Towards the Implementation of Personalised

Medicine in Europe: the Strategic Research

and Innovation Agenda (SRIA)Tallinn, 26 October 2015

Erica Hackenitz

The Netherlands Organisation for Health Research and Development

(ZonMw)

For CSA PerMed

Why are our health interventions still not so

successful?

Only 15% are evidence-based

Patients react different on the same intervention

• Diseases are due to genome-environmental

interactions. Examples: cancer, CVDs, obesity, addiction

• There is no „one size fits all“

We need more targeted, “personalised“ interventions

A paradigm shift is needed

• From common complex diseases to “multiple rare diseases”

• From diseases to “diseasomes”

• From risk factor to “risk pattern”

• From clinical utility to “personal utility”

(A.Brand, 2008)

• “Some countries have already developed their national

strategy on PM while others have only started” (Inventory EC

DG Research 2015)

• A range of activities are ongoing in different countries and

consortia in Europe

• Joint forces will be necessary for the implementation of PM

State-of-the-Art

AIMS:

• To step up coordination efforts between European key

stakeholders already active in the field of PM

• To prepare a strategic research and innovation agenda

(SRIA)

EC: Coordination & Support Action (CSA) PerMed

• Research and Health Ministries

• Funding Bodies

• Research Institutes

• Industry and SMEs

• Foundations and Societies

• Connected to important other

European initiatives related

Personalized Medicine (e.g.

EuroBioForum, ESF, EAPM, CASyM,

EPEMED)

Who?

Ministry of

Social

Affairs

(MoSA-

Estonia)

Cooperating

partner

18 Partners, 9 cooperating partners

Evaluation of

Personalised Medicine

(PM) Reports (over

20)

Interviews with

PM Stakeholders

(around 40)

PerMed Partners

(27)

Further input

From a.o. Publications

and Meetings on PM

Workshop 1, Berlin~ 90 participants

• Basic Research & New Technologies

• Translational Research

• Regulation, Reimbursement & Market

access

• Health System in General

• PM in Europe & Canada

• HTA and Citizens perspective

Workshop 2, Gastein, EHFG Forum 4 ~ 120 participants

• Patient involvement

• General Practitioner (GP)

• Hospitals

• Legal & Ethical aspects

• Regulatory aspects

• Rare Diseases (example)

• Nutrition & life style (example)

SRIA Recommendations to:

• European Commission

• Member States

• Research Communities

• Industry

• Funding and Regulatory Bodies

• Providers

How?

• 5 Challenges

• 35 Recommendations

Strategic Research and Innovation Agenda

SRIA - Challenges

• Challenge 1 – Developing Awareness and Empowerment

• Challenge 2 – Integrating Big Data and ICT Solutions

• Challenge 3 – Translating Basic to Clinical Research and Beyond

• Challenge 4 – Bringing Innovation to the Market

• Challenge 5 – Shaping Sustainable Health Care

I. Provide further evidence for the benefit delivered by PM to health

systems.

II. Develop and promote models for individual responsibility, ownership

and sharing of personal health data.

III. Develop mobile health applications to maximize engagement of

patients with their treatment pathways and track the safety and

effectiveness of these interventions.

IV. Understand how the changes related to PM will impact public health

and ensure they translate directly to benefits for individual citizens and

society.

1. Developing Awareness and Empowerment(excerpt)

I. Promote strategies to make sense of ‘big data’.

II. Develop and encourage the fast uptake of technologies for data

capture, storage, management and processing.

III. Support translational research infrastructures and enforce data

harmonisation fostered by specific ICT infrastructures designed to the

health data.

IV. Support analytical methods and modelling approaches to develop new

disease models, e.g. ‘Computerised Twins’ or a ‘Virtual Patient’.

2. Integrating Big Data and ICT Solutions(excerpt)

I. Develop methods to better integrate and evaluate the information

provided by genomic, epigenetic, transcriptomic, proteomic,

metabolomic and microbiome analyses.

II. Support research in preclinical models to validate hypotheses resulting

from molecular analyses of patient samples and treatment outcomes.

III. Promote collaborative pre-competitive and trans-disciplinary research

in all disease areas to gain trustworthy and objective information.

IV. Instigate a European-wide biomarker evaluation and validation process.

3. Translating Basic to Clinical Research and Beyond(excerpt)

I. Formalise a risk-based approach for the evaluation of PM.

II. Optimise individual drug therapies and poly-pharmacy especially in the

case of multi-morbidity.

III. Support research on an adequate regulatory and legal framework for

PM.

IV. Encourage a systematic early dialogue between innovators, patients

and decision-makers throughout all regulatory steps to provide

guidance and clarity.

4. Bringing Innovation to the Market(excerpt)

I. Support health economics research of PM to support decision makers.

II. Develop prospective surveillance systems for personal health data that

facilitate accurate and on-going assessment of highly dynamic health

information across the life course.

III. Develop training programmes on PM for health professionals.

IV. Encourage a citizen-driven framework for the adoption of electronic

health records.

V. Develop a framework for pricing and reimbursement for PM that

ensures equitable access for all patients – regardless of economic or

geographic status – and is sustainable for health systems.

5. Shaping Sustainable Health Care(excerpt)

Published and launched in June

2015 in Brussels.

• There will be a need to include more communication and training

modules, more outreach activities, and more non-research cross-sectoral

projects to complement ‘classical’ basic and translational research

activities.

• Funding needs to provide incentives to include specialists from a wide

range of areas such as: Big data, information and communication

technologies, data sharing and integration, public health, ethical, legal

and social aspects, regulatory affairs, industry

Towards PM ImplementationThe challenge for research funders and decision-makers will be to fund

research beyond the classical funding schemes

DG Research has organized several workshops with MS to join forces for PM

research in Europe.

Towards a

European

roadmap for

Personalised

Medicine

“European Personalised Medicine

Consortium” (EPMC)

The basis for

the EPMC

initiative of

the

European

Commission

Each Challenge Group is composed of Experts

and lead by a Facilitator from one of the Member States..

ESTONIA?

Possible Structure EPMC

Shaping Europe’s Vision for Personalised Medicine

Thank you very much for your attention