Orphan Drug Act
An orphan drug is a pharmaceutical product developed to treat an especially rare medical condition.
A research paper on the Orphan Drug Act may want to examine the history of the legislation. Other ways to examine the Orphan drug Act in a research paper include the following:
- Previous Acts that fell short
- Previous drugs that were once orphan drugs
- The incentives, tax breaks and fast-track approval processes for orphan drugs
- Orphan diseases
The Orphan Drug Act of 1983 was legislation passed by Congress in order to facilitate the development and production of drugs designed to treat very rare conditions.
Orphan Drug Act
and Testing
In 1962, in response to the thalidomide crisis, Congress passed the Kefauver-Harris Amendment to ensure that all drugs manufactured for use in the United States pass rigorous trials. However, certain drugs, known as orphan drugs, often cannot meet some of the statistical burdens for testing, since the conditions that they treat are extremely rare. In 1982, Representative Henry Waxman sponsored the Orphan Drug Act (ODA) in order to approve drugs designed to treat less than 200,000 people. These drugs must still follow FDA regulations regarding safety and efficacy, but there are incentives, tax breaks and fast-track approval processes for these orphan drugs.
Orphan Drug Act
Success
The Orphan Drug Act has been seen as a success story in the field of pharmaceuticals. Before passage, only 38 drugs were approved to treat orphan diseases in the United States. Thanks to the ODA some 1100 drugs were approved in the U.S. by 2004. Drug maker Pfizer specifically established an orphan drug division to focus on research and development. Pharmaceuticals such as Abilify, Botox, Vioxx, and Cialis originally started out as orphan drugs.