What is meant by an "investigational drug"?

An "investigational drug" refers to a substance that is in the process of being studied and evaluated for safety and efficacy but has not yet received approval from regulatory authorities, such as the Food and Drug Administration (FDA), for general use in the population. This designation is critical because it indicates that the drug is part of research aimed at understanding its potential benefits and risks before it can be made widely available to patients.

Investigational drugs are typically administered in the context of clinical trials, where researchers gather necessary data to support potential approval. During these trials, the drugs are assessed through multiple phases to ensure they meet safety and efficacy standards. It's important to understand that, although investigational drugs may be used in clinical trials, they are not available for general prescribing or use outside these tightly controlled studies until they gain official approval.

The concept of an investigational drug is relevant to patient safety, regulatory compliance, and the process of drug development, making it a fundamental aspect of pharmaceutical law and practice.