US Conference on Rare Diseases and Orphan Products

US Conference on Rare Diseases and Orphan Products

07-10-2013 - 09-10-2013 Bethesda North Marriott Hotel and Conference Center, Bethesda, MD

This year, the US Conference on Rare Diseases and Orphan Products will focus specifically on the changing health care environment.

This year, the US Conference on Rare Diseases and Orphan Products will focus specifically on today’s changing health care environment, addressing questions such as: What effect will the implementation of the Affordable Care Act have on rare disease patients? What is the investment outlook for orphan product development? And what will our health care system look like in the future?   

Sponsored by NORD (the National Organization for Rare Disorders) and DIA (the Drug Information Association), this major event brings together all stakeholders in the rare disease community to address shared goals, concerns, challenges and opportunities. 

This year, the third annual meeting of stakeholders in the rare disease community comes at an opportune time. Two new laws – the Affordable Care Act and the FDA Safety and Innovation Act – are being implemented. Together, these laws will shape the future for the rare disease community for years to come. In addition, this year marks the 30th anniversary of the Orphan Drug Act, as well as, the 30th anniversary of NORD (National Organization for Rare Disorders).

NORD president and chief executive Peter Saltonstall said: “This conference provides an opportunity to share our various perspectives and collaborate in ways that are absolutely essential for the millions of Americans living with rare diseases.” 

The conference, which is hosted in collaboration with the US Food and Drug Administration and EURORDIS (Rare Diseases Europe), will include keynote, plenary and smaller group sessions. Plenary sessions will open a dialogue on topics of shared interest among medical researchers, industry thought leaders, patient organizations, government officials and investors. 

The meeting will include four major themes:

  1. Research & Regulation
  2. Access and Reimbursement
  3. The Role of the patient in the research and regulatory process
  4. The Implementation of the Affordable Care Act

Who Should Attend

  • 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 disease
  • 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






UK drug discovery firm Summit Pharmaceuticals is an Oxford-based drug discovery and development company targeting high-value areas of unmet medical need including Duchenne Muscular Dystrophy and C. difficile infection.

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