Orphan drug designation

Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. These diseases are so rare that the pharmaceutical industry would be less likely to develop the medicine under usual marketing conditions. The designation provides certain benefits to the drug developer to encourage the development of drugs for the small market of individuals with these conditions.

Pronunciation

Orphan drug designation: /ˈɔːrfən drʌg ˌdɛzɪɡˈneɪʃən/

Etymology

The term "orphan drug" comes from pharmaceutical drugs that remained underdeveloped, or "orphaned," due to the lack of financial incentives and market demand. The term "designation" refers to the status assigned by regulatory authorities like the Food and Drug Administration (FDA) or the European Medicines Agency (EMA).

Related Terms

Rare disease: A disease that affects a small percentage of the population. Most rare diseases are genetic and are present throughout a person's entire life, even if symptoms do not immediately appear.
Food and Drug Administration (FDA): A federal agency of the United States Department of Health and Human Services that is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, dietary supplements, prescription and over-the-counter pharmaceutical drugs, vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices, cosmetics, animal foods & feed, and veterinary products.
European Medicines Agency (EMA): A European agency for the evaluation of medicinal products. It operates as a decentralized agency of the European Union and its main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use.
Pharmaceutical industry: The industry responsible for the research, development, production, and distribution of drugs.