For Industry

FDA/EMA Orphan Product Designation and Grant Workshop

 
 
FDA White Oak Campus
Silver Spring, Maryland
October 12, 2012


  
On Friday, October 12, 2012, the Food and Drug Administration (FDA) Office of Orphan Products Development along with the European Medicines Agency (EMA) Orphan Medicines Section held an Orphan Product Designation and Grant Workshop at the FDA White Oak Campus in Silver Spring, Maryland.  The workshop provided information about the EMA and FDA Orphan Drug Designation programs, the FDA Humanitarian Use Device (HUD) Designation program, the FDA Orphan Products Grants program, and European Union (EU) rare disease research programs to over 200 participants from over 11 countries representing pharmaceutical, biotechnology, device companies, academia, drug and device regulators. The workshop was held in partnership with the European Organisation for Rare Diseases (EURORDIS), Genetic Alliance and the National Organization for Rare Disorders (NORD).
 
The morning opened with two simultaneous morning plenary sessions for on-site and webcast viewers. One session provided an overview of the EMA and FDA Orphan Drug Designation programs, the Orphan Products Grants program, and EU rare disease research programs as it relates to drugs and biologics. Another session provided an overview of the FDA HUD Designation Program and the Orphan Products Grants program as it relates to medical devices.
 
The afternoon session provided an opportunity for 29 preregistered on-site participant teams 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, a HUD or orphan drug designation. These participants brought information for at least one potential orphan drug or device that holds promise for the treatment of a rare disease to the one-on-one meetings in order to discuss the processes for compiling an application.
 
The workshop was well received.  Attendees of the morning sessions noted that the workshop provided a good overview of the EMA and FDA Orphan Drug programs as well as the HUD Designation program.   Participants of the afternoon one-on-one sessions found the face-to-face interactions with the FDA and EMA staff members to be beneficial in developing their orphan product grant, HUD or orphan drug designation applications.
 
Post Conference Resources:
  
To view a video recording of the Drug Designation and Grant Session, please choose: Plenary Session, or Panel Session 

To view slide presentations please choose from the following:

FDA Orphan Designation 101
G
ayatri R. Rao, MD, JD, Office of Orphan Products Development

EMA Orphan Designation 101
Jordi Llinares, MD, MSc, European Medicines Agency/Orphan Medicines

Transition Talk- FDA Resources and Meetings
Larry Bauer, RN, Office of New Drugs/Rare Disease Program

OOPD Grant Program Overview
Katherine Needleman, PhD, Office of Orphan Products Development

EU/EC Publicly Funded Research Programs 
Jordi Llinares, MD, MSc, European Medicines Agency/Orphan Medicines

Closing Remarks
Gayatri R. Rao, MD, JD, Office of Orphan Products Development 

 

To view a video recording of the Humanitarian Use Device and Grant Session, please choose: Plenary Session, or Panel Session 

To view slide presentations please choose from the following:

FDA HUD Designation 101
Eric Chen, MS, Office of Orphan Products Development

FDA HDE Applications 101
Elizabeth Hillebrenner, MSE, Center for Devices and Radiological Health

Transition Talk- FDA Resources and Meetings
William Sutton, Center for Devices and Radiological Health 

OOPD Grant Program/PDC Overview
Linda Ulrich, MD, Office of Orphan Products Development
 

Closing Remarks
Debra Lewis, OD, MBA, Office of Orphan Products Development 

 

Meeting Evaluation: We invite your comments and suggestions by completing an evaluation form for this workshop. The evaluation form for either the Orphan Drug and Grant Session or Humanitarian Use Device and Grant Session can be accessed through the following links: Evaluation Forms

 

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