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Meeting Program

The US Conference on Rare Diseases and Orphan Products features keynote presentations, high-level plenary and smaller group sessions, including comprehensive coverage of today's hottest topics in sessions intended to promote advancements in science, care and other considerations that will address the needs of the 30 million Americans with rare diseases.  These topics include:

  • Access and Reimbursement
  • Challenges of Reimbursement for the Rare Disease Patient
  • Comparative Effectiveness Research and Health Technology Assessments (HTA)
  • Facing the Crisis in Biomedical Innovation: A Venture Investor’s Perspective
  • Impact of FDASIA on Orphan Product Development
  • Investing in Orphan Products: Is the Environment getting Better Or Worse
  • Natural History Studies
  • New Relationship with the Patient Community – FDA/NIH and the Patient
  • New Relationship with the Patient Community – Industry and the Patient
  • NIH Updates
  • Postmarketing Period-Opportunities for Continued Learning
  • Shaping the Future of Health Policy Now
  • Working with the FDA

Rare Diseases and Orphan Products


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