Clinical Studies (Overview)

The new drug application (NDA) is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale in the United States. To obtain this authorization, a drug manufacturer submits in an NDA nonclinical (animal) and clinical (human) test data and analyses, drug information, and descriptions of manufacturing procedures.

An NDA must provide sufficient information, data, and analyses to permit FDA reviewers to reach several key decisions, including:

• Whether the drug is safe and effective for its proposed use(s), and whether the benefits of the drug outweigh its risks.

• Whether the drug's proposed labeling is appropriate, and, if not, what the drug's labeling should contain.

• Whether the methods used in manufacturing the drug and the controls used to maintain the drug's quality are adequate to preserve the drug's identity, strength, quality, and purity.

The purpose of preclinical work--animal pharmacology/toxicology testing--is to develop adequate data to undergird a decision that it is reasonably safe to proceed with human trials of the drug. Clinical trials represent the ultimate premarket testing ground for unapproved drugs. During these trials, an investigational compound is administered to humans and is evaluated for its safety and effectiveness in treating, preventing, or diagnosing a specific disease or condition. The results of this testing will comprise the single most important factor in the approval or disapproval of a new drug.

Although the goal of clinical trials is to obtain safety and effectiveness data, the overriding consideration in these studies is the safety of those in the trials. CDER monitors the study design and conduct of clinical trials to ensure that people in the trials are not exposed to unnecessary risks.