Insights into Rare Disease Drug Approval:Trends and Recent Developments Mike Lanthier Operations Research Analyst FDA Office of the Commissioner NORD Rare Diseases & Orphan Products Breakthrough Summit, October 17, 2017
The Orphan Drug Act was signed into law, on Jan. 4, 1983 Drug/biologic may be “designated” as an “orphan drug” if it is to prevent, treat, or diagnose a disease/condition that occurs in <200,000 patients in U.S. Designations are granted for a given drug for use in a given diseases
Incentives associated with designation: Tax Credits –50% tax credit for clinical research and testing expenses Waiver of User Fees 7 years of marketing exclusivity upon FDA approval of a specific orphan drug for a specific indication
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