Personalized Medicine 2020 and beyond – Preparing Europe for leading the global way (PerMed)

Project Information

PERMED

Grant agreement ID: 602139

Closed project

Start date 1 September 2013

End date 31 August 2015

Funded under

• Specific Programme "Cooperation": Health

Total cost € 645 115,00

EU contribution € 497 378,00

Coordinated by

DEUTSCHES ZENTRUM FUR LUFT - UND RAUMFAHRT EV
Germany

Final Report Summary - PERMED (Personalized Medicine 2020 and beyond – Preparing Europe for leading the global way (PerMed))

Executive Summary:
The implementation of Personalised Medicine (PM) was and still is a major challenge in Europe and beyond. It calls for appropriate governance strategies at the European and global level as it challenges the way in which healthcare systems worldwide are set up.

Based on many existing initiatives in the area of PM, a Coordination and Support Action (CSA) of 27 partners was initiated representing a well-balanced consortium of governmental and funding organisations, research societies and organisations, industry/SME as well as representatives of European patient organisations. In order to step up coordination efforts, allow synergies and avoid duplication or competition, a dialogue platform for these and other stakeholders in PM was established.

Based on a systematic analysis of published key reports as well as direct contact with experts in workshops and semi-structured interviews, a strategic research and innovation agenda (SRIA) was published. The SRIA addresses European decision makers and documents current challenges and recommendations for solutions to foster research and implementation in the area of Personalised Medicine.

Taken together, the established interdisciplinary and international dialogue platform plus the SRIA have prepared the ground for succeeding measures and initiatives, e.g. a European consortium for Personalised Medicine.
Project Context and Objectives:
By definition of the European Commission, Personalised Medicine (PM) refers to a medical model using characterization of individuals’ phenotypes and genotypes (e.g. molecular profiling, medical imaging, lifestyle data) 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. The Health Directorate of the European Commission's DG Research and Innovation stated already in 2012 that PM is one of the most innovative areas in the future of health research with a high potential for patients, citizens and the economy.

However, its full potential could not be developed due to fragmented activities, insufficient communication and lack of generic solutions in the different areas of Personalised Medicine. To promote optimal Personalised Medicine implementation details of national strategic initiatives, research activities and best practices along the entire value chain have to be exchanged and, where feasible, reasonably aligned. This requires a joint effort on a European or even international level.

The CSA PerMed aimed to prepare the ground for such a joint effort by creating a dialogue platform for all relevant stakeholders and by providing guidelines to decision makers about current challenges and possible solutions in the implementation of PM.
The main objectives of the CSA PerMed were to:
• Identify relevant fields, organisations, current national, European and selected international initiatives, policies and capacities related to Personalised Medicine based on an inventory and synthesis of existing relevant information
• Step up coordination efforts between European key players and stakeholders in the different areas of Personalised Medicine (e.g. governmental and funding bodies, researchers, private sector, regulators and policy makers, payers and insurers, service providers and healthcare professionals as well as citizens/patients) to create synergies
• Highlight European and national showcases and best practice examples for already successful approaches in all areas related to Personalised Medicine
• Complement existing activities by identifying and promoting promising research topics
• Develop a strategic research and innovation agenda with general recommendations and research activities which could foster the further implementation of Personalised Medicine (PM) in Europe and beyond.
• Disseminate workshop results and recommendations to the European Commission, European initiatives, national ministries and funding organisations, and other relevant stakeholders identified in the workshops, including the general public (WP4 and WP5).
• Explore the potential for a European or global Personalised Medicine initiative.

Project Results:
Inventory of Activities, Key Players and Identification of Gaps and Needs

Currently, the uptake of PM in Europe is stagnant, despite its increasingly abundant benefits. The full advantages and the potential of PM can only be achieved when it is used in clinical decision making in an informational, structured framework, but the discrepancy between the ability to sequence a genome and to identify a small amount of relevant genes for translation into clinical practice is a major bottleneck. The translation of biomarker research into clinical practice is also challenging, since it affects various areas of healthcare. It involves a wide range of stakeholders and entails tackling numerous barriers for implementation. In order to integrate PM in clinical practice, these barriers must be lifted.

In order to identify existing barriers in Europe, different sources and viewpoints were combined. Many national and European key players, networks and organisations, as well as industry and SMEs, are actively engaged in the emerging field of Personalised Medicine and have produced strategy documents. However, these parallel activities remained poorly connected and there was an urgent need for coordination to avoid duplication of work.

In a systematic literature review, relevant reviews and strategic reports were analysed for gaps-and-needs and SWOT (strengths, weaknesses, opportunities and threats) aspects. Additional sources of information were surveys and interviews conducted with relevant stakeholders in the different areas of PM. The involved stakeholder groups represented basic science, translational research, the regulatory field, health systems and patient perspectives. They were identified from the strategic reports as well as by nomination from the 27 PerMed partners. All in all, 36 interviews were carried out and a list of SWOTs, challenges and needs drawn from the interviews was prepared for WS1.

To assess the most relevant barriers, an index was developed. The prioritisation of relevant barriers took place at the PerMed meeting in Berlin (March 2014) with around 90 participants representing different organisations attending the meeting. Recommendations were developed to facilitate the integration of PM into the European and national research strategies in a timely, effective and efficient manner. The details of this approach were published (Horgan D. at al., 2014, s. attachments) and presented at the PerMed workshops in Berlin and the European Health Forum Gastein (EHFG, October 2014). They were used as a starting point for the development of the strategic research and innovation agenda (s. WP3).

Dialogue Platform for Relevant Stakeholders in Personalised Medicine

The goals of this platform were to strengthen the cross-border networking between users and providers as well as purchasers and performers in the field. The aim was to overcome national, European and international limitations and to illustrate the benefits, needs and potential for cooperation in Personalised Medicine for research, healthcare systems, economic development and European citizens.

The dialogue platform was based on the “Round table PerMed”, a forum for ministries and funding organisations. Its aims were to exchange information about on-going and designated measures, as well as national and regional strategic agendas and interests. Furthermore it was monitoring the necessary framework for the successful translation of research. The activities of this “Round Table PerMed” also prepared the ground for a European Personalised Medicine Consortium.

An Internet platform was established to support internal and external communication (s. also below). Two successive workshops were organised to connect key players and stakeholders in the different areas of PM across Europe and beyond (e.g. governmental and funding bodies, researchers, the private sector, regulators and policy-makers, payers and insurers, service providers and healthcare professionals, and citizens/patients).

The first workshop took place on March 27-28, 2014 in Berlin (Germany). It was dedicated to review, discuss and adapt with stakeholders the recommendations distilled from strategic reports and semi-structured interviews (s. above). Expert lectures were organized to open up the discussion and give important impulses by international best-practise examples. Special care was taken to integrate new input and complementary suggestions and to avoid duplication to existing publications and previous events. To foster the discussion among the participants four key areas were defined:
1. “Basic Research & New Technologies”,
2. “Translational Research”,
3. “Regulation, Reimbursement & Market access”,
4. “Health Care System in general”.
Participants were actively distributed to the different sessions to reach a balanced representation of competence respectively. In addition, representatives of related projects were invited (e.g. CSA 3GB-Test, CSA CASyM, E-Rare, ERA- Nets Transcan and Neuron). In the key note presentations participants were introduced into the topic (international and EU perspective) and made familiar with the results of the analysis so far. The sessions served as discussion space to prioritize, add and/or delete recommendations in the respective field of expertise of the participants.
To highlight important topics and to trigger the discussion as well as the work within the Session the following lectures where held by the invited speaker and PerMed representatives:
• Pierre Meulien: Towards Implementing Personalised Medicine in Canada
• Irene Norstedt: Personalised Medicine: an EU Perspective
• Angela Brand, Lada Leyens and Erica Hackenitz: SWOT and Gaps & Needs analysis for Personalised Medicine
• Reiner Banken: Health Technology Assessment (HTA) for adding Value to Innovation
• Helmut Brand: Personalised Medicine for the European citizens
• Christof von Kalle: Personalised Cancer Therapy (unfortunately the speaker could not attend the meeting due to public transport problems)
Outcomes of the sessions were presented on the second day and discussed with the entire audience to ensure that cross-sectional issues were adequately addressed. The workshop was attended by around 90 persons.
As a result of the analysis of WP1 and the Berlin workshop, recommendations for improving the implementation of PM in Europe were developed. The results of the workshop were also taken into account for the concept of the second workshop at the European Health Forum in Gastein (EHFG, October 2014) and the development of the EAPM (European Alliance for Personalised Medicine) Road Map on research topics to bring forward Personalised Medicine.
The second workshop was hosted in cooperation with the international Forum Gastein (IFG), embedded as a Parallel Forum at the conference of the European Health Forum Gastein (EHFG) in Gastein, Austria: Forum 4 “Personalised medicine 2020”. It had a special focus on ethical issues; citizen/patients aspects and the view of the general practitioners (GPs). An additional objective of the second workshop was to discuss with the participants of the PerMed Forum the 16 shortlisted recommendations, selected from the 33 recommendations that were identified during the first WS in Berlin. The debate was enriched by the speeches of the invited speakers and contributions by the audience.
To highlight important topics which are adding to the focus of this second workshop and to trigger the discussion as well as the work within the Session, the following lectures where held by the invited speaker and PerMed representatives:
• Angela Brand and Erica Hackenitz on behalf of PerMed (A first set of 16 recommendations)
• Falk Ehmann, European Medicines Agency (EMA): Early Dialogue and Regulatory Aspects
• Effy Vayena, University of Zurich: Legal and Ethical Aspect
• Kaisa Immonen-Charalambous, European Patients' Forum (EPF): Citizens’ Perspective and Needs
• Antonio L. Andreu Periz, Instituto de Salud Carlos III (ISCIII): A Hospital’s Perspective
• Ferenc Hajnal, European Union of General Practitioners (UEMO): The General Practitioner's perspective
• Christoph Klein, University Munich: Best Practice Example - Rare Diseases
• André Boorsma, Netherlands Organisation for Applied Scientific Research (TNO): Best Practice Example - Nutrition

