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Rare Alliance Canada
International Impact
David King, PhD
Shire
March 9,2016
2
Players Involved
Health
Economists
Academic
Researchers
Industry
Regulatory
Agencies
Payers
Rare Disease
Patients
Policy
MakersClinical
Experts
Successful Rare
Disease Treatment
3
Why do we need to talk?
Perspective
What is a meaningful
endpoint?
Value
Coordination
Disease
Understanding
Research
Breakthroughs
Ensuring
Access
Diagnosis
4
IRDiC
International Rare
Disease Research
Consortium
Working to bring
200 new rare
disease treatments
by 2020
International with a
long history of
Canadian
involvement
Adapt-Smart
Private-public
consortium
Address drivers,
opportunities and
obstacles to
adaptive pathways
for earlier access
Green Park
Collaborative
Multi-stakeholder
forum looking at
ways to incorporate
real world data into
development
EUPATI
IMI initiative
involving 33
institutions
Educate patients on
drug development
and registration
Existing Collaborations
5
•  Brings all players to the table
•  Work towards a common
understanding
•  Build a sustainable solution
•  Regulatory framework that
provides a common reference
point
Benefits For Canada
6
•  A single integrated
message
•  Highlights the scientific
expertise found in
Canada
•  Brings Canadian biotech
to the forefront
•  Provides a potential
roadmap for other
countries
International
7
The End Game

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