Looking Back at the Kefauver-Harris Drug Amendments and their Meaning

By: John P. Swann, Ph. D.

The Drug Amendments of 1962, also known as the Kefauver-Harris Drug Amendments, became law five decades ago. But this law’s importance grows with each passing year, making Americans safer than ever from unsafe and ineffective medicines.

John P. Swann, Ph. D.To understand why this law stands today as a pillar of public health in America, it helps to look at how our history shaped it.

There was a system of drug controls in place as early as 1905 that took effectiveness into account, but it was voluntary and administered privately by the American Medical Association. Congress passed laws that required effectiveness from the early 1940s on, but only for selected medicines, such as insulin and penicillin. In 1941, FDA developed regulations to ensure good manufacturing practices to ensure a product’s quality and purity, but only for one drug category. Technically, the Federal Trade Commission had been regulating drug advertising since 1938, but there was little strength in its hold on this industry. And the 1938 Food, Drug, and Cosmetic Act required evidence of a drug’s safety, but the nature of that proof and oversight over how it should be developed were not that clear.

In 1959 Sen. Estes Kefauver began hearings that focused on the high cost of medicines—reflected in the comprehensive bill he introduced in April 1961. But priorities shifted substantially in the next year with the global thalidomide disaster, narrowly averted here, in which a sedative used to treat morning sickness caused thousands of birth defects around the world. Substantial legislative input from FDA helped shape the law that President Kennedy signed on October 10, 1962. And it changed everything:  requirements for therapeutic viability of drugs, veracity in marketing, the proper conduct of investigations, verifiable production controls, patient protections, actual FDA assent to constitute approval, and rigorous proof as the essential element of a drug application.

FDA assembled clinical experts to advise the Agency on drugs previously approved for safety only. They reviewed the available evidence on the effectiveness of those drugs and found that on average 4 out of 10 drugs approved before 1962 and still marketed—medicines that physicians prescribed to their sick patients—either did not work or needed more—often much more—evidence that they did. In the following years FDA removed more than 1,000 of these from the market. At the same time, the agency further called upon therapeutic experts through the systematic use of advisory committees to offer their insights into approval decisions, decisions that still ultimately rested with FDA.

In sorting out this therapeutic mess from the pre-1962 era, the investigational, manufacturing, and regulatory communities reached an understanding about what constituted acceptable evidence, which generally meant randomized, well-controlled clinical trials. While that definition shifted over the following years to accommodate, for example, the needs of gravely ill patients facing few if any treatment options, these changes did not come at the expense of good clinical evidence. Science remained the benchmark of Kefauver-Harris’s legacy.

So, the Drug Amendments of 1962 elevated medical practice, pharmaceutical manufacturing, and public health by inserting a much greater degree of certainty in the way drugs are tested, manufactured, approved, advertised, prescribed, dispensed, and taken.

John P. Swann, Ph. D., is an FDA historian