Orphan Drugs

The term orphan drug refers to a product that treats a rare disease affecting fewer than 200,000 Americans. Orphan drugs help the companies that manufacture them, under the Orphan drug act. Under the act a small company can pick up a product that would be worth anywhere from $5 million to $20 million a year. The orphan drug act has helped in the development of products to treat drug addiction, leprosy, hemophilia, and rare cancers, as well as diseases most people have never heard of, such as cryptosporidiosis (an infection caused by a protozoan parasite found in animals’ intestines that causes diarrhea, fever, weight loss, and lymph node enlargement) and neurocysticerosis. In the past the FDA under the drug act has approved few years ‘ 41 orphan drugs. One of the premier examples of how well the Orphan Drug Act can work came with the approval of the drug PEG-ADA (adenosine deaminase and enzyme the body usually produces on its own). “This was a breakthrough” FDA’s Haffner said of PEGnology, the enzyme replacement process developed by Enzon. “If it works the way it’s thought it will, this technology will change the way we can provide drugs to the patients”.
The history of the Orphan Drug Act
FDA set up the offices of Orphan Product Development in 1982 to focus on drugs, medical devices, foods for medical purposes, and biologics such as immune globulin for rare disorders. President Reagan signed the Orphan Drug Act into law on JAN. 3 1983. It guarantees the developer of an orphan drug seven years of market exclusivity and 50 percent tax credit for certain clinical research expenses. Imagine Pepsi or Coke having a monopoly on each other’s soft drink, that would be the equivalent on that. Initially, the act applied to only patient populations when it could be shown there was little or no hope of recovering development cost from sales in the United States. A later amendment to the act defined 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.”
The Orphan Drug Act has been a major success and a model federal program. The FDA has designated Fifteen years ago after its passage, more than 150 orphan drugs have been approved for marketing in the United States, and 800. Additionally, Japan, Singapore, and Australia have enacted orphan drug legislation, and the European Union is expected to enact a similar law in 1999. Approximately 85% of orphan drugs are treatments for diseases affecting fewer