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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.

PEER REVIEW: Review of a clinical trial by experts chosen by the study sponsor. These experts review the trials for scientific merit, participant safety, and ethical considerations.

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.

PHASE IV TRIALS: Post-marketing studies to delineate additional information including the drug's risks, benefits, and optimal use.

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. (See Placebo Controlled Study).

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.

PREVENTION TRIALS: Refers to trials to find better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.

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 (See Inclusion/Exclusion Criteria).

QUALITY OF LIFE TRIALS (or Supportive Care trials): Refers to trials that explore ways to improve comfort and quality of life for individuals with a chronic illness.

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 (See Arm).

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. (See Arm and Placebo).

RECRUITING: The period during which a trial is attempting to identify and enroll participants. Recruitment activites can include advertising and other ways of solicting interest from possible particpants. (See recruitment status and enrolling).

RECRUITMENT STATUS: Indicates the current stage of a trial, whether it is planned, ongoing, or completed. Possible values include:
Not yet recruiting: participants are not yet being recruited or enrolled
Recruiting: participants are currently being recruited and enrolled
Enrolling by invitation: participants are being (or will be) selected from a predetermined population
Active, not recruiting: study is ongoing (i.e., patients are being treated or examined), but enrollment has completed
Completed: the study has concluded normally; participants are no longer being examined or treated (i.e., last patient's last visit has occurred)
Suspended: recruiting or enrolling participants has halted prematurely but potentially will resume
Terminated: recruiting or enrolling participants has halted prematurely and will not resume; participants are no longer being examined or treated
Withdrawn: study halted prematurely, prior to enrollment of first participant

RISK-BENEFIT RATIO: The risk to individual participants versus the potential benefits. The risk/benefit ratio may differ depending on the condition being treated.

SCREENING TRIALS: Refers to trials which test the best way to detect certain diseases or health conditions.

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 (See Adverse Reaction).

SINGLE-BLIND 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. (See Blind and Double-Blind Study).

SINGLE-MASKED STUDY: See Single-Blind 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.

STANDARDS OF CARE: Treatment regimen or medical management based on state of the art participant care.

STATISTICAL SIGNIFICANCE: The probability that an event or difference occurred by chance alone. In clinical trials, the level of statistical significance depends on the number of participants studied and the observations made, as well as the magnitude of differences observed.

STUDY ENDPOINT: A primary or secondary outcome used to judge the effectiveness of a treatment.

STUDY TYPE: The primary investigative techniques used in an observational protocol; types are Purpose, Duration, Selection, and Timing.

SUSPENDED: See Recruitment Status

TERMINATED: See Recruitment Status

TOXICITY: An adverse effect produced 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.

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. To be considered for a treatment IND a participant cannot be eligible to be in the definitive clinical trial.

TREATMENT TRIALS: Refers to trials which test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.

WITHDRAWN: See Recruitment Status

Glossary Sources:

AIDSinfo: Glossary of HIV/AIDS-Related terms 4th Edition.
CenterWatch, Inc. Patient Resources: Glossary.
ECRI (formerly the Emergency Care Research Institute).
Eli Lilly and Company: Lilly Clinical Trials Glossary.
MediStudy.com Inc: ClinicalTrials: A-Z Glossary.
National Cancer Institute: Cancer.gov Dictionary.

Background Information

Last Updated: March 18th, 2008
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