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DAY ONE – WEDNESDAY, JANUARY 30, 2019

7:45

Registration and Continental Breakfast

8:45

Chairman’s Welcome and Opening Remarks

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

JOINT PLENARY SESSIONS

9:00

The Future of Registries and Real World Evidence (RWE) —
Realizing Mutual Goals for Patients, Payers, Providers and Pharma

Jane Fang
MedImmune R&D IS Head for Clinical Biologics
AstraZeneca

9:45

Stanford-Inspire Case Study The Real World Online — Understanding and Using Patient-Reported, Real World Evidence from Social Media

Monica St. Claire
Product Lead, Insights Innovation
Inspire

10:30

Networking and Refreshment Break

11:00

Leveraging Big Data and Artificial Intelligence (AI) for Registries, RWE Generation and Personalized Decision Making

Richard Gliklich, MD
CEO
OM1

11:45

Build Common Data Models Across Multiple Data Sources

Omar Dabbous
Vice President, Global Health Economics and
Outcomes Research and Real World Evidence
AveXis, Inc.

12:30

Networking Luncheon

1:30

Celgene Case Study Utility of Real World Data (RWD) for Prospective Observational Studies —
Lessons Learned and Data Evaluations of Pilot Oncology “EHR Registries”

E. Dawn Flick, RPh, MPH, ScD
Senior Director, Registries and Real-World Data Collaborations, US Medical Affairs
Celgene Corporation

Susan Fish
Associate Director, Registries and Real-World Data Collaborations, US Medical Affairs
Celgene Corporation

2:15

UPDATE FROM AHRQ Defining and Standardizing Outcomes Measures for Better Registry Data Utilization

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

3:00

Networking and Refreshment Break

TRACK 1 PATIENT REGISTRIES

3:30

A Practical Guide for Launching an Effective Registry

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

4:15

Parent Project MD
Case Study
Key Considerations for a Sustainable Registry Model

Pat Furlong
Founding President & CEO
Parent Project Muscular Dystrophy

 
TRACK 2 REAL WORLD EVIDENCE

3:30

Enriching Drug Development in the Era of Real World Evidence

Christoph Koenen, MD
Head of Cardiovascular Development
Bristol-Myers Squibb

4:15

What Happens When You Dramatically Lower the Cost to Collect and Analyze the Data Required to Create RWE and Patient Registries? It Changes Everything.

Aaron Leibtag
CEO, Cofounder
Pentavere Research Group Inc.

5:00

Close of Day One

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

DAY TWO – THURSDAY, JANUARY 31, 2019

8:15

Continental Breakfast

8:45

Chairman’s Review of Day One

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

TRACK 1 PATIENT REGISTRIES

9:00

Tackle Operational and Organizational Complexities When Implementing Patient Registries

Ashley Winslow, PhD
Senior Director, Translational Research & Portfolio Development, Orphan Disease Center
University of Pennsylvania School of Medicine

9:45

Patient Centricity — Strategies for Incorporating Patient-Reported Outcomes

Vanessa Boulanger
Director of Research Programs
National Organization for Rare Disorders (NORD)

10:30

Networking and Refreshment Break

11:00

Case Study Update Experience of Planning and Conducting an Observational Registry in a Rare Disease
(MF-Cutaneous T-Cell Lymphoma)

Badri Rengarajan, MD
Medical Lead, Aspire Unit
Actelion

11:45

Case Study The Use of Registry Data to Support Quality Improvements and Meet Regulatory Requirements

Diane Alejo
Director, Information Technology
Johns Hopkins University School of Medicine

 
TRACK 2 REAL WORLD EVIDENCE

9:00

Utilizing Patient Experience Data to Characterize the Real World Clinical Reality, Measure Outcomes and Improve Decisions on Drug Discovery, Market Access and Value

Renee Willmon, MSc, PhD Candidate
Manager of Behavior Science
Self Care Catalysts Inc.

9:45

Biopharmaceutical Acceleration Model for Product Development and Commercialisation

David Thompson, PhD
Senior Vice President, Head of
Thought Leadership
Syneos Health, Real World & Late Phase

10:30

Networking and Refreshment Break

11:00

Develop a Strategy for Comprehensive Evidence Generation

Thomas A. Abbott
Head, Capabilities & Alliances
Astellas US LLC

11:45

The Promise of AI and Machine Learning for Real World Data Applications

Larry Pickett
CEO
RxDataScience

12:30

Networking Luncheon

JOINT PLENARY SESSIONS

1:30

Address Registry Obstacles Post-Launch

Hugh K. Findlay, RN, BS, CCRA
Associate Director, Regional Registry Site Operations
Sanofi Genzyme

2:15

Gain Insight into Using Real World Registry Data for Label Expansion

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

3:00

Chairman’s Wrap-Up

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

3:15

Close of Conference

Want more agenda details? Download the brochure.

