1. http://www.patientsacademy.eu – info@patientsacademy.eu European Patients’ Academy on Therapeutic Innovation The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n° 115334, resources of which are composed of financial contribution from the European Union's Seventh Framework Programme (FP7/2007-2013) and EFPIA companies.

2. Health research & policy is changing at a fast pace Innovation transforms the lives of patients with serious, lifelong conditions: Molecular targets/pathways Genome sequencing, Translational research Personalized medicine Small trial populations Biomarkers, companion diagnostics Need for post-marketing data Health Technology Assessment, QoL, endpoints, comparators BUT long term pressure on health budgets – here to stay Window of opportunity trial design relationship between researchers, regulators, industry, patients 2

3. Unmet need of patient and public on information on medicines R&D Patients… seek up-to-date, credible, understandable information about innovation in treatments are largely unaware about clinical trials, translational research, personalized medicine, pharmaco- economics, their key role Patient advocates… like to advise on protocol design, informed consent, ethical review, marketing authorization, value assessment, health policy lack the education and training required to participate as a partner in medicines R&D FP7-funded PatientPartner project demonstrated a clear need & willingness to contribute

4. Patients' organisations key role in building a new environment for the development of new medicines Patient‘s organisations have unique insights in „real life“ and „real needs“ of patients: Gap analysis in research priorities Clinical trial design Priority setting Research policy Training required to get expertise required to contribute to medicines research & development (R&D) projects Research subject Info provider Advisor Reviewer Co-researcher Driving force Source: PatientPartner FP7 Project (2010) 4

5. Public Research Ethics Committees Competent authorities Policy makers /Research Policy HTA agencies & committees Research subject Info provider Advisor Reviewer Co-researcher Driving force Clinical Research Patients have a key role in all aspects of health-related research Trial protocol design, informed consent, ethical review, marketing authorization, value assessment, health policy Are there enough patient advocates to engage in R&D?

6. European Patients’ Academy: Paradigm shift in empowering patients on medicines R&D  Launched Feb ’12, runs for 5 years, 30 consortium members, Funded by IMI JU  will develop and disseminate objective, credible, correct and up-to-date public knowledge about medicines R&D  will build competencies & expert capacity among patients & public  will facilitate patient involvement in R&D to collaborate in academic research, industry research, authorities and ethics committees

7. The Patients‘ Academy addresses unmet needs European Patients' Academy on Therapeutic Innovation is… initiated and led by key European Patient Advocacy organisations the key European initiative to develop and provide objective, credible, correct and up-to-date public knowledge about medical research a paradigm shift in empowering patients and the public to understand medical research and how to contribute to it a strong multi-stakeholder consortium of patient organisations, academia, NGOs and industry

8. Within the next 5 years, the Patients‘ Academy will…  develop and disseminate accessible, well-structured and user- friendly information and education on medicines R&D  build expert capacity by training patient advocates, and competencies among patients and the public  create the leading public library on medicines R&D: 7 languages, “creative commons” license  facilitate patient involvement in R&D to partner up with academia, authorities, industry, ethics committees …and NOT: develop indication- or therapy-specific information!

9. Areas covered by the European Patients’ Academy 1. Discovery of Medicines & Planning of Medicines Development 2. Non-Clinical Testing and Pharmaceutical Development 3. Exploratory and Confirmatory Clinical Development 4. Clinical Trials 5. Regulatory Affairs, Medicinal product Safety, Pharmacovigilance and Pharmaco-epidemiology 6. Health Technology Assessment and the economics …and NOT: develop indication- or therapy-specific information!

10. EUPATI Patient Experts Training Course -- for expert patients To bring this to life, EUPATI develops education targeted at different levels 100 patient advocates 12.000 patient advocates 100.000 individuals EUPATI Educational Toolbox -- for patient advocates EUPATI Internet Library -- for the health-interested public English French German Spanish Polish Italian Russian English

11. EUPATI Patient Experts Training Course has started on 6 Oct 2014 Outline 150+ hours of e-learning and two 4-day sessions Two cycles of 50 participants Open to patients, carers, patient advocates and volunteers Selection By the application deadline for the first 50 trainees, more than 300 applications were received! Great demand from the patient community! Selection was done by selection panel based on solid criteria Start of course: Oct 2014 (1 st 53 trainees), Sept 2015 (2 nd 50 trainees) e-Learning EUPATI Training Programme 10/2014 – 11/2015 Application Phase 3-4/2014 e-LearningF2f e-Learningf2f Internet Library Toolbox Courses

