Orphan Drug
定義
A drug developed to treat a rare disease or condition affecting fewer than 200,000 people in the United States. The Orphan Drug Act provides manufacturers with incentives including tax credits, grant funding, and 7 years of market exclusivity to encourage development.
例
Ivacaftor (Kalydeco) was approved as an orphan drug for cystic fibrosis affecting fewer than 30,000 Americans.
関連用語
ガイドでの言及
-
The Orphan Drug Act ExplainedWhy rare diseases were historically neglected by drug developers and how the 1983 Orphan Drug Act transformed treatment options for …
-
Patient Assistance ProgramsPharmaceutical manufacturers and nonprofits operate programs that provide free or discounted medications to qualifying patients. This guide walks through who …
-
Gene Therapy DrugsGene therapy aims to treat disease by modifying the genetic material inside a patient's cells. Learn how approved gene therapies …