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2012 Orphan Disease Forum: Addressing Today’s Opportunities and Challenges

April 6, 2012
If all of the people with rare disease lived in one country, it would be the world’s third most populous country! Through dedicated companies such as the National Organization for Rare Disorders (NORD) and Centric Health Resources, the pressing issue of orphan diseases and its patients are being addressed.

A rare disease is defined by any disease affecting fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting over 350 million people worldwide.  Furthermore, 95% of rare diseases have no FDA approved drug treatments, according to data published by the Kakkis EveryLife Foundation.

One leader devoted to helping people with rare or orphan diseases is NORD.  NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. Working closely with many government agencies, including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), all NORD programs are focused on one ultimate goal – to improve the lives of individuals and families affected by rare diseases.

NORD and its co-organizer, Centric Health Resources, a Patient Centered Health Management organization that delivers prescribed therapies and specialized health management services to affected individuals, will host a two-day forum at the 2012 BIO International Convention to provide unique perspective on the orphan drug marketplace, regulatory considerations and much more.

Session topics include:

  • Orphan Markets are Different: A Primer

  • Are We Heading into a Pharmaceutical and Biotechnology Market of Orphan and Narrow Indications?

  • Best Practices in Targeted Drug Development: A Rare Disease Case Study

  • Innovations in Supply Chain Economics and Value Creation in U.S. Orphan Markets

  • Considerations for Novel Approval Pathways for Rare and Orphan Diseases

  • Specialized Marketing & Sales: A Vision for Orphan and Targeted Therapies

  • Rare Diseases: Orphaned, but Not Alone

  • Is Big Pharma the Preferred Partner for Orphan Drug Biotechs?

BIO is pleased to recognize the leadership provided by the 2012 Orphan Disease Forum sponsors.  The sponsors include: Alnylam Pharmaceuticals, Genzyme, a Sanofi Company and Millennium: The Takeda Oncology Company.

The two-day forum will be held Tuesday, June 19th and Wednesday, June 20th at the Boston Exhibition and Convention Center.  To learn more about the event and get up-to-date program and speaker information, visit