12. Reflecting European diversity: 7 languages, 12 countries 7 most frequently spoken languages: English, French, German, Spanish, Polish, Italian, Russian Serving 12 European countries: UK, Ireland, Malta, France, Luxemburg, the francophone Belgium, Germany, Austria, Switzerland, Spain, Italy and Poland, plus Russian-speaking population in CEE

13. EUPATI National Platforms: Partnership on the country level EUPATI National Platforms will... make sure EUPATI understands educational needs in R&D on national level when developing content disseminate EUPATI’s existing training material and information on the national level To raise public interest about EUPATI in 12 countries To identify training faculty, logistics and financial support on the national level AT BE CH DE ES FR IT IE LUX MT PL UK

14. Project led by 4 key pan-European patient associations ◦ EUPATI Project Coordination ◦ >50 umbrella patient organisations. ◦ Linking national and regional patient alliances ◦ >600 rare disease organisations in >45 countries ◦ >100 members in over 30 countries Additional partners in other patient organisations and "members of members" via "EUPATI Network"

15. Coordinated by patients (EPF) Leading pan-EU patient umbrella groups involved in all key activities Strong impetus from key academic partners and research organisations Industry expertise in medicines R&D Advisory bodies & codes committed to ensure independence and good governance EMA, Swissmedic, MHRA, BfArM, AIFA Key experts in bioethics, genetics, HTA, economics, evidence based medicine, patient advocacy provide feedback and expertise Strong consortium & strong governance

16.  Quality control in task forces and by experts: Materials will be reviewed and tested prior to release (multi-stakeholder editorial board, Ethics Panel, external advisers and experts)  EUPATI Regulatory Advisory Panel involving 5 regulatory bodies EMA, BfArM, Swissmedic, MHRA, AIFA. o ensures objectivity, transparency, impartiality and independence of EUPATI's content o supervises adherence to the 'Core Quality Principles of information to patients on diseases and treatment options' (Pharmaceutical Forum) o Provides guidance and advice to the project teams  EUPATI Project Advisory Board with leading independent experts from different areas  EUPATI Ethics Panel with genuine ethics expertise,  Effective Codes of Practices in place o EUPATI Code of Practice o EUPATI Ethical Framework o EFPIA Code for industry partners and 'Code of Practice between patient organisations and healthcare industry' endorsed by patient organisations Mechanisms to ensure independence and credibility of EUPATI

17. Preparation Phase M1-18 Confirmation Phase M19-48 Sustain Phase M49-60 WP1 Coordination Project Infrastructure Project Management, Communication, Evaluation, Finances WP2 Network Implem. Establishment of EUPATI Network Public conferences, Regional Workshops, EUPATI Network, EUPATI National Platforms WP3 Needs Assessment & Gap An. Needs analysis, Review of material, focus groups Quantitative survey, Literature review Publication of results Quality Assurance WP4 Content Dev. Syllabus Editorial process, Content Development, translation Refinement, quality control WP5 IT Infrastr. Design, development and support of technical infrastructure for eLearning/IT Platform WP6 Deploy- ment & Quality Assurance First course performed EUPATI IT Platform launch Deployment, dissemination, quality assessm‘t WP7 Sustainability Research on Patient Partnership models Best practice guideline dev., Recommendation of new teaching methods Development of code of conduct for patient involvement Sustainability concept dev. New technologies and future remit strategy Moved from preparation phase into implementation/development phase 17

18. Reminder: Governance structure WP2: Network Implementation Lead: EFGCP, Co-lead: Janssen WP3: Needs Assessment, Gap Analysis Lead: Nowgen, Co-lead: GSK, Novo Nordisk WP4: Content Development Lead: DIA Europe, Co-lead: Uni Copenhagen, GSK WP5: IT Infrastructure Lead: Hibernia, Co-lead: Amgen WP6: Deployment & Quality Control Lead: EPF, Co-lead: Amgen WP7: Sustainability Lead: EATG, Co-lead: Bayer Project Secretariat / WP1 EUPATI Director, Coordinator, Assistant, Communication Officer Lead: EPF, Co-Lead: VFA Project Steering Committee (Project coordinator, 1 representative of each Consortium Partner) Innovative Medicines Initiative Joint Undertaking Project Advisory Board Ethics Panel Regulatory Advisory Panel EMA, BfArM, AIFA, Swissmedic, MHRA 18

