Orphan Drugs

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Chapter: Essential pharmacology : Introduction And Routes Of Administration

These are drugs or biological products for diagnosis/treatment/ prevention of a rare disease or condition, or a more common disease (endemic only in resource poor countries) for which there is no reasonable expectation that the cost of developing and marketing it will be recovered from the sales of that drug.


ORPHAN DRUGS

 

These are drugs or biological products for diagnosis/treatment/ prevention of a rare disease or condition, or a more common disease (endemic only in resource poor countries) for which there is no reasonable expectation that the cost of developing and marketing it will be recovered from the sales of that drug. The list includes sodium nitrite, fomepizole, liposomal amphotericin B, ancrod, rifabutin, succimer, somatropin, digoxin immune Fab (digoxin antibody), liothyronine (T3) and many more. Though these drugs may be life saving for some patients, they are commercially difficult to obtain. Governments in developed countries offer tax benefits and other incentives to pharmaceutical companies for developing and marketing orphan drugs (e.g. Orphan Drug Act in USA).

 

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