DAY ONE – WEDNESDAY, JANUARY 30, 2019

7:45

Registration and Continental Breakfast

8:45

Chairman’s Welcome and Opening Remarks

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

JOINT PLENARY SESSIONS

9:00

The Future of Registries and Real World Evidence (RWE) —
Realizing Mutual Goals for Patients, Payers, Providers and Pharma

Jane Fang
MedImmune R&D IS Head for Clinical Biologics
AstraZeneca

9:45

Stanford-Inspire Case Study The Real World Online — Understanding and Using Patient-Reported, Real World Evidence from Social Media

Monica St. Claire
Product Lead, Insights Innovation
Inspire

10:30

Networking and Refreshment Break

11:00

Leveraging Big Data and Artificial Intelligence (AI) for Registries, RWE Generation and Personalized Decision Making

Richard Gliklich, MD
CEO
OM1

11:45

Build Common Data Models Across Multiple Data Sources

Omar Dabbous
Vice President, Global Health Economics and
Outcomes Research and Real World Evidence
AveXis, Inc.

12:30

Networking Luncheon

1:30

Celgene Case Study Utility of Real World Data (RWD) for Prospective Observational Studies —
Lessons Learned and Data Evaluations of Pilot Oncology “EHR Registries”

E. Dawn Flick, RPh, MPH, ScD
Senior Director, Registries and Real-World Data Collaborations, US Medical Affairs
Celgene Corporation

Susan Fish
Associate Director, Registries and Real-World Data Collaborations, US Medical Affairs
Celgene Corporation

2:15

UPDATE FROM AHRQ Defining and Standardizing Outcomes Measures for Better Registry Data Utilization

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

3:00

Networking and Refreshment Break

TRACK 1 PATIENT REGISTRIES

3:30

A Practical Guide for Launching an Effective Registry

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

4:15

Parent Project MD
Case Study
Key Considerations for a Sustainable Registry Model

Pat Furlong
Founding President & CEO
Parent Project Muscular Dystrophy

 
TRACK 2 REAL WORLD EVIDENCE

3:30

Enriching Drug Development in the Era of Real World Evidence

Christoph Koenen, MD
Head of Cardiovascular Development
Bristol-Myers Squibb

4:15

What Happens When You Dramatically Lower the Cost to Collect and Analyze the Data Required to Create RWE and Patient Registries? It Changes Everything.

Aaron Leibtag
CEO, Cofounder
Pentavere Research Group Inc.

5:00

Close of Day One

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

DAY TWO – THURSDAY, JANUARY 31, 2019

8:15

Continental Breakfast

8:45

Chairman’s Review of Day One

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

TRACK 1 PATIENT REGISTRIES

9:00

Tackle Operational and Organizational Complexities When Implementing Patient Registries

Ashley Winslow, PhD
Senior Director, Translational Research & Portfolio Development, Orphan Disease Center
University of Pennsylvania School of Medicine

9:45

Patient Centricity — Strategies for Incorporating Patient-Reported Outcomes

Vanessa Boulanger
Director of Research Programs
National Organization for Rare Disorders (NORD)

10:30

Networking and Refreshment Break

11:00

Case Study Update Experience of Planning and Conducting an Observational Registry in a Rare Disease
(MF-Cutaneous T-Cell Lymphoma)

Badri Rengarajan, MD
Medical Lead, Aspire Unit
Actelion

11:45

Case Study The Use of Registry Data to Support Quality Improvements and Meet Regulatory Requirements

Diane Alejo
Director, Information Technology
Johns Hopkins University School of Medicine

 
TRACK 2 REAL WORLD EVIDENCE

9:00

Utilizing Patient Experience Data to Characterize the Real World Clinical Reality, Measure Outcomes and Improve Decisions on Drug Discovery, Market Access and Value

Renee Willmon, MSc, PhD Candidate
Manager of Behavior Science
Self Care Catalysts Inc.

9:45

Biopharmaceutical Acceleration Model for Product Development and Commercialisation

David Thompson, PhD
Senior Vice President, Head of
Thought Leadership
Syneos Health, Real World & Late Phase

10:30

Networking and Refreshment Break

11:00

Develop a Strategy for Comprehensive Evidence Generation

Thomas A. Abbott
Head, Capabilities & Alliances
Astellas US LLC

11:45

The Promise of AI and Machine Learning for Real World Data Applications

Larry Pickett
CEO
RxDataScience

12:30

Networking Luncheon

JOINT PLENARY SESSIONS

1:30

Address Registry Obstacles Post-Launch

Hugh K. Findlay, RN, BS, CCRA
Associate Director, Regional Registry Site Operations
Sanofi Genzyme

2:15

Gain Insight into Using Real World Registry Data for Label Expansion

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

3:00

Chairman’s Wrap-Up

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

3:15

Close of Conference