CORD Rare Drug Conference: June 8-9, 2022 Session 4: Patient Engagement and Patient Empowerment

1. Rare One Canada:
Canada’s Smart Rare Disease and
Rare Drug Strategy
Patient Engagement and Patient
Empowerment
9-June-2022
Oxana Iliach, PhD

2. PATIENTS
Diagnostic
Access to
Treatment
Regulatory
and Health
Authorities
Academic
Research
Industry
Insurance
Companies
Health
Care
Providers
Additional
Services
Centers of
Excellence
Replace “Patient Journey” with “No Travel”

3. PATIENT ADVOCACY ROLES
HAVE CHANGED
Barbarian
at the Gates
Beggars at the
Table
Strange
Bedfellows
Everybody
Wants a Patient
Innmates are
Running the
Asylum

4. Promise of Innovative
Transformative
Therapies
Reality of Access
to Breakthrough
Medicines

5. Cut Costs
Fast Track R&D
Coordinated
Regulatory Review
Early Dialogue to
Determine Value
and Patient
Outcome Measures
Cooperative
Framework for Fair
Prices and
Sustainable
Healthcare Budgets
Engage Patients in
Continuum of
evidence generation
linked to healthcare
outcomes
3
Structured Cooperation between Developers, Payers & Patients

6. PATIENT PARTNER:
UNMET NEEDS TO OPTIMAL USE

7. CORD’s Strategy from 2015 and Ontario 2017
1

8. Quebec’s strategy
• A definition of rare diseases – a condition affecting 1 in 2,000 people
• A vision – Optimize access to quality health care for rare disease
patients, and
• Guiding principles:
1. Taking a patient and family-centred approach
2. Improving health equity
3. Improving access to care and services
4. Sustaining care and services and
5. Learning approach
• Focused on the three following themes:
1. Awareness building and training
2. Ensuring equitable access to diagnosis and care and
3. Promoting research, innovation and data collection
2

9. 3

10. 4

11. 5
effective drugs and better health outcomes
Preliminary underlying principles to
help guide the strategy:
• Patient-centered
• Transparent and accountable
• System alignment and sustainability
• Ethical
• Efficient and effective
• Evidence-informed
• Collaborative and inclusive
• Adaptive
NOW “STRATEGIC PILLARS”
Improve access to RD
treatments and make it
consistent across Canada
Optimize, collect, and use
evidence that meets the needs
of decision-makers along the
pharmaceutical management
continuum and across the
lifecycle of the drug
Support optimal patient
outcomes and sustainability
of the Cdn health care
system by ensuring
spending on drugs for rare
diseases brings value for
money
Strengthen alignment
of research and
innovation systems
with drugs for RDs
access objectives
• Adopt common
vision/commitment for the
strategy
• Enhance coordination and
shared decision-making for the
strategy
• Develop data standards to enable
collection of data and knowledge
of RDs and effective treatments
• Leverage / analyze health data,
including RWE, to improve
ongoing decision-making
• Streamline process and
efforts for DRD
• Promote risk-sharing and
equitable sharing of costs
among payers
• Explore innovative drug
reimbursement models
• Support research by
building on Canada’s RD
research capacity
• Support access to data
that can spur clinical trials
Notable differences/changes:
• More focus on “effectiveness” e.g.,
the VISION and “value-for-money”
• Previous version included an
objective to “invest in innovation”
including a sub—tactic to “support
the life sciences sector”; this
language has been watered down in
Pillar 4

12. www.Canada4Rare.ca