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Clinical Trials Glossary

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Clinical Trials Glossary www.eurordis.org G L O S S A R Y CLINICAL TRIALS GLOSSARY 18 June 2007 Rare Diseases Europe Analysis of Variance (ANOVA) [Statistics]: A statistical Absorption [Pharmacology] : In pharmacology, absorption is technique for defining and segregating the cause of the process that results in the transmission or circulation of variability affecting a set of observations. This technique the drug from the site of administration to the blood. More provides a basis for analyzing the effects of various commonly, absorption is the process where orally treatments or variables on the subjects or patients being administered drugs enter the blood through the intestinal investigated (see also Variance, Latin square). epithelial cells that has toxic effects on a living organism, when that organism is exposed to a lethal dose of a Analysis plan [Methodology, Statistics]: The strategy for substance once. This results in short-term, chronic toxicity. analysis predefined in the statistical section of the protocol and/or protocol amendments. The plan may be elaborated in Academic research: Research and reports written by a separate (sponsor’s) document to cover technical details leading education experts. and procedures for implementing the statistical analyses. Adaptive clinical trials [Methodology]: A process for The plan should be reviewed and possibly updated as a improving the efficiency of clinical trials based on interim result of the blind review of the data analysis of clinical data. This potentially leads to reductions Animal model [Pharmacology]: An animal with the same or in overall sample size, shorter project duration, improved a similar disease in humans. Animal models are used to quality of results, and reduced costs (see also Bayesian study the development and progression of diseases and to approaches). test new treatments before they are given to humans. Addiction [Pharmacology]: dependence. Animals with transplanted human cancers or other tissues are called xenograft models (see also Preclinical study). Adequate and well-controlled Trial [Methodology]: An adequate and well-controlled trial has the following Anonymous data [Database]: Information that does not characteristics: 1) a design that permits a valid comparison permit re-identification of individuals no matter how much with a control to provide a quantitative assessment of additional information is available (see also Identifiable treatment effect; 2) the use of methods to minimize bias in personal information, Indirectly identifying data, Confidentiality). the allocation of patients to treatment groups and in the ANOVA [Statistics]: Analysis of Variance. measurement and assessment of response to treatment; and 3) an analysis of the study results appropriate to the design Approved drugs [Regulation]: Regulatory authority must in order to assess the effects of the treatment. approve a substance as a drug before it can be marketed. The approval process involves several steps including pre- Adherence [Methodology]: The extent to which the patient clinical laboratory and animal studies, clinical trials for continues the mode of treatment previously agreed upon or safety and efficacy, filing of a New Drug Application by the intervention as prescribed (see also Compliance). drug’s manufacturer. Adverse Event (AE) [Pharmacology]: An AE is any Area Under Curve (AUC) [Pharmacology]: The area under untoward medical occurrence in a patient or clinical the plot of plasma concentration of drug (not logarithm of investigation subject administered with a pharmaceutical the concentration) against time after drug administration. product which does not necessarily have a causal relationship with this treatment. An AE can therefore be any Arm [Methodology]: Any of the treatment groups in a unfavourable and unintended sign (including an abnormal randomized trial. Most randomized trials have two "arms," laboratory finding, symptom, or disease temporally but some have three "arms," or even more (see also Treatment associated with the use of a medicinal [investigational] group, control subjects). product), whether or not related to the medicinal Aspect Voluntary agreement without legal value but [investigational] product. ethically pertinent, e.g. child assessment. Aliquot [Pharmacology]: A portion of a specimen used for Assent [Ethics, Regulation]:Agreement by an individual not testing. competent to give legally valid informed consent (ie a child All randomized subjects [Methodology]: The analysis set or cognitively impaired person). Ex: to obtain consent for that includes all patients who were given randomized research involving a child between the ages of 7 and 18, an treatment, with the patients assigned to the treatment group assent form written in language understandable to the child to which they were randomized. Practical considerations, is signed by the child, and a separate Child Consent form is such as missing data, may lead to some subjects in this set read and signed by the parent(s) or legally authorized not being included in the corresponding analysis. representative allowing the child to participate Alpha error [Statistics]: Type I Error (see also Beta error). Attributable risk [Methodology]: measure of the proportion of disease risk that is attributable to exposure (see also Risk). Alternative hypothesis [Statistics]: This usually refers to the proposal expressing the particular way the null AUC [Pharmacology]: Area Under Curve. hypothesis is held to be false. Sometimes referred to as the Audit [Methodology, Regulation]: Audit A systematic and motivated hypothesis, it usually reflects a difference the independent examination of trial-related activities and researcher hopes to demonstrate. In the ANOVA setting, the documents to determine whether the evaluated trial-related usual alternative hypothesis is that the true means of the activities were conducted, and the data were recorded, various groups are unequal (see also Null hypothesis). analyzed, and accurately reported according to the protocol, sponsor’s standard operating procedures (SOP’s), good clinical practice (GCP), and the applicable regulatory requirement(s) (see also Review, Inspection). Page 2 - Clinical Trials Glossary Authorized third party / authorized representative Benefit [Ethics]: A valued or desired outcome; an [Ethics]: A representative of an individual who is not advantage. Human research application requests information competent to provide free and informed consent. The about the direct benefits accruing to the research authorized third party acts in the interest of the individual participants. concerned (see also Competence, Consent). Beta error [Statistics]: Type II Error (see also Alpha error). Autonomy [Ethics]: Literally, self ruling, related to, and sometimes used in lieu of the bioethical principle of respect Bias [Methodology, Statistics]: The systematic tendency of for persons. Implies intentionality and freedom from any factors associated with the design, conduct, analysis, coercion or undue influence. In the Kantian tradition, and evaluation of the results of a clinical trial to make the autonomy implies freely embracing a moral obligation. 2) In estimate of a treatment effect deviate from its true value. public health, individual autonomy may be limited by Bias introduced through deviations in conduct is referred to interventions applied to populations (see also: Respect for as ‘‘operational’’ bias. The other sources of bias listed above Persons). An autonomous being has the power of self- are referred to as ‘‘statistical’’ (see also Selection bias). direction, possessing the ability to act as it decides, Bid [Pharmacology]: Twice a day. independent of the will of others and of other internal or external factors (see also Belmont principles). Binary variable [Statistics]: A special categorical variable for which only two possible categories exist. For example, Average: Mean. the variable gender would be a binary variable. The variable ice cream flavour would NOT be a binary variable because Baseline [Methodology]: Information gathered at the beginning of a study from which variations found in the it has more than two categories for the responses (see also Continuous variable). study are measured. 2). A known value or quantity with which an unknown is compared when measured or assessed. Bio-analytical assays: Methods for quantitative 3). The initial time point in a clinical trial, just before a measurement of a drug, drug metabolites or chemicals in participant starts to receive the experimental treatment biological fluids. which is being tested. Safety and efficacy of a drug are often determined by monitoring changes from the baseline values. Bioavailability [Pharmacology]: The percentage of dose entering the systemic circulation after administration of a Baseline assessment [Ethics, Methodology]: Assessment of given dosage form. More explicitly, the ratio of the amount subjects as they enter a trial before receiving any treatment. of drug "absorbed" from a test formulation to the amount "absorbed" after administration of a standard formulation. Bayes' theorem [Statistics]: A theorem describing how the Frequently, the "standard formulation" used in assessing conditional probability of a set of possible causes for a given bioavailability is the aqueous solution of the drug, observed event can be computed from knowledge of the administered intravenously. probability of each cause and the conditional probability of the outcome of each cause. Bioequivalence [Pharmacology]: Term in pharmacokinetics
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