Day 1: RD 2022 Fall Conference Clinical Networks of Excellence: Co-Chairs: Leanne Ward, CHEO; Craig Campbell, London Health Sciences; Francois Bernier, Alberta Health Services

1. TRANSFORMING VISION TO REALITY
SMART Investment in Rare Disease Infrastructure to Optimize Diagnosis,
Prescribing, and Life-long Treatment Management
CLINICAL CENTRES/NETWORKS OF EXCELLENCE
CHAIRS: FRANCOIS BERNIER, CRAIG CAMPBELL, LEANNE WARD
DAY 1

2. CLINICAL CENTRES/NETWORKS OF EXCELLENCE
CHAIRS: FRANCOIS BERNIER, CRAIG CAMPBELL, LEANNE WARD
PANELISTS:
DR. ANGELA GENGE, THE NEURO
DR. THIERRY LACASE, MICYRN
DR. LAURA MCADAM, HOLLAND BLOORVIEW
SMART Investment in Rare Disease Infrastructure to Optimize
Diagnosis, Prescribing, and Life-long Treatment Management

3. OBJECTIVES
• Defining the roles of the Canadian Organization for Rare Disorders (CORD) and
Maternal Infant Children and Youth Research Network (MICYRN)
• Agreeing on Canadian criteria for Centers of Excellence and Allied Centres of
Distinction, adapted from international best practices (WHO model, NORD COEs)
• Developing Terms of Reference for Rare Disease Specialty Networks Groups
(based on European Reference Networks)
• Developing Proposal for Rare Disease Community Networks (Family Practitioners
and Allied Professionals)
• Developing Proposal for Rare Disease Nurses/Genetic Counsellors Network
• Agreeing on and implementing procedures for application, review and
designation
• Identifying and requesting resource requirements: Human resources, facilities,
technology, management, financial

4. OUTLINE
PANELISTS
15 MIN
DR. LEANNE WARD TO
MODERATE
STAGE SETTING
5 MIN
DR. CRAIG CAMPBELL
GROUP WORK
THREE QUESTIONS
30 MIN
DR. FRANCOIS BERNIER

5. PANELISTS
Dr. Angela Genge
• Executive Director of
the Neuro Clinical
Research Unit
• Montreal
Neurological Institute
• Director of ALS Clinic
Dr. Thierry Lacaze
• Scientific Director of
MICYRN
• Head of Neonatology,
Cumming School
Medicine Calgary
• PI of CHEER Initiative
Dr. Laura McAdam
• Physician Director of
Child Development
Holland Bloorview
Kids Rehabilitation
Hospital
• Lead for NM and Rett
Syndrome clinics

6. RATIONALE AND VISION
• VARIOUS RARE DISEASE NETWORKS AROUND THE WORLD
• EVOLVE FROM THE LANDSCAPE IN WHICH THEY FUNCTION, AND FROM
THE UNIQUE OBJECTIVES THEY HAVE SET FOR THEIR COUNTRY/REGION
• CENTRES OF EXCELLENCE AND NETWORKS HAVE VARIOUS COMPONENTS
• AWARENESS AND ADVOCACY
• PATIENT JOURNEY
• CLINICAL CARE
• RESEARCHEX
• EDUCATION
• LEARNING FROM OTHER NETWORKS IS KEY BUT CANADA REQUIRES A
NOVEL APPROACH - WHY?
• EXPERTISE EXISTS, STARTING POINT, GEOGRAPHY, HEALTH SYSTEM

7. RATIONALE AND VISION
• AWARENESS AND ADVOCACY
• WITH ABSENCE OF A NATIONAL RARE DISEASE STRATEGY THIS IS CRITICAL FOR
CANADA
• PATIENT JOURNEY
• CLINICAL CARE GUIDELINES, CASE CONFERENCES, NAVIGATION, DECISION MAKING
SUPPORT
• CLINICAL CARE
• DIAGNOSTICS, RARE DISEASE REGISTRIES, SMOOTHING REGULATORY PATHWAYS,
COLLECTING METRICS
• RESEARCH
• GRANTS, INNOVATION, MEASURING OUTCOMES
• EDUCATION
• TRAINING VARIOUS HCP TO ADDRESS RARE DISEASE ISSUES, PROVIDE CONFIDENCE
TO NAVIGATE FOR PATIENTS IN PARTNERSHIP WITH TERTIARY CARE CENTRES

