EUROPEAN REFERENCE NETWORKS Matt Bolz-Johnson, EURORDIS Rare - - PowerPoint PPT Presentation

EUROPEAN REFERENCE NETWORKS

Rare Disease Ireland, Dublin, Ireland 23 October 2018

Matt Bolz-Johnson,

EURORDIS

2

Challenges we face as a community

Rare diseases and rare cancers can be chronic, progressive, degenerative, disabling and frequently life-threatening:

• Patients and experts are few, geographically scattered and often isolated
• Reliable information is scarce
• Resources are costly and access limited
• Lack of treatments and challenges to access adequate care
• Fragmented research, data and information
• Poor outcomes and in some cases reduced life expectancy

300

No one country, no one continent, can solve alone the problems posed by rare diseases

DIAGNOSTIC JOURNEY

25% wait from early symptoms to diagnosis 40% wrong diagnosis + inappropriate treatment Travel to different region

• r (2%) another country

50% received genetic counselling

<30yrs 40% 25% <Half

https://www.eurordis.org/IMG/pdf/Fact_Sheet_Eurordiscare2.pdf

Rare Bone - Rare Cancer - Peadatric Cancer - Rare Cardiac - Rare Connective Tissue - Rare Craniofacial & ENT - Rare Endocrinology - Rare & Complex Epilepsy - Rare Eye - Rare Gastrointestinal Diseases - Rare Hematology - Rare Hepatic Diseases - Rare Immunodeficiency, Auto Inflammatory & Autoimmune Disease - Genetic Tumours - Rare Malformations & Development Anomalies - Rare Hereditary Metabolic Diseases - Rare Neurology - Rare Neuromuscular - Rare Pulmonary Diseases - Rare & Undiagnosed Skin Disorders - Rare Renal - Transplantation in children - Rare Urogenital Diseases - Rare Multi-systemic Vascular Diseases -

Success of Patient + Clinical Partnership

26/10/2018

5

Patient representatives were co-founders, co-authors, co-chairs and equal partners in making ERNs a reality for all !!!

• Groundswell of collaboration in the rare disease

community:

• 24 European Reference Networks (ERNs) linking >300

hospitals and 900 expert centres

• 24 European Patient Advocacy Groups (ePAG) of >300

patient organsations & <1000 ePAG member

• rganisations

European Patient Advocacy Groups are:

• Forums to optimise involvement of patients
• Aligned with RD ERN scope
• Patients are voting Members of all ERN Boards & >150

Disease Specific Network Committees

6

European Patient Advocates

• 24 European Patient Advocacy Groups (ePAG)
• >250 patient organsations
• <1700 ePAG member organisations

7 Networks harness the collective knowledge and experience of experts, focusing on a common goal to drive improve access to diagnosis and treatment by providing high-quality healthcare to patients.

ERN Spearhead EU Expertise

→ Connect expert, anchored into national health systems → Creating a critical mass of cases and data → Inclusive, not exclusive, multi-professional, not single professional → Common language, common platform enabling mobility of expertise → Collaborative spirit, trust and sharing with a common goal → Culture of learning → Increase patient outcomes and survival

European Reference Network

Specialist Treatment Specialist Advice

• Virtual healthcare:

specialist advice

• Knowledge generation:

sharing experience and expertise, research and innovation

• Knowledge

dissemination: clinical guidelines, healthcare pathways, education and training Patients RD Specific ERN National Healthcare Provider

Note: Slide inspired from Dr Enrique Terol, DG SANTE

9

24 Approved ERNs : Our Optimal Structure

European Reference Networks European Reference Networks Rare Bone Diseases Rare Immunodeficiency, Auto Inflammatory & Autoimmune Disease Adult Cancer Genetic Tumours Peadatric Cancer Rare Malformations & Development Anomalies Rare Cardiac Rare Hereditary Metabolic Diseases Rare Connective Tissue Rare Neurology Rare Craniofacial & ENT Rare Neuromuscular Rare Endocrinology Rare Pulmonary Diseases Rare & Complex Epilepsy Rare & Undiagnosed Skin Disorders Rare Eye Rare Renal Rare Gastrointestinal Diseases Transplantation in children Rare Hematology Rare Urogenital Diseases Rare Hepatic Diseases Rare Multi-systemic Vascular Diseases

