Skip navigation

7th Annual Rare Disease and Orphan Drug Leadership Congress

Ensure Access to Orphan Drug Therapies through Collaborative Efforts with Government, Researchers and Patient Groups

July 18-19, 2012
  • Philadelphia, Pennsylvania

Agenda

Want more agenda details? Download the brochure.
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

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

5:00 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

9:00 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

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

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

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

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

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

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

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

Close of Conference


Agenda

Want more agenda details? Download the brochure.