From Safety Studies to Human Trials

The nonclinical safety assessment for marketing approval of a pharmaceutical usually includes pharmacology studies, general toxicity studies, toxicokinetic and nonclinical pharmacokinetic studies, reproduction toxicity studies, genotoxicity studies and, for drugs that have special cause for concern or are intended for a long duration of use, an assessment of carcinogenic potential. Other nonclinical studies to assess phototoxicity, immunotoxicity, juvenile animal toxicity and abuse liability should be conducted on a case-by-case basis.

Clinical trials are research studies performed on people to evaluate a medical, surgical, or behavioral intervention. They are the primary way that researchers find out if a new treatment, like a new drug or diet, or medical device (for example, a pacemaker) is safe and effective in people. Often a clinical trial is used to learn if a new treatment is more effective and/or has fewer harmful side effects than the standard treatment.

Other clinical trials test ways to find a disease early, sometimes before there are symptoms. Still, others try ways to prevent health problems. A clinical trial may also look at how to make life better for people living with a life-threatening disease or a chronic health problem. Clinical trials sometimes study the role of caregivers or support groups.

Before the U.S. Food and Drug Administration (FDA) approves a clinical trial, scientists perform laboratory tests and studies in animals to test a potential therapy's safety and efficacy. If these studies show favorable results, the FDA gives approval for the intervention to be tested in humans.