The FDA Office of Orphan Products Development along with the European Medicines Agency (EMA) Orphan Medicines Section held an Orphan Product Designation and Grant Workshop on Friday, October 12, 2012, from 8:30 a.m. to 5:30 p.m. The workshop was held on the FDA's White Oak Campus in Building 31 (The Great Room), 10903 New Hampshire Avenue, Silver Spring, Maryland, 20993.
This one-day workshop was designed to provide valuable information about the EMA and FDA Orphan Drug Designation programs, the FDA Humanitarian Use Device (HUD) Designation program, the FDA Orphan Products Grant program, and European Union (EU) rare disease research programs to participants representing pharmaceutical, biotechnology, and device companies, as well as to academics. The workshop was held in partnership with the European Organisation for Rare Diseases (EURORDIS), Genetic Alliance and the National Organization for Rare Disorders (NORD).
All participants were able to register for one of two simultaneous morning sessions – the first provided an overview of the EMA and FDA Orphan Drug Designation programs, the Orphan Products Grant program, and EU rare disease research programs as it relates to drugs and biologics, while the second provided an overview of the FDA Humanitarian Use Device (HUD) Designation Program and the Orphan Products Grant program as it relates to medical devices. Both of these morning sessions were available by webcast.
The afternoon session provided a unique opportunity for appropriately registered on-site participants to have one-on-one meetings with FDA or EMA staff members to discuss the specifics on how to apply for an orphan product grant, or a HUD or orphan drug designation. Participants requesting one-on-one meetings underwent a second registration process with FDA, and were expected to bring information for at least one candidate orphan drug or device that holds promise for the treatment of a rare disease to the one-on-one meeting in order to discuss the processes for putting together an application. In addition, participants of the HUD or orphan drug designation one-on-one sessions were highly encouraged to come prepared with a working draft submission of their particular promising therapy in order to maximize utility of the one-on-one meetings.