Orphan drugs are given special status by the government due to the rarity of the conditions they treat. Because these diseases are so rare, the market for them is small, making orphan drugs economically undesirable to produce. Government assistance, via tax breaks, subsidy for research, and exclusivity gives companies the incentive to produce these drugs.
In 1983, the United States legislature enacted the Orphan Drug Act, with the aim of encouraging drug companies to provide a home for promising treatments for such disorders. The government offers incentives to encourage manufacturers to develop and market drugs that might not have been developed otherwise.
These drugs go through the normal FDA approval process. Once an orphan drug is approved for marketing, a doctor may prescribe it to his or her patients. If the drug is still in the experimental stage, the manufacturer may make it available to individuals on a compassionate-use basis, or individuals may be able to enroll in a clinical trial.
Do not take XYREM if you:
Talk with a XYREM Patient Connection Mentor
The XYREM Patient Connection program provides an opportunity for you to talk one-on-one with a mentor who has been diagnosed with excessive daytime sleepiness and/or cataplexy with narcolepsy, and has been treated with XYREM.