Clinical trials represent the ultimate premarket testing ground for unapproved drugs. During these trials, an investigation 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.
This website provides guidance for conducting clinical trials with investigational drugs and information for compliance with the regulations of the Food and Drug Administration. For clinical investigators who are not familiar with FDA regulations and the guidance that the Agency provides for good clinical practice, there are two sources of information which investigators should read first. Federal Regulations for Clinical Investigators contains Code of Federal Regulations pertaining to the responsibilities of clinical investigators. The second source of information for new clinical investigators is Guidance for Industry: Good Clinical Practice Consolidated Guideline. This guidance, developed with the European Union, Japan, and other countries represents the Agency's current thinking on good clinical practice, and contains sixty-two definitions of terms used in clinical trials. Additional information on new drug development and review process, plus organization, contact, and meeting information can be found on the Drug Approval Application webpage.