The following glossary was prepared to help the consumer become familiar with many of the common terms used in clinical trials.
ADVERSE REACTION: (Adverse Event.) An unwanted effect caused by the administration of drugs. Onset may be sudden or develop over time (See Side Effects).
ADVOCACY AND SUPPORT GROUPS: Organizations and groups that actively support participants and their families with valuable resources, including self-empowerment and survival tools.
APPROVED DRUGS: In the U. S., the Food and Drug Administration (FDA) must approve a substance as a drug before it can be marketed. The approval process involves several steps including pre-clinical laboratory and animal studies, clinical trials for safety and efficacy, filing of a New Drug Application by the manufacturer of the drug, FDA review of the application, and FDA approval/rejection of application (See Food and Drug Administration).
ARM: Any of the treatment groups in a randomized trial. Most randomized trials have two “arms,” but some have three “arms,” or even more (See Randomized Trial).
BASELINE: 1. Information gathered at the beginning of a study from which variations found in the study are measured. 2. A known value or quantity with which an unknown is compared when measured or assessed. 3. The initial time point in a clinical trial, just before a participant starts to receive the experimental treatment which is being tested. At this reference point, measurable values such as CD4 count are recorded. Safety and efficacy of a drug are often determined by monitoring changes from the baseline values.
BIAS: When a point of view prevents impartial judgment on issues relating to the subject of that point of view. In clinical studies, bias is controlled by blinding and randomization (See Blind and Randomization).
BLIND: A randomized trial is “Blind” if the participant is not told which arm of the trial he is on. A clinical
trial is “Blind” if participants are unaware on whether they are in the experimental or control arm of the study; also called masked. (See Single Blind Study and Double Blind Study).
CLINICAL: Pertaining to or founded on observation and treatment of participants, as distinguished from theoretical or basic science.
CLINICAL ENDPOINT: See Endpoint.
CLINICAL INVESTIGATOR: A medical researcher in charge of carrying out a clinical trial’s protocol.
CLINICAL TRIAL: A clinical trial is a research study to answer specific questions about vaccines or new therapies or new ways of using known treatments. Clinical trials (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people. Trials are in four phases: Phase I tests a new drug or treatment in a small group; Phase II expands the study to a larger group of people; Phase III expands the study to an even larger group of people; and Phase IV takes place after the drug or treatment has been licensed and marketed. (See Phase I, II, III, and IV Trials).
COHORT: In epidemiology, a group of individuals with some characteristics in common.
COMMUNITY-BASED CLINICAL TRIAL (CBCT): A clinical trial conducted primarily through primary-care physicians rather than academic research facilities.
COMPASSIONATE USE: A method of providing experimental therapeutics prior to final FDA approval for use in humans. This procedure is used with very sick individuals who have no other treatment options.