Glossary

The following glossary explains some terms commonly used in clinical trials.

Adverse event (AE)

Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product wand which does not necessarily have a causal relationship with this treatment any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment.

An adverse event (AE) can, therefore, be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. See also serious adverse event

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.
Clinical

Pertaining to or founded on observation and treatment of participants, as distinguished from theoretical or basic science.

Cohort

In epidemiology, a group of individuals with some characteristics in common.

Contraindication

A specific circumstance when the use of certain treatments could be harmful.

Control Group

The standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

Ascending Dose 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.

Consent Form

A document that describes the rights of the study participants, and includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document.

The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

Eligibility Criteria

Summary criteria for participant selection; includes Inclusion and Exclusion criteria

Food and Drug Administration (FD)

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, including those used in the diagnosis, treatment, and prevention of HIV infection, AIDS, and AIDS-related opportunistic infections. The FDA also works with the blood banking industry to safeguard the nation’s blood supply. Internet address: www.fda.gov

Inclusion/Exclusion Criteria

The medical or social standards determining whether a person may or may not be allowed to enter a clinical trial. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. It is important to note that inclusion and exclusion criteria are not used to reject people personally, but rather to identify appropriate participants and keep them safe.

Investigator

A medical researcher in charge of carrying out a clinical trial’s protocol.

Open Label Study

A clinical trial in which doctors and participants know which drug or vaccine is being administered.

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 III TRIALS

Expanded controlled and uncontrolled trials after preliminary evidence suggesting the 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 an adequate basis for physician labelling.

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 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.

Randomisation

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.

Risk-Benefit Ratio

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

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.

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.

Arm

Any of the treatment groups in a randomized trial. Most randomized trials have two “arms,” but some have three “arms,” or even more.

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 of 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.

Confidentiality

Refers to maintaining the confidentiality of trial participants including their personal identity and all personal medical information. The trial participants’ consent to the use of records for data verification purposes should be obtained prior to the trial and assurance must be given that confidentiality will be maintained.

Control

A control is the nature of the intervention control.

Controlled Trial

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.

Double Blind 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.

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.

Empirical

Based on experimental data, not on a theory.

Experimental Drug

A drug that is not FDA licensed for use in humans, or as a treatment for a particular condition

Hypothesis

A supposition or assumption advanced as a basis for reasoning or argument, or as a guide to experimental investigation.

Informed Consent

The process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study.

Ethics Committee

A committee of physicians, statisticians, researchers, community advocates, and others that ensures that a clinical trial is ethical and that the rights of study participants are protected. All clinical trials in the New Zealand must be approved by an IRB before they begin.

Ethics committee may recommend that a trial is stopped if there are safety concerns or if the trial objectives have been achieved.

Pharmacokinetics

The processes (in a living organism) of absorption, distribution, metabolism, and excretion of a drug or vaccine.

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 IV TRIALS

Post-marketing studies to delineate additional information including the drug’s risks, benefits, and optimal use.

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.

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.

Randomised 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.

Side Effects

Any undesired actions or effects of a drug or treatment. Negative or adverse effects may include a headache, nausea, hair loss, skin irritation, or other physical problems. Experimental drugs must be evaluated for both immediate and long-term side effects.

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.

Treatment Trials

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