What is an orphan drug?

An orphan drug is a medication or other medicinal product used to treat a rare disease or disorder. It is called an “orphan drug” because due to its limited market, it is unlikely to be adopted by a pharmaceutical company. Selling to only a small group of patients, a drug manufacturer would likely not be able to recover development and marketing costs, let alone make a profit by producing the product.

The following link offers additional information about orphan drugs:


Orphan Drugs: A Global Crusade

Orphan Drugs: A Global Crusade

Ms. Abbey Meyers wrote her memoirs in this book in an effort to document the issues that led to the enactment of the “Orphan Drug Act of 1983” in the United States and the extraordinary scientific and industrial progress that ensued, including the development of Orphan Drug legislation in other industrialized countries around the world.

abbey s meyers



Ms. Meyers is the recipient of the FDA Commissioner's Special Citation for Exceptional Dedication and Achievements on Behalf of All People Afflicted with Rare Disorders (1988), and the Department of Health and Human Services Public Health Service Award for Exceptional Achievements in Orphan Drug Development (1985). She holds an Honorary Doctorate from Alfred University in New York and is considered the primary consumer advocate responsible for passage of the Orphan Drug Act of 1983.