10

Ireland: 1 Hospitals in 3 ERNs

European Reference Networks European Reference Networks Rare Bone Diseases Rare Immunodeficiency, Auto Inflammatory & Autoimmune Disease Adult Cancer Genetic Tumours Peadatric Cancer Rare Malformations & Development Anomalies Rare Cardiac Rare Hereditary Metabolic Diseases Rare Connective Tissue Rare Neurology Rare Craniofacial & ENT Rare Neuromuscular Rare Endocrinology Rare Pulmonary Diseases Rare & Complex Epilepsy Rare & Undiagnosed Skin Disorders Rare Eye Rare Renal Rare Gastrointestinal Diseases Transplantation in children Rare Hematology Rare Urogenital Diseases Rare Hepatic Diseases Rare Multi-systemic Vascular Diseases

Useful websites

http://ec.europa.eu/health/ern/policy_en

European Commission ERN webpage RD Action (Joint Action for Rare Diseases)

http://www.rd-action.eu/european- reference-networks-erns/coordination-

• f-rare-disease-erns/

UK HCP Members in ERNs

24 ERNs connecting 900 HCP Members in 300 Hospitals, across 26 Member States:

• 70% ERN HCP Members from

5 MS: France, Italy, Germany, The Netherlands and the United Kingdom

• 10% (c. 100) ERN HCP

Members from 40 UK Hospitals

• 23/24 ERNs include UK ERN

HCP Members

• 6/24 ERNs are lead by UK

HCP Members

EURORDIS Statement on ERN Post-Brexit

EURORDIS Calls for UK HCP Members to continue to be Members in ERNs. Securing strong mutual benefit for both European Union and United Kingdom, specifically:

• EU and UK patients benefiting from the

pooling of scarce expertise in rare and complex diseases under European Reference Networks;

• Creating the critical mass of expertise,

patients and their data in ‘ready-made communities’ attracting investment in the EU & UK market, accelerate research and therapeutic innovation development;

• Securing safe and sustainable supra-

specialised workforce through ERNs cross-border training and education activities.

15

Structure cross-border cooperation is needed to ensure that patients have access to the diagnosis, care and therapies they need and deserve:

• Each country individually has a low cohort of patients

and their data, making it’s impossible to conduct clinical trials.

• Pan-EU population allows the critical mass of expertise,

patients and their data in ‘ready-made communities’

• Offer us the opportunity be the integrated research

infrastructure that will optimise research and clinical trials

• Attracting investment in the EU & UK as a world-leading

market for innovation and research.

• Accelerating research and therapeutic innovation

development in the EU.

Research & Registries

Rare diseases represent an area with high European added value, cooperation must be enhanced in a strategic and structured way.

ERN Stage of Development

ERN Implementation (2015/17) ERN Deployment (2018/20) ERN Consolidation (2020/22)

ERNs Deployment (2018-2020): Building Infrastructure & Interoperability

Build Network Governance & Core Networks Raise Awareness of ERNs & Referrals Universal Coverage: Identification of experts to apply to join ALL ERNs Virtual Healthcare ERN & NHS Integration: National Networks & Adopt ERN Referral Pathways National Coordinating Hub linked to All ERNs Sustainable Funding: MS support of MFF2020-25

Types of ERN Membership

European Reference Network

HCP Members

National Coordination Hub

Associated National Centre

• MS Competency through a

designation process

• Clinicians, hospitals, laboratories, …
• No formal definition
• No defined designation process in

MSs

• ‘Endorsed’ by their MS under

their legislation

• European Commission

Competency

• Complete a full application and

independent European assessment

• Role explicitly laid out in the EC

ERN Delegated Acts

• MS Competency through a

designation process

• Coordination and information

role

• No formal definition
• No defined designation process

in MSs

Full HCP Member: Open to all expert centres that meets the specific criteria for expertise in a specific RD Affiliated Partners: Member State with without a Member of a Network may decide to designate healthcare providers with a special link to a given Network.

