What is an orphan drug?

Orphan drugs are medications or other medicinal products used to treat  rare diseases or disorders. They are called  “orphan drugs” because due to their limited market, few pharmaceutical companies pursue research into such products. Selling to only a small group of patients, drug manufacturers would be unlikely to recover development and marketing costs, let alone make a profit by producing the product. 

Although the Orphan Drug Act of 1983 introduced incentives for drug companies to develop treatments for rare diseases, today, it remains that no treatments exist for the vast majority of rare diseases.

The following link offers additional information about orphan drugs:

Orphan Drugs: A Global Crusade

Orphan Drugs: A Global Crusade

Abbey S. Meyers wrote this memoir to document the issues that led to the enactment of the “Orphan Drug Act of 1983” 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 founder and Past-President of the National Organization for Rare Disorders (NORD), a coalition of national volunteer health agencies and a clearinghouse for information about little-known illnesses. Ms. Meyers currently holds the honorary title of President Emeritus of NORD. She also served as Honorary President of the European Organization for Rare Disorders (EURORDIS) before retiring from role as a leader of the rare disease consumer movement in 2009.

During her professional career in the United States, Ms. Meyers served as Consumer Representative on the National Commission on Orphan Diseases (1986-89), the NIH Human Gene Therapy Subcommittee (1989-92), the NIH Recombinant DNA Advisory Committee (RAC) (1993-96), the FDA Biological Response Modifiers Committee (1995-99) and the HHS National Human Research Protections Advisory Committee (2000-2002).

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 to be the primary American consumer advocate responsible for the passage of the Orphan Drug Act of 1983.

Throughout her career, Ms. Meyers has sat on numerous committees, boards and commissions dealing with orphan diseases at the national and international levels.

She is the author of many articles and papers related to this topic and has been a frequent speaker at conferences across North America, Europe and Asia.

For more information about her work, please visit abbeysmeyers.com.