Orphan Drug Act

A recent national survey of 2,000 US adults living in households with a member with a rare disease weighted to reflect our population found 8% reporting that they or a household member have been diagnosed with a rare disease.

When I joined the FDA to be its “biotechnology czar” more than 40 years ago, genetic engineering was a new concept.

The theme for this year’s Rare Disease Day is “bridging health and social care” and The Lancet Diabetes & Endocrinology, in addition to

It is Game On! at Commissioner Scott Gottlieb's U.S. Food and Drug Administration.