FDA Harnesses Technology and Collaboration to Support Rare Disease Product Development

By: Amy Abernethy, M.D., Ph.D., Principal Deputy Commissioner and Acting Chief Information Officer and Janet Maynard, M.D., M.H.S., Director, Office of Orphan Products Development

Patients and their families are the focus of our work at the FDA. We ensure that treatments for diseases are safe and effective. Patients and their families are experts in their diseases and help inform many aspects of medical product development. This is especially notable in rare diseases, where product development can be challenging given the small number of patients affected with a given disease and the potential for limited understanding of the pathophysiology of some rare diseases.

While rare diseases are individually rare, collectively they are not. It is estimated that rare diseases affect 25 to 30 million Americans. […]

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