orphan drugs


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Related to orphan drugs: orphan disease

or·phan pro·ducts

drugs, biologicals, and medical devices (including diagnostic in vitro tests) that may be useful in either common or rare diseases but that are not considered commercially viable.
Synonym(s): orphan drugs

or·phan prod·ucts

(ōr'făn prod'ŭkts)
Drugs, biologicals, and medical devices (including diagnostic in vitro tests) that may be useful in treating rare diseases but are not considered commercially viable.

orphan drugs

A drug used to treat a rare disease and for which the market is so small that manufacturers have little incentive to go to the expense of producing them. In1983 US legislation provided financial incentives, in the form of tax credits and 7 year exclusive rights, to firms willing to produce such drugs. This has been effective and, by mid-2003, 229 orphan drugs were available.
References in periodicals archive ?
"This review concludes that two of every three orphan drugs approved show the FDA's flexibility in its review of effectiveness data on orphan drug therapies," says Sasinowski.
To further accelerate the development of orphan drugs by the pharmaceutical and biotechnology industries, the NCOD recently asked Congress to approve greater incentives for orphan drug companies.
But companies that produce them under the orphan drug designation are offered several advantages, including research grants, tax benefits, protocol assistance, marketing exclusivity (seven years in the United States) and regulatory challenges (such as a smaller number of clinical trials) that are less daunting than those faced by other new medicines.
The PMA's commission on drugs for rare diseases is encouraging the development of orphan drugs, and finding corporate sponsors for promising therapies.
The Orphan Drug Act of 1983 has worked well in achieving the purpose for which it was enacted--to provide incentives that encourages orphan drug development, says PMA president Gerald Mossinghoff.
Genentech's human growth hormone, used to treat dwarfism, and Amgen's EPO, used to treat anemia in kidney dialysis patients, would retain their orphan drug status.
Some 36 orphan drugs have been approved by the FDA under the Orphan Drug Act and are available to patients.
According to a new report published by Allied Market Research, titled, "Orphan Drugs Market by Disease Type and Indication: Global Opportunity Analysis and Industry Forecast, 2014-2022," the global orphan drugs market was valued at $106 billion in 2015, and is projected to reach $169 billion by 2022, growing at a CAGR of 6.8% from 2016 to 2022.
So, besides enacting an orphan drugs legislation with provisions for incentives for orphan drug developers, the government needs to create a framework that is conducive for the manufacturing of orphan drugs in India, according to ORDI.
Orphan Drugs Market, by Disease Type, Revenue (US$ Bn) 2015-2025
Seven of the top ten companies by orphan drug sales are global industry players."
For those interested in attending Orphan Drugs 2016, registration is live online at http://www.orphandrugs-event.com/prcom.