September 11, 2012

2012 Conference on Rare Diseases and Orphan Products

U.S. Conference on Rare Diseases and Orphan Products

Register By October 1 For Early Bird Rate!

Join patient advocates, drug and device industry leaders, researchers, government partners and investors at this major conference to address together "Shaping the Future Now" for rare diseases and orphan products.  Sponsored by NORD and DIA, with major input from FDA and NIH, this event will include general and breakout sessions with three themes:  policy, research and regulation, and special challenges.
The 2nd annual U.S. Conference on Rare Diseases and Orphan Products will take place October 22-24 in Washington DC. Everyone is welcome, and the program should be of particular interest to:
  • Researchers from academia and drug and device companies
  • Patient organizations and those interested in creating one
  • Senior managers from drug and device companies interested in rare diseases
  • Investors focused on the future of orphan product development
  • Policy experts who are concerned about federal or state policies that affect patients with rare diseases
  • Providers of services to the rare disease community, including insurance providers and healthcare professionals
  • Government officials responsible for rare disease research and orphan product oversight

Online Registration Is Open Now!

A full description of the conference and online registration are available now.  Register by October 1 for the Early Bird rate.  Topics and speakers will include:
  • Impact of FDASIA on Orphan Product Development (Andrew J. Emmett, MPH, Managing Director, Science and Regulatory Affairs, BIO; Cassie A. Scherer, JD, Policy Advisor, Office of the Center Director, CDRH, FDA)
  • Investing in Orphan Products: Is the Environment Getting Better or Worse? (session chaired by Thomas M. Burton, JD, Staff Reporter, The Wall Street Journal)
  • Special Challenges in Rare Diseases (John J. Castellani, President & CEO, PhRMA)
  • Well-Designed & Well-Conducted Clinical Trials (session chaired by Gayatri Rao, JD, MD, Director, FDA Office of Orphan Products Development)
  • The New Relationship With the Patient Community (session chaired by Jayne C. Gershkowitz, Senior Director, Patient Advocacy & Public Policy, Amicus Therapeutics)
  • TRND and Translational Development (Christopher P. Austin, MD, Scientific Director, NIH Center for Translational Therapeutics, National Center for Advancing Translational Sciences)
  • Endpoint Development (session chaired by Anne R. Pariser, MD, Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA)
  • Facing the Crisis in Biomedical Innovation: A Venture Investor's Perspective (Jonathan S. Leff, MBA, Managing Director, Warburg Pincus)
  • Research and Regulation (Robert M. Califf, MD, MACC, Vice Chancellor for Clinical and Tanslational Medicine; Director, Duke Translational Medicine Institute; Duke University School of Medicine)

At a Critical Moment in Time: Bringing All Stakeholders Together

With a major new law (the FDA Safety and Innovative Act) soon to be implemented, this forward-looking conference brings together all stakeholders to "Shape the Future Now" for rare diseases and orphan products.
The conference is co-sponsored by the National Organization for Rare Disorders (NORD), the voice of 30 million Americans with rare diseases, and the Drug Information Association (DIA), a neutral, nonprofit, global professional association for those working in discovery, development and management of pharmaceuticals, medical devices and related products.
Collaborators include Duke University School of Medicine, EURORDIS (Rare Diseases Europe), the Food and Drug Administration (FDA) and the National Institutes of Health (NIH).


  1. If you do come to DC, I'd love to meet you in person: sfox at in case there's a chance!

  2. I only heard about this confab from an online pharmacy reviews forum (although that was after the conference) and I figured last year's theme was a discussion starter. Be looking forward to next year's conference.