Drugs that the pharmaceutical companies do not wish to develop either because they cannot be patented, because they are used only in rare conditions by very few people, or because of a variety of economic reasons. In such cases, the federal government may work with the companies to make the drugs available to those persons who need them.
The name of the legal status granted by the Food and Drug Administration’s Office of Orphan Products Development (to certain pharmaceuticals). This classification provides the sponsors of those pharmaceuticals with special tax and other financial incentives (e.g., market monopoly for a limited time). If companies feel that they possess a cure (drug) for a certain disease, but the number of potential patients is below a certain number and there is potential competition from rival companies, then the high cost of developing and shepherding the drug through the FDA would be such that the company would not be able to regain its development costs and make a profit. Hence, orphan drug status was designed to encourage drug development efforts for otherwise noneconomic harmaceuticals with less than 200,000 patients a year.
A drug that typically is used for the treatment of a rare condition, making it exorbitantly expensive and/or difficult to obtain.
A medication that pharmaceutical companies generally do not produce because it is used to treat a rare condition affecting relatively few people. This is so especially if the patent has run out on the drug and competitors might enter the field at any time they wish. In 1983, the federal government passed the Orphan Drug Act, which offers financial inducements to encourage the development, testing (including testing of drugs developed in other countries), and distribution of such drugs to the people who need them, such as medication for Tourette’s syndrome.
Drugs used to treat rare diseases. Orphan drugs are defined by the Food and Drug Administration (FDA) as those used to treat diseases or conditions affecting fewer than 200,000 people in the United States. Because so few people need them, the drugs are unlikely to prove profitable for manufacturers, and it is sometimes difficult to find sponsors for them.
Any drug that is effective for certain illnesses but, for a variety of reasons, is not profitable for manufacturers to produce.
A medication utilized to treat a condition categorized as a rare disease, with the Food and Drug Administration defining “rare disease” as an affliction that affects fewer than 200,000 individuals in the United States. Orphan drugs, including both approved and experimental medications, fall within this classification.
Medications created to address uncommon disorders, yet not produced on a widespread scale.