How New Drugs Get Approved by the FDA
Drugs go through several phases of clinical (human) testing after initial laboratory studies are complete.
• Phase I studies: Involve a small group of people, usually 20 to 80. Those trials test safety of the new drug, determine the maximum dose as well as a safe range of doses that can be given. Phase I trials also start collecting information on side effects.
• Phase II studies: Involve several hundred people to determine whether the new drug is effective, as well as to continue to test safety, best dosage and determine side effects.
• Phase III studies: Involve several thousand people to confirm what was learned in phase II studies, monitor side effects or compare the new drug to a similar treatment.
After successful Phase III studies, the pharmaceutical company will submit a New Drug Application to the U.S. Food and Drug Administration (FDA). An FDA committee will review the data and decide whether the drug should be approved or should be tested further.
Even if the drug is approved, clinical trials usually continue. These trials, sometimes called Phase IV studies, help determine the best use of the drug, new uses for the drug (called new indications), or guidelines for prescribing certain doses. Information is continually collected regarding risks and benefits for using the drugs. That information helps doctors decide which patients will benefit from the drug, and which should avoid it.
When pharmaceutical companies start testing new compounds that could become drugs, the compound is said to be “in the pipeline.” The pharmaceutical company will assign the compound a series of letters and numbers, then a generic name. For instance, compound AMG162 has moved far enough along the pipeline to be named denosumab. When denosumab is ready for FDA review, it will get a trade (brand) name. For example, Enbrel is the trade name for generic etanercept.
