What does the term orphan drugs mean ?
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.or example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.or example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.iktionary (0.00 / 0 votes) Rate this definition: orphan drug (Noun). A medicinal drug which is effective in the treatment of some disease(s), but which is not manufactured or marketed because the demand is insufficient to cover the costs of supply. orphan drug (Noun). A drug as defined under the Orphan Drug Act (1983). Wiktionary (0.00 / 0 votes) Rate this definition: orphan drug (Noun). A medicinal drug which is effective in the treatment of some disease(s), but which is not manufactured or marketed because the demand is insufficient to cover the costs of supply.orphan drug (Noun). A drug as defined under the Orphan Drug Act (1983).iktionary (0.00 / 0 votes) Rate this definition: orphan drug (Noun). A medicinal drug which is effective in the treatment of some disease(s), but which is not manufactured or marketed because the demand is insufficient to cover the costs of supply. orphan drug (Noun). A drug as defined under the Orphan Drug Act (1983). orphan drug. noun. : a drug that is not developed or marketed because its extremely limited use (as in the treatment of a rare disease) makes it unprofitable. a drug that is not developed or marketed because its extremely limited use makes it unprofitable. ADVERTISEMENT. In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive.or example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. One type of incentive is the orphan drug status, which provides tax reductions and the exclusive right to develop the cure for a specific condition for a period of seven years to companies attempting to cure rare diseases.eveloping drugs to treat the vast number of diseases in the world is a line of business that can lead to massive fortunes, but unfortunately not all developed drugs become the golden ticket. This status is referred to as orphan designation (or sometimes “orphan status”). For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations at 21 CFR Part 316. sponsor seeking orphan designation for a drug must submit a request for designation to OOPD with the information required in 21 CFR 316.20 and 316.21. Each designation request must stand on its own merit. The orphan drug market has become a lucrative one for pharmaceutical companies, with an estimated market size of over $50 billion in 2011, and a growth rate of 25.8% annually in the first decade of this millennium, compared with 20.1% for non-orphan drugs.RELATED TERMS.rphan drugs routinely cost in the six figures per year, and as a result, almost a third of orphan drugs have annual sales of more than $1 billion, according to Thomson Reuters. A sponsor seeking orphan designation for a drug must submit a request for designation to OOPD with the information required in 21 CFR 316.20 and 316.21. Each designation request must stand on its own merit. sponsor seeking orphan designation for a drug must submit a request for designation to OOPD with the information required in 21 CFR 316.20 and 316.21. Each designation request must stand on its own merit.
A pharmaceutical agent.