From an antacid to a new cancer treatment, every drug must be proven safe and effective in controlled clinical trials before the U.S. Food and Drug Administration (FDA) allows it to be sold in the United States.

When federal law first required controlled clinical trials in 1962, most people didn’t know how to do them and they were rampant with problems, according Dr. Robert Temple, director of the FDA’s Office of Medical Policy. Over the years, FDA experts evaluated the many shortcomings of the clinical trials they saw and worked to fix them. To this end, the FDA established regulations and guidance to ensure that sponsors of clinical trials, medical researchers (investigators), and ethics committees (institutional review boards) understand how they can effectively carry out their responsibilities and comply with federal law.

"As FDA expertise evolved, so did controlled clinical trials, and the quality of their design, conduct, analysis, and interpretation improved," says Robert T. O’Neill, Ph.D., director of the FDA’s Office of Biostatistics, who has seen much of the evolution of controlled trials since he joined the FDA in 1971. "FDA has been the major force behind the development of good principles for the design and interpretation of controlled trials. We’ve promoted, fed, and cared for controlled clinical trials as a critical force in drug development, and we continue to do so today."

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