa.eu/research/health

www.ec.europa.eu/research/horizon2020


REACHING OUT TO OTHER SECTORS


The determinants of health


The determinants of health – an example

The Journal of The Royal Society for the Promotion of Health JRSH, November 2006 Vol 126 No 6


Reaching out to other sectors

• Health is not the responsibility of the health sector only

• Health is a result of a number of factors, individual and social

• There is solid evidence that health can be influenced by policies in other sectors, and that health has, in turn, important effects on the realization of the goals of other sectors, such as economic wealth.

• Need for action at many levels for disease prevention and health promotion

• Break down the silos

• Capitalise on various fields of expertise


Inter-sectoral Governance for Health in all policies

• Health in All Policies encourages an inter-sectoral approach which is necessary to tackle challenges such as climate change, NCDs, ageing etc….

• It goes beyond the boundaries of the health sector, to link with policies such as transport, housing, the environment, agriculture, education, fiscal policies, tax policies and economic policies.


Reaching out to other sectors – call topics –

• PHC 4 - Health promotion and disease prevention: improved inter-sector co-operation for environment and health based interventions

• PHC 31 - Foresight for health policy development and regulation



PHC 4

Health promotion and disease prevention: improved inter-sector

co-operation for environment and health based interventions


Personalised exposure

monitoring

Early intervention

s

Testing

EMISSION SOURCES

CLIMATE CHANGE

LIFE-STYLE

SOCIO-ECONOMICS

EXTERNAL EXPOSURE

INTERNAL EXPOSURE

HEALTH/ DISEASE

TREATMENT

DIAGNOSIS

Mechanisms

Cohorts

Omics

Surveys

Bio-markers

Epigenetic programming

Environment andHealth Research

Molecular epi-

demiology Mixture toxicity

Challenges

Tools for integrated environment and health impact assessment


The SCOPE of this topic is limited to the integration of environment, climate and health sectors (including but not limited to air quality, water and sanitation, chemicals, occupational factors, etc.).

APPROACH:

multidisciplinary involving relevant stakeholders such as policy makers,

the private sector, civil society organisations and so on

PHC 4 - Health promotion and disease prevention: improved inter-sector

co-operation for environment and health based interventions - in 2015


Proposals should address all of the following elements:

1. Develop inter-sector interventions (and/or policy initiatives) to promote health or prevent disease based on known environmental stressors.

• These inter-sector interventions will address key environmental stressors for which changes in relevant EU and international policies related to environment, climate and health would have the greatest impact.

• In the development of these interventions, age and gender aspects should be taken into account where appropriate.

PHC 4 - SCOPE (continued)


2. Document success characteristics of the abovementioned inter-sector interventions, including those factors that help to overcome barriers to inter-sector co-operation; contextual factors such as the interplay between politics and economics should be addressed;

3. Assess these inter-sector interventions for their health, economic and social benefits and their impact on reducing inequalities.

To note: Research activities will be developed as a European contribution to existing international activities and those under development



PHC 4 - Expected impact and budget

Impact:

• On the basis of quantitative and qualitative indicators, evidence on effective interventions taking a ‘health in all’ approach, linking environment, climate and health, allowing informed decisions on multi-sector interventions and related policies.

• Impact on health and care systems and other public services in terms of their sustainability,

• Contribution to the EU commitment to the Rio+20 agenda and the new UN Sustainable Development Goals (SDGs), as well as to the Parma declaration 2010

Budget: proposed EU contribution of between € 4-6 M (total budget € 18 M)


Time for questions



PHC 31

Foresight for health policy development and regulation


Foresight to…

• Support more informed decisions for planning with a 5-20 years perspective – basis for policy dialogue

• Explore multiple futures and their consequences

• Assess risks – known risks/unknown risks

• Consider long-term changes in social, economic, technological and ecological environment

• help select best indicators to allow forecasting


Reaching out to other sectors- Foresight for health -

• Invest now in research to address the health challenges of the future - Foresight can facilitate the definition of strategic research and innovation goals for the short, mid- and long-term

• Challenges to health come from outside and within the sector


SCOPE

• to identify key driving forces- (external and internal to the health systems) likely to influence health and wellbeing in Europe and beyond in the future.

• to contribute to the understanding of the inter-relationships between these factors; analyse their economic and social impact and suggest alternative policy options to respond to the challenges they pose.

PHC 31 - Foresight for health policy development and regulation



• to present a comprehensive, structured and participatory framework of analysis, integrating and quantifying key factors impacting health, health inequalities and demand for health services taking Europe's diversity into account. Use of Copernicus data, products and information may be considered where relevant.

• Use should be made of current techniques for foresight such as: horizon scanning, trend monitoring, and analysis based on epidemiological surveillance (of health and health determinants), weak signal analysis, expert opinion (to create collective intelligence), scenario development, back-casting and wildcards (to help define alternative futures).


Proposals should

• Include quantitative analysis, such as environment, health, economic and other modelling and sensitivity analysis to measure variation in impact of different factors.

• Include the identification and validation of relevant measures & indicators and the development of (common) standards.

• Capitalise on existing good practice in Europe as well as international level experiences.



• The usefulness of current health data and statistics for these modelling exercises should be assessed and suggestions for improvement made.

• Proposals should also include networking between centres with existing expertise in (health) foresight, both public and private, and partnerships with centres aspiring to develop this expertise.

• If more than one proposal is successful, proposals should collaborate.



PHC 31 - Expected impact & Budget

Impact:• Through the use of a validated analytical framework with a

robust set of standardised indicators, the ability to model and track the impact of various factors (internal and external to the health systems) on population health should be improved.

• A basis for policy dialogue, facilitating timely decision making in the EU MS and beyond with regards to health sector reform and guide investments in health care to improve population health.

