ned an orphan product as one with a potential patient population of fewer than 200,000 people. Under the Orphan Drug Act, the FDA makes grants for drug development, assists the drug developer in the designing the clinical studies required for marketing and can speed up the drug approval process. “A lot of these drugs are for very serious and life threatening disorders, in some cases affecting only a few hundred people. You have the smaller NDA (new drug application) and a group of very needy people, and that’s what expedites it. But the safety and the efficacy requirements are the same” Marlene Haffner FDA’s director of orphan product development. Since the enactment of the Orphan Drug Act, 41 drugs for rare diseases have been developed and brought onto the market. Activity in orphan products, which, in addition to drugs, can include biologics, medical devices, and foods marketed for medical purposes, is researching an all time high. According to Haffner, FDA has designated as orphans 375 drugs and at least 150 are being actively developed or are going through the approval process. The federal award grant available to fund orphan drug development have increased steadily from $500,000 in 1983 to $7.5 million in 1990. However hardly any of the federal grants go to private organizations such as NORD. Today’s more common definition of Orphan Drug”. Another definition of “Orphan Drug” refers to a drug that will serve so few patients that it will be commercially impractical for a manufacturer to sponsor it. Orphan drug also refers to compounds in the public domain for which there can be no patent protection once a company goes through expensive efficacy test and gets FDA approval, so that a competitor could immediately begin producing a generic copy at a fraction of the cost. The phrase was coined in a 1968 editorial in American Journal of Pharmacy, “Homeless or Orphan Drugs.”Accom...