8. QUESTION 1
• ASSUMING WE ALL AGREE THAT THE RATIONALE IS SOUND FOR A RARE DISEASE
NETWORK, AND THE VISION INCLUDES ELEMENTS DISCUSSED…
• THEN KNOWING THE CANADIAN CONTEXT WHAT IS/ARE THE KEY
ELEMENTS OF A NETWORK OF RARE DISEASE CENTRES OF
EXCELLENCE?
• AWARENESS AND ADVOCACY
• PATIENT JOURNEY ENABLER
• CLINICAL CARE ENHANCER
• DRIVE RESEARCH
• EDUCATION FUNCTION
• OTHER?

9. RARE DISEASE CENTRES/NETWORKS OF EXCELLENCE
DIFFERENT MODEL SYSTEMS HAVE BEEN BUILT TO CREATE RARE
DISEASE CENTRES/NETWORKS OF EXCELLENCE
• NETWORK MODEL (ERN)
• CENTRE OF EXCELLENCE MODEL – FOCUSED ON INDIVIDUAL CENTRES
(NORD)
• FORMAL (ERN) OR INFORMAL (WHO )
• SPOKE AND HUB MODEL

10. QUESTION 2
•WHAT IS THE BEST MODEL FOR CANADIAN CENTRE
OF RARE DISEASE?
• NETWORK MODEL VS. CENTRE OF EXCELLENCE MODEL – FOCUSED ON
INDIVIDUAL CENTRES?
• FORMAL OR INFORMAL GOVERNANCE?
• SPOKE AND HUB MODEL?
•WHAT WOULD BE THE MAIN CRITERIA FOR BEING A
CENTRE/NETWORK OF EXCELLENCE?

11. PROOF OF CONCEPT PLAN
KNOWING THAT THE GOVERNMENT HAS PUT MONETARY COMMITMENT
FOR DRUGS FOR RARE DISEASE
….AND THAT CORD AND STAKEHOLDERS HAVE IMPRESSED THE NEED FOR
INFRASTRUCTURE RATHER THEN JUST DRUG COVERAGE
….AND THAT CENTRES OF EXCELLENCE WOULD BE A VITAL PART OF THAT
INFRASTRUCTURE
• DOES IT MAKE SENSE TO PROPOSE A LAUNCH TEAM TO CREATE A
CONCRETE PLAN FOR CENTRES/NETWORKS OF EXCELLENCE?
• WHAT IS THE FEASIBILITY OF A LAUNCH TEAM TO START US OFF?
• WHO WOULD BE ON THAT LAUNCH TEAM?
• STEERING COMMITTEE, PROJECT MANAGER, EDUCATION LEAD, PATIENT
NAVIGATOR LEAD, RESEARCH LEAD

12. QUESTION 3.
•WHAT IS THE FEASIBILITY OF A LAUNCH TEAM TO
START US OFF?
• IN ORDER THAT GOVERNMENT COULD NOT RESIST
FUNDING/REOURCING A LONGER TERM EFFORT WHAT WOULD
BE THE THREE MAIN 6-12 MONTH DELIVERABLES OF
A LAUNCH TEAM?
• EG. SETTING CRITERIA FOR A COE, ASSET MAP OF KEY INFRASTRUCTURE EG.
REGISTRIES, EDUCATION MODEL, PATIENT NEEDS ASSESSMENT

13. QUESTIONS
1. WHAT IS/ARE THE KEY ELEMENTS OF A NETWORK OF
RARE DISEASE CENTRES OF EXCELLENCE?
2. WHAT IS THE BEST MODEL FOR CANADIAN CENTRE OF
RARE DISEASE?
• WHAT WOULD BE THE MAIN CRITERIA FOR BEING A CENTRE OR NETWORK OF
EXCELLENCE?
3. WHAT WOULD BE THE THREE MAIN 6-12 MONTH
DELIVERABLES OF A LAUNCH TEAM?

14. THANK YOU!