European Science Foundation www.esf.org A Forward Look on Personalised Medicine Stephane Berghmans European Health Forum Gastein, 4-5 October 2012

1. www.esf.org
A Forward Look on Personalised Medicine
Stephane Berghmans
1
European Health Forum Gastein, 4-5 October 2012
ESF Forward Look - EHFG, 4-5 October 2012

2. European Science Foundation
Member Organisations
ESF is an independent association
of 78 Member Organisations
• research funding organisations
• research performing organisations
• academies and learned societies
in 30 countries
2 ESF Forward Look - EHFG, 4-5 October 2012

3. Standing Committee domains
•Biomedical Sciences
•Humanities
•Social Sciences
•Life, Earth & Environmental
Sciences
•Physical and Engineering
Sciences
Expert Board/Committee domains
•Marine Sciences • Radio Astronomy
•Polar Sciences • Nuclear Physics
•Space Sciences • Material Sciences
& Engineering
3 ESF Forward Look - EHFG, 4-5 October 2012

4. Forward Look on Personalised Medicine
Foresight instrument
● Multidisciplinary topics or
complex areas viewed at a
European level
● Medium to long-term scientific
perspectives
● Link scientists, stakeholders and
policy makers
● Wide consultation – identify
major opportunities and
constraints
● Produce major reports and
action plans
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5. ESF Forward Look on Personalised Medicine
Governance
Scientific Committee Management Committee
Chairs
• Aarno Palotie, Head of Medical • Liselotte Højgaard EMRC, Chair
Sequencing at the Wellcome Trust • Milena Zic-Fuchs SCH, Chair
Sanger Institute, UK; Director of the
Finnish Genome Center, University of • Michel Salzet LESC, member
Helsinki , Finland • Rainer Kattel SCSS, core group member
• Stephen Holgate, Medical Research • Jukka Corander PESC, representative
Council, School of Medicine, University of
Southampton, UK
• Heyo K. Kroemer, DFG
• Barbara Prainsack, Centre for
Biomedicine & Society - CBAS Brunel, Deutsche Forschungsgemeinschaft
UK • Jacques Grassi, Inserm
Institut National de la Santé et de la
• Angela Brand, IPHG, Faculty of
Recherche Médicale
Health, Medicine and Life Sciences,
Maastricht University, The Netherlands • Joaquin Arenas Barbero, MICINN
• Hans Lehrach, Max Planck Institute Spanish ministry for science and innovation
for Molecular Genetics, Berlin, Germany
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6. Forward Look on Personalised Medicine
Methodology
In
-d
in e p
te th
rv e
ie xp
w e
s rt
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7. Forward Look on Personalised Medicine
TABLE OF CONTENT
1.Introduction
2.How do we define personalised medicine?
3.What can personalised medicine offer?
4.What are the technological considerations for personalised
medicine?
5.What are the disciplinary considerations for personalised
medicine?
6.What are the key issues affecting the development and
implementation of personalised medicine?
7.Recommendations
8.Can we predict a timeline for the development and
implementation of personalised medicine?
7 ESF Forward Look - EHFG, 4-5 October 2012

8. Forward Look on Personalised Medicine
TABLE OF CONTENT
1.Introduction
2.How do we define personalised medicine?
3.What can personalised medicine offer?
4.What are the technological considerations for personalised
medicine?
5.What are the disciplinary considerations for personalised
medicine?
6.What are the key issues affecting the development and
implementation of personalised medicine?
7.Recommendations
8.Can we predict a timeline for the development and
implementation of personalised medicine?
8 ESF Forward Look - EHFG, 4-5 October 2012

9. Forward Look on Personalised Medicine
80
recommendations
…
resulting in a
Personalised
Medicine
“Target”
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10. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“At the heart of
personalised medicine
lies the individual
citizen…”
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11. Forward Look on Personalised Medicine
WHAT CAN PERSONALISED MEDICINE
OFFER?
For Personalised Medicine to be effectively introduced
stakeholders must be clear about its benefits
THE EUROPEAN CITIZEN
•Promise of safer, more effective treatments
Examples in stratified medicines to treat specific tumour subtypes
•Longer-term promise: prevention of disease
Genetic prediction
Monitoring of physiological status over the life course
•Empowerment: more control over their own health
Today citizens don’t feel in control of decisions about their wellbeing
Issues: less direct involvement of healthcare professionals
health literacy and access
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12. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“… the citizen’s health status
will be reflected by a new
disease taxonomy informed by
the multi-layered
characterisation of
physiological and
pathological processes”
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13. Forward Look on Personalised Medicine
WHAT CAN PERSONALISED MEDICINE
OFFER?
For Personalised Medicine to be effectively introduced
stakeholders must be clear about its benefits
FOR THE HEALTHCARE PROFESSION
•Opportunities in diseases subclassification
Possible greater insight into complex & chronic diseases
More effective use of existing drugs
Drug efficacy in unstratified patient groups
•Potential for a life-course approach to healthcare
Individual baseline physiological data
Improved recognition of relevant pathological changes
Opportunity for greater prevention; early intervention
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14. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“Comprehensive, accessible,
and interoperable datasets
must be generated to
support the development of a
new disease taxonomy and
allow for its ongoing
refinement and
application"
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15. Forward Look on Personalised Medicine
WHAT ARE THE TECHNOLOGICAL CONSIDERATIONS
FOR PM?
Defining feature of PM will be data integration
ICT ICT
solutions solutions
Global solutions
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16. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“Models and decision-making
processes must be revised to
reflect a focus on the
individual citizen at all levels,
from assessment of the safety
and efficacy of interventions,
through HTA and
reimbursement, to diagnosis,
treatment and prevention”
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17. Forward Look on Personalised Medicine
WHAT CAN PERSONALISED MEDICINE
OFFER?
For Personalised Medicine to be effectively introduced
stakeholders must be clear about its benefits
FOR REIMBURSEMENT BODIES
•Will PM reduce health expenditure: promise or benchmark?
e.g. investment in new technology and infrastructure
e.g. drug reprofiling based on stratification
Scepticism: PM has potential to increase healthcare expenditure
•Cost of increased emphasis on diagnostics and preemptive testing?
Direct cost of investment in diagnostics technology
False-positive diagnosis (offset by long-term reductions through prevention?)
•Realistic promise and expectations
Cost containment vs. cost reduction: improved return on investment
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18. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“Emphasis must be placed on
stakeholder participation,
interdisciplinary interaction,
public-private and
precompetitive
partnerships, and
translational research in
order to develop the
frameworks that support the
vision of personalised
medicine and healthcare”
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19. Forward Look on Personalised Medicine
WHAT ARE THE DISCIPLINARY CONSIDERATIONS
FOR PM?
THE CHALLENGE
OF
INTERDISCIPLINA
RITY
3 disease areas
Challenge
Achieve cross-disciplinary
consensus to allow PM
challenges to be addressed
*approved by FDA as of September 2011
in each context
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20. Forward Look on Personalised Medicine
Personalised
Medicine
“Target”
“Dedicated funding and
governmental support must be
provided to ensure the
availability of core
infrastructure, including
access to core technology and
frameworks for education and
training of professionals and
the wider community”
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21. Forward Look on Personalised Medicine
Personalised
Health Care
Transformation
Reform
Change
Management Stakeholder
Engagement
Critical
Timing Proof of
Principle
...
Global
Stakeholder Group
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22. Stephane Berghmans
Formerly Currently
Head of Unit CEO
Biomedical Sciences Center for Innovation in Medicine
European Science Foundation Liege, Belgium
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