Drugs and Biologics


What is a drug?

The Federal Food Drug and Cosmetic Act (FD&C Act) and the FDA regulations define the term drug, in part, by reference to its intended use, as “articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease” and “articles (other than food) intended to affect the structure or any function of the body of man or other animals.” Therefore, almost any ingested or topical or injectable product that, through its label or labeling (including internet websites, promotional pamphlets, and other marketing material), is claimed to be beneficial for such uses will be regulated by FDA as a drug. The definition also includes components of drugs, such as active pharmaceutical ingredients.
See FDA Guidance on Human Drugs.
Biological products may also be considered drugs within the meaning of the FD&C Act. Biological products (42 U.S.C. 262(i)) include, among other products, bacterial vaccines, allergenic extracts, gene therapy products, growth factors, cytokines, and monoclonal antibodies.

What is an investigational drug?

Investigational new drug means a new drug or biological drug that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes. The terms "investigational drug" and "investigational new drug" are deemed to be synonymous for purposes of this part. (21 CFR 312.3(b))

What is a clinical investigation?

Clinical investigation means any experiment in which a drug is administered or dispensed to, or used involving, one or more human subjects. For the purposes of this part, an experiment is any use of a drug except for the use of a marketed drug in the course of medical practice. (21 CFR 312.3(b))
Note: The fact that a clinical investigation can be any experiment in which a drug is administered or dispensed to, or used involving, one subject distinguishes it from the Common Rule’s definition of research under 45 CFR 46. 102, which requires “a systematic investigation, including research development, testing, and evaluation, designed to develop or contribute to generalizable knowledge.”

What is a sponsor?

Sponsor means a person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. The sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator. (21 CFR 312.3(b))