Wednesday July 18, 2012
7:30 am
Conference Registration and Continental Breakfast
8:30 am
Chairperson’s Welcome and Opening Remarks
Stephanie S. Okey M.S.
Vice President and General Manager, Rare Diseases, U.S Genetic Diseases
Genzyme
8:45 am
Congressional Video Address • FAST Act — What Would Approval Mean for Rare Diseases?
Cliff Stearns
U.S. Representative of Florida’s 6th Congressional District
9:00 am
National Health Council Address • MODDERN Cures Act — Game Changing Legislation
Marc Boutin JD
Executive Vice President and Chief Operating Officer
National Health Council
9:45 am
FDA Address • Review Proposed Rulemaking Changes and EMA Harmonization Efforts by the FDA
James H. Reese Ph.D., RAC
Reviewer, EMA Coordinator, Office of Orphan Products Development
Food & Drug Administration (FDA)
11:00 am
NIH Address • NIH Initiatives Supporting Orphan Drug Development and Collaboration
Elizabeth Ottinger Ph.D.
Project Manager Team Lead, Therapeutics for Rare & Neglected Diseases (TRND), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
11:30 am
Spotlight Session • Promising Initiatives in the Rare Disease Sector
Dean Suhr
President, MLD Foundation; Chairman of the Patient Advocacy Advisory Board
RARE Project
Emil D. Kakkis M.D., Ph.D.
President
EveryLife Foundation for Rare Diseases
12:15 pm
Networking Luncheon
1:30 pm
Developing Treatments for Rare Diseases — Land Mine or Gold Mine?
Roger J. Garceau M.D., FAAP
Senior Vice President and Chief Medical Officer
NPS Pharmaceuticals
2:15 pm
Case Study • Create a Successful Drug Development Path
Ron C. Falcone Ph.D.
Vice President, Regulatory Affairs
Incyte Corporation
3:00 pm
Networking and Refreshment Break
3:30 pm
Utilizing Discoveries in Research and Development to the Benefit of All Patients
Jeffrey Kralstein
Vice President and Global Head, Clinical and Scientific Excellence
Novartis Pharmaceuticals
4:15 pm
Ensuring Access and Driving Outcomes Through New Patient Care and Distribution Models
Craig Kephart
President
Centric Health Resources, Inc.
5:00 pm
2012 — “The Year of High Priced Drugs” and the Potential Implications on Future Pricing Strategies
Douglas Paul Pharm.D., MS
Vice President and Partner
Medical Marketing Economics (MME), LLC
Jack Mycka
Global President and CEO
Medical Marketing Economics (MME), LLC
5:45 pm
Close of Day One
5:45 pm - 6:45 pm
Wine and Cheese Reception Hosted by Compass
Thursday July 19, 2012
7:30 am
Continental Breakfast
8:00 am
Chairperson’s Review of Day One
Stephanie S. Okey M.S.
Vice President and General Manager, Rare Diseases, U.S Genetic Diseases
Genzyme
8:15 am
Virtual Work Environments: A New Way to Optimize Resources and Improve Physician Access
Lance Hill
CEO
Within3
9:00 am
Reimbursement Hub, Specialty Pharmacy, Patient Support, Oh My!
Peter H. Nalen
President, CEO
Compass Healthcare Marketers
Kristin Keller
Executive Vice President
Compass Healthcare Marketers
9:45 am
Foundation Address • Develop Patient Relationships for Improved Rare Disease Treatments
Ann Brazeau
Vice President of Development
MPN Research Foundation
10:30 am
Networking and Refreshment Break
Access World Shared Content
11:00 am
Case Study • Implement a Managed Access Program for an Orphan Therapy
Simon Estcourt
Senior Vice President, Strategy and Corporate Development
IDIS
11:30 am
Panel Discussion • How to Interact with Advocacy Groups to Facilitate Access
John Lagus
Vice President, New Product and Market Development
IDIS
Dean Suhr
President, MLD Foundation; Chairman of the Patient Advocacy Advisory Board
RARE Project
Kim Ryan
Director of Patient Information Services
Fight Colorectal Cancer Foundation
Julie Fleshman JD, MBA
President & CEO
Pancreatic Cancer Action Network
Jill Panetta Ph.D.
Chief Scientific Officer
12:45 pm
Networking Luncheon
2:00 pm
Leverage Social Media in the Rare Disease Community for Patient Recruitment and Awareness
Wendy White
Founder and President
Siren Interactive
Robert Derham
President
CheckOrphan
Kristina Broadbelt
Director, Global Patient Advocacy and Corporate Reputation
Viropharma Incorporated
Katherine Leon
SCAD Patient Advocate
WomenHeart Champion
3:30 pm
Chairperson’s Closing Remarks
Stephanie S. Okey M.S.
Vice President and General Manager, Rare Diseases, U.S Genetic Diseases
Genzyme
3:30 pm
