“I’m From the Government, and I’m Here to Help” Debra Y. Lewis, O.D. Director, Orphan Products Grant Program Office of Orphan Products Development Food and Drug Administration
Current FDA Responsibilities
The Orphan Disease Problem
The Nature of Rare Diseases
Barriers to Orphan Drug Development
The Orphan Drug Act Signed in 1983
The FDA Office of Orphan Products Development (OPD)
Orphan Designation Incentives
A Rare Disease is Determined by the Size of the Population Affected
The Role of OPD Staff
The FDA Office of Orphan Products Development
Assistance For Orphan Product Sponsors
The Designation Process
Orphan Products Include
Orphan Designations
Orphan Products Must Be Just as Safe and Effective
The Office of Orphan Products Development Grant Program
Grant Program Objective
Orphan Products Research Grants
Grant Support for Investigation of Rare Disease Treatment
Orphan Products Grant Program
Orphan Grant Application Process
Grant Program Accomplishments
“One additional orphan drug approval is estimated to have prevented 211 deaths in the subsequent year.” NBER Working Paper No. 8677
The Office of Orphan Products Development, FDA
Home Page: http://www.kennedysdisease.org