U.S. Conference on Rare Diseases and Orphan Products
Register By October 1 For Early Bird Rate!
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!
- 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
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).