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DAY ONETUESDAY, DECEMBER 5, 2017

7:30

Registration and Continental Breakfast

8:30

Chairmain’s Welcome and Opening Remarks

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

Developing Sustainable Registry Models — Understanding Global Regulatory Guidelines

8:45

Global Regulatory Perspectives on Registries for Clinical Development and
Post-Approval Assessment

J. Alexander Cole, DSc, MPH
Senior Director and Global Head, Epidemiology
Alexion Pharmaceuticals

9:30

Informing and Improving Care Using the Cystic Fibrosis Foundation Patient Registry

Aliza Fink, DSc
Director, Epidemiology
Cystic Fibrosis Foundation

10:15

Networking and Refreshment Break

10:45

Best Practices in Designing Sustainable Registry Models

Karen M. Anderson, PhD
Senior Director, Medical Affairs, Rare Genetic Diseases
Agios Pharmaceuticals

Christopher Jones, PhD
Co-founder & Partner
iTakeControl Health

11:30

PANEL
Promoting and Executing Sustainable Registry Models Through Collaborations Between Public, Private and Industry Partners

MODERATOR:

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

PANELISTS:

Christopher Scalchunes
Vice President, Research
Immune Deficiency Foundation

Elise Berliner, PhD
Director, Technology Program, Center for Outcomes and Research Evidence
AHRQ (Agency for Healthcare Research and Quality) (invited)

Megan Lynch O’Boyle
Principal Investigator
Phelan-McDermid Syndrome International Registry (PMSIR)

12:15

Networking Luncheon

Scaling the Benefits of Patient Registries — Harmonizing Multiple Data Sources and Promoting Collaboration

1:30

Harmonizing Outcomes Measures Across Registries to Promote the Validity and
Use of Registry Data

Elise Berliner, PhD
Director, Technology Program, Center for Outcomes and Research Evidence
AHRQ (Agency for Healthcare Research and Quality) (invited)

2:30

Integrating and Preparing Data for Patient Registries —
Solutions for Overcoming Common Hurdles

Gary Palgon, CISSP
Vice President, Healthcare and Life Sciences Solutions
Liaison Technologies, Inc.

3:15

Networking and Refreshment Break

3:45

The Role of Registries in Active Surveillance in Support of a Biologic Product in EU

Yun Gu, PhD
Director, Epidemiology, Worldwide Safety & Regulatory
Pfizer Inc

4:30

Evolving Rare Disease Registries — One Company’s Experience

Michael Keating, PharmD, RPh
Associate Medical Director
Janssen Pharmaceutical Company

5:00

Close of Day One

Networking, Wine and Cheese Reception immediately following the final session on day one

DAY TWO WEDNESDAY, DECEMBER 6, 2017

7:30

Continental Breakfast

8:15

Chairmain’s Review of Day One

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

New Models and Best Practices in
Registry Design and Launch

8:30

FIRESIDE CHAT
Getting It Right from the Get-Go — Best Practices for Starting a Registry

MODERATOR:

Christopher Jones, PhD
Co-founder & Partner
iTakeControl Health

PANELISTS:

Rezaul Khandker
Director, Health Outcomes
Merck

Michael Keating, PharmD, RPh
Associate Medical Director
Janssen Pharmaceutical Company

Christiane Denzel
Global Head, Rare Disease Registries
Sanofi Genzyme

9:15

PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism)
A Registry for Subjects with Chronic Hypoparathyroidism —
Implementing a Hub and Satellite Model to Overcome Study Start-Up, Communication and Long-Term Engagement Challenges

Nicole Robichaud
Associate Director, Real World Late Phase Studies
Shire

10:00

Networking and Refreshment Break

10:30

Registries’ Roles in Supporting Rare Disease Clinical Development Programs — State-of-the-Art and Future Trends

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

Ashley Winslow, PhD
Senior Director, Translational Research, Orphan Disease Center
University of Pennsylvania

11:15

PANEL
Rare Disease Registries — Expanding the Discussion

MODERATOR:

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

PANELSITS:

Ashley Winslow, PhD
Senior Director, Translational Research, Orphan Disease Center
University of Pennsylvania

Liz Kennerly
Patient and Patient Advocate
United Mitochondrial Disease Foundation

Nicole Robichaud
Associate Director, Real World Late Phase Studies
Shire

Christiane Denzel
Global Head, Rare Disease Registries
Sanofi Genzyme

12:00

Luncheon Roundtable Sessions
A Manage the Complexities Around Regulatory and Privacy Protection in Healthcare Registries
B Address Registry Obstacles Post-Launch
C Patient-Centric Registries — Best Practices for Incorporating Patient-Reported Outcomes

Driving Better Treatment Options —
Enabling PatientCentric Outcomes Data

1:15

Paving the Way for Meaningful Outcomes and Better Treatment —
Designing Patient-Centric Registry Models

Michelle Durborow
Senior Director, Scientific Operations
The Life Raft Group — a global patient group focused on ensuring the survival of gastrointestinal stromal tumors (GIST)

2:15

PI CONNECT — Reaping the Benefits of Combining Patient-Reported and
Clinical Data into One Registry

Tara Caulder
USIDNET Project Director
Immune Deficiency Foundation

Christopher Scalchunes
Vice President, Research
Immune Deficiency Foundation

3:00

Close of Conference

Want more agenda details? Download the brochure.

