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This is the EUPATI Glossary which explains key terms used in medicines research and development. This is work in progress - we are still adding additional terms, and we are reviewing the descriptions to make them more understandable. This email address is being protected from spambots. You need JavaScript enabled to view it.



Glossary

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absorption
In pharmacology, absorption is the process that results in the transport or diffusion of the drug from the site of administration to the blood. More commonly, absorption is the process where orally administered drugs get into the blood through the intestinal epithelial cells.
adaptive clinical trials
A newer flexible process for improving the efficiency of clinical trials based on interim analyses of clinical data, potentially leading to reductions in trial size, shorter trial duration, improved quality of results, and reduced costs (see also Bayesian approaches).
adaptive licensing
Stepwise procedure to approve a new drug, with repeated cycles of data gathering, evaluation and regulatory approval to reduce uncertainty about the drug’s safety and efficacy. It allows earlier access to new therapies, particularly relevant in situations with high unmet need.
adequate and well-controlled trial
An adequate and well-controlled trial has the following characteristics: 1. a design that permits a valid comparison of the intervention to an arm without the intervention to provide a quantitative assessment of treatment effect; 2. the use of methods to minimize bias in the allocation of patients to treatment groups and in the measurement and assessment of response to treatment; and 3. an analysis of the study results in order to appropriately assess the effects of the intervention.
adherence
The degree to which the patient continues a treatment or intervention, factors of which include number of pills, number of doses per day, types of administration (oral vs injection, etc), necessity of food or water, taste of product, ability to do so with other people around, etc (see also Compliance).
adverse event
An adverse event (or AE) is any untoward medical occurrence in a person who is taking a pharmaceutical product, an unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product. AEs should be reported to the doctor in order to find out if it is associated with the medical product and what can be done to stop or lessen these negative effects.
AE
An AE (or adverse event) is any untoward medical occurrence in a person who is taking a pharmaceutical product, an unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product. AEs should be reported to the doctor in order to find out if it is associated with the medical product and what can be done to stop or lessen these negative effects.
AEMPS
Agencia Española de Medicamentos y Productos Sanitarios, the Spanish Competent Authority (see NCA).
aetiology
The cause of a disease; the study of the causes of disease.
AGES
Agentur für Gesundheit und Ernährungssicherheit, the Austrian Competent Authority (see NCA).
AIFA
Agenzia Italiana del Farmaco, the Italian Competent Authority (see NCA).
aliquot
A portion of a specimen used for testing.
all randomised subjects
All participants in a trial who have been randomly (by chance) assigned to one intervention arm or another of that trial. Practical considerations, such as missing data over time, may lead to some participants not being included in the final analysis (see Flowchart).
Alpha error
Also known as a Type I error, it is is the incorrect rejection of a true null hypothesis, a false positive, ie, a trial finds that a medical treatment should cure a disease, when in fact it doesn't.
alternative hypothesis
In general, the proposition that the null hypothesis is held to be false. Sometimes referred to as the motivated hypothesis, it usually reflects a difference the researcher hopes to demonstrate. In the ANOVA setting, the usual alternative hypothesis is that the true means of the various groups are unequal (ie, the negation of the Null hypothesis).
analysis of variance
Also known as ANOVA, the Analysis of Variance is a statistical technique for looking at multiple groups and multiple factors, to help define the cause of any variability affecting a set of observations. This technique provides a basis for analysing the effects of various treatments or other parameters (variables) that may have an effect on the patients being investigated (see also Variance).
analysis plan
The strategy for analysis predefined in the statistical section of the protocol and/or protocol amendments. The plan may be elaborated in a separate document (internal to the sponsor) to cover technical details and procedures for implementing the statistical analyses
animal model
An animal (rat, mouse, monkey, woodchuck, etc) with a disease either the same as or very similar to a disease in humans. Animal models are used to study the development and progression of diseases as well as to test new treatments before they are given to humans.
ANM
Agentia Nationala a Medicamentului i a Dispozitivelor Medicale, the Romanian Regulatory (Competent) Authority (see NCA).
ANOVA
Also known as Analysis of Variance, ANOVA is a statistical technique for looking at multiple groups and multiple factors, to help define the cause of any variability affecting a set of observations. This technique provides a basis for analysing the effects of various treatments or other parameters (variables) that may have an effect on the patients being investigated (see also Variance).
ANSM
Agence Nationale de Sécurité du Médicament et des produits de santé, the French Regulatory (Competent) Authority (see NCA).
approved drugs
Before a drug can be marketed, it must be approved by a regulatory (competent) authority, who scientifically evaluates it and then continues to supervise or monitor its use once in the market. 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 (USA) or Marketing Approval Application (EU) by the manufacturer of the drug.
area under the curve
The area under the curve is the concentration of the amount of a drug in chart form. It is useful in measuring the amount of drug absorbed by the body and so is helpful in calculating the correct dosage; it is often divided by time after drug administration to measure the drug's concentration.
arm
Any of the treatment groups in a randomised trial. Many randomised trials have two "arms" or groups, but some may have three or even more (see also treatment group, control subjects).
attributable risk
Measure of the proportion of disease risk attributable to an exposure (see risk).
AUC
Also known as area under the curve, the AUC is the concentration of the amount of a drug in plot form. It is useful in measuring the amount of drug absorbed by the body and so is helpful in calculating the correct dosage; it is often divided by time after drug administration to measure the drug's concentration.
audit
A systematic and independent examination of trial-related activities and documents to determine whether the evaluated trial-related activities were conducted, and the data recorded, analysed, and accurately reported following the protocol, sponsor's standard operating procedures (SOPs), good clinical practice (GCP), and the applicable regulatory requirement(s) (see review).
authorized representative
A representative of an individual who is not competent to provide free and informed consent. The authorized third party acts in the interest of that individual (see also competence, Informed Consent).
authorized third party
A representative of an individual who is not competent to provide free and informed consent. The authorized third party acts in the interest of that individual (see competence, Informed Consent).
autonomy
Literally, self-ruling. Related to, and sometimes used instead of, the bioethical principle of respect for persons. Implies intentionality and freedom from force or undue influence (coercion). In public health, individual autonomy may be limited by interventions applied to populations (see respect for persons). An autonomous being is one who has the power of self-direction, possessing the ability to act as he/she decides, independent of the will of others and of other internal or external factors (see Belmont Principles).
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 assigned treatment. Safety and efficacy of a drug are often determined by monitoring changes from baseline (BL) values.
baseline assessment
Assessment of subjects as they enter a trial and before they receive any treatment.
basic research
Research that advances scientific knowledge but does not have specific immediate commercial or practical objectives.
Bayes' Theorem
A theorem describing how the conditional probability of a set of possible causes for a given observed event can be computed from knowledge of the probability of each cause and the conditional probability of the outcome of each cause.
Bayesian approach to statistics
Approaches to data analysis that provide a posterior probability distribution for some parameter (eg, treatment effect), derived from the observed data and a prior probability distribution for the parameter. The posterior distribution is then used as the basis for statistical inference (see adaptive clinical trials, Bayes' Theorem, frequentist methods, odds ratio).
BDA
Bulgarian Drug Agency, the Bulgarian Regulatory (Competent) Authority (see NCA).
Belmont Principles
The Belmont Report, a document identifying the basic ethical principles that should underlie the conduct of biomedical and behavioural research involving human subjects, includes guidelines to ensure that research is conducted in accordance with beneficence, respect for persons and justice. They should be given consideration in any research situation (http://www.hhs.gov/ohrp/humansubjects/guidance/belmont.html).
beneficence
Literally, doing good; in bioethics, a prima facie principle underlying utilitarian approaches. Implies an obligation to promote benefits of things judged to be good, typically balancing produced goods against risks or harms. In public health, beneficence implies acting in the best interest of the population or society as a whole (see Belmont principles).
benefit
A valued or desired outcome; an advantage. Human research looks for information about the direct benefits accruing to the research participants (see harms - or risks - and benefits).
benefit-risk assessment
An assessment which compares the risk of administration to the benefits gained in order to judge whether there is a greater health risk or a greater health benefit to the user of a medicinal product, the benefit-risk balance (see benefit-risk balance). Benefit-risk assessments are one of the fundamental activities in licensing medicines and in clinical research. Human beings (whether as individuals or as a population) should not be administered any medicinal product where the risk of negative effects outweighs the potential benefit.
benefit-risk balance
The result of the benefit-risk assessment must be non-negative (of greater benefit than risk or at least the same benefit as risk) to enable a medicinal product to be licensed and keep it on the market or to continue a clinical trial (see benefit-risk assessment).
Beta error
Also known as a Type II error, a Beta error is the failure to reject a false null hypothesis, or a false negative, ie, a clinical trial of a medical treatment failing to show that the treatment works when really it does.
BfArM
Bundesinstitut für Atrzmittel und Medizinprodukte, one of the German Regulatory (Competent) Authorities (see NCA).
bias
The systematic tendency of any factors associated with the design, conduct, analysis, and evaluation of the results of a clinical trial to make the estimate of a treatment effect deviate from its true value. Bias introduced through deviations in conduct is referred to as ''operational'' bias. Other sources of bias are referred to as ''statistical'' (see selection bias).
BID
Twice a day
binary variable
A special categorical variable for which only two possible categories exist. For example, the variable "light" would be a binary variable with the possible states being on or off. The variable "ice cream flavour" would NOT be a binary variable because there are more than two categories for the responses (chocolate, strawberry, vanilla, banana, etc) (see continuous variable).
bioavailability
Bioavailability is the amount of an administered dose of medicine that reaches the systemic circulation (blood stream). By definition, when medicine is administered intravenously, its bioavailability is 100%. However, when medicine is administered via other routes, eg, orally, its bioavailability generally decreases (due to gastrointestinal factors, bacteria and liver enzymes). Bioavailability is a key parameter in pharmacokinetics and must be calculated before oral administration in order to secure the correct dose.
bioequivalence
Bioequivalence is a term in pharmacokinetics used to assess the expected biological equivalence of two distinct preparations of a given medicine. If two products are said to be bioequivalent it means that they would be expected to be, for all intents and purposes, the same.
bioethics
A field of inquiry and academic discipline at the intersection of ethics and the life sciences. Emerging with an emphasis upon problems faced in the practice of medicine and biomedical research, bioethics has overlapping areas of scholarship and application: theory and method, clinical practice, regulatory policy, research practice, cultural and social concerns, and recently, public health and epidemiology (see ethics).
biologicals/biopharmaceuticals
Biologicals/biopharmaceuticals are biologically active molecules such as proteins (including antibodies), nucleic acids (DNA, RNA) and living microorganisms like viruses and bacteria. Biologicals are administered by intravenous (IV) injection. A classical biopharmaceutical is insulin.
biomarker

A biomarker is anything that can be used as an indicator of a particular disease state or some other physiological state of an organism. Biomarkers can be specific cells, molecules, or genes, gene products, enzymes, or hormones. Biomarkers help in early diagnosis, disease prevention, drug target identification, drug response, etc, eg, cholesterol is a biomarker for risk of coronary heart disease.

biopharmaceutical research

Research based on complex macromolecules derived from recombinant DNA technology, cell fusion, or processes involving genetic manipulation. They include recombinant proteins, genetically engineered vaccines, therapeutic monoclonal antibodies, and nucleic acid-based therapeutics, including gene therapy vectors. The term may be used for all types of molecules and not just complex macromolecules.

biostatistics

Sometimes known as biometrics, biostatistics is the application of statistics to biology and, most commonly, to medicine. Because research questions in biology and medicine are various, biostatistics has expanded its domain to include any quantitative, not just statistical, model that may be used to answer these questions.

biotransformation

Chemical alteration of an drug that occurs by virtue of the passage of the drug through a biological system.

blind review

The checking and assessment of data during the course of a study, before the breaking of the blind, for the purpose of confirming and finalising the analysis plan.

blinding

A procedure in which one or more parties in a trial are kept unaware of the treatment assignment(s). This is done in order to avoid bias, whether it be intentional or unconscious. Single-blinding usually refers to the subject/participant being unaware, and double-blinding usually refers to the subject, investigator, monitor, and in some cases, data analysts being unaware of the treatment assignment(s) (see double-blind study, single-blind study). Opposite of an open or open-label trial.

bridging data package

Selected information from the Complete Clinical Data Package that is relevant to the population of a new geographical region, including pharmacokinetic data, and any preliminary pharmacodynamic and dose-response data and, if needed, supplemental data obtained from a bridging study in the new region that will allow extrapolation of the foreign safety and efficacy data to the population of the new region.

bridging study

A bridging study is defined as a supplemental study performed in a new geographical region to provide pharmacodynamic or clinical data on efficacy, safety, dosage and dose regimen in the new region that will allow extrapolation of the foreign clinical data to the new region. Such studies could include additional pharmacokinetic information.

Bulgarian drug agency

Bulgarian drug agency, the Bulgarian Regulatory (Competent) Authority (see NCA).

c2

C2 or Chi2 is a statistical method used to compare frequencies of two or more groups. Researchers often use chi-squares to test nominal data (see nominal scale).

case or case study

A detailed description of a concrete situation requiring analysis and a resultant judgment or action. Cases provide specific circumstances involving a patient (in medical ethics), a participant or group (in research ethics) or a population (in public health ethics). There is usually a unifying situation/circumstance in the cases as to why they are grouped as they are (if there is more than one).

case report form
CRF - A printed, optical, or electronic document designed to record all of the protocol required information to be reported to the sponsor on each trial subject.
case-control study
A study that compares two groups of people: those with the disease or condition under study (the 'cases') and a very similar group of people who do not have the disease or condition (the 'controls'). Researchers study the medical and lifestyle histories of the people in each group to learn what factors may be associated with the disease or condition (see retrospective study).
casuistry
The application of general ethical priciples to particular cases of conscience or conduct, including the conduct of a clinical trial
causality
The relating of causes to the effects they produce. Epidemiology is mostly concerned with causality; several types of causes may be distinguished. It must be emphasized, however, that epidemiological evidence by itself is insufficient to establish causality, although it can provide powerful circumstantial evidence.
CBG
Protocol required information to be reported to the sponsor on each trial subject.
Chi-squared
Chi-squared is a statistic used to compare frequencies of two or more groups. Researchers often use chi-squares to test nominal data (see nominal scale).
children
Persons who have not attained the legal age for consent to treatment or procedures involved in the research, as determined under the applicable law of the jurisdiction in which the research will occur (see assent, permission, vulnerable subjects).
chronic toxicity
A continuous or repeated toxic effect of an agent on a living organism exposed to the agent.
CI
The CI or Confidence Interval quantifies the uncertainty in a measurement. It is usually reported as 95% CI, which is the range of values within which we can be 95% sure that the true value for the whole population lies. For example, for a measured value of 10 that has a 95% CI from 5 to 15, we would have 95% confidence that the true value was between 5 and 15 (see variance, standard deviation).
CIOMS
CIOMS, or the Council for International Organizations of Medical Sciences, is an international, non-governmental, non-profit organization established jointly by WHO and UNESCO in 1949 to serve the scientific interests of the international biomedical community.
clearance
Is a term in pharmacokinetics which describes the drug elimination process time from the body without identifying the mechanism of the process. Clearance is defined as the volume of fluid cleared of drug from the body per unit of time (see pharmacokinetics).
clinical endpoint
Clinical endpoints are distinct measurements or analyses of disease characteristics reflecting the effect of a therapeutic intervention in a clinical trial or study. It is often a characteristic or variable that reflects how a patient feels, functions, or survives (see endpoint).
clinical investigator
A medical researcher who takes part in a clinical trial protocol.
clinical pharmacology
The study of pharmacology in relation to clinical science. It is a science which deals with the effects of drugs in healthy volunteers and in patients. In the evaluation process the action and adverse affects of drugs can be measured and compared.
clinical research assistant
A clinical research assistant (CRA) is a person employed by a sponsor, or by a contract research organization (CRO) acting on a sponsor's behalf, who monitors the progress of investigator sites participating in a clinical study. At some (primarily academic) sites, clinical research coordinators are called CRAs.
clinical trial/study
Any investigation in human subjects intended to discover or verify the clinical, pharmacological, and/or other pharmacodynamic effects of an investigational product(s), and/or to identify any adverse reactions to an investigational product(s), and/or to study absorption, distribution, metabolism, and excretion of an investigational product(s) with the object of ascertaining its dosage, safety and/or efficacy. The terms clinical trial and clinical study are synonymous.
co-PI
The co-PI or the co-principal investigator is the second scholar or researcher involved in conducting the research; if the project is for a thesis or dissertation, the student is the co-PI.
co-principal investigator
The co-principal investigator or the co-PI is the second scholar or researcher involved in conducting the research; if the project is for a thesis or dissertation, the student is the co-PI.
cognitively impaired
Having either a psychiatric disorder (eg, psychosis, neurosis, personality or behaviour disorders, or dementia) or a developmental disorder (eg, mental retardation, intellectual disability) that affects cognitive or emotional functions to the extent that capacity for judgment and reasoning is significantly diminished. Others, including persons dependent on drugs or alcohol, those suffering from degenerative diseases affecting the brain, terminally ill patients, and persons with severely disabling physical handicaps, may also be compromised in their ability to make decisions in their best interests (see vulnerable subjects).
cohort study
A form of longitudinal study used in medicine and social science. In medicine, it is a type of study design usually undertaken to obtain additional evidence to refute or support the existence of an association between suspected cause and disease. The cohorts are identified prior to the appearance of the disease under investigation. The study groups are observed over a period of time to determine the frequency of disease among them. The main characteristic is that the study proceeds from cause to effect (see cross-sectional study).
combination therapy
Combination therapy or polytherapy is the use of more than one medication or other therapy (vs. monotherapy, which is any therapy taken alone)
comorbidity
The presence of coexisting or additional diseases with reference to the primary diagnosis. Comorbidity may affect the ability of affected individuals to function as well as their survival; may be used as a prognostic indicator for length of hospital stay, cost factors, and outcome or survival (see morbidity).

comparator
The reference arm in a clinical trial, it can be an investigational or marketed product (ie, active control), or under certain circumstances, placebo.
compassionate use
Method of providing an unlicensed drug prior to final approval by a regulatory (competent) authority for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained for compassionate use of a drug or therapy.
compensation
Payment or medical care provided to subjects injured in research; does not refer to payment (remuneration) for participation in research (see remuneration), nor does it refer to reimbursement of expenses incurred by a participant taking part in a specific research study.
competence
The ability of prospective subjects to give informed consent in accord with their own fundamental values. It involves the ability to understand information presented, appreciate the potential consequences of the decision, and provide free and informed consent (See incompetence, incapacity, vulnerable subjects).
competent authorities
In medicine, the competent authority, also known as the regulatory authority, is a body who acts on behalf of a government on matters related to advice, evaluation and approval of the medicine. On a European level the competent authorities are the European Commission and the European Medicines Agency (EMA).
complete clinical data package
A clinical data package from the sponsor to regulators containing clinical data that should fulfill regulatory requirements.
compliance
Adherence to all the trial-related requirements, good clinical practice (GCP) requirements, and the applicable regulatory requirements (see observance, adherence). Also, for patients on a regimen of any sort, the following of the requirements of taking the drug(s) as prescribed.
compounds insensitive to ethnic factors
A compound whose characteristics suggest minimal potential for clinically significant impact by ethnic factors on safety, efficacy, or dose response.
compounds sensitive to ethnic factors
A compound whose characteristics suggest the potential for clinically significant impact by intrinsic and/or extrinsic ethnic factors on safety, efficacy, or dose response.
confidence interval
CI - Quantifies the uncertainty in measurement. It is usually reported as 95% CI, which is the range of values within which we can be 95% sure that the true value for the whole population lies. For example, for an NNT of 10 with a 95% CI of 5 to 15, we would have 95% confidence that the true NNT value was between 5 and 15(see also Variance, Standard deviation)
confidentiality
One of the important components of medical ethics and a fundamental component in the doctor-patient relationship. Refers to maintaining personal identity and all personal medical information of trial participants. 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. No personal information about an individual is to be revealed to third parties without the individual's consent (see respect for persons, anonymous data, indirectly identifying data, confidentiality).
confirmatory studies
Studies conducted in patients (1000–5000) to gather additional confirmatory information about safety and efficacy of the drug, by possibly studying different populations and different dosages and by using the drug in combination with other drugs. Phase III studies are pivotal for getting Marketing Authorisation (approval) of the drug.
confounding variable
A factor that distorts the true relationship of the study variables of central interest by virtue of being related to the outcome of interest but extraneous to the study question (possibly due to an unequal distribution among the groups/arms being compared). For example, age might confound a study of the effect of a toxin on longevity if individuals exposed to the toxin were older than those not exposed.
consent
Consent, better known as informed consent is a person's voluntary agreement, based upon adequate knowledge and understanding of relevant information, to participate in research or to undergo a diagnostic, therapeutic, or preventive procedure. In giving informed consent, subjects do not waive any of their legal rights, or release the investigator, the sponsor, the institution or their representatives from liability for negligence. Before carrying out any form of research on anyone, the participant must be informed about the study's aims, methods, anticipated benefits and any potential risks. The individual must also be informed that they are free to decline to participate in the study and are free to leave at any time without any impact on their treatment. The researcher must obtain this consent, usually in writing. If this is not possible, non-written consent must be formally documented (see autonomy).
consortium
An association or grouping of institutions, businesses, or financial organisations, usually set up for a common purpose that would be beyond the capabilities of a single member of the group.
content management system
A technical web platform designed to make it easy for non-technical users to add, edit and manage a website.
content validity
The extent to which a variable (eg, a rating scale) measures what it is supposed to measure.
contingency table
A table (usually 2 rows x 2 columns) often used in epidemiology to show the relationship between disease and exposure. The table can be used to divide persons into the categories of diseased and exposed (a), diseased and not exposed (b), not diseased and exposed (c), and not diseased and not exposed (d) (see chi-square, confidence interval).
continuous variable
A variable that can be expressed by a large (sometimes infinite) number of score values. For example, height, temperature, and grade point average are continuous variables. Contrast with dichotomous variable (see also binary variable).
contract
A written, dated, and signed agreement between two or more involved parties that sets out any arrangements on delegation and distribution of tasks and obligations and, if appropriate, on financial matters. A protocol of a clinical study/trial may serve as the basis of a contract.
contract research organization
A Contract Research Organisation or CRO is a person or an organization (commercial, academic, or other) contracted by the sponsor to perform one or more of a sponsor's trial-related duties and functions.
control
The control is the standard against which experimental observations may be evaluated.
control subjects
Subject(s) used for comparison who are not taking the experimental treatment or who do not have a given condition, background, or risk factor that is the object of study. Control conditions may be concurrent (occurring more or less simultaneously with the condition under study) or historical (preceding the condition under study). When the present condition of subjects is compared with their own condition on a prior regimen or treatment, the study is considered historically controlled. The control group serves as a measuring stick to gauge the effectiveness of the experimental treatment (see also arm, treatment group, control group).
controlled trial
In clinical trials, one group of participants is given an experimental drug/agent, while another group (ie, the control group) is given either a standard treatment for the disease or a placebo (see also placebo, comparator, descriptive study).
coordinating committee
A committee that a sponsor may organize to coordinate the conduct of a multicenter trial.
correlation
The simultaneous change in value of two numerically valued random variables: the positive correlation between cigarette smoking and the incidence of lung cancer; the negative correlation between age and normal vision.
course
In education, a course is a structure of lectures and other accompanying learning methods dealing with a particular topic.
CRA
A CRA or clinical research assistant is a person employed by a sponsor or by a contract research organization (CRO) acting on a sponsor's behalf who monitors the progress of investigator sites participating in a clinical study. At some (primarily academic) sites, clinical research coordinators are called CRAs.
creative commons license
With Creative Commons Public License (CCPL), the EUPATI consortium grants any public third party a worldwide, royalty-free, non-exclusive license to reproduce, adapt, transform, distribute and publicly perform EUPATI's project results as long as all copyright notices for the work - including original sources - are kept intact, and as long as the use is non-commercial. The license used in EUPATI is "Attribution-Non Commercial- ShareAlike 3.0 Unported" - http://creativecommons.org/licenses/by-nc-sa/3.0/legalcode.
CRF
A CRF or case report form is a printed, optical, or electronic document designed to record all of the protocol-required information to be reported to the sponsor on each trial subject.
CRO
A CRO or contract research organization is a person or an organization (commercial, academic, or other) contracted by the sponsor to perform one or more of a sponsor's trial related duties and functions.
cross-over design
A clinical trial design in which patients receive, in sequence, the treatment (or the control), and then, after a specified time, switch to the control (or treatment). In this design, patients serve as their own controls. Randomization is used to determine the order in which a patient receives the treatment vs. the control.
cross-sectional study
In survey research, a study in which data are obtained at one point in time. Contrast with longitudinal studies in which individuals are followed (ie, interviewed repeatedly) over a length of time. Cross-sectional studies provide a 'snapshot' of the outcome and the characteristics associated with it, at a specific point in time,and may give an idea of the exposure/disease outcome relationship, although it does not clarify causality. Note that a cross-sectional study can ask questions about previous periods of time (see cohort study).
curriculum
The planned interaction of students with instructional content, materials, resources, and processes for evaluating the attainment of educational objectives.
data and safety monitoring board
An independent data monitoring committee that can be composed of community representatives and clinical research experts that may be established by the sponsor to assess at intervals the progress of a clinical trial, the safety data, and the critical efficacy endpoints, and to recommend to the sponsor whether to continue, modify, or stop a trial. Also known as a DMC (Data Monitoring Committee).
data monitoring committee
An independent committee that can be composed of community representatives and clinical research experts that may be established by the sponsor to assess at intervals the progress of a clinical trial, the safety data, and the critical efficacy endpoints, and to recommend to the sponsor whether to continue, modify, or stop a trial. Also known as a DSMB (Data and Safety Monitoring Board).
debriefing
Giving subjects previously undisclosed information about the research project following completion of their participation in research. (This usage, in the behavioural sciences, departs from standard English, in which debriefing is obtaining rather than imparting information.)
Declaration of Helsinki
A series of guidelines adopted by the 18th World Medical Assembly in Helsinki, Finland in 1964. The Declaration addresses ethical issues for physicians conducting biomedical research involving humans. Recommendations include the procedures required to ensure subject safety in clinical trials, including informed consent and Ethics Committee reviews. It has been revised eight times, most recently in 2008. A comment period in 2013 just closed to revise the Declaration once again.
deductive reasoning
(or deduction). Reasoning from the general to the particular (or from cause to effect) (See inductive reasoning).
dependent variable
A variable that is not under the experimenter's control -- the data. It is the variable that is observed and measured in response to the independent variable. For example, a test score could be a dependent variable because it could change depending on several factors such as how much you studied, how much sleep you got the night before you took the test, or even how hungry you were when you took it. The dependent variable (the test score) cannot change the independent variable (how well you slept) (see independent variable).
descriptive study
Any study that is not truly experimental (eg, quasi-experimental studies, correlational studies, record reviews, case histories, and observational studies) (see controlled trial).
dichotomous variable
Also known as a binary or dummy variable, used as a device to sort data into mutually exclusive categories (such as smoker/non-smoker, etc) (see continuous variable).
directly identifying data
Information that allows for individuals to be identified or located easily without the need for any additional information. Examples of directly identifying data items include names and residential addresses
dissent
Refusal to participate in research by an individual not able to provide free and informed consent
documentation
All records, in any form (including, but not limited to, written, electronic, magnetic, and optical records; and scans, x-rays, and electrocardiograms) that describe or record the methods, conduct, and/or results of a trial, the factors affecting a trial, and the actions taken
dosage
The quantity of a medicine given per administration, or per day
dose regimen
The route, frequency and duration of administration of the dose of a medicine over a period of time
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. (see also Blinding, Single blind study)
Double dummy
A technique for retaining the blinding of a study when administering supplies in a clinical trial, when the two treatments cannot be made identical. Supplies are prepared for Treatment A (active and indistinguishable placebo) and for Treatment B (active and indistinguishable placebo). Subjects then take two sets of treatment; either A (active) and B (placebo), or A (placebo) and B (active).
drop out
A participant in a clinical trial who for any reason does not continue in the trial until the last visit required of him/her by the study protocol (see loss to follow-up).
drug dependence
The compulsive use of a substance, despite its negative or dangerous effects; being tolerant to (and so needing a higher dose) as well as dependent on something that is psychologically or physically habit-forming (ie, alcohol or narcotics). Abruptly stopping the drug leads to symptoms of withdrawal.
drug development process
The entire process of bringing a new medicines to patients. It includes drug discovery/product development, pre-clinical research (microorganisms/animals), clinical trials (on humans), the marketing authorisation process and post-authorisation studies
drug distribution
The process by which a drug reversibly leaves the blood stream and enters extracellular fluid and/or the cells of tissue
DSMB
Data safety monitoring board - An independent committee, composed of community representatives and clinical research experts, that reviews data while a clinical trial is in progress to ensure that participants are not exposed to undue risk. A DSMB may recommend that a trial be stopped if there are safety concerns or if the trial objectives have been achieved
duty of care
A duty to do everything reasonably practicable to protect individuals from harm. Individuals such as doctors or researchers are often held to a higher standard of care, as they are in a position where large numbers of individuals may unquestioningly trust their motives and judgements due to their roles in society. (see also Obligation)
EBM
Applies the scientific method to medical practice. According to the Centre for Evidence-Based Medicines using techniques from science, engineering and statistics, such as meta-analysis of scientific literature, risk-benefit analysis, and randomized controlled trials, it aims for the ideal that healthcare professionals should make "conscientious, explicit, and judicious use of current best evidence" in their everyday practice
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. (see also Inclusion criteria, Exclusion criteria)
EMA
European Medicines Agency, a European agency located in London. EMA is responsible for the scientific evaluation of medicinal products which should be assessed through the centralised procedure
emancipated minor
A legal status conferred upon persons who have not yet attained the age of legal competency as defined by state law (for such purposes as consenting to medical care), but who are entitled to treatment as if they had by virtue of assuming adult responsibilities such as being self-supporting and not living at home, marriage, or procreation. (See also: Mature Minor)
endpoint
Overall outcome that the protocol is designed to evaluate
EOF
National Organization for Medicine, the Greek Competent Authority. (See NCA)
epedited review
Review of proposed research by the IRB chair or a designated voting member or group of voting members rather than by the entire IRB. Federal rules permit expedited review for certain kinds of research involving no more than minimal risk and for minor changes in approved research
epidemiology
The study of the various factors influencing the occurrence, distribution, prevention and control of disease, injury and other health-related events in a defined human population
equipoise
A condition where there is a balance between different social, emotional, or intellectual influences; something that creates a balanced state, usually by counterbalancing some other force or thing
equitable
Fair or just; used in the context of selection of subjects to indicate that the benefits and burdens of research are fairly distributed. (see also Justice)
equivalence trial
A trial with the primary objective of showing that the response to two or more treatments differs by an amount which is clinically unimportant. This is usually demonstrated by showing that the true treatment difference is likely to lie between a lower and an upper equivalence margin of clinically acceptable differences
ethics
The practice of applying a code of conduct based on moral principles to day-to-day actions, on how one should act. May be used to balance what is fair to individuals or organizations and what is right for society (see bioethics).
ethics committee
The Ethics Committee or Independent Ethics Committee (IEC) or Research Ethics Committee (REC) is an independent body (a review board or a committee, institutional, regional, national, or supranational), whose membership comprises medical/scientific professionals and non-medical/non-scientific members. Their responsibility it is to ensure the protection of the rights, safety, and well-being of the people involved in a trial and to provide public assurance of that protection. They review and provide opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting informed consent of the trial subjects, among other things. The legal status, composition, function, operations, and regulatory requirements pertaining to Independent Ethics Committees may differ between countries, but should allow such a committee to act in agreement with the tenets of Good Clinical Practice (GCP).
ethnic factors
Ethnic factors relate to races or large populations grouped according to common traits and customs. Therefore "ethnicity", by virtue of its cultural as well as genetic implications, has a broader meaning than "race". Ethnic factors may be classified as either intrinsic or extrinsic.
EUPATI
European Patients' Academy on Therapeutic Innovation
event
Any medical occurrence in a person during a clinical investigation/trial. This is not the same as "serious event," which is based on patient/event outcome.
evidence-based medicine
Applying the scientific method to medical practice, it uses techniques from science, engineering and statistics, such as meta-analysis of scientific literature, risk-benefit analysis, and randomized controlled trials. It aims for the goal that healthcare professionals should make "conscientious, explicit, and judicious use of current best evidence" in their everyday practice.
ex vivo
Ex vivo is an experimental technique in an artificial environment outside the living organism with a minimum alteration of natural conditions, ie, measuring a physical property at high temperatures (see in vivo and in vitro).
exclusion criteria
The medical or social standards determining whether a person may be allowed to enter a clinical trial. These criteria are based on such factors as age, gender, the type and stage of disease, previous treatment history, and other medical conditions. Inclusion criteria are used to identify appropriate participants, keep them safe, and have a homogeneous population to measure, and is not meant to reject people personally (see inclusion criteria).
excretion
The process of eliminating waste products of metabolism and other materials from an organism that are of no use.
explanatory trials
Explanatory questions ask whether a carefully selected group of patients can benefit from a treatment and, if so, by what biological mechanism. Explanatory trials define a population of roughly equivalent risk by imposing strict eligibility criteria, and test a precise hypothesis about the treatment's biological mode of action. The treatment's effect is assessed through endpoints based on the biological mode of action, so follow-up is generally short and the endpoints are usually laboratory measurements, not clinical outcomes. The protocol gives rigid rules for patient management and study drug discontinuation, and any deviation from the protocol must be carefully described, because it may affect the interpretation of the trial.
exploratory studies
Exploratory studies look at a problem that has not been completely defined and the results can give insight into future directions of research. It helps to gain experience in a new or not-well-defined area to then go on and hypothesize in future studies. The results are rarely generalisable.
extrapolation of clinical data
The projection of known data into a new area, for example, the generalisation and application of the safety data generated in a different population (from a foreign region) to our population.
factorial design
A factorial design evaluates more than one intervention in a single experiment. For example, in a simple 2x2 factorial design, participants are allocated to one of four possible combinations of nicotine replacement and counseling - participants can be allocated to (a) nicotine replacement alone, (b) counseling alone, (c) both, or (d) neither.
FAGG
Federaal Agentschap voor Geneesmiddelen en Gezondheidsproducten, the Belgian Competent Authority, (see NCA).
FDA
US Food and Drug Administration, the US Regulatory (Competent) Authority (see NCA). Like all NCAs, they protect and promote the public's health, and with the EMA, due to the size of the population specifically covered, are considered the global standard.
fimea
Läälealan turvallisuus ja kehittämiskeskus, the Finnish Competent Authority (see NCA).
follow-up
Process of periodic contact with participants after a randomised trial to determine longer term consequences of the intervention.
foreign clinical data
Clinical data generated outside of the region where product registration is sought.
frequentist methods
Statistical methods, such as significance tests and confidence intervals, which can be interpreted in terms of the frequency of certain outcomes occurring in hypothetical repeated realizations of the same experimental situation. (see also Bayesian approaches)
full board review
Review of proposed research at a convened meeting at which a majority of the membership of the IRB are present, including at least one member whose primary concerns are in non-scientific areas. For the research to be approved, it must receive the approval of a majority of those members present at the meeting
GCP
Good clinical practice - A standard for the design, conduct, performance, monitoring, auditing, recording, analyses, and reporting of clinical trials that provides assurance that the data and reported results are credible and accurate, and that the rights, integrity, and confidentiality of trial subjects are protected
generalisability
The extent to which the findings of a clinical trial can be reliably extrapolated from the subjects who participated in the trial to a broader patient population.
generalisation
The extent to which the findings of a clinical trial can be reliably extrapolated from the subjects who participated in the trial to a broader patient population
generic drug
Generic drug (or generics) is a drug defined as "a drug product containing the same active ingredients as the original formulation and is comparable to dosage form, strength, route of administration, quality and performance characteristics (bioequivalence), and intended use
geometric mean
The appropriate measure of central tendency on a multiply-divide scale. On the Standard Celeration Chart you derive a geometric mean by multiplying N number of frequencies and then taking the Nth root of that (see also Mean, Harmonic mean, Median, Mode)
global assessment variable
A single variable, usually a scale of ordered categorical ratings that integrates objective variables and the investigator's overall impression about the state or change in state of a subject
GLP
Good Laboratory Practice - International regulations that must be observed to ensure high quality experimental standards and reliable data
good clinical practice
GCP - A standard for the design, conduct, performance, monitoring, auditing, recording, analyses, and reporting of clinical trials that provides assurance that the data and reported results are credible and accurate, and that the rights, integrity, and confidentiality of trial subjects are protected
good laboratory practice
GLP - International regulations that must be observed to ensure high quality experimental standards and reliable data
guardian
An individual who is authorized under applicable state or local law to give permission on behalf of a child to general medical care
GYEMSZI
Gyógyszerészeti és Egészségügyi Minöség- és Szervezetfejlesztési Intézet the Hungarian Competent Authority. See NCA
half life
The time required for half the amount of a drug to be eliminated from the body
HALMED
Agencija za Lijekove i Medicinske Proizvode, Pharmacovigilance, the Croatian Competent Authority
harmonic mean
A harmonic mean is used when the influence of extreme values is to be minimized. The harmonic mean of n numbers expressed as the reciprocal of the arithmetic mean of the reciprocals of the numbers (see also Mean, Geometric mean, Median, Mode)
harms and benefits
The physical, psychological, social, economic or legal impact of research on a research subject and/or on society. Harms and benefits vary according to the research discipline and the methodology used. They may be difficult to predict
health technology assessment
A multi-disciplinary field of policy analysis that examines the medical, economic, social and ethical implications of the incremental value, diffusion and use of a medical technology in health care
healthy volunteer
A participant in a trial who is not affected by a specific disease or condition. Often, healthy volunteers are used in Phase I studies where efficacy is not the issue, but safety and sometimes dosing.
Helsinki declaration
A series of guidelines adopted by the 18th World Medical Assembly in Helsinki, Finland in 1964. The Declaration addresses ethical issues for physicians conducting biomedical research involving humans. Recommendations include the procedures required to ensure subject safety in clinical trials, including Informed Consent and Ethics Committee reviews. It has been revised eight times, the last two in 2004 and 2008, see http://www.wma.net/en/30publications/10policies/b3/.
historical control
A person, or group of persons, for whom data were collected earlier than for the group being studied. Because of changes over time in risks, prognosis, healthcare, etc, there is a large risk of bias (in studies that use historical controls) due to systematic differences between the comparison groups.
human subjects
Individuals whose physiological or behavioural characteristics and responses are the object of study in a research project. Human subjects are defined as living individuals about whom an investigator conducting research obtains: (1) data through intervention or interaction with the individual; or (2) identifiable private information.
hypersensitivity reactions
Undesirable reactions produced by the immune system, including allergies and autoimmunity. These reactions may be damaging, uncomfortable, or occasionally fatal, and provoke characteristic symptoms whenever they subsequently happen.
IB
Investigator's brochure - A compilation of the clinical and nonclinical data on the investigational product(s) that is relevant to the study of investigational product(s) in human subjects
ICH
International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use: a unique project that brings together the regulatory authorities of Europe, Japan and the United States and experts from the pharmaceutical industry in the three regions to discuss scientific and technical aspects of product registration. The purpose is to make recommendations on ways to achieve greater harmonisation in the interpretation and application of technical guidelines and requirements for product registration in order to reduce or obviate the need to duplicate the testing carried out during the research and development of new medicines.
idiosyncrasy
Specific (and usually unexplained) reaction of an individual to a chemical exposure to which most other individuals do not react at all. For example, some people react to their first aspirin with a potentially fatal shock. General allergic reactions do not fall into this category.
IEC
The Independent Ethics Committee or Ethics Committee or Research Ethics Committee (REC) is an independent body (a review board or a committee, institutional, regional, national, or supranational), whose membership comprises medical/scientific professionals and non-medical/non-scientific members. Their responsibility it is to ensure the protection of the rights, safety, and well-being of human subjects involved in a trial and to provide public assurance of that protection. They review and provide opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and materials to be used in obtaining and documenting informed consent of the trial subjects, among other things. The legal status, composition, function, operations, and regulatory requirements pertaining to Independent Ethics Committees may differ between countries, but should allow such a committee to act in agreement with the tenets of Good Clinical Practice (GCP).
IMB
Irish Medicines Board, the Irish Competent Authority (see NCA).
IMI
Innovative Medicines Initiative is a joint undertaking between the European Commission and EFPIA (European Federation of Pharmaceutical Industry Associations) and is Europe's largest public-private initiative aiming to speed up the development of better and safer medicines for patients. IMI frequently includes academia and other public organisations to enhance R&D in Europe, see www.imi.europa.eu.
impartial witness
A person who is independent of the trial, who cannot be unfairly influenced by people involved with the trial, who attends the informed consent process if the subject or the subject's legally acceptable representative cannot read, and who reads the informed consent form and any other written information supplied to the subject.
in silico
An experiment performed on computer or via computer simulation, for example, finding new drug candidates by screening by computer. The phrase is parallel to the Latin phrases in vivo and in vitro.
in vitro
(lat. "In glass") - An artificial environment created outside a living organism (eg, a test tube or culture plate) used in experimental research to study a disease or process
in vivo
Pertaining to a biochemical process or reaction taking place in a living cell or organism. Compare in vitro
in-kind
material, service or cash contributions but no manpower providing a contribution in goods, commodities or services instead of monetary payments
incapacity
Refers to a person's mental status and means inability to understand information presented, to appreciate the consequences of acting (or not acting) on that information, and to make a choice. Often used as a synonym for incompetence. (See also: Incompetence.)(see also Vulnerable Subjects)
incidence
The frequency of new occurrences of disease within a defined time interval. Incidence rate is the number of new cases of a specified disease divided by the number of people in a population over a specified period of time, usually one year. (see also Prevalence)
inclusion criteria
The medical or social standards determining whether a person may 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 criteria are not used to reject people personally, but rather to identify appropriate participants and keep them safe (see exclusion criteria)
incompetence
A legal term meaning inability to manage one's own affairs, and often used as a synonym for incapacity. (See also: Incapacity.)(see also Vulnerable Subjects)
Independent Ethics Committee
(IEC) or Ethics Committee or Research Ethics Committee (REC) - An independent body (a review board or a committee, institutional, regional, national, or supranational), whose membership comprise smedical/ scientific professionals and nonmedical/ non-scientific members, whose responsibility it is to ensure the protection of the rights, safety, and well-being of human subjects involved in a trial and to provide public assurance of that protection, by, among other things, reviewing and approving/providing favourable opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting informed consent of the trial subjects. The legal status, composition, function, operations, and regulatory requirements pertaining to Independent Ethics Committees (and Research Ethics Committees) may differ among countries, but should allow such a committee to act in agreement with GCP as described in this guideline
independent variable
A variable that is manipulated, measured, or selected by the researcher as an antecedent condition to an observed behaviour. In a hypothesized cause-and-effect relationship, the independent variable is the cause and the dependent variable is the outcome or effect. (see also Dependent variable)
indication
A sign, symptom, or medical condition that leads to the recommendation of a treatment, test, or procedure
induction
Is the process of reasoning to a conclusion about an entire class by examining some of its members. See deductive reasoning (see also Deductive reasoning)
inductive reasoning
Is the process of reasoning to a conclusion about an entire class by examining some of its members. See deductive reasoning (see also Deductive reasoning)
infarmed
Autoridade Nacional do Medicamento e Produtos de Saude, the Portuguese Competent Authority. See NCA
informed consent
or consent - A person's voluntary agreement, based upon adequate knowledge and understanding of relevant information, to participate in research or to undergo a diagnostic, therapeutic, or preventive procedure. In giving informed consent, subjects may not waive or appear to waive any of their legal rights, or release or appear to release the investigator, the sponsor, the institution or agents thereof from liability for negligence. Before carrying out any form of research on an individual, they must be informed about the study's aims, methods, anticipated benefits and any potential risks. The individual must also be informed that they are free to decline to participate in the study and are free to leave at any time without any impact on their treatment. The researcher must then obtain the individual's consent, usually in writing. If this is not possible, non-written consent must be formally documented. (see also Autonomy)
INN
International Non proprietary Name(See also trade name)
innovative medicine initiative
IMI - Joint undertaking between the European Commission and EFPIA (European Federation of Pharmaceutical Industry Associations) to support the pre-competitive collaboration between industry and academia/other public organisations to enhance R&D in Europe, see www.imi.europa.eu
inspection
The act by a regulatory authority(ies) of conducting an official review of documents, facilities, records, and any other resources that are deemed by the authority(ies) to be related to the clinical trial and that may be located at the site of the trial, at the sponsor's and/or contract research organization's (CRO's) facilities, or at other establishments deemed appropriate by the regulatory authority(ies). (see also Audit)
institution
Any public or private entity or agency or medical or dental facility where clinical trials are conducted
institutional review board
IRB - An independent body constituted of medical, scientific, and non-scientific members, whose responsibility it is to ensure the protection of the rights, safety, and wellbeing of human subjects involved in a trial by, among other things, reviewing, approving, and providing continuing review of trials, of protocols and amendments, and of the methods and material to be used in obtaining and documenting informed consent of the trial subjects
intention to treat
ITT - Results of a study is based on the initial treatment assignment and not on the treatment eventually received. Analysing the intention-to-treat population of a study provides unbiased comparisons among the treatment groups. These analyses are done to avoid the effects of crossover and dropout in trials, which may break the random assignment to the treatment groups in a study
intention-to-treat principle
The principle that asserts that the effect of a treatment policy can be best assessed by evaluating on the basis of the intention to treat a subject (i.e., the planned treatment regimen) rather than the actual treatment given. It has the consequence that subjects allocated to a treatment group should be followed up, assessed, and analysed as members of that group irrespective of their compliance to the planned course of treatment. (see also Per protocol)
interaction
qualitative and quantitative - The situation in which a treatment contrast (e.g., difference between investigational product and control) is dependent on another factor (e.g., centre). A quantitative interaction refers to the case where the magnitude of the contrast differs at the different levels of the factor, whereas for a qualitative interaction the direction of the contrast differs for at least one level of the factor
interim analysis
Any analysis intended to compare treatment arms with respect to efficacy or safety at any time prior to the formal completion of a trial
interim clinical trial/study report
A report of intermediate results and their evaluation based on analyses performed during the course of a trial
interpreter reliability
Inter- and interpreter reliability - The level of consistency of a rater (intra) or a group of raters (inter) in making an assessment of treatment outcome
Interreliability
Inter- and interpreter reliability - The level of consistency of a rater (intra) or a group of raters (inter) in making an assessment of treatment outcome
intervention
Includes both physical procedures by which data are gathered (for example, venepuncture) and manipulations of the subject or the subject's environment that are performed for research purposes
intramuscular
injection of a substance directly into a muscle
intravenous
infusion of liquid substances directly into a blood vein
investigational new drug
A new therapeutic product that has been approved for clinical development. The data generated in lab and animal tests suggest its effectiveness in treating a human disease
investigational product
A pharmaceutical form of an active ingredient or placebo being tested or used as a reference in a clinical trial, including a product with a marketing authorization when used or assembled (formulated or packaged) in a way different from the approved form, or when used for an unapproved indication, or when used to gain further information about an approved use
investigator
A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator. See also Subinvestigator. (see also Sponsor/investigator)
investigator's brochure
IB - A compilation of the clinical and nonclinical data on the investigational product(s) that is relevant to the study of investigational product(s) in human subjects
investigator/institution
An expression meaning ''the investigator and/or institution
IRB
Institutional Review Board (US terminology) - An independent body constituted of medical, scientific, and non-scientific members, whose responsibility it is to ensure the protection of the rights, safety, and wellbeing of human subjects involved in a trial by, among other things, reviewing, approving, and providing continuing review of trials, of protocols and amendments, and of the methods and material to be used in obtaining and documenting informed consent of the trial subjects. See Ethics Committee
ITT
Intention of treat - Results of a study is based on the initial treatment assignment and not on the treatment eventually received. Analysing the intention-to-treat population of a study provides unbiased comparisons among the treatment groups. These analyses are done to avoid the effects of crossover and dropout in trials, which may break the random assignment to the treatment groups in a study
IV
Intravenous
jazmp
Javna agencija Republike Slovenije za zdravila in medicinske pripomočke, the Slovenian Competent Authority. See NCA
justice
An ethical principle discussed in the Belmont Report requiring fairness in distribution of burdens and benefits; Justice involves considering the fair distribution of the benefits and burdens of research within society. The duty of researchers is to conduct studies that advance knowledge in a given area to potentially benefit all members of the population. However, the advancement of knowledge cannot be at the expense of individual research participants or particular sub-groups of the population. (see also Belmont principles)
latin square
A Latin square is an n × n table filled with n different symbols in such a way that each symbol occurs exactly once in each row and exactly once in each column. Here are two examples (see also Analysis of variance)
LD 50
The dose of a substance that will kill half (50%) of the treated test animals when given as a single dose. A measure of acute toxicity
legal incompetence
A legal state, defined by procedures spelled out in provincial law, that an individual is unable to consent for him or herself. (see also Vulnerable Subjects)
legally authorized representative
A person authorized either by statute or by court appointment to make decisions on behalf of another person. In human subject research, an individual or judicial or other body authorized under applicable law to consent on behalf of a prospective subject to the subject's participation in the procedure(s) involved in the research
libertarianism
Places the personal freedom of an individual at the centre of analysis. Personal freedom and autonomy are claimed as rights, unless there is sufficient reason to limit them
life expectancy
The average number of years remaining for a living being (or the average for a class of living beings) of a given age to live. Life expectancy is also called average life span or mean life span, in distinction to maximum life span. Life expectancy should not be confused with median survival time (the time at which 50% of a cohort will have died)
logistic regression
A statistical technique that predicts the probability of a dichotomous dependent variable (eg, dead or alive) using, typically, a combination of continuous and categorical independent variables (see also Regression)
loss to follow-up
Loss of contact with some participants, so that researchers cannot complete data collection as planned. Loss to follow-up is a common cause of missing data, especially in long-term studies (see also Drop out)
MA
Marketing Authorisation - A medicinal product must have a valid marketing authorisation before it can be launched onto the market and sold to consumers. Marketing authorisation for a medicinal product can be applied for through the mutual recognition procedure, centralized procedure or national procedure and is in Europe given either by the Commission for products licensed via the centralized procedure or by the National Competent Authorities for product licensed via the other procedures
MAA
Marketing Authorisation Application - A medicinal product must have a valid marketing authorisation before it can be launched onto the market and sold to consumers. Marketing authorisation for a medicinal product can be applied for through the mutual recognition procedure, centralized procedure or national procedure
marketing authorisation
MA - A medicinal product must have a valid marketing authorisation before it can be launched onto the market and sold to consumers. Marketing authorisation for a medicinal product can be applied for through the mutual recognition procedure, centralized procedure or national procedure and is in Europe given either by the Commission for products licensed via the centralized procedure or by the National Competent Authorities for product licensed via the other procedures
marketing authorisation application
MAA - A medicinal product must have a valid marketing authorisation before it can be launched onto the market and sold to consumers. Marketing authorisation for a medicinal product can be applied for through the mutual recognition procedure, centralized procedure or national procedure
mature minor
Someone who has not reached adulthood (as defined by state law) but who may be treated as an adult for certain purposes (e.g., consenting to medical care). (see also Children)
me too drug
A compound that is structurally very similar to already known drugs, with only minor pharmacological differences
mean
A type of average (measure of central tendency), which is defined as the sum of all the values in a set of numerical data divided by n; this value is usually the most common usage of the word "average," and typically it is labelled as either µ (lowercase Greek letter "mu") or m to denote a population mean or x (referred to a "x bar") to denote a sample mean(see also Mean, Geometric mean, Harmonic mean, Median, Mode)
median
The midpoint in a series of numbers; half the data values are above the median, and half are below. For example, in the odd series 1, 4, 9, 12 and 33, 9 is the median. In the even series 1, 4, 10, 12, 33 and 88, 11 is the median (halfway between 9 and 12). Note the median is not necessarily the same as the average (or mean). For example, the median of 2, 6, 10, 22 and 40 is 10 but the average is 18(see also Mean, Geometric mean, Harmonic mean, Median, Mode)
median survival time
The time from either diagnosis or treatment at which half of the patients with a given disease are found to be, or expected to be, still alive. In a clinical trial, median survival time is one way to measure how effective a treatment is
Medicines Authority
Medicines Authority, the Maltese Competent Authority. See NCA
medicines development process
The entire process of bringing a new medicines to patients. It includes drug discovery/product development, pre-clinical research (microorganisms/animals), clinical trials (on humans), the marketing authorisation process and post-authorisation studies
meta-analysis
The formal evaluation of the quantitative evidence from two or more trials bearing on the same question. This most commonly involves the statistical combination of summary statistics from the various trials, but the term is sometimes used to refer to the combination of the raw data
metabolism
The sum of the processes by which a particular substance is handled (as by assimilation and incorporation or by detoxification and excretion) in the living body
MHRA
Medicines and Healthcare products Regulatory Agency, The UK Competent Authority. See NCA
minimal effective dose
Minimum dose required to produce a given effect
minimal risk
A risk is minimal where the probability and magnitude of harm or discomfort anticipated in the proposed research are not greater, in and of themselves, than those ordinarily encountered in daily life or during the performance of routine physical or psychological examinations or tests. For example, the risk of drawing a small amount of blood from a healthy individual for research purposes is no greater than the risk of doing so as part of routine physical examination. The definition of minimal risk for research involving prisoners differs somewhat from that given for non-institutionalized adults
Ministère de la Santé, Direction de la Santé, Divi
Ministère de la Santé, Direction de la Santé, Division de la Pharmacie et des Médicaments, the Luxemburg Competent Authority. See NCA
Ministry of Health , Pharmaceutical Services
Ministry of Health , Pharmaceutical Services, the Cypriote Competent Authority. See NCA
mode
The mode is the value that has the largest number of observations, namely the most frequent value or values. The mode is not necessarily unique, unlike the arithmetic mean. (see also Mean, Geometric mean, Harmonic mean, Median, Mode)
monitoring
The act of overseeing: the progress of a clinical trial, and of ensuring that it is conducted, recorded, and reported in accordance with the protocol, standard operating procedures (SOP's), GCP, and the applicable regulatory requirement(s)
monitoring Report
A written report from the monitor to the sponsor after each site visit and/or other trial related communication according to the sponsor's SOP's
morbidity
Any departure, subjective or objective, from a state of physiological or psychological well-being. In this sense, sickness, illness, and a morbid condition are synonymous
morbidity rate
Number of individuals who become ill with a particular disease within a susceptible population during a specified time period
morbidity table
Actuarial statistics showing the frequency and duration of a sickness. (see also Mortality table)
mortality rate
The annual number of deaths (from a disease or at general) per 1000 people. (see also Morbidity rate)
mortality table
A table showing the incidence of death at specified ages. It shows the number of persons in each age group that die, expressed in terms of deaths per thousand, and based on the deaths in a population of a million persons(see also Morbidity table)
MPA
Läkemedelsverket, the Swedish Competent Authority. See NCA
multicenter trial
A trial involving two or more study centres, a common study protocol, and a single analysis plan pooling the data across all centres. (see also Trial site)
multiple logistic regression
Kind of logistic regression in which there are many variables, including several exponential factors
mutagenicity
The property of a chemical that causes the genetic characteristics of an organism to change in such a way that future generations are permanently affected
national competent authority
NCA - or Regulatory Authority. Bodies having the power to regulate. In the ICH GCP guideline, the expression "Regulatory Authorities" includes the authorities that review submitted clinical data and those that conduct inspections). These bodies are sometimes referred to as competent authorities
national liaison team
A group of national representatives from a patient organisation, academia and industry that work very closely to establish the EUPATI National Platforms
national platforms
Groups established through EUPATI's Work Package 2 to facilitate the implementation, roll-out and awareness raising of EUPATI courses in 12 countries. The platforms aim to develop momentum by encouraging stakeholders to identify the priorities and best national pathways to raise awareness and use of EUPATI's results
NCA
National Competent Authorities - or Regulatory Authorities. Bodies having the power to regulate. In the ICH GCP guideline, the expression ''Regulatory Authorities'' includes the authorities that review submitted clinical data and those that conduct inspections). These bodies are sometimes referred to as competent authorities
negative predictive value
The probability that an individual does not have the tested condition, given that the test result is negative. (see also Positive predictive value)
new chemical entity
In the fields of medicine a “small molecule” is a low molecular weight organic compound typically below 500 Daltons. Most medicines today are “small molecules” which typically interacts with enzymes or receptors in the body. Small molecules are typically administered orally (by mouth) as tablets or fluids
nocebo
The nocebo effect is the phenomenon whereby a patient who believes that a treatment will cause harm actually experiences adverse effects. (see also Placebo)
non-maleficence
Literally, not causing harm, and is a prima facie principle in bioethics, sometimes subsumed under the principle of beneficence. An obligation traditionally at the heart of medical ethics-the "first do no harm" component of the Hippocratic Oath-non-maleficence can be seen as distinct from the obligation to produce good (see also Belmont principles, Beneficence)
normal distribution
The frequency of a data distribution simulating a bell-shaped curve that is symmetrical around the mean and exhibits an equal chance of a data point being above or below the mean. (syn: Gaussian distribution) (see also Poisson distribution)
null hypothesis
The proposal that no difference exists between groups or that there is no association between risk indicator and outcome variables. If the null hypothesis is true then the findings from the study are the result of chance or random factors. In clinical trials, the prediction that there is no relationship between your treatment and your outcome. see alternative hypothesis (see also Alternative hypothesis)
Nüremberg code
As a result of the medical experiments conducted by Nazis during World War II, the Military Tribunal in Nuremberg in 1947 set forth, for the first time, a code of medical ethics for researchers conducting clinical trials. The code is designed to protect the safety and integrity of study participants
obligation
Used interchangeably with duty. That which is required, although tempered by competing duties. Obligations are correlated with rights. In epidemiology and public health, professional role obligations derive from basic ethical principles and are articulated in codes of professional conduct.(see also Duty of care)
observational study
Type of study in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome (for example, no treatment is given)
OD
Once a day
odds ratio
The odds ratio is a measure of effect size particularly important in Bayesian statistics and logistic regression. It is defined as the ratio of the odds of an event occurring in one group to the odds of it occurring in another group, or to a sample-based estimate of that ratio. The increased use of logistic regression in medical and social science research means that the odds ratio is commonly used as a means of expressing the results in some forms of clinical trials, such as case-controlled trials, and in survey research. It is often abbreviated "OR" in reports. (see also Bayesian approaches)
oncogenicity
The ability to transform cells to a neoplastic (proliferative) state
open label trial
A clinical trial in which doctors and participants know which drug or vaccine is being administered
Opt in
Tick box or similar is provided on a consent form asking the individual if they are willing for their personal information to be used for the research purposes. A website or phone number is also provided where the individual can obtain more information
Opt out
Individuals are informed that their personal information may be used for research purposes. Other times, individuals are automatically included in a research database (eg - cervical screening). Instructions are provided as to what should be done if the individual does not wish for their data to be used
order effect
In a repeated measures design, the effect that the order of introducing treatment has on the dependent variable
orphan drug
Medicine developed for rare diseases and conditions which, in the U.S., affect fewer than 200,000 people or, in the EU, affect 5 or fewer per 10,000 people. Because sales of orphan drugs are likely to be small compared to their development costs, pharmaceutical companies are awarded exclusive rights to market these medicines for a period of time as an incentive to develop them
outcome
The impact of care provided to a patient. Outcomes can be positive, such as the ability to walk freely as a result of rehabilitation, or negative, such as the occurrence of bedsores as a result of lack of mobility of a patient(see also Endpoint)
P value
Refers to the probability that the result obtained could have happened by chance. Usually refers to a number derived from a calculation in the study and is displayed as p_ 0.05 or p_ 0.01 or such. This means the likelihood of such a result by chance is less than one in twenty or one in a hundred. The custom is to consider p values of 0.05 or less to signify a significant result, one highly unlikely to happen by chance. (see also Significance level, Statistical significance)
paired data
Data that consists of pairs of related observations eg blood pressure measurement before and after exercise, or data from a matched case-control study. For each observation in one sample there is a corresponding observation in the other sample. The samples are usually of equal size (see also Case control)
parametric test
A test that measures the value of a parameter on the output or a particular point of an assembly. (see also Statistical test)
paternalism
The theory of overriding the decisions of an individual with the intention of benefiting that individual
patient advocacy groups
Organisations, generally non-profit, that focus on supporting and representing patients in various aspects of health care. Patient advocacy groups usually operate on three levels: 1. supporting individual patients by addressing their needs and providing educational resources, 2. representing patients in health policy and public healthcare, 3. collaborating with stakeholders e.g. in research
patient advocate
In the context of EUPATI, a patient advocate is a representative of a patient organisation who influences the health policy environment to ensure it reflects the views of the organisation and a genuine patient perspective
patient ambassador
Patient who is empowered to represent a patient perspective in the medicines development processes by interacting with scientific committees, HTA agencies, industry, regulatory bodies, academia and other relevant actors and has the knowledge to inform other patients about new treatment development process aspects
patient involvement
patients role in activities or decisions that will have consequences for the patient community, because of their specific knowledge and relevant experience as patients
patient journalist
Patient who raises awareness amongst the lay patients, hard-to-reach patients and the lay public implementing a series of communication activities defined by the communication strategy
PD
Pharmacodynamics
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
PEI
Paul Erlich Institut, one of the German Competent Authorities. See NCA
percentile
A descriptive measure used to characterize a set of numbers. For example, if the 8th percentile of a set of 200 numbers has the value 27, then 8 percent of the numbers (16 numbers) are less than 27; the median of the set is the 50th percentile(see also Distribution, Quartile)
periodic safety update report
PSUR - For a medicinal product with a marketing authorisation: All records of adverse reactions shall be submitted to the competent authorities in form of a periodic safety update report, either immediately upon request or periodically as follows: six monthly for the first two years after authorisation, annually for the subsequent two years, and at the time of the first renewal. Thereafter the periodic safety update report shall be submitted at five-yearly intervals together with the application for renewal of the authorisation. The periodic safety update report shall include a scientific evaluation of the benefit and risks afforded by the medicinal products
permission
The agreement of parent(s) or guardian to the participation of their child or ward in research
personalised medicine
a medical model emphasizing in general the customization of healthcare, with all decisions and practices being tailored to individual patients in whatever ways possible
pharmaceutical form
The way the drugs are delivered to the patient
pharmaceutical innovation
Technological progress that can lead to the creation of entirely new therapies, an improvement of the process or administration, or an increase in the therapeutic value of an existing therapy for patients
pharmacodynamics
Is the understanding of the biochemical and physiological effects of medicines on the organism and the relationship between medicine concentration and effect, i.e. what medicine does to the body
pharmacoeconomics
A science which helps determine what benefits a new medicine offers to off set its costs
pharmacokinetic study
A study of how a medicine is handled by the body, usually involving measurement of blood concentrations of drug and its metabolite(s) (sometimes concentrations in urine or tissues) as a function of time. Pharmacokinetic studies are used to characterize absorption, distribution, metabolism and excretion of a drug, either in blood or in other pertinent locations. When combined with pharmacodynamic measures (a PK/PD study) it can characterize the relation of blood concentrations to the extent and timing of pharmacodynamic effects
pharmacokinetics
Pharmacokinetics is a discipline describing the course of substances in the body and their relationship with an organism or system over time. A pharmacokinetic study is a systematic assessment of what the body does to a drug after it has been introduced into the body
pharmacology
The study of how chemical substances interact with living systems. If substances have medicinal properties, they are considered pharmaceuticals
pharmacovigilance
The science of collecting, monitoring, researching, assessing and evaluating information from healthcare providers and patients on the adverse and long term effects of medications
phase I
Exploratory study involving very limited human exposure to the drug, with no therapeutic or diagnostic goals (for example, screening studies, microdose studies). Phase 1: Studies that are usually conducted in healthy volunteers (50-100) with special emphasis on safety assessment. The goal is to find out what the drugs most frequent and serious adverse events are and how the drug is metabolized and excreted
phase II
Studies are conducted in patients (200-700) with the aim to gather data on efficacy i.e. does the drug possess a pharmacological effect for a given disease condition at a tolerable dose. For example, participants receiving the drug may be compared with similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied
phase III
Studies are conducted in patients (1000–5000) to gather more information about safety and efficacy of the drug by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 3 studies are pivotal for getting Marketing Authorisation approval of the drug
phase IV
These studies, also known as post marketing surveillance trials, are usually conducted after Marketing Authorisation of a new drug in order to gather additional information about safety, efficacy, or optimal use
PK
Pharmacokinetics
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. (see also Nocebo, Control group)
PoC
Proof of Concept - Classically, proof of concept can be viewed as studies that demonstrate the clinical relevance of a novel mechanism to treat a disease. The objectives of Proof of Concept: validation of the relevance of novel therapeutic targets and vivo preclinical models to man; define potential biological markets for clinical efficacy or toxicity
poisson distribution
A mathematical expression giving the probability of observing various numbers of a particular event in a sample when the mean probability of that event on any one trial is very small. (see also Distribution, Normal distribution)
PoM
Proof of Mechanism - Relates to the earliest stages of drug development, often pre-clinical (i.e., before the drug is given to humans, or before given to research animals). It could be based on showing that the drug interacts with the intended molecular receptor or enzyme, and/or affects cell biochemistry in the desired manner and direction
PoP
Proof of Principle - Relates to later clinical development, Phase III, typically involving larger numbers of patients treated at doses and durations representative of marketed use, and in randomised comparison to placebo and/or existing active drugs. They aim to show convincing, statistically significant evidence of efficacy and to give a better assessment of safety than is possible in smaller, short term studies
population pharmacokinetic methods
Population pharmacokinetic methods are a population-based evaluation of measurements of systemic drug concentrations, usually two or more per patient under steady state conditions, from all, or a defined subset of, patients who participate in clinical trials
population representative of the new region
A population that includes the major racial groups within the new region
positive predictive value
The probability that an individual with a positive test has, or will develop, a particular disease, or characteristic, that the test is designed to detect. (see also Negative predictive value)
preclinical study
Study that uses live animals or cell cultures to determine the effectiveness and toxicity of a treatment. Preclinical studies take place before any testing in humans is done. (see also Clinical study, In vitro, In vivo, In silico, Animal model)
predictive medicine
A rapidly emerging field of medicine that entails predicting disease and instituting preventive measures in order to either prevent the disease altogether or significantly decrease its impact upon the patient (such as by preventing mortality or limiting morbidity
predictive value
The statistic generated by dividing the number of true positives by the sum of the true positives and false positives (eg the number of cases with truly good care divided by the sum of the cases with truly good care plus those cases classified with good care who did not receive it - i.e., the likelihood that a patient classified as the recipient of good care actually received good care)
prevalence
Number of cases of a disease, infected persons, or persons with some other attribute present during a particular interval of time. It is often expressed as a rate (for example, the prevalence of diabetes per 1,000 persons during a year). See related Incidence (see also Incidence)
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, vitamins, vaccines, minerals, or lifestyle changes
prima facie
A term used to describe bioethical principles as neither rules of thumb nor absolute prescriptions but rather as binding in all cases unless an obligation found in one principle conflicts with another. In such situations, balancing of competing principles is undertaken using the technique of specification
principles
Four prima facie principles (derived from the Belmont Report) remain at the centre of education and debate in bioethics: beneficence, nonmaleficence, respect for persons, and justice. They provide the source of rules for ethical decisions (for example, truth telling, privacy, informed consent, etc). Rules are not deduced from principles (that is, principalism) but rather arise from specification. Users of casuistry may also refer to principles when selecting maxims (see Belmont principles)
prisoner
An individual involuntarily confined in a penal institution, including persons: (1) sentenced under a criminal or civil statue; (2) detained pending arraignment, trial, or sentencing; and (3) detained in other facilities (e.g., for drug detoxification or treatment of alcoholism) under statutes or commitment procedures providing such alternatives to criminal prosecution or incarceration in a penal institution. (see also Vulnerable Subjects)
privacy
The right of an individual to be secure from unauthorized disclosure to third parties of information about oneself, such as that which is contained in medical records
probabilistic matching
The linking of records using mathematical algorithms to determine the likelihood that two or more records from various data sets represent the same individual. The decision on whether two records matched is based on the total 'match score'; often clerical review is undertaken to match scores close to the cut-off value set by the researchers
prodrug
A prodrug is a pharmacological substance (drug) which is administered in an inactive (or significantly less active) form. Once administered, the prodrug is metabolised in the body (in vivo) into the active compound