• Guidance for future health research

Budget: proposed EU contribution of between € 2-3 M (total budget € 6M)


Time for questions


LOOKING FOR EFFICIENCY GAINS


Health systems under stress

• Health spending is growing at a faster pace than GDP

• Driven by: ageing population; increasing patients expectations and demands on health care…

• Health inequalities are rising

=> need for transformation to ensure cost effectiveness & sustainability and access to high quality healthcare


Wanted:

• Through better health promotion and disease prevention, fewer ‘chronic patients’

• Simpler, more personalised and cost-effective technologies and treatments

• Integrated and patient-centred services

• Safety, quality and continuum of care

• Diverse and skilled health workforce

Health System Transformation

Investing now in the right

research will pay off


Health System Transformation – starting point –

A report from the US Preventive Services Task

Force recommends against prostate cancer screening

In the past 12 months alone, the BMJ, the Lancet, and the

New England Journal of Medicine have published 24 articles or communications

debating the value of breast cancer screening

"We need a continued focus on the cost-effectiveness of population

based disease prevention interventions, including the

‘deprogramming’ of interventions that are known

to be ineffective" Eur J Public Health (2013) 23 (5): 722. doi:

10.1093/eurpub/ckt102

More efficient use of available resources

Disinvesting in ineffective or not cost effective

screening programmes


Evaluating screening & prevention programmes

• It is about reassessing a programme in light of new

evidence and practice

• Allows further programming or deprogramming

where relevant

=> More efficient use of resources


• Proposals should assess existing screening and disease prevention strategies and programmes, at the level of the individual or stratified population groups and across Europe, on the basis of:

health outcomes,

quality-of-life,

equity and cost-effectiveness

and ethical considerations,

• The gender dimension should be taken into account where relevant.

PHC 6 - Evaluating existing screening and prevention programmes

SCOPE


• Comparison between different countries and regions, demographic groups and cultures should be made in order to identify specific contextual links as well as to identify opportunities for exchange of knowledge and experience between countries and regions.

• Proposals should include the development of new methods or the adaptation of existing ones for this type of assessment. These methods and tools (including self-assessment tools) should be applied in different health systems and organisational infrastructures to test their applicability in different political, economic and societal contexts.



• Due attention should be paid from the outset to the further development and dissemination of methodological expertise, including capacity building across Europe, in order that the expertise generated is fully exploited.



PHC 6 - Expected impact & budget

Impact:• Evidence for the increased use, or discontinuation of,

existing screening and prevention programmes allowing informed decisions by policymakers

• Capacity building in the assessment of such screening and prevention programmes.

• Improved health outcomes, greater health equity and cost effectiveness based on the implementation of effective screening and prevention programmes

Budget: proposed EU contribution of between € 2-3 M (total budget € 15M)


Time for questions


Health System Transformation – starting point –

More efficient use of available resources

In search of new models, more prevention oriented


Health System Transformation – In search of new models…..

• "At the same time, Primary Health Care (PHC) reforms, and the PHC movement that promotes them, have to be more responsive to social change and rising expectations that come with development and modernization.(...) This requires delicate trade-offs and negotiation with multiple stakeholders that imply a stark departure from the linear, top-down models of the past". (The World Health Report 2008; Primary Health Care Now More Than Ever.)

• Press release of the American Medical Association - Nov. 18, 2013WASHINGTON -- Team-based health care delivery models are quickly emerging as the preferred method for providing coordinated, cost-effective, high-quality health care for patients and today the American Medical Association's House of Delegates adopted new recommendations for creating payment mechanisms to sustain these promising new models of care.

• In the long term, the sustainability of Europe’s healthcare systems will depend on two factors: reorienting its focus from treating illness to promoting health, and improving the management of chronic diseases to make it both more effective and more cost-efficient. (Economist Intelligence Unit (2012): Never too early: Tackling chronic disease to extend healthy life years)

• ”… the way to get costs lower is to move care farther and farther from the hospital setting — and even out of doctors’ offices." George C. Halverson – CEO Kaiser Permanente


SCOPE:

• As action oriented research, proposals should develop new, or improve on existing, models for health systems, in order to make these systems more patient-centred, prevention oriented, efficient, resilient to crises, safe and sustainable.

• The models’ applicability and adaptation to different European health systems and EU regions should be assessed, and their value, including individual and societal benefits, demonstrated.

PHC 23 - Developing and comparing new models for safe and efficient, prevention

oriented, health and care systems:


• Models may apply to different levels within the health system (micro – the patient interaction level, meso- the health care organization and community level, and macro - the policy level).

• They must be compared with alternatives (including existing models), capitalising on Europe's diversity.

• Views of relevant stakeholders such as policy makers and citizens should be taken into account in the design of and evaluation of these models.

• The gender dimension should be duly addressed.

• Capacity building and awareness raising activities for the adoption and further use of models developed should be included.



• Proposals should address the related challenge of ensuring appropriate and sufficient resources (human, financial, infrastructural, equipment (or consumables) and technology) for these new models and develop adequate governance mechanisms.

• Proposals may include methodological work in the field of health technology assessment, health systems performance assessment, health workforce analysis as well as indicators and measures to describe and monitor the quality of life of European citizens adequately, taking into account the diverse socio-demographic groups and cultural backgrounds, and should track costs.



PHC-23 - Expected impact and budget

Impact:

• On the basis of quantitative and qualitative indicators, evidence for new or improved patient-centred, prevention oriented, safe and efficient models for health care systems and services.

• Evidence to be used by policy makers and decision makers in making improvements to health and care systems, health and other policies

Budget: proposed EU contribution of between € 4-6 M (total budget € 30 M)


Time for questions


Thank you

www.ec.europa.eu/research/health

www.ec.europa.eu/research/horizon2020

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