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Orphan Drugs


The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.

FDA's Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access) sometimes called "compassionate use," is the use of an investigational drug outside of a clinical trial to treat a patient or group of patients with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options.

The FDA's Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use also allows a physician who would like to submit an Investigational New Drug application (IND) to obtain an unapproved drug for an individual patient, he or she should first ensure that the manufacturer of the unapproved drug is willing to provide the drug.


Orphan Drug Designations and Approvals from FDA allows searches to be run by entering the product name, orphan designation, and dates. Silver Spring (MD): U.S. Food and Drug Administration; 2013.

Orphan Drug Product Designations from Facts & Comparisons eAnswers. St. Louis: Wolter Kluwers Health; 2013-. Online requires subscription. Available also in annual print editions.

Orphan Drugs from Orphanet may be searched by the name of the substance in their composition, or by their commercial name (trade name). Substance names include chemical names, code names of the substance or common names (INN : International Non-proprietary Name). Also available are tabs for List of Orphan Designations and List of Orphan Drugs with MA (marketing authorization). Available in seven languages. Paris: Orphanet-INSERM; 2013.

Other Resources

Hernberg-Stahl E, Reljanovic M. Orphan Drugs: Understanding the rare disease market and its dynamics. Oxford: Woodhead Publishing; 2013.

Pryde DC, Palmer MJ, editors. Orphan Drugs and Rare Diseases. London: Royal Society of Chemistry; 2014.

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