Orphan drug


Also found in: Dictionary, Thesaurus, Legal, Encyclopedia, Wikipedia.

orphan drug

[ôr′fən]
Etymology: Gk, orphanos, without parents; ME, drogge
a term that generally refers to drugs needed to treat rare diseases but can encompass any pharmaceutic product available to physicians and patients in countries other than the United States that has not been "adopted" by a domestic pharmaceutic manufacturer or distributor. An orphan drug may not be available in the United States because total sales would not justify the expense of research and development or because the medication may be a natural substance that cannot be effectively protected by patent laws against competition from a similar form of the product. The U.S. Orphan Drug Act of 1983 offers federal financial incentives to commercial and nonprofit organizations to develop and market drugs previously unavailable in the United States for rare diseases affecting fewer than 200,000 people. The FDA assists in the process with its office of Orphan Product Development.
Any drug, biological, medical device, or food of potential or actual use in treating ‘orphan’ diseases—diseases regarded by the pharmaceutical industry as too rare for developing commercially viable products

orphan drug/product

Any drug, biological, medical device, or food of potential or actual use in treating 'orphan' diseases–diseases regarded by the pharmaceutical industry as too rare for developing commercially viable products. See Orphan disease. Cf Pseudoorphan drug.

Orphan drug

A term for a drug that treats a rare disease, defined by the Food and Drug Administration (FDA) as one that affects fewer than 200,000 Americans. The FDA has an Office of Orphan Products Development (OOPD), which offers grants to researchers to develop these products.
References in periodicals archive ?
The report is a study on the global orphan drugs market along with an in-depth analysis of Asian, North-American, European and other regional markets.
Understand market trends: Identify the top 20 brands with FDA orphan drug approval and their forecast values for 2017.
Several brands of the pooled human blood product IVIG have previously received orphan drug designation for CIDP.
Persons afflicted with rare disease are not the only beneficiaries of the Orphan Drug Act.
Company activity: summarises leading and emerging companies and their established orphan drug products and pipelines
US Orphan Drug Pipeline Insight 2014 - Report Highlight:
While orphan drug laws in Asia may provide benefits for companies registering new drugs, such as marketing exclusivity, an expedited registration process, and a reduction in the number of clinical trials required, each registration process is unique and can pose a variety of challenges.
About Orphan Drug Designation Orphan Drug designation is a status assigned to a medicine intended for use in rare diseases.
Orphan Drug Pipeline by Phase, Orphan Designated Disease & Country
Outlook - summarises the future outlook for the orphan drug sector
The returns on investment from orphan drugs are expected to help the pharma companies to overcome the impact of revenue loss due to expiry of patents of blockbuster drugs.