International – Orphan product designation requests climb in the US, remain steady in the EU

The number of new requests for orphan drug designation in the United States increased 41% from 2019 to 2020, bringing the number of requests to 753 for 2020, according to the U.S. Food and Drug Administration.
There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. The agency received 20 humanitarian use device designation requests, similar to the number received in 2019.
“Orphan drug designation is an important incentive in rare disease product development and this growth will continue to support development of treatments for rare diseases. Given the ongoing increase in volume and complexity of designations, we continue to enhance and modernize our processes,” Acting FDA Commissioner Janet Woodcock, MD, and Director of the Office of Orphan Products Development Janet Maynard, MD, wrote in a recent FDA Voices article