THE NATIONAL ACADEMIES
Institute of Medicine
Board on Health Sciences Policy
Committee on Accelerating Rare Diseases Research and Orphan Product Development
Rare Diseases and Orphan Products: Accelerating Research and Development
While the number of people affected by each rare disease is relatively small, the number of these diseases is so large -- an estimated 5,000 to 8,000 conditions -- that a host of challenges complicates the development of safe and effective drugs, biologics, and medical devices to prevent, diagnose, treat, or cure these conditions. This report recommends an integrated national strategy to improve rare disease research and product development.
This briefing was for members of Congress and congressional staff only. The report was publicly released on October 4, 2010 and can be found, in its entirety, on the Web site of the National Academies Press.