TIMELINE
"Quincy, M.E." Seldom Silent, Never Heard
March 1981
Actor Jack Klugman portrays a man with Tourette's Syndrome, who attempts to convince Congress to act on passing an Orphan Drug Act.
Congress Passes the Orphan Drug Act
January 1983
The Orphan Drug Act was passed in January of 1983 (mainly due to Republican Congressman Henry Waxman and Abbey S. Meyers) to motivate further research and development of orphan drugs. Before the Orphan Drug Act, many developers did not work on developing treatments for orphan diseases.
First Orphan Drug Developed
February 1983
Panhematin, by Ovation Pharmaceuticals, is the first orphan drug approved under the Orphan Drug Act, and is used to treat acute porphyrias. The condition typically affects only 10 out of every 100,000 people, and can lead to permanent paralysis when untreated.
NORD Founded
May 1983
Abbey S. Meyers, who worked with Henry Waxman to pass the Orphan Drug Act, founds the National Organization for Rare Disorders. She is also named president.
Orphan Drug Amendments
August 1985
The definition of a rare disease changes to one that either affects less that 200,000 individuals in the U.S.A., or that affects over 200,000 individuals in the U.S.A. without sufficient funds for development and distribution.
Focus on AIDS
March 1987
With the increasing death rate for AIDS, President Ronald Reagan was quick to accept the FDA's decision to give orphan approval to zidovudine (AZT or Retrovir), and Congress approved emergency funding to assist states in their process of purchasing the drug.
Rise of Botox
December 1989
Botulinum toxin type A was approved and given marketing approval on December 29, 1989. It was originally designated on March 22, 1984. As an orphan drug, the purpose of Botox is to treat strabismus and biepharospasms associated with dystonia. These are facial muscle contractions, in this case specifically related to movements in the eye.
Orphan Drugs in...Japan
October 1993
The Japanese government revised the pharmaceutical law to aid in the research/development of specifically orphan drugs on October 1, 1993. The definition of an orphan disease was one that affects 4 in every 10,000 people.
Orphan Drugs in...Australia
January 1998
Australia's Orphan Drug Policy was set up in early 1998, and was initiated in late 1997. Australia defines an orphan drug as one that affects at most 2 of every 10,000 people, or about 2000 people of the total population of 18 million. Research and development for orphan medicine in this country is not supported by grants.
Orphan Drugs in...the European Union
December 1999
The European Parliament adopted Regulation (EC) No 141/2000 on December 16, 1999. This is more commonly known as the Orphan Regulation. It establishes the procedure for the designation of orphan medicines and lists incentives for developing and marketing these drugs.
Orphan Drugs in...South Korea
2003
In 2003, South Korea adopted an Orphan Drug Policy. The known prevalence for an orphan disease has to be equal to or less than 100/10,000,000.
Orphan Drug Restatement
2013
The FDA starts to fear that people are misusing the Orphan Drug Act and its rules. As a result of this, they restate the the definitions and make revisions to their policies.
Orphan Drugs in...the Philippines
March 2016
Before the end of his presidential term, President Aquino of the Philippines signed a law that assists people in obtaining treatment for rare and orphan diseases.
