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Day One-October 17, 2016

7:00

Conference Registration and Continental Breakfast

8:00

NORD’s Welcome & Opening Remarks
Peter L. Saltonstall
President and CEO, NORD

8:15

Patient Keynote Address
Kristen Gray
Co-Founder, CureBatten.org

9:00

Exploring Frontiers-Telemedicine and Rare Diseases
David Flannery, MD, FACMG, FAAP
Medical Director,
American College of Medical Genetics

9:30

Keynote Address
Robert Califf, MD
Commissioner,
FDA

10:15

Networking and Refreshment Break

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11:00

Patient Advances through Genetic Innovation

Moderator:
Nora Yang, PhD MBA
Director, Portfolio Management and Strategic Operations, Therapeutics for Rare and Neglected Diseases (TRND), NCATS, NIH

Panelists:
Jane Juusola, PhD, FACMG
Director, Whole Exome Sequencing Program, GeneDx

John Leonard, MD
Chief Medical Officer, Intellia Therapeutics

Jeffrey Marrazzo
Co-Founder and Chief Executive Officer, Spark Therapeutics

Celia Witten, MD, PhD
Director of Office of Cellular, Tissue and Gene Therapy, CBER, FDA 

12:15

Lunch and Learn Breakout Roundtables
*download the agenda for details on roundtable discussions*

Sponsored by:pfizer

1:30

Choose Between Three Breakout Sessions
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2:45

Networking and Refreshment Break

3:30

The Challenge of Access and Reimbursement – Rising Concerns Regarding Affordability, Innovation and Quality of Care

Moderator:
Catherine Blansfield, MA, BS, RN
Vice President of Access and Outcomes,
NORD

Panelists:
Lori Reilly
Executive Vice President, Policy & Research, PhRMA

Loreen M. Brown, MSW
Senior Vice President, Product, Strategy and Commercialization Excellence,
Lash Group, a part of AmerisourceBergen

Karen L. Erickson
Associate Executive Director, Community Engagement, The Alpha-1 Foundation

Peter Dehnel
Medical Director, Blue Cross and Blue Shield of Minnesota

4:30

The Landscape for Investment

Moderator:
Jim Geraghty
Entrepreneur-In-Residence,
Third Rock Ventures

Panelists:
Ted W. Love, MD
Chief Executive Officer,
Global Blood Therapeutics

Peter Kolchinsky
Managing Director and Portfolio Manager,
RA Capital Management, LLC

Amanda L. Hayward
Independent

5:30

Close of Day One
Networking Cocktail Reception to follow

Day One Breakout Sessions

A

The Crucial Role of Data in Advancing Diagnosis and Clinical Drug Development

B

Collaborations Across Borders-Addressing Rare Diseases as a Global Public Health Challenge

C

Focus on Pediatric Diseases-Advancing Research and Treatments

Day Two-October 18, 2016

7:30

Continental Breakfast Opens

8:00

Day Two Insights and Keynote Introduction
Peter L. Saltonstall
President and CEO, NORD

8:10

Update from NORD Board of Directors
Marshall Summar
Division Chief, Genetics and Metabolism, Children’s National Health System

 

8:20

National Election Implications Keynote Address
Kate Rawson
Senior Editor, Prevision Policy

9:00

Driving Progress Through Policy

Moderator: 
Martha Rinker
Vice President of Public Policy,
NORD

Panelists:
Tim Boyd
Associate Director of State Policy, NORD 

Paul Melmeyer
Associate Director of Public Policy, NORD 

 

10:00

Networking and Refreshment Break

10:45

Choose Between Three Tracks
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1:00

Networking Luncheon

2:15

NORD and Trio Health Partnership to Improve Quality of Care and Outcomes

Speakers:
Pamela K. Gavin
Chief Operating Officer,
NORD

Brent Clough
CEO,
Trio

3:00

Predicting the Pipeline – Orphan Product Development and Progress in 2017

Moderator:
Philip J. Brooks, PhD
Program Director, Division of Clinical Innovation, NCATS,
NIH

Panelists:
Eric Floyd, MS, MBA, PhD
Chief Scientific Officer, Dohmen

Mike Lanthier
Operation Research Analyst, Office of Planning, Office of the Commissioner, FDA

Noel Southall, PhD
Leader, Informatics, Division of Pre-Clinical Innovation, NCATS, NIH

3:45

The FDA Commitment to Rare Diseases

Moderator:
Peter L. Saltonstall
President and CEO,
NORD

Speakers:
Janet Woodcock MD
Director, CDER,
FDA

Peter Marks, MD, PhD
Director, CBER,
FDA

5:00

Closing Remarks 
Peter L. Saltonstall
President and CEO, NORD

Day Two Tracks

I

Trending Topics from FDA

II

Strategies to Address Patient Challenges

III

Breaking down Barriers to Access

Key Contacts

General Inquiries
events@rarediseases.org

Sponsorship Inquiries
Derek Gavin
dgavin@rarediseases.org