Hospitals

Comprehensive Care + 24 Hour Emergency

Glossary of Terms

The following glossary is to help you become familiar with the most common terms used in clinical trials, taken from "Taking Part in Clinical Trials:  What Pet Owners Need to Know" from the The Perseus Foundation:

ADVERSE REACTION: (Adverse Event.) An unwanted effect caused by the administration of drugs. Onset may be sudden or develop over time.
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.
ARM: Any of the treatment groups in a randomized trial. Most randomized trials have two “arms,” but some have three “arms,” or even more.
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.
BLIND: A randomized trial is “Blind” if the participant is not told which arm of the trial he is on. A clinical trial 1s “Blind” if participants are unaware on whether they are in the experimental or control arm of the study; also called masked.
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.
COMPLEMENTARY AND ALTERNATIVE THERAPY: Broad range of healing philosophies, approaches, and therapies that Western (conventional) medicine does not commonly use to promote well-being or treat health conditions. Examples include acupuncture, herbs, etc.
CONTRAINDICATION: A specific circumstance when the use of certain treatments could be harmful.
CONTROL: A control is the nature of the intervention control.
CONTROL GROUP: The standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental by a drug that is detrimental to the participant’s health. The level of toxicity associated with a drug will vary depending on the condition which the drug is used to treat.
CONTROLLED TRIALS: Control is a standard against which experimental observations may be evaluated. In clinical trials, one group of participants is given
an experimental drug, while another group (i.e., the control group) is given either a standard treatment for the disease or a placebo.
DIAGNOSTIC TRIALS: Refers to trials that are conducted to find better tests or procedures for diagnosing a particular disease or condition. Diagnostic trials usually include people who have signs or symptoms of the disease or condition being studied.
DOSE-RANGING STUDY: A clinical trial in which two or more doses of an agent (such as a drug) are tested against each other to determine which dose works best and is least harmful. 
DOUBLE-BLIND STUDY/DOUBLE-MASKED STUDY: A clinical trial design in which neither the participating individuals nor the study staff knows which participants are receiving the experimental drug and which are receiving a placebo (or another therapy). Double-blind trials are thought to produce objective results, since the expectations of the doctor and the participant about the experimental drug do not affect the outcome; also called double-masked study.
DRUG-DRUG INTERACTION: A modification of the effect of a drug when administered with another drug. The effect may be an increase or a decrease in the action of either substance, or it may be an adverse effect that is not normally associated with either drug.
EFFICACY: (Of a drug or treatment). The maximum ability of a drug or treatment to produce a result regardless of dosage. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.  In the procedure mandated by the FDA, Phase II clinical trials gauge efficacy, and Phase III trials confirm it
ELIGIBILITY CRITERIA: Summary criteria for participant selection; includes Inclusion and Exclusion criteria.
ENDPOINT: Overall outcome that the protocol is designed to evaluate. Common endpoints are severe toxicity, disease progression, or death.
EPIDEMIOLOGY: The branch of medical science that deals with the study of incidence and distribution and control of a disease in a population.
EXCLUSION/INCLUSION CRITERIA: See Inclusion/Exclusion Criteria.
EXPERIMENTAL DRUG: A drug that is not FDA licensed for use in humans, or as a treatment for a particular condition. 
FOOD AND DRUG ADMINISTRATION (FDA): The U.S. Department of Health and Human Services agency responsible for ensuring the safety and
effectiveness of all drugs, biologics, vaccines, and medical devices
INVESTIGATIONAL NEW DRUG: A new drug, antibiotic drug, or biological drug that is used in a clinical investigation. It also includes a biological product used in vitro for diagnostic purposes.
OFF-LABEL USE: A drug prescribed for conditions other than those approved by the FDA.
OPEN-LABEL TRIAL: A clinical trial in which doctors and participants know which drug or vaccine is being administered.
ORPHAN DRUGS: An FDA category that refers to medications used to treat diseases and conditions that occur rarely. There is little financial incentive for the pharmaceutical industry to develop medications for these diseases or conditions. Orphan drug status, however, gives a manufacturer specific financial incentives to develop and provide such medications.
PHARMACOKINETICS: The processes (in a living organism) of absorption, distribution, metabolism, and excretion of a drug or vaccine.
PHASE I TRIALS: Initial studies to determine the metabolism and pharmacologic actions of drugs in humans, the side effects associated with
increasing doses, and to gain early evidence of effectiveness; may include healthy participants and/or patients.
PHASE II TRIALS: Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition under study and to determine the common short-term side effects and risks.
PHASE III TRIALS: Expanded controlled and uncontrolled trials after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional information to evaluate the overall benefit-risk relationship of the drug and provide and adequate basis for physician labeling.
PLACEBO: A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment. No sick participant receives a placebo if there is a known beneficial treatment.
PLACEBO CONTROLLED STUDY: A method of investigation of drugs in which an inactive substance (the placebo) is given to one group of participants,   while the drug being tested is given to another group. The results obtained in the two groups are then compared to see if the investigational treatment is more effective in treating the condition.
PLACEBO EFFECT: A physical or emotional change, occurring after a substance is taken or administered, that is not the result of any special property of the substance. The change may be beneficial, reflecting the expectations of the participant and, often, the expectations of the person giving the substance.
PRECLINICAL: Refers to the testing of experimental drugs in the test tube or in animals - the testing that occurs before trials in humans may be carried out.
PROTOCOL: A study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment
RANDOMIZATION: A method based on chance by which study participants are assigned to a treatment group. Randomization minimizes the differences among groups by equally distributing people with particular characteristics among all the trial arms. The researchers do not know which treatment is better. From what is known at the time, any one of the treatments chosen could be of benefit to the participant.
RANDOMIZED TRIAL: A study in which participants are randomly (i.e., by chance) assigned to one of two or more treatment arms of a clinical trial. Occasionally placebos are utilized.
RISK-BENEFIT RATIO: The risk to individual participants versus the potential benefits. The risk/benefit ratio may differ depending on the condition being treated.
SIDE EFFECTS: Any undesired actions or effects of a drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental drugs must be evaluated for both immediate and long-term side effects.
SINGLE-BLIND STUDYSINGLE-MASKED STUDY: A study in which one party, either the investigator or participant, is unaware of what medication the participant is taking; also called single-masked study. 
STANDARD TREATMENT: A treatment currently in wide use and approved by the FDA, considered to be effective in the treatment of a specific disease or condition.
STATISTICAL SIGNIFICANCE: The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance spends on the number of participants studied and the observations made, as well as the magnitude of differences observed.
STUDY TYPE: The primary investigative techniques used in an observational protocol; types are Purpose, Duration, Selection, and Timing.
TOXICITY: An adverse effect produced.
TREATMENT IND: IND stands for Investigational New Drug application, which is part of the process to get approval from the FDA for marketing a new prescription drug in the U.S. It makes promising new drugs available to desperately ill participants as early in the drug development process as possible. Treatment INDs are made available to participants before general marketing begins, typically during Phase III studies.
TREATMENT TRIALS: Refers to trials which test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

Share this: