the patients perspective and role of epags

Transcription

1 Patients Registry in ERNs: the patients perspective and role of epags 5th International Summer School Rare Disease & Orphan Drug Registries VirginieBros-Facer, Research Infrastructure Project Manager 18 September 2017, ISS, Rome

2 The European Organisation for Rare Diseases, EURORDIS, is a patient-driven alliance of patient organisations and individuals active in the field of rare diseases. Our vision is to enable better lives and cures for people living with a rare disease. Our mission is to work across borders and diseases to improve the livesof people living with a rare disease 2 2

3 738rare disease patient organisations in 65 countries covering over 4000 diseases. 38 National Alliances of RD Patients Organisations 55 European Federations of specific rare diseases Outreach to over 1800 patient groups 35 staff, offices in Paris, Brussels and Barcelona with also Geneva, Zagreb, Belgrade Over 320 Volunteers: +80 patient advocates and +250 moderators 3 3

4 Advocating for patients Health Policy & Healthcare services Medicines & Therapies Patient Empowerment and training Information & Networking Research Policy & Actions 4 4

5 RD Patient registries: an advocacy priority since 2006 EURORDIS-NORD-CORD Joint Declaration of 10 key principles for RD patients registries (2012) Contribution to the EUCERD Core Recommendations on RD Patient Registration and Data Collection (2013) Contribution to the CEG-RD Recommendation on Ways to Improve Codification for RD in Health Information Systems (2014) EPIRARE Patient Survey (2012/2013) Partner in RD Connect ( ): chairing the Patient Advisory Council, bringing the patients perspective to the project activities including WP2 (registries) Partner in RD-Action 5

6 Patient representation and involvement in 24 ERNs

7 Patient-centred& Empowerment European Reference Networks (ERNs) created on founding principles of patient created on founding principles of patient- centred care, patient advocate empowerment, patient engagement European Patient Advisory Groups (epag): 24 forums for dialogue, unity & solidarity to optimise involvement of patients Represent patients and engage in governance of RD ERNs Open to members & non-member patient groups in EU Aligned with RD ERN scope Composed of +>150 elected epag representatives & <1000 epag member organisations Democratically established & progressively expanded with agreed Terms of Reference 7

8 Common Objectives of epags Ensure care is patient-centred & respects patients rights & choice Ensure transparencyin quality of care, safety standards, clinical outcomes & treatment options Ensure ethical issues for patients are addressed, balancing patient & clinical needs Contribute to the development of patient information, policy, good practice, care pathways& guidelines Advise on planning, monitoring& evaluation of ERN initiatives 8

9 epagrepresentativesin the governance structure of ERNs Different approaches to engage patients as formal members in ERNs: epag representatives can be formal voting members and co-chairs of: oern Boards & committees: oscientific Advisory Board, Steering Committee and Management Committee oclinical Committees (single disease or sub-group of diseases) supporting ERN Boards oparent/patient Advocacy Committee/Board otransversal Focus Working groups 9

10 European Patient Advocacy Group Representatives European Reference Network No. epagreps No. Disease Specific Networks No.DSN without epag Rep Rare Bone ERN Adult Cancer ERN Paediatric Cancer ERN* Rare Cardiac ERN Rare Connective Tissue ERN Craniofacial & ENT Rare Endocrine ERN Rare Epilepsies ERN Rare Eye ERN Rare Gastrointestinal ERN Genetic Tumour ERN Rare Haematological ERN Rare Immunodeficiency, Autoinflammatory and Autoimmune ERN

11 European Patient Advocacy Group Representatives European Reference Network No. epag Reps No. Disease Specific Networks No.DSN without epag Rep Rare Kidney ERN Rare Liver ERN Rare Malformations ERN 7 3 Rare Metabolic ERN 7 7 Rare Neurology ERN Rare Neuromuscular ERN 7 5 Rare Pulmonary ERN Rare Skin ERN Transplantation ERN Rare Urogenital ERN Rare Vascular ERN Total

12 ERN Governance Structure Overarching ERN Disease specific networks Group Number epag Representives Network Boards per Network Board Clinical committees 115 >150 (and growing) Gaps - 21 Network Boards: Patient representatives are voting members of ERN Network Boards and co-chairs of committees and working groups. Patient Advisory Council are being established for each ERN Thematic topics: In process of identifying patient representatives for the ERN transversal working groups for: 1. Research 2. ehealth and Registries 3. Education & Training 4. Quality, Outcomes & Guidelines 12

13 Cross-ERN Research & Cross-ERN Research & Registries SUMMARY

14 ERN Clinical Research Activities The main goals of the ERNs regarding Clinical Research include: Fostering Clinical Trials to enable the development of therapies Promoting translational research activities to enable successful progression from bench to bedside 14

15 ERN Registry Activities More specifically regarding registries, many ERNs plan to: Natural history & Epidemiol ogical Studies Quality Data Collection Extend national registries into European patient registries Develop quality data collection in patient registries Promote the development of European patient registries using existing national registries for specific conditions Foster natural history and epidemiological studies 15

16 Priority Research Activities The first activities of the networks to enable cross-border research will include: 1. Identifying the research needs of the network and develop a strategic research plan highlighting research priority areas 2. Mapping and contributing to the development of existing research infrastructures (databases, registries, biobanks relevant to the disease areas covered by the Networks) 3. Supporting core networks with access to open source registry software and support tools (including interoperability tools) 16

17 Priority Research Activities (continued) 4. Setting up European/Network database for designing clinical and translational research studies (building on existing databases and registries) 5. Developing a framework for public private partnerships with regards to sponsoring clinical trials 6. Promoting innovative methodologies and designs for clinical trials 7. Facilitating sharing of data, knowledge, standardised protocols and technologies across disciplines and stakeholders 17

18 Roles of patients and patient organisations Leaders Drivers Partners Governance role, selection of outcome measures (Co-funding role/experience) Advisors Steering committee, advisory committee, methodology, assessment panel (eligibility criteria), input in study design, privacy/ethical issues, PROMs Participants PROMs 18

19 Capacity building to support epagsin their roles

20 epag Leadership Programme Webinar Capacity buidling Transversal Focus Groups Across ERNs Peer Coaching epagfocused Mentoring Programme individual focus Training courses 1. Virtual Healthcare; 2. Data Sharing; 3. Outcomes & Indicators; 4. Clinical Guidelines; 5. ERN & OrphanDrug Development 1. ERN Education & Training FG 2. ERN Research & Registries FG 3. ERN Outcomes& Guidelines FG Quarterly epag Coaching Groups with clinical lead Mentoring Programme: Pilot with 12 mentors 1. Presenting with Impact training; 2. Influencing without authority training 20

21 Takehome messages Patients have a clear vision of the added value and benefits of a comprehensive European approach to Rare Disease Registries Patients demand that the legal aspects regarding registries should be regulated at EU level Need to ensure a coherent transversal structure and governance across ERNs for Task force/wg on registries and research Patients empowerment and capacity building are needed for adequate and full involvement of patient representatives in the governance and activities of registries Registries should include data directly reported by patients along with data reported by healthcare professionals. 21 Need to ensure that all ERNs include & increase patient rep in Task Force/WP on registries and research

22 Thank you for your attention. Virginie Bros-Facer, PhD Research Infrastructure Project Manager Tel: virginie.bros-facer@eurordis.org