Embedding the workshop into the EHFG conference enabled the recommendations to be revised and discussed with the target audience of policy makers (of around 120 participants). These refined recommendations were a preparatory step for the development of the strategic research and innovation agenda (SRIA, s. WP3). In addition, five overarching challenges were defined in preparation for the workshop and discussed and refined during the PerMed partner meetings as well as the round table PerMed meetings. These challenges represent the entire health care value chain, beginning with the citizen and patient, through researchers and manufacturers of drugs and medical devices, and finally to healthcare providers, regulatory bodies and governmental agencies.
Parallel to the EHFG event an article describing PerMed activities was published in a special issue of the EuroHealth magazine (Leyens L. et al., 2014, s. attachment). This issue was included in the EHFG hand out to all participants.

Guidance and Recommendation for strategic development - Strategic Research & Innovation Agenda

Using the inventory described in WP1 and the results of the dialogue platform of WP2 as input, a Strategic Research & Innovation Agenda (SRIA) was developed as a guideline for European decision makers. Its main objective is to show relevant current challenges in the implementation of PM, to give recommendations for addressing these challenges and to suggest key research priorities in PM.

Based on the results of the inventory and the workshops, the PerMed identified running and planned PM activities and identified enablers and best practices. It proposed common activities and potential joint funding in PM research, as well as opportunities for the alignment of existing regional, national, European and, where possible, international strategies. After the compilation and revision of the challenges and recommendations arising from the workshops, a consultation process within the scientific community as an external validation step was initiated to allow further refinement of the recommendations. Comments and input from over 25 experts covering all relevant areas of expertise were integrated into the SRIA. In summary, the SRIA identified five main challenges for the implementation of PM in Europe:
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 Healthcare
Based on recent and future developments, the SRIA contains 35 recommendations. These are grouped into the five overarching challenges, taking into account that some recommendations apply to more than one challenge. They include suggestions for creating the necessary framework regarding e.g. economic, organisational, regulatory, ethical, legal and social aspects for the successful translation of PM research results into clinical practice.
Highlighted in the SRIA are nine prioritised recommendations by PerMed Round Table, which have the highest potential impact and outcome in facilitating the introduction of PM for the benefit of patients, citizens and society as a whole. These prioritized recommendations are to:
• Demonstrate the impact and potential benefits of PM for health systems, citizens and society by supporting public health evaluations to assist decision- making and develop appropriate, equitable and sustainable access for all patients
• Incorporate patient participation and responsibility in all phases of research and development in the healthcare system and in the ownership and control of personal health data
• Develop common principles and regulatory frameworks that enable sharing of personal data for research in a way that is ethical and acceptable to patients and the public.
• Promote the development of high quality sustainable databases including clinical, environmental, social, health and wellbeing information.
• Support translational research infrastructures and enforce data harmonisation fostered by specific ICT infrastructures designed to health data.
• Develop new decision support tools and methodologies of ICT to analyse and interpret data in order to support physicians and other key stakeholders in their decision-making process.
• Develop methods to better integrate and evaluate the information provided by genomic, epigenetic, transcriptomic, proteomic, metabolomic and micro-biome analyses.
• Support development of new clinical trial designs taking into account best available evidence on the individual level and promote integration with concomitant preclinical testing.
• Encourage a systematic early dialogue between innovators, citizens and decision-makers throughout all regulatory steps to provide guidance and clarity.

