Conclusions
The Orphan Drug Act of 1983 recognized the reality of the marketplace as an important factor influencing the development of new medications. Since 1983, hundreds of orphan compounds have been "adopted" by pharmaceutical developers and shepherded to FDA approval. Specialized centers such as CODR have emerged to address the unmet needs of orphan diseases, often in collaboration with private industry. As the Orphan Drug Act approaches its 30th anniversary, it continues to help people with epilepsy and many other rare disorders.
For more information, a recent book, Orphan Drugs in Epilepsy, addresses this topic as well.
Suggested Reading
Coles LD, Cloyd JC. The role of academic institutions in the development of drugs for rare and neglected diseases. Clin Pharmacol Ther. 2012;92:193-202.
Epilepsy Foundation. Incidence and prevalence of epilepsy. http://epilepsyfoundation.org/aboutepilepsy/whatisepilepsy/
statistics.cfm Accessed October 11, 2012.
Gandey A. FDA approves clobazam add-on for Lennox syndrome. October 24, 2011. http://www.medscape.com/viewarticle/752080 Accessed October 11, 2012.
Nevarez AK. Orphan drugs. May 26, 2009. http://www.ice-epilepsy.org/orphan-drugs.html Accessed October 11, 2012.
US Food and Drug Administration. FDA approves new drug to treat severe form of epilepsy. November 20, 2008. http://www.fda.gov/NewsEvents/Newsroom/
PressAnnouncements/2008/ucm116980.htm Accessed October 11, 2012.