Federal Food, Drug, and Cosmetic Act and the Kefauver-Harris Amendments

United States, federal statute. The Federal Food, Drug, and Cosmetic Act was signed in June 1938 after the Elixir Sulfanilamide poisonings of 1937 and is codified at Title 21 of the United States Code, chapter 9. The Kefauver-Harris Drug Efficacy Amendments of 1962, passed after thalidomide, added the demand that a drug be shown effective through 'substantial evidence' from 'adequate and well-controlled investigations', required informed consent from trial participants, and gave the FDA control over prescription drug advertising. Primary text: the FDA's FD&C Act page links the current code.

What it changed: before 1938 a medicine could be sold with no safety review; before 1962 it could be sold with no proof of benefit. Section 505 of the Act is the legal home of the new drug application, and the 'substantial evidence' standard is the yardstick against which every later shortcut (accelerated approval, breakthrough designation, single-arm approvals in rare cancers) is measured. The FDA has interpreted the standard as usually two adequate and well-controlled trials, or one trial plus confirmatory evidence since the 1997 Modernization Act, and oncology is where the one-trial reading is most often used.

The arguments: patient groups and some economists say the effectiveness requirement slows access and that the 1962 amendments lengthened development by years; defenders answer that it is what stops ineffective drugs reaching patients who cannot afford wasted time. The 1962 provisions also created the Drug Efficacy Study Implementation review, which removed hundreds of pre-1962 products from the market for lacking evidence.