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Rep. Henry Waxman and FDA Commissioner Margaret Hamburg Are Among Headliners to Speak at NORD Summit

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Friday, September 26, 2014

The principal author of the Orphan Drug Act -- Congressman Henry Waxman (CA) -- and Food and Drug Administration (FDA) Commissioner Margaret Hamburg, MD, will be among the thought leaders speaking at the National Organization for Rare Disorders Breakthrough Summit on Rare Diseases and Orphan Products in October.

The Summit will take place Oct. 21 and 22 at the Hilton Alexandria Mark Center in Alexandria, VA. It will feature general sessions as well as special tracks for patient advocates, medical researchers, and those seeking to develop new therapies and assure patient access to them.

Rep. Waxman played a major role in advancing the development of treatments for patients with rare diseases with the Orphan Drug Act, which created federal incentives making it possible for companies to invest in products for small patient populations. 

As the senior official of the Food and Drug Administration, Commissioner Hamburg leads the federal agency charged with review and approval of potential new therapies.  Approximately one-third of the new products approved in recent years have been "orphan products" -- or those for rare diseases.

"This program truly has something for everyone interested in rare diseases or orphan products," said NORD President and CEO Peter L. Saltonstall. "With senior officials from FDA, more than 80 patient advocates, and key industry innovators participating, we are looking forward to NORD's best conference ever."

Other Summit speakers in addition to Rep. Waxman and Commissioner Hamburg will include:

  • Janet Woodcock, MD, Director of FDA's Center for Drug Evaluation and Research
  • David I. Scheer, President, Scheer & Company, Inc.
  • James C. Greenwood, President and Chief Executive Officer, Biotechnology Industry Organization (BIO)
  • Hank Fuchs, Chief Medical Officer, BioMarin
  • Jess G. Thoene, MD, University of Michigan Health System
  • Audrey Gordon, President, Executive Director, The Progeria Research Foundation
  • John C. McKew, PhD, NIH National Center for Advancing Translational Sciences
  • Dennis Jackman, Senior VP, Public Affairs, CSL Behring
  • Marshall L. Summar, MD, Children's National Health System
  • Caroline Pearson, Vice President, Avalere Health
  • Stephanie Okay, Senior VP and General Manager, Genzyme, a Sanofi Company
  • Nancy Goodman, Founder and Executive Director, Kids v Cancer

... and many more rare disease community innovators.

General and special track sessions will focus on topics of current interest such as:

  • a social media session to examine recent phenomena, such as the ice bucket challenge, and how to harness the power of social media
  • new insights on the patient's role in the approval process
  • a panel of innovators discussing enterprising breakthroughs in the pursuit of life-transforming rare disease treatments
  • a session on advancing therapies for pediatric rare diseases
  • a look at the impact of the ACA and exchanges on market accessibility for orphan drugs
  • a symposium on the U.S. Expanded Access system for patient access to investigational therapies

Established in 1983, NORD is the primary nonprofit organization representing all patients and families affected by rare diseases in the U.S.  It provides programs of advocacy, education, research and patient/family services to improve the lives of all people living with rare diseases.  NORD represents 215 disease-specific member organizations and partners with many other organizations in specific causes of importance to the rare disease patient community.

The NORD Summit is open to everyone interested in rare diseases and orphan products.  Find additional information about the Summit, including online registration, here


Mary Dunkle

Tel: 203 744-0100)

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