Rare Disease Report

Who's Who of the Rare Disease Community to Speak at NORD's Conference

AUGUST 25, 2015
James Radke, PhD
Acting FDA Commissioner Stephen Ostroff among the speakers

The agenda and speakers scheduled for the NORD 2015 Rare Diseases & Orphan Products Breakthrough Summit has been finalized and it is going to be another stellar line up of industry, advocacy, and regulatory leaders speaking at the summit being held in Arlington, Virginia on October 21-22, 2015.

The summit is also the only rare disease conference to be co-sponsored by the FDA. Among the speakers will be FDA dignitaries Stephen Ostroff, MD (Acting Commissioner, FDA) and Janet Woodcock, MD, (Director, Center for Drug Evaluation and Research, FDA). 

Scheduled speakers include:

  • Taher Abbasi, co-founder, CEO, Cellworks Group
  • Jeff Allen, executive director, Friends of Cancer Research
  • Christopher P Austin, MD, Director, NCATS
  • Ron Bartek, President and Cofounder, Friedreich’s Ataxia Research Alliance
  • Don Bobbear, Principal-Managing Director, The Patient Experience Project
  • Marc Boutin, JD, Chief Executive Officer, National Health Council
  • Tim Boyd, Associate Director of State Policy, NORD
  • Catherine Brady, Vice President, JPA Health Communications
  • Christine Brown, MS, Executive Director, National PKU Alliance
  • Barbara Conley, MD, Associate Director, Cancer Diagnosis Program, National Cancer Institute
  • Derek Cothran, Vice President, Lash Group
  • Peter Day, Managing Director, Healthcare Investment Banking, Piper Jaffray
  • Joshua Denny, MD, MS, FACMI, Associate Professor of Bioinformatics and Medicine, Vanderbilt University Medical Center
  • Leigh Fazzina, Social Media Counselor, Sam Brown Healthcare Communications
  • Pat Furlong, Founding President and CEO, Parent Project Muscular Dystrophy
  • Pamela Gavin, Chief Operating Officer, NORD
  • Nicholas Galakatos, Managing Director, Clarus Ventures
  • James Geraghty, Entrepreneur in Residence, Third Rock Ventures
  • Jonathon Goldsmith, Rare Diseases Program, Office of New Drugs, CBER, FDA
  • Nancy Goodman, Founder and Executive Director, Kids v. Cancer
  • Nicole Hebbert, Vice President, Commercial Services Operations
  • Dennis Jackman, Senior Vice President, Public Affairs, CSL Behring
  • John Jenkins, MD, Director, Office of New Drugs, FDA
  • Carrie Jones, Principal, JPA Health Communications
  • Steve Kaminsky, PhD, Scientific Director, International Rett Syndrome Association
  • Richard Klein, Patient Liason Program Director, Office of Health and Constituent Affairs, FDA
  • Jono Lancaster, Patient Advocate, Treacher Collins Syndrome
  • Ricki Lewis, PhD, Geneticist, DNA Science Blog
  • Naomi Lowy, MD, Senior Medical Officer, Professional Affairs and Stakeholder Engagement, CDER, FDA
  • Peter Marks, MD, PhD, Deputy Director, CBER, FDA
  • Janet McUlsky, Senior Director, National Alliance Development, Pfizer
  • Jules T. Mitchel, MBA, PhD, Co-Founder, Target Health
  • Richard Moscicki, MD, Deputy Center Director for Science Operations, CDER, FDA
  • Theresa Mullin, PhD, Director, Office of Strategic Programs, CDER, FDA
  • Jan Neilsen, Division President, Access and Patient Support, Sonexus Health
  • Will Nolan, Director of Communications and Administrations, Parent Project Muscular Dystrophy
  • Stephanie Okey, MS, Consultant, Former Genzyme
  • Stephen Ostroff, MD, Acting Commissioner, FDA
  • Jim Palma, Executive Director, Target Cancer Foundation
  • Elektra Papadopoulos, Acting Associate Director, Clinical Outcomes Staff, Office of New Drugs, CDER, FDA
  • Bray Patrick-Lake, MFS, Director of Stakeholder Engagement, Clinical Trials Transformation Initiative
  • Dave Penake, Vice President, Commercial Operations, Corcept Therapeutics
  • Eleanor M Perfetto, PhD MS,  Professor, University of Maryland School of Pharmacy, Senior Vice President of Strategic Initiatives, National Health Council
  • Lisa Phelps, MPH, Director of Marketing and Community Relations, NORD
  • Richard Peters, MD, PhD, Senior Vice President, Global Head of Rare Diseases, Genzyme
  • Kevin A Prohaska, DO, MPH, Captain, Medical Officer, Office of Good Clinical Practice, FDA
  • Gayatri R. Rao, MD, JD, Director, Office of Orphan Products Development, FDA
  • Amit Sachdev, Executive Vice President, Policy, Access and Value, Vertex
  • Peter L Saltonstall, President and Chief Executive Officer, NORD
  • Robert Saul, MD, Co-Director, AAP “Genetics in Primary Care”
  • Tina Schlect, PhD, MBA, President, Center Point Clinical Services
  • Roslyn Schneider, Senior Director, Global Patient Affairs, Pfizer
  • Maddie Shaw, Pediatric Patient, Primary Immune Disorder
  • Jeffrey W Sherman, MD, FACP, Chief Medical Officer and Executive Vice President, Research and Development, Horizon Phama, Inc.
  • Sue Sherman, President, LAM Foundation
  • Jeffrey Shuren, MD, JD, Director, Center for Devices and Radiological Health, CDRH, FDA
  • Shelden Shuster, PhD, President and Professor, Keck Graduate Institute
  • Mary Smith, Vice President, Product Development, United Therapeutics
  • Marshall L Summer, MD, Division Chief, Genetics and Metabolism, Children's National Health System
  • Farra Trompeter, Vice President, Big Duck
  • Sophia Walker, Medical Student, University of Connecticut
  • Mike S Watson, PhD, FACMG, Executive Director, ACMG
  • John Whyte, Director, Professional Affairs and Stakeholder Engagement, CDE, FDA
  • Janet Woodcock, MD, Director, Center for Drug Evaluation and Research, FDA
  • Lynne P Yao, Acting Director, Division of Pediatric and Maternal Health/ODE IV/CDER, FDA

Scheduled Topics include:

  • Optimizing clinical trial success and patient engagement from start to finish
  • Patient-centered outcomes research (PCOR) and practical methods to incorporate the patient voice into research and development
  • FDA presentations on current topics in rare diseases, including expanded access
  • Physician education to facilitate early diagnosis and treatment, featuring perspectives from the American Academic of Pediatrics and Genetics in Primary Care Institute
  • Precision and personalized medicine and what it means for rare diseases
  • Rare cancer treatment challenges
  • The legislative path ahead on national and state levels
  • Marketing and communications strategies for patient organizations, featuring proven techniques to increase awareness and fundraising
  • Fueling progress and access with specialty pharmacies, hub and distribution strategies
For more information about the conference, visit http://rarediseases.org/summit-overview/

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.