DAY ONETUESDAY, DECEMBER 5, 2017

7:30

Registration and Continental Breakfast

8:30

Chairmain’s Welcome and Opening Remarks

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

Developing Sustainable Registry Models — Understanding Global Regulatory Guidelines

8:45

Global Regulatory Perspectives on Registries for Clinical Development and
Post-Approval Assessment

J. Alexander Cole, DSc, MPH
Senior Director and Global Head, Epidemiology
Alexion Pharmaceuticals

9:30

Informing and Improving Care Using the Cystic Fibrosis Foundation Patient Registry

Aliza Fink, DSc
Director, Epidemiology
Cystic Fibrosis Foundation

10:15

Networking and Refreshment Break

10:45

Best Practices in Designing Sustainable Registry Models

Karen M. Anderson, PhD
Senior Director, Medical Affairs, Rare Genetic Diseases
Agios Pharmaceuticals

Christopher Jones, PhD
Co-founder & Partner
iTakeControl Health

11:30

PANEL
Promoting and Executing Sustainable Registry Models Through Collaborations Between Public, Private and Industry Partners

MODERATOR:

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

PANELISTS:

Christopher Scalchunes
Vice President, Research
Immune Deficiency Foundation

Elise Berliner, PhD
Director, Technology Program, Center for Outcomes and Research Evidence
AHRQ (Agency for Healthcare Research and Quality) (invited)

Megan Lynch O’Boyle
Principal Investigator
Phelan-McDermid Syndrome International Registry (PMSIR)

12:15

Networking Luncheon

Scaling the Benefits of Patient Registries — Harmonizing Multiple Data Sources and Promoting Collaboration

1:30

Harmonizing Outcomes Measures Across Registries to Promote the Validity and
Use of Registry Data

Elise Berliner, PhD
Director, Technology Program, Center for Outcomes and Research Evidence
AHRQ (Agency for Healthcare Research and Quality) (invited)

2:30

Integrating and Preparing Data for Patient Registries —
Solutions for Overcoming Common Hurdles

Gary Palgon, CISSP
Vice President, Healthcare and Life Sciences Solutions
Liaison Technologies, Inc.

3:15

Networking and Refreshment Break

3:45

The Role of Registries in Active Surveillance in Support of a Biologic Product in EU

Yun Gu, PhD
Director, Epidemiology, Worldwide Safety & Regulatory
Pfizer Inc

4:30

Evolving Rare Disease Registries — One Company’s Experience

Michael Keating, PharmD, RPh
Associate Medical Director
Janssen Pharmaceutical Company

5:00

Close of Day One

Networking, Wine and Cheese Reception immediately following the final session on day one

DAY TWO WEDNESDAY, DECEMBER 6, 2017

7:30

Continental Breakfast

8:15

Chairmain’s Review of Day One

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

New Models and Best Practices in
Registry Design and Launch

8:30

FIRESIDE CHAT
Getting It Right from the Get-Go — Best Practices for Starting a Registry

MODERATOR:

Christopher Jones, PhD
Co-founder & Partner
iTakeControl Health

PANELISTS:

Rezaul Khandker
Director, Health Outcomes
Merck

Michael Keating, PharmD, RPh
Associate Medical Director
Janssen Pharmaceutical Company

Christiane Denzel
Global Head, Rare Disease Registries
Sanofi Genzyme

9:15

PARADIGHM (Physicians Advancing Disease Knowledge in Hypoparathyroidism)
A Registry for Subjects with Chronic Hypoparathyroidism —
Implementing a Hub and Satellite Model to Overcome Study Start-Up, Communication and Long-Term Engagement Challenges

Nicole Robichaud
Associate Director, Real World Late Phase Studies
Shire

10:00

Networking and Refreshment Break

10:30

Registries’ Roles in Supporting Rare Disease Clinical Development Programs — State-of-the-Art and Future Trends

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

Ashley Winslow, PhD
Senior Director, Translational Research, Orphan Disease Center
University of Pennsylvania

11:15

PANEL
Rare Disease Registries — Expanding the Discussion

MODERATOR:

Joseph D. Giuliano
Senior Director, Global Medical Operations and Patient Registry
Amicus Therapeutics

PANELSITS:

Ashley Winslow, PhD
Senior Director, Translational Research, Orphan Disease Center
University of Pennsylvania

Liz Kennerly
Patient and Patient Advocate
United Mitochondrial Disease Foundation

Nicole Robichaud
Associate Director, Real World Late Phase Studies
Shire

Christiane Denzel
Global Head, Rare Disease Registries
Sanofi Genzyme

12:00

Luncheon Roundtable Sessions
A Manage the Complexities Around Regulatory and Privacy Protection in Healthcare Registries
B Address Registry Obstacles Post-Launch
C Patient-Centric Registries — Best Practices for Incorporating Patient-Reported Outcomes

Driving Better Treatment Options —
Enabling PatientCentric Outcomes Data

1:15

Paving the Way for Meaningful Outcomes and Better Treatment —
Designing Patient-Centric Registry Models

Michelle Durborow
Senior Director, Scientific Operations
The Life Raft Group — a global patient group focused on ensuring the survival of gastrointestinal stromal tumors (GIST)

2:15

PI CONNECT — Reaping the Benefits of Combining Patient-Reported and
Clinical Data into One Registry

Tara Caulder
USIDNET Project Director
Immune Deficiency Foundation

Christopher Scalchunes
Vice President, Research
Immune Deficiency Foundation

3:00

Close of Conference