Glossary

GLOSSARY

Adverse Event (AE)

A negative experience encountered by an individual during the course of a clinical trial, that is associated with the drug. An AE can include previously undetected symptoms, or the exacerbation of a pre-existing condition. When an AE has been determined to be related to the investigational product, it is considered an Adverse Drug Reaction.


Blinding

The process through which on or more parties to a clinical trial are unaware of the treatment assignments. In a single-blinded study, both the subjects and the investigators are unaware of the treatment assignments. Also, in a double-blinded study, the monitors and sometimes the data analysts are unaware. “Blinded” studies are conducted to prevent the unintentional biases that affect subject data treatment assignments are known.


Clinical Investigation

A systematic study designed to evaluate a product (drug, device, or biologic) using human subjects, in the treatment, prevention, or diagnosis of a disease or condition, as determined by the product’s benefits relative to its risks.


Clinical Research

Study of drug, biologic or device in human subjects with the intent to discover potential beneficial effects and/or determine its safety and efficacy. Also called clinical study and clinical investigation. Note that in this manual, this term is used in its narrow sense as used by the FDA. Thus, it does not encompass all the research that is carried out in the clinical setting (e.g., health services research).


Clinical Research Coordinator (CRC)

Site administrator for the clinical study. Duties are delegated by the investigator. Also called research, study or healthcare coordinator, and data manager, research nurse or protocol nurse.


Clinical Trial

Any investigation in human subjects intended to determine the clinical pharmacological, pharmacokinetic, and/or other pharmacodynamics effects of an investigational agent, and/or to identify any adverse reactions to an investigational agent to assess the agent’s safety and efficacy.


Consent Form

A document explaining all relevant study information to assist the study volunteer in understanding the expectations and requirements of participation in a clinical trial. This document is presented to and signed by the study subject.


Double-Blind

The design of a study in which neither the investigator or the subject knows which medication (or placebo) the subject is receiving.


Drug

As defined by the Food, Drug and Cosmetic Act, drugs are “articles (other than food) intended for the use in diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animals, or to affect the structure or any function of the body of man or other animals.”


Ethics Committee

An independent group of both medical and non-medical professionals who are responsible for verifying the integrity of a study and ensuring the safety, integrity, and human rights of the study participants.


Food and Drug Adninistration (FDA)

Within the Department of Health and Human Services. Enforces Food, Drug and Cosmetics Act and related federal public health laws. Grants IND, IDE, PMA and NDA approvals.


Good Clinical Practice (GCP)

International ethical and scientific quality standard for designing, conducting, monitoring, recording, auditing, analyzing and reporting studies. Insures that the data reported is credible and accurate, and that subject’s rights and confidentiality are protected.


Human Subject

A patient or healthy individual participating in a research study. A living individual about whom an investigator obtains private information or data through intervention or interaction.


Informed Consent

The voluntary verification of a patient’s willingness to participate in a clinical trial, along with the documentation thereof. This verification is requested only after complete, objective information has been given about the trials, including an explanation of the study’s objectives, potential benefits, risks and inconveniences, alternation therapies available, and of the subject’s rights and responsibilities in accordance with the current revision of the Declaration of Helsinki.

Institutional Review Board (IRB)

An independent group of professionals designated to review and approve the clinical protocol, informed consent forms, study advertisements, and patient brochures, to ensure that the study is safe and effective for human participation. It is also the IRB’s responsibility to ensure that the study adheres to the FDA’s regulations.


Investigator

A medical professional, usually a physician but may also be a nurse, pharmacist or other health care professional, under whose direction an investigational drug is administered or dispensed. A principal investigator is responsible for the overall conduct of the clinical trial at his/her site.


Open-Label Study

A study in which all parties, (patient, physician and study coordinator) are informed of the drug and dose being administered. In an open-label study, none of the participants are given placebos. These are usually conducted with Phase I & II studies.


Phase I Study

The first of four phases of clinical trials, Phase I studies are designed to establish the effects of a new drug in humans. These studies are usually conducted on small populations of healthy humans to specifically determine a drug’s toxicity, absorption, distribution and metabolism.


Phase II Study

After the successful completion of phase I trials, a drug is then tested for safety and efficacy in a slightly larger population of individuals who are afflicted with the disease or condition for which the drug was developed.


Phase III Study

The third and last pre-approval round of testing of a drug is conducted on large populations of afflicted patients. Phase III studies usually test the new drug in comparison with the standard therapy currently being used for the disease in question. The results of these trials usually provide the information that is included in the package insert and labeling.


Phase IV Study

After a drug has been approved by the FDA, phase IV studies are conducted to compare the drug to a competitor, explore additional patient populations, or to further study any adverse events.


Placebo

An inactive substance designed to resemble the drug being tested. It is used as a control to rule out any psychological effects testing may present. Most well-designed studies include a control group which is unwittingly taking a placebo.


Protocol

A detailed plan that sets forth the objectives, study design, and methodology for a clinical trial. A study protocol must be approved by an IRB before investigational drugs may be administered to humans.


Randomization

Study participants are usually assigned to groups in such a way that each participant has an equal chance of being assigned to each treatment (or control) group. Since randomization ensures that no specific criteria are used to assign any patients to a particular group, all the groups will be equally comparable.


Research

Systematic investigation designed to develop or contribute generalizable knowledge. Includes Clinical Research.


Research Team

Investigator, subinvestigator and clinical research coordinator involved with study.


Sponsor

Individual, company, institution or organization taking responsibility for initiation, management and financing of study.


Sub-investigator

Helps design and conduct investigation at a study site.


Subject/Study Subject

Participants in a study: See “Human Subject.”