PCR terms

the process by which medications reach the blood stream when administered other than intravenously, for example, through nasal membranes

any noxious and unintended response associated with the use of a drug in humans

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

the likelihood that a relationship observed between two variables is due to chance. The probability of a type 1 error

an organized collection of data or information with a common theme arranged in rows and columns and represented as a single file; comparable to a database table

a set of subjects whose data are to be included in the main analyses. This should be defined in the statistical section of the protocol

personal data which have been processed to make it impossible to know the person with whom the data are associated. Applicable particularly for secondary use of health data.

a measurement, evaluation, or judgment for a study variable pertaining to the status of a subject

a systematic and independent examination of trial-related activities and documents to determine whether the evaluated trial-related activities were conducted and the data were recorded, analyzed, and accurately reported according to the protocol, sponsors standard operating procedures, good clinical practice and the applicable regulatory requirements

trial in which a particular type of subject is equally represented in each study group

assessment of subjects as they enter a trial and before they receive any treatