Glossaries

Early Phase 2 trials that focus on a proof-of-concept assessment of efficacy and safety in a small number of patients. [After FDA Guidance for industry end of Phase 2a meetings, September 2009] See also phase, phase 2, phase 2b.

Phase that includes the controlled clinical trials conducted to evaluate the safety and efficacy of the drug in a limited number of patients with the disease or condition under study. Objectives can be dose-ranging (dose-response, frequency of dosing), type of patients, or numerous other characteristics of safety and efficacy. [After 21 CRF Part 312.21 Phases of an investigation] See also phase, phase 2a, phase 2b.

The initial introduction of an investigational new drug into humans. Phase 1 studies are closely monitored and are most often conducted in normal healthy volunteer subjects but in specific cases also in patients. NOTE: These studies are designed to determine the metabolism and pharmacologic actions of the drug in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness. During Phase 1, sufficient information about the drug's pharmacokinetics and pharmacological effects should be obtained to permit the design of well-controlled, scientifically valid Phase 2 studies. The total number of subjects and patients included in Phase 1 studies varies with the drug, but is generally in the range of 20 to 80. Phase 1 studies also include studies of drug metabolism, structure-activity relationships, and mechanism of action in humans, as well as studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes. [after ICH E8; After ICH Topic E8 NOTE FOR GUIDANCE ON GENERAL CONSIDERATIONS FOR CLINICAL TRIALS, CPMP/ICH/291/95 March 1998] See also phase.

Originally described as an exploratory study with no safety or efficacy targets. It is not cited in current FDA guidance and no longer in common usage. See also phase.

A stage in the sequence of activities in a clinical study (e.g., Screening, Randomization, Treatment, Follow-up). See also arm, visit, phase (of clinical development), epoch.

A stage in the clinical research and development of a therapy from initial clinical trials to post-approval studies. NOTE: Clinical trials are generally categorized into four (sometimes five) phases. A therapeutic intervention may be evaluated in two or more phases simultaneously in different trials, and some trials may overlap two different phases. [21 CFR section 312.21; After ICH Topic E8 NOTE FOR GUIDANCE ON GENERAL CONSIDERATIONS FOR CLINICAL TRIALS, CPMP/ICH/291/95 March 1998] See also Phase 0-5, epoch (if reference is to a single trial), phase (within a study), clinical research and development.

Process and science of monitoring the safety of medicines and taking action to reduce their risks and increase their benefits. NOTE: Pharmacovigilance is a key public health function that comprises: collecting and managing data on the safety of medicines; looking at the data to detect 'signals' (any new or changing safety issue); evaluating the data and making decisions with regard to safety issues; acting to protect public health (including regulatory action);communicating with stakeholders; auditing of both the outcomes of action taken and the key processes involved. [After IDMP] See also postmarketing surveillance.

Science that deals with the characteristics, effects, and uses of drugs and their interactions with living organisms.

Study of the processes of bodily absorption, distribution, metabolism, and excretion (ADME) of medicinal products.

Science that examines inherited variations in genes that dictate drug response and explores the ways such variations can be used to predict whether a person will respond favorably, adversely, or not at all to an investigational product.

An assay intended to study interindividual variations in whole genome or candidate gene maps, biomarkers, and alterations in gene expression or inactivation that may be correlated with pharmacological function and therapeutic response. Compare to pharmacogenetic test.

Study of the way drugs interact with genetic makeup or the study of genetic response to a drug.

An assay intended to study interindividual variations in DNA sequence related to drug absorption and disposition or drug action. Compare to pharmacogenomic test.

Branch of economics that applies cost-benefit, cost-utility, cost-minimization, and cost-effectiveness analyses to assess the utility of different pharmaceutical products or to compare drug therapy to other treatments.

Branch of pharmacology that studies reactions between drugs and living structures, including the physiological responses to pharmacological, biochemical, physiological, and therapeutic agents.