Office of Rare Diseases Research (US)

Rare diseases collectively affect millions of Americans of all ages. They often are serious and life-altering; many are life-threatening or fatal. But because each rare disease affects a relatively small population, it can be challenging to develop drugs and medical devices to prevent, diagnose, and treat these conditions. Many rare diseases still lack even a basic understanding of the cause or underlying molecular mechanisms. To help in accelerating rare diseases research and product development, the National Institutes of Health (NIH), with support from the Food and Drug Administration (FDA), asked the IOM to examine the opportunities for and obstacles in developing drugs and medical devices for treating rare diseases.

On Monday, October 04, 2010, the Institute of Medicine released its report titled “Rare Diseases and Orphan Products: Accelerating Research and Development.” Information about IOM’s activities in support of this report as well as the report itself can be found at www.iom.edu/rarediseaseresearch.