proof of concept
PoC - Classically, proof of concept can be viewed as studies that demonstrate the clinical relevance of a novel mechanism to treat a disease. The objectives of Proof of Concept: validation of the relevance of novel therapeutic targets and vivo preclinical models to man; define potential biological markets for clinical efficacy or toxicity
proof of mechanism
PoM - Relates to the earliest stages of drug development, often pre-clinical (i.e., before the drug is given to humans, or before given to research animals). It could be based on showing that the drug interacts with the intended molecular receptor or enzyme, and/or affects cell biochemistry in the desired manner and direction
proof of principle
PoP - Relates to later clinical development, Phase III, typically involving larger numbers of patients treated at doses and durations representative of marketed use, and in randomised comparison to placebo and/or existing active drugs. They aim to show convincing, statistically significant evidence of efficacy and to give a better assessment of safety than is possible in smaller, short term studies
proportionism
Acknowledges rules and values, but does not regard them as universal. The theory takes into account the human nature of the individual, the situation, and the intentions behind an individual's actions. A broad idea of doing good is aimed for. Proportionism can be viewed as a compromise between the extremes of absolutism and relativism
prospective study
Study designed to observe outcomes or events that occur subsequent to the identification of the group of subjects to be studied. Prospective study needs not involve manipulation or intervention but may be purely observational or involve only the collection of data. (see also Retrospective study)
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
protocol amendment
A written description of a change(s) to or formal clarification of a protocol
PSUR
Periodic Safety Update Report - For a medicinal product with a marketing authorisation: All records of adverse reactions shall be submitted to the competent authorities in form of a periodic safety update report, either immediately upon request or periodically as follows: six monthly for the first two years after authorisation, annually for the subsequent two years, and at the time of the first renewal. Thereafter the periodic safety update report shall be submitted at five-yearly intervals together with the application for renewal of the authorisation. The periodic safety update report shall include a scientific evaluation of the benefit and risks afforded by the medicinal products
QA
Quality Assurance - All those planned and systematic actions that are established to ensure that the trial is performed and the data are generated, documented (recorded), and reported in compliance with GCP and the applicable regulatory requirement(s)
QALY
A measure of the outcome of actions (either individual or treatment interventions) in terms of their health impact. If an action gives a person an extra year of healthy life expectancy, that counts as one QALY. If an action gives a person an extra year of unhealthy life expectancy (partly disabled or in some distress), it has a value of less than one. Death is rated at zero
QC
Quality Control - The operational techniques and activities undertaken within the quality assurance system to verify that the requirements for quality of the trial-related activities have been fulfilled
QD
Once a day (from the Latin "quaque die")
quality assurance
QA - All those planned and systematic actions that are established to ensure that the trial is performed and the data are generated, documented (recorded), and reported in compliance with GCP and the applicable regulatory requirement(s)
quality control
QC - The operational techniques and activities undertaken within the quality assurance system to verify that the requirements for quality of the trial-related activities have been fulfilled
quality of life QOL
The overall enjoyment of life. Many clinical trials measure aspects of a patient's sense of well-being and ability to perform various tasks to assess the effects that cancer and its treatment have on the patient
quartile
One of four segments of a distribution that has been divided into quarters. For example, the second-from-the-bottom quartile of an income distribution is those whose income exceeds the incomes of from 25% to 50% of the population. The three quartile points that lie between the extremes of the distribution are designated as Q 1, Q 2, and Q 3 and are defined in terms of the distribution function F(x) as follows: Thus, Q 2 coincides with the median. (see also Distribution, Percentile, Q1, Q2, Q3)
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
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
Raviamiamet
State Agency of Medicines, the Estonian Competent Authority. See NCA
REC
Research Ethics Committee or Independent Ethics Committee (IEC) or Ethics Committee - An independent body (a review board or a committee, institutional, regional, national, or supranational), whose membership comprise smedical/ scientific professionals and nonmedical/ non-scientific members, whose responsibility it is to ensure the protection of the rights, safety, and well-being of human subjects involved in a trial and to provide public assurance of that protection, by, among other things, reviewing and approving/providing favourable opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting informed consent of the trial subjects. The legal status, composition, function, operations, and regulatory requirements pertaining to Independent Ethics Committees (and Research Ethics Committees) may differ among countries, but should allow such a committee to act in agreement with GCP as described in this guideline
recruitment
Process used by investigators to enrol appropriate subjects into a clinical study, i.e. those selected on the basis of the protocol's inclusion and exclusion criteria
registry
A record that chronicles information about all new disease cases, to gain an understanding of the demographic patterns and aetiology of the disease
regulatory authorities
Bodies having the power to regulate. In the ICH GCP guideline, the expression ''Regulatory Authorities'' includes the authorities that review submitted clinical data and those that conduct inspections). These bodies are sometimes referred to as competent authorities
reimbursement
The reimbursement of legitimate expenses incurred by a subject taking part in a specific research project
relativism
This theory claims that an individual's personal belief or approval is what makes an action morally right. The basic belief is that morality is not grounded in reasoning, and thus it is the individual's feelings or beliefs that are the only possible means for morally justifying an action
remuneration
Payment for participation in research. Remuneration should be appropriate for the amount of effort involved, and not excessive and thereby coercive. Remunerations are not considered a benefit
research
A systematic investigation (i.e., the gathering and analysis of information) designed to develop or contribute to generalisable knowledge
Research Ethics Committee
(REC) or Independent Ethics Committee (IEC) or Ethics Committee- An independent body (a review board or a committee, institutional, regional, national, or supranational), whose membership comprise smedical/ scientific professionals and nonmedical/ non-scientific members, whose responsibility it is to ensure the protection of the rights, safety, and well-being of human subjects involved in a trial and to provide public assurance of that protection, by, among other things, reviewing and approving/providing favourable opinion on the trial protocol, the suitability of the investigator(s), facilities, and the methods and material to be used in obtaining and documenting informed consent of the trial subjects. The legal status, composition, function, operations, and regulatory requirements pertaining to Independent Ethics Committees (and Research Ethics Committees) may differ among countries, but should allow such a committee to act in agreement with GCP as described in this guideline
respect for persons
An ethical principle discussed in the Belmont Report. Respect for persons concerns individual autonomy and seeks to ensure that an individual's wishes are respected, even if they differ from those of the researcher. Allowing individuals to provide informed consent, cease participating in the study if they so desire, and keeping any personal information confidential are processes that uphold the dignity and individuality of research participants. Persons should be treated as ends in themselves and not as means to an end. Implies two distinct moral requirements: acknowledge autonomy and protect those with diminished autonomy. (see also Autonomy, Belmont principles, Confidentiality, Prima facie, Principles,)
retrospective study
Research conducted by reviewing records from the past (e.g., birth and death certificates, medical records, school records, or employment records) or by obtaining information about past events elicited through interviews or surveys. Case control studies are an example of this type of research. (see also Case control study)
review
(of research). The concurrent oversight of research on a periodic basis. In addition to the at least annual reviews mandated by the federal regulations, reviews may, if deemed appropriate, also be conducted on a continuous or periodic basis. (see also Audit)
rights
Justified claims made by individuals or groups upon others and based on a system of rules authorising us to affirm or demand what is due. Possessing a right validly constrains others from interfering with the exercise of that right. Moral rights are claims justified by moral principles and are correlated with obligations. In public health, a broadly defined set of human rights are often asserted
rights-based ethics
Concerns whether any proposed actions violate any fundamental human rights or legal rights, such as the right to privacy or the right to free speech. The focus is on moral principle rather than on consequences
risk
The probability of harm or injury (physical, psychological, social, or economic) occurring as a result of participation in a research study. Both the probability and magnitude of possible harm may vary from minimal to significant. Federal regulations define only "minimal risk." (See also: Minimal Risk.)
risk factor
Something that may increase the chance of developing a disease. For instance, some examples of risk factors for cancer include age, a family history of certain cancers, use of tobacco products, certain eating habits, obesity, exposure to radiation or other cancer-causing agents, and certain genetic changes
risk-benefit assessment
An assessment which compares the benefit gained through administration to the risk in order to judge whether there is a greater health risks or a greater health benefits to the user of a medicinal product resulting in a benefit risk balance.( See risk-benefit balance). Risk - benefit assessment is one of the fundamental activities in licensing of medicines and in clinical research. Human beings (as individuals or population) should not be administered any medicinal products where the risk of negative affects outweighs the potential benefit. This is the opposite of benefit - risk assessment which looks at the risks compared to the benefit
risk-benefit balance
The result of the risk-benefit assessment which must be negative (of greater benefit than risk or the same benefit as risk) to have medicinal product licensed and keep it on the market or to continue a clinical trial. See risk-benefit assesment
route of administration
It can be intravenous, intramuscularly, subcutaneous, oral, topically, etc
SAE
Serious Adverse Event or Serious Adverse Drug Reaction (Serious ADR). Any untoward medical occurrence that at any dose: - Results in death, - Is life-threatening, - Requires inpatient hospitalization or prolongation of existing hospitalization, - Results in persistent or significant disability/incapacity, or - Is a congenital anomaly/birth defect. (See the ICH Guideline for Clinical Safety Data Management: Definitions and Standards for Expedited Reporting.)
safety and tolerability
The safety of a medical product concerns the medical risk to the subject, usually assessed in a clinical trial by laboratory tests (including clinical chemistry and haematology), vital signs, clinical adverse events (diseases, signs and symptoms), and other special safety tests (e.g., electrocardiograms, ophthalmology). The tolerability of the medical product represents the degree to which overt adverse effects can be tolerated by the subject
screening trials
Refers to trials which test the best way to detect certain diseases or health conditions
SD
Standard deviation - In probability and statistics, the standard deviation is the most common measure of statistical dispersion. Simply put, standard deviation measures how spread out the values in a data set are. More precisely, it is a measure of the average distance of the data values from their mean. If the data points are all close to the mean, then the standard deviation is low (closer to zero). If many data points are very different from the mean, then the standard deviation is high (further from zero). If all the data values are equal, then the standard deviation will be zero
selection bias
An error in choosing the individuals or groups to take part in a study. Ideally, the subjects in a study should be very similar to one another and to the larger population from which they are drawn (for example, all individuals with the same disease or condition). If there are important differences, the results of the study may not be valid (see also Bias)
sequential analysis
A statistical technique in which the sample size is not fixed in advance, rather, sampling is stopped as soon as significant results are observed. The criteria for stopping the trials at each sample size are set so that the overall probability (for all sample sizes) of falsely rejecting the null hypothesis at any step is held to a preset level. Cf. hypothesis test
serious ADR
Serious Adverse Drug Reaction or Serious Adverse Event (SAE). Any untoward medical occurrence that at any dose: - Results in death, - Is life-threatening, - Requires inpatient hospitalization or prolongation of existing hospitalization, - Results in persistent or significant disability/incapacity, or - Is a congenital anomaly/birth defect. (See the ICH Guideline for Clinical Safety Data Management: Definitions and Standards for Expedited Reporting.)
serious adverse drug reaction
Serious Adverse Drug Reaction (Serious ADR)or Serious Adverse Event (SAE). Any untoward medical occurrence that at any dose: - Results in death, - Is life-threatening, - Requires inpatient hospitalization or prolongation of existing hospitalization, - Results in persistent or significant disability/incapacity, or - Is a congenital anomaly/birth defect. (See the ICH Guideline for Clinical Safety Data Management: Definitions and Standards for Expedited Reporting.)
serious adverse event
Serious Adverse Event (SAE) or Serious Adverse Drug Reaction (Serious ADR). Any untoward medical occurrence that at any dose: - Results in death, - Is life-threatening, - Requires inpatient hospitalization or prolongation of existing hospitalization, - Results in persistent or significant disability/incapacity, or - Is a congenital anomaly/birth defect. (See the ICH Guideline for Clinical Safety Data Management: Definitions and Standards for Expedited Reporting.)
significance level
A test is the maximum probability that the observed statistic would be observed under the null hypothesis that is considered consistent (see also P value, Statistical significance)
significant difference
A "significant difference" is a difference that is of clinical or public health importance, based on substantial scientific data. This definition differs from the commonly used "statistically significant difference," which refers to the event that, for a given set of data, the statistical test for a difference between the effects in two groups achieves statistical significance. Statistical significance depends upon the amount of information in the data set. With a very large amount of information, one could find a statistically significant, but clinically small difference that is of very little clinical importance. Conversely, with less information one could find a large difference of potential importance that is not statistically significant
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 also Blinding, Double blind study)
site visit
A visit by agency officials, representatives, or consultants to the location of a research activity to assess the adequacy of IRB protection of human subjects or the capability of personnel to conduct the research
small molecules
In the fields of medicine a “small molecule” is a low molecular weight organic compound typically below 500 Daltons. Most medicines today are “small molecules” which typically interacts with enzymes or receptors in the body. Small molecules are typically administered orally (by mouth) as tablets or fluids
SMR
