Phocomelia is a “congenital malformation” of development where the upper appendage of an arm or leg is absent so the hands or feet are attached to the body like stumps. The word phocomelia combines phoco—denoting seal and melia—denoting limb to signify a “limb like a seal’s flipper".

From 1956 to 1962, approximately 10,000 children were born with severe malformities, including phocomelia, because their mothers had taken thalidomide during pregnancy. In 1962, in reaction to the tragedy, the United States Congress enacted the Kefauver-Harris Amendment.

Among other things, the amendment requires drug manufacturers to show the effectiveness of their products as well as their safety, to report adverse events to the FDA, and to ensure that their advertisements to physicians disclose the risks as well as the benefits of their products. Informed consent was required from participants in clinical studies. The FDA also was given jurisdiction over prescription drug advertising. In addition, the agency was required to approve a regulatory submission known as a new drug application before a company could market a new drug and be allowed to issue good manufacturing practice guidelines governing how drugs were to be manufactured. Inspection of drug manufacturers was mandated every 2 years.

The law was signed by President John F. Kennedy on October 10, 1962.

The Kefauver-Harris Amendment provided the modern scenario for approving new drugs:

• Initial drug discovery.
• Preclinical (animal) testing.
• An investigational new drug application (IND) outlines what the sponsor of a new drug proposes for human testing in clinical trials.
• Phase 1 studies (typically involve 20 to 80 healthy people).
• Phase 2 studies (typically involve a few dozen to about 300 people with the ailment that the new drug is intended to treat).
• Phase 3 studies (typically involve several hundred to about 3,000 people with the ailment that the new drug is intended to treat).
• The pre-NDA period, just before a new drug application (NDA) is submitted. A common time for the FDA and drug sponsors to meet.
• Submission of an NDA is the formal step asking the FDA to consider a drug for marketing approval.
• After an NDA is received, the FDA has 60 days to decide whether to file it so it can be reviewed.
• If the FDA files the NDA, an FDA review team is assigned to evaluate the sponsor's research on the drug's safety and effectiveness.
• The FDA reviews information that goes on a drug's professional labeling (information on how to use the drug).
• The FDA inspects the facilities where the drug will be manufactured as part of the approval process.
• FDA reviewers will approve the application or find it either "approvable" or "not approvable."
• Post marketing study commitments, also called Phase 4 commitments.

See also

Federal pharmacy law

References