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Rare Alliance Canada International Impact David King, PhD Shire March 9,2016

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Page 1: David King: Rare Disease Day 2016 Conference

Rare Alliance Canada International Impact

David King, PhD Shire March 9,2016

Page 2: David King: Rare Disease Day 2016 Conference

2

Players Involved

Health Economists

Academic Researchers

Industry

Regulatory Agencies

Payers

Rare Disease Patients

Policy Makers Clinical

Experts

Successful Rare Disease Treatment

Page 3: David King: Rare Disease Day 2016 Conference

3

Why do we need to talk?

Perspective

What is a meaningful endpoint?

Value

Coordination

Disease Understanding

Research Breakthroughs

Ensuring Access

Diagnosis

Page 4: David King: Rare Disease Day 2016 Conference

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

Page 5: David King: Rare Disease Day 2016 Conference

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

Page 6: David King: Rare Disease Day 2016 Conference

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

Page 7: David King: Rare Disease Day 2016 Conference

7

The End Game


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