Furthermore 22 PM research activities and their expected impact have been suggested. All of them are based on PerMed research recommendations and examples of national measures aimed at the development of PM approaches. As a result of the discussions of the Round Table, the instrument of an ERA-Net is suggested for the near future in the SRIA.
More details can be found in the attached SRIA document.
Potential Impact:
WP4 – Dissemination of CSA PerMed Dialogue Platform and Results

A first step for dissemination of the CSA PerMed activities and results was the creation of a project website (www.permed2020.eu). In addition to giving an introduction into the project, the results of the two workshops including all presentations can be accessed there. A PerMed newsletter was compiled and disseminated as an upload on the webpage as well as distributed via the partners’ channels (http://www.permed2020.eu/1418.php). The SRIA and the three PerMed publications are also available for download. Facilitating internal communication, the website contained a sharepoint that was accessible for PerMed partners only and used for sharing documents and other information. Cross-links to other relevant communication platforms such as EuroBioForum and the EAPM were established.

The two workshops described above were not only a means to bring together stakeholders from all relevant areas of expertise, but were additionally important for dissemination. Especially the second workshop that took place at the conference of the European Health Forum Gastein reached a large audience of relevant experts, decision makers and other stakeholders.

PerMed results were published in three peer-reviewed papers (s. attachments; Horgan D. at al., 2014; Leyens L. et al., 2014; Leyens L. et al. 2015).
In addition, PerMed results were presented during several workshops, conferences and meetings ( e.g. EuroBioForum, May 2014 in Munich; annual meeting of the EAPM September 2014 in Brussels; the High level Conference “Making Access to Personalised Medicine a Reality for Patients”, July 2015 in Luxembourg).
The SRIA mentioned above was introduced to the general public in a press conference in Brussels that took place during the EAPM (European Alliance for Personalised Medicine) annual conference in June 2015. This setting ensured a maximum outreach to relevant experts and policy makers. Drafts and final versions of the SRIA were channelled into the research and policy agenda setting process at the level of European institutions, e.g. as input to the Horizon2020 work group “Personalised medicine, mechanisms, systems medicine, biomarkers and diagnostics” and the EU Presidency Conference of Luxembourg.
The Europe-wide network established by the CSA PerMed and hereby especially the “Round Table PerMed” will have an impact far beyond the actual duration of the PerMed project. Initiatives based on the CSA PerMed activities and results are currently planned by the European Commission and PerMed partners are taking an active role in this. During the project, it became increasingly clear that the implementation of Personalised Medicine is a European or even global task. Indeed, a European consortium for Personalised Medicine is currently being planned by the European Commission (s. Horizon 2020 call H2020-SC1-2016-2017) that might later be expanded to an international consortium.
Transnational collaborative research actions are needed that implement at least some of the recommendations identified by the SRIA. The Round Table PerMed considered an ERA-Net Cofund on PM with training and outreach modules as well as support for cross-sectional projects to be the most suitable instrument in the European context and included this suggestion into the SRIA. In fact, such an ERA-Net Cofund is now part of the current Horizon2020 program (s. Horizon2020 call H2020-SC1-2016-2017).
List of Websites:
http://www.permed2020.eu

Contact of Coordinator: Wolfgang Ballensiefen, German Aerospace Center Project Management Agency (DLR-PT), Health Research, Heinrich-Konen-Str. 1, 53227 Bonn Germany, Phone: [phone], mail: [e-mail]