Standardised Mortality Ratio - The rate of deaths relative to national average rates after adjustments have been made for the age structure and relative social status (or deprivation) of the study population.(see also Mortality rate)
SOP
Standard Operating Procedures - Detailed, written instructions to achieve uniformity of the performance of a specific function
source data
All information in original records and certified copies of original records of clinical findings, observations, or other activities in a clinical trial necessary for the reconstruction and evaluation of the trial. Source data are contained in source documents (original records or certified copies)
specification
A methodological technique for interpreting a more general ethical principle to bring its implications closer to-to better "apply" it to-actions and decisions. Specification may be used to resolve conflicts among, to balance, or to rank principles. In public health, the Precautionary Principle is a specified version of the more general principle of beneficence (see also: Principles and Principalism)
sponsor
An individual, company, institution, or organization that takes responsibility for the initiation, management, and/or financing of a clinical trial
sponsor-investigator
An individual who both initiates and conducts, alone or with others, a clinical trial, and under whose immediate direction the investigational product is administered to, dispensed to, or used by a subject. The term does not include any person other than an individual (e.g., it does not include a corporation or an agency). The obligations of a sponsor-investigator include both those of a sponsor and those of an investigator
SST
Sundhedsstyrelsen, The Danish Competent Authority. See NCA
standard deviation
SD - In probability and statistics, the standard deviation is the most common measure of statistical dispersion. Simply put, standard deviation measures how spread out the values in a data set are. More precisely, it is a measure of the average distance of the data values from their mean. If the data points are all close to the mean, then the standard deviation is low (closer to zero). If many data points are very different from the mean, then the standard deviation is high (further from zero). If all the data values are equal, then the standard deviation will be zero
standard error mean
A measure of variability. The standard error of the mean quantifies how accurately the true population mean is known. A measure of the variability of the mean of the sample as an estimate of the true value of the population mean. The larger the sample size the smaller the standard error of the mean. Used in computing confidence intervals. In a clinical trial, the larger the sample size the tighter the 95% CI is around the point estimate of the study (see also Standard deviation, Variance)
standard operating procedures
SOP's - Detailed, written instructions to achieve uniformity of the performance of a specific function
standard treatment
A treatment currently in wide use and approved, considered to be effective in the treatment of a specific disease or condition. (see also Control group)
standardised mortality ratio
SMR - The rate of deaths relative to national average rates after adjustments have been made for the age structure and relative social status (or deprivation) of the study population.(see also Mortality rate)
statistical distribution
The frequency of occurrence of the values of a variable. For each possible value of the variable, there is an associated frequency with which the variable assumes that value. frequency histogram: a graphic that displays how many measures fall into different classes, giving the frequency at which each category is seen observed. frequency polygon: a graphic presentation of frequency of a phenomenon that typically uses straight lines and points. (see also Normal distribution, Poisson distribution)
statistical significance
In statistics, a result is significant if it is unlikely to have occurred by chance, given that in reality, the independent variable (the test condition being examined) has no effect, or, formally stated, that a presumed null hypothesis is true. See also Significant Difference. (see also Significance level, Significance level)
statistical test
Type of statistical procedure that is applied to data to determine whether the results are statistically significant (that is, the outcome is not likely to have resulted by chance alone) (see also t test)
statistics
Branch of mathematics that deals with the collection and interpretation of numerical information
stratified sampling
A method of sampling from a population. When sub-populations vary considerably, it is advantageous to sample each subpopulation (stratum) independently. Stratification is the process of grouping members of the population into relatively homogeneous subgroups before sampling. The strata should be mutually exclusive: every element in the population must be assigned to only one stratum. The strata should also be collectively exhaustive: no population element can be excluded. Then random or systematic sampling is applied within each stratum. This often improves the representativeness of the sample by reducing sampling error. It can produce a weighted mean that has less variability than the arithmetic mean of a simple random sample of the population
study coordinators
Are typically members of a research team that are responsible for such things as, recruiting, screening, and enrolling study participants, as well as ensuring the adherence to Good Clinical Practice guidelines
subcutaneous
Administration of a drug into the layer of skin directly below the dermis and epidermis
subinvestigator
Any individual member of the clinical trial team designated and supervised by the investigator at a trial site to perform critical trial-related procedures and/or to make important trial-related decisions (e.g., associates, residents, research fellows). See also Investigator. (see also Investigator)
subject
A volunteer who participates in a clinical trial, either as a patient recipient of the experimental treatment, or as a control who receives the standard treatment, or as a healthy volunteer recipient who receives either the experimental treatment or no treatment
SUKL(CZ)
Státní ústav pro kontrolu léčiv, the Czech Competent Authority. See NCA
SUKL(SK)
Štátny ústav pre kontrolu liečiv, the Slovakian Competent Authotity. See NCA
superiority trial
A trial with the primary objective of showing that the response to the investigational product is superior to a comparative agent (active or placebo control)
surrogate endpoint
An outcome measure that is used in place of a primary endpoint (outcome). Examples are decrease in blood pressure as a predictor of decrease in strokes and heart attacks in hypertensive patients, and increase in T-cell (a type of white blood cell) counts as an indicator of improved survival of AIDS patients. Use of a surrogate endpoint assumes that it is a reliable predictor of the primary endpoint(s) of interest (see also Clinical endpoint, Endpoint)
surrogate variable
A variable that provides an indirect measurement of effect in situations where direct measurement of clinical effect is not feasible or practical
surveys
Studies designed to obtain information from a large number of respondents through written questionnaires, telephone interviews, door-to-door canvassing, or similar procedures
survival analysis
A branch of statistics which deals with death in biological organisms and failure in mechanical systems. This topic is called reliability theory or reliability analysis in engineering, and duration analysis or duration modelling in economics. Death or failure is called an "event" in the survival analysis literature, and so models of death or failure are generically termed time-to-event models
SUSAR
A SUSAR is a Suspected Unexpected Serious Adverse Reaction: serious adverse reactions in subjects given a drug, that may or may not be dose related, but are unexpected, ie an adverse reaction, the nature or severity of which is not consistent with the applicable product information (e.g., Investigator's Brochure for an unapproved investigational product or package insert/summary of product characteristics for an approved product). A SUSAR may occur during clinical trials or clinical care. Reporting is mandatory for clinical investigators in the EU
swissmedic
Swissmedic, the Swiss Competent Authority. See NCA
syllabus
A summary or list of the main topics of a course of study, text, or lecture
t test
A statistical test which computes the probability (p) that two groups of a single parameter are members of the same population. The population must follow at distribution. However, at test is robust enough to allow considerable deviation from normality
teratogenicity
Potential side effect of some drugs producing physical defects in offspring in utero (i.e., birth defects)
therapeutic dose range
The difference between the lowest effective dose and the highest dose that gives further benefit
therapeutic innovation
Technological progress that can lead to the creation of entirely new therapies, an improvement of the process or administration, or an increase in the therapeutic value of an existing therapy for patients
tid
three times a day
tolerance
A condition in which higher doses of a drug are required to produce the same effect as during initial use; often leads to physical dependence
toxicology
Study of the adverse effects of chemicals on living organisms. It is the study of symptoms, mechanisms, treatments and detection of poisoning, especially the poisoning of people. The chief criterion regarding the toxicity of a chemical is the dose, i.e. the amount of exposure to the substance. Almost all substances are toxic under the right conditions
trainer of trainer
expert patients trained to become trainers in order to scale up the education programme in their countries
treatment effect
An effect attributed to a treatment in a clinical trial. In most clinical trials, the treatment effect of interest is a comparison (or contrast) of two or more treatments
treatment emergent event
An event that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state
treatment group
The group of participants that receives an experimental treatment. See also control group, standard treatment. *(see also arm, control subjects)
treatment trials
Refers to trials which test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy
trial site
The location(s) where trial-related activities are actually conducted. (see also Multicentre trial)
Type I Error
Alpha error - A rejection of a hypothesis when it is true. An example of Type 1 Error is finding a substance efficient when it is not. This is an error of "seeing too much in the data
Type II Error
Beta error - An acceptance of a hypothesis when it is false. An example of Type II Error is not finding a substance present when it is present. This is an error of "not seeing enough in the data."
Type III error
A Type III error consists of correctly rejecting the null hypothesis, but incorrectly attributing the cause. In other words, the researcher correctly identifies an effect, but incorrectly attributes the cause of the effect
unexpected adverse drug reaction
An adverse reaction, the nature or severity of which is not consistent with the applicable product information (e.g., Investigator's Brochure for an unapproved investigational product or package insert/summary of product characteristics for an approved product). (See the ICH Guideline for Clinical Safety Data Management: Definitions and Standards for Expedited Reporting.)
unexpected adverse reaction
An adverse reaction, the nature, or severity of which is not consistent with the applicable product information (e.g. investigator's brochure for an unapproved investigational product or summary of product characteristics (SmPC) for an authorised product)
URPL
Urząd Rejestracji Produktów Leczniczych, Wyrobow Medycznych i Produktów Biobójczych, the Polish Competent Authority. See NCA
utilitarian
And utility - an approach to ethics asserting that one should always strive to produce the greatest possible balance of good over harm. Classically expressed as the obligation to produce the greatest good for the greatest number; more recent accounts emphasise optimisation of benefits and harms
utilitarianism
Concerns the notion that the 'right' thing to do is that which maximises the good. The outcome of an action is what is considered to be ethically important, with the greatest good for the greatest number of people being pursued
utility
and utilitarian - An approach to ethics asserting that one should always strive to produce the greatest possible balance of good over harm. Classically expressed as the obligation to produce the greatest good for the greatest number; more recent accounts emphasise optimisation of benefits and harms
valid analysis
This term means an unbiased assessment. Such an assessment will, on average, yield the correct estimate of the difference in outcomes between two groups of subjects. Valid analysis can and should be conducted for both small and large studies. A valid analysis does not need to have a high statistical power for detecting a stated effect. The principal requirements for ensuring a valid analysis of the question of interest are: allocation of study participants of both sexes/genders (males and females) and from different racial/ethnic groups to the intervention and control groups by an unbiased process such as randomization; unbiased evaluation of the outcome(s) of study participants; and use of unbiased statistical analyses and proper methods of inference to estimate and compare the intervention effects among the gender and racial/ethnic groups
valid consent
In order for an individual to provide valid consent they must be capable of communicating their wishes effectively. Age, language difficulties and physical and mental disabilities may prevent a person from either giving or withholding consent. Consent must be given voluntarily and without coercion. The individual must have a complete understanding of the implications and potential consequences of giving consent. The consent given must be specific and current, in that consent given in particular circumstances cannot be generalised to other similar situations that the individual may be involved in. (see also Consent)
values
Concepts used to explain how and why things matter. Values are involved wherever we distinguish between things good and bad, better or worse. Values are characterised as scientific, professional, cultural, social, personal, family, religious, and organic (for example, health). Scientific values include: objectivity, accuracy, generalisability, validity and others. Values are pervasive in epidemiology and public health.2
variance
A measure of the spread of the values in a distribution. The larger the variance, the larger the distance of the individual cases from the group mean. (see also Standard deviation, Analysis of variance, Confidence interval)
virtues
Character traits-neither skills nor techniques- that make an individual a good professional practitioner, and help her to do her work well. For epidemiologists, the virtues of excellence, integrity, honesty, self effacement, and prudence are important examples. Virtue is not easily taught but may be learned by example from mentors
voluntary
Free of coercion, duress, or undue inducement or influence. Used in the research context to refer to a subject's decision to participate (or to continue to participate) in a research activity
vulnerable subjects
Individuals whose willingness to volunteer in a clinical trial may be unduly influenced by the expectation, whether justified or not, of benefits associated with participation, or of a retaliatory response from senior members of a hierarchy in case of refusal to participate. Examples are members of a group with a hierarchical structure, such as medical, pharmacy, dental and nursing students, subordinate hospital and laboratory personnel, employees of the pharmaceutical industry, members of the armed forces, and persons kept in detention. Other vulnerable subjects include patients with incurable diseases, persons in nursing homes, unemployed or impoverished persons, patients in emergency situations, ethnic minority groups, homeless persons, nomads, refugees, minors, and those incapable of giving consent. (see also Prisoner)
VVKT
the Lithuanian Competent Authority. See NCA
wash out period
Interruption in ongoing treatment
well-being
(of the trial subjects) - The physical and mental integrity of the subjects participating in a clinical trial
Wiki
a website that allows the creation and editing of any number of interlinked web pages via a web browser using a simplified mark-up language or a WYSIWYG text editor
work package
Organizational structure within EUPATI that has its own defined area of work, objectives, tasks and deliverables. The EUPATI project is divided into seven different work packages
xenobiotic
A biologically (pharmacologically, endocrinologically, and/or toxicologically) active substance that is not produced endogenously and is, therefore, foreign to a living organism. Examples include drugs, dietary components, carcinogens, and chemicals that have been introduced into the environment by artificial means
ZVA
Zāļu valsts aģentūra, the Latvian Competent Authority. See NCA