Term of the Day
Natural History Study
A natural history study is a preplanned intended to track the course of the disease. Its purpose is to identify demographic, genetic, environmental, and other variables (e.g., treatment modalities, concomitant medications) that correlate with the disease’s development and outcomes. Natural history studies are likely to include patients receiving the current standard of care and/or emergent care, which may alter some manifestations of the disease.
Orphan Drug Designation (ODD)
An Orphan Drug Designation (ODD) is a regulatory designation granted by the FDA (Food and Drug Administration) in the United States, and similar agencies in other countries, to drugs and biologics that are intended to treat rare diseases or conditions.The Orphan Drug Act was passed in the United States in 1983 to encourage the development of drugs for rare diseases, defined as those affecting fewer than 200,000 individuals in the U.S. Orphan Drug Designation provides various benefits to drug developers, including tax credits, grants for clinical trials, and a period of market exclusivity for the drug if it is approved by the FDA.
To receive Orphan Drug Designation, a drug must meet specific criteria established by the FDA, including the rarity of the disease or condition it is intended to treat, and the potential for the drug to provide significant clinical benefit over existing therapies.