title: Incentives for orphan drug research and development in the United States.
abstract: BACKGROUND
  The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007.
  
  
  METHODS
  Primary data sources were the FDA Orange Book, the FDA Office of Orphan Drugs Development, and the US Patent and Trademark Office. Data included all orphan designations and approvals listed by the FDA and all NMEs approved by the FDA during the study period.
  
  
  RESULTS
  The FDA listed 1,793 orphan designations and 322 approvals between 1983 and 2007. Cancer was the main group of diseases targeted for orphan approvals. Eighty-three companies concentrated 67.7% of the total orphan NMEs approvals. The average time from orphan designation to FDA approval was 4.0 +/- 3.3 years (mean +/- standard deviation). The average maximum effective patent and market exclusivity life was 11.7 +/- 5.0 years for orphan NME. OD market exclusivity increased the average maximum effective patent and market exclusivity life of ODs by 0.8 years.
  
  
  CONCLUSIONS
  Public programs, federal regulations, and policies support orphan drugs R&D. Grants, research design support, FDA fee waivers, tax incentives, and orphan drug market exclusivity are the main incentives for orphan drug R&D. Although the 7-year orphan drug market exclusivity provision had a positive yet relatively modest overall effect on effective patent and market exclusivity life, economic incentives and public support mechanisms provide a platform for continued orphan drug development for a highly specialized market.
authors.affiliation: Division of Pharmacy Practice & Administration, College of Pharmacy, Ohio State University, Columbus, Ohio, USA. pharmacoeconomics@osu.edu
authors.forename: Enrique
authors.initials: E
authors.lastname: Seoane-Vazquez
authors.forename: Rosa
authors.initials: R
authors.lastname: Rodriguez-Monguio
authors.forename: Sheryl L
authors.initials: SL
authors.lastname: Szeinbach
authors.forename: Jay
authors.initials: J
authors.lastname: Visaria
mesh_terms: D017277:Drug Approval / Q000706:statistics & numerical data*; D004345:Drug Industry / Q000191:economics / Q000706:statistics & numerical data; D006291:Health Policy; D006801:Humans; D009042:Motivation; D009369:Neoplasms / Q000188:drug therapy; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence; D010330:Patents as Topic / Q000706:statistics & numerical data*; D035583:Rare Diseases / Q000188:drug therapy; D012106:Research; D014481:United States; D014486:United States Food and Drug Administration