Operational Criteria

Organisation of the Operational Criteria Networks Healthcare Providers

General Criteria and Conditions to be fulfilled: 1. Highly Specialised Healthcare 2. Governance and Coordination 3. Patient Care 4. Multidisciplinary Approach 5. Good Practice, Outcome Measures, and Quality Control 6. Contribution to Research 7. Continuous Education, Training, and Development 8. Networking and Collaboration General Criteria and Conditions to be fulfilled: 1. Patient Empowerment and Patient-Centred Care 2. Organisation, Management, and Business Continuity 3. Research, Education and Training 4. Expertise, Information Systems, and e- Health Tools 5. Quality and Safety Specific Criteria and Conditions to be fulfilled: 1. Competence, Experience and Outcomes of Care 2. Human Resources 3. Organisation of Patient Care 4. Facilities and Equipment

Defined in the Network proposal and fulfillment assessed for each applicant healthcare provider. Based on the evidence and consensus of the scientific, technical and professional community

Call for New Members

• f Existing ERNs

Stage Activity Expected Date Preparation

• ERN NC mapping exercise of gaps in expertise and

geography Summer - Autumn

• EC amendment to the ERN Implementation

Decision End 2018 Pre-application

• ERN NC advise to BoMS

Winter 2018 Application Call

• Call for New HCP Members

Summer 2019

• Application developed and validated by member

states 3 months process Assessment

• Eligibility checking of the application by EC

Q3 2019

• Technical assessment by Independent Assessment

Body Q4 2019

• Final ERN BoMS approval

Spring 2020

Application Process

ERN Application Form:

• Expression of interest

to the ERN Coordinator!

• Member State

Endorsement Letter

• Complete Application

to be HCP Member of a Specific ERN

Self-Assessment Form:

• Against General

Criteria for HCP Member

• Against Specific

Criteria for ERN set by each ERN (clinical competencies)

• Evidence to be

collected

EC Assessment Stages:

• Self-Assessment with sign
• ff from ERN Coordinator
• European Commission

Application Check

• Independent Assessment

Body (IAB) to complete 1. Documentation Review Check

• IAB complete an On-Site

Visit.

Application Stage = 3 months Assessment Stage = 3 months

Delivering benefits for all

• Patients: ERNs should be a major equalising force & should spread best practices
• Hospitals: European Reference Networks (and their HCP Members) are:
• Legally recognised as expert centres and formalising networking structures/practices,

national coverage and population catchment area

• Enabling safe and sustainable workforce & services provision. Build expert capacity,

service competency, securing sustainability and investment.

• Regulators, payers, HTA bodies: unprecedented access to expertise and experts
• Industry: ERNs are ready-made expert communities, sitting at interplay of ‘care’ & ‘research’

1. Build on European wide collaboration with nearly 1000 HCP from 26 Member States in 24 ERNs 2. Connecting centres enabling peer learning through centralisation of complex cases 3. Creating a critical mass of patients and data, turning data into knowledge banks to push the pace of research and clinical practice 4. Magnetising patients needs to the right expert, leading to faster diagnosis and treatment 5. Transparency on patient health outcomes from care received as a driver for clinical excellence 6. Increased evidence and adherence to clinical guidelines 7. Increased access to better therapies, with faster development of new therapies for ALL RD

Our unmissable opportunity

Our hope is that 30 million lives affected by rare diseases will be improved through:

National Action in Ireland

Raise awareness and communication of ERN and hospital being a HCP Member Develop information about ERNs for the Ireland patient community Identification of experts / hospitals to apply to join ERNs either as Full or Affiliated Centres. Collaborate with Ireland’s ERN Board of Member State representative to ensure representation in all ERNs Establish collaborative partnership working with ERN HCP Members Actively develop national networks and promote links with local hospitals into ERNs Identify and endorse a hospital / unit to be ‘National Coordinationg Hubs’ into all ERNs

go raibh maith agat