19. Task Forces within EUPATI WP2: Network Implementation WP3: Needs Assessment, Gap Analysis WP4: Content Development WP5: IT Infrastructure WP6: Deployment & Quality Control WP7: Sustainability WP1: Project Secretariat WP4 Task Forces 4.1 Medicines development process from research to approval 4.2 Personalized and predictive medicine 4.3 Drug safety and risk/benefit assessment of (novel and existing) medicines 4.4 Pharmaco-economics and health technology assessment 4.5 Design and objectives of clinical trials (& involved stakeholders) 4.6 Patients' roles and responsibilities in innovative medicines development (4.7) Editorial Board WP5 Task Forces 5.1 Functional specification 5.2 Implementation of the signed-off functional specification 5.3 Support and training to defined users in the organisation 5.4 Handover of infrastructure and hosting of the portal to EUPATI WP2 Task Forces 2.1 Establishment and Coordination of the EUPATI Network 2.2 Organisation of EUPATI Conferences and Regional Workshops 2.3 Organisation of National Liaison Teams to support other WPs' activities 2.4 Design, management and maintenance of a digital communication platform for all Network Members 2.5 EUPATI Ethics Panel WP1 Task Force Communications Task Force

20. Unique and unprecedented partnership between patient organisations, other public partners and pharmaceutical industry based on the philosophy of the IMI Unique in IMI that EUPATI is patient- and not industry-led Opportunity to establish an effective, transparent and credible partnership Exceptional learning experience for industry representatives to work with patient organisations in a constructive and reflective manner, which could serve as a role model in other environments as well Making best use of industry expertise in medicines R&D, incl. e.g. the legislative environment and development of ITP as required by law Opportunity to strengthen the voice of the patients in the search for innovation and new medicines Chance to reduce fear, lack of trust and misconception among patients regarding the pharmaceutical industry Uniqueness of the partnership

21. What we bring in and what we produce will be owned by the public EUPATI material will be provided under the "Creative Commons License" Content can be copied, distributed, edited, remixed, and built upon, all within the boundaries of copyright law on non-commercial basis Authorship and licensing needs to stay intact and mentioned on all derivatives Similarly applied by e.g. WikiPedia, Google, Whitehouse.gov and many others See http://creativecommons.org/licenses/by/3.0/ Public license model guarantees ownership and re-use by the public

22. Key stakeholders of the European Patients’ Academy Patient Advocacy Orgs (national/ int‘l) Industry & CROs Academia Research networks, & projects Regulatory authorities Policy makers (EU & Member States) Ethics Com- mittees Journa- lists (medical, lay press) Health- related NGOs

23. EUPATI 2013 Conference “A Vision for 2020”, Rome, 18/19 April 2013 183 delegates from 28 countries, great enthusiasm, best practice and healthy criticism 23

24. Beyond EUPATI: Creating sustainable impact beyond 2016 EUPATI will develop:  New and innovative concepts that will ensure more active involvement of patient experts in pharma R&D, HTA  Guidelines for interaction between stakeholders, ethical principles and best practice procedures.  Best practice guidelines for patient involvement in research and drug development from trial design to study implementation, safety monitoring, approval, access, reimbursement, pharmacovigilance and benefit/risk assessment.  Road map for future collaboration with highly innovative sectors of industry, including and beyond the pharmaceutical industry, and Health Authorities

25. EUPATI in 2016: What we will have achieved  EUPATI platform fully loaded with training, education, information material in multiple languages  EUPATI Patient Ambassador, Patient Journalist, Train-the-Trainer Programme in place  Good practice guideline for patient involvement released  Annual Conferences, Focus Groups and Regional Workshops performed. Expert network established.

26. Get to know us! Web: www.patientsacademy.eu Twitter: @eupatients as well as:

27. Backup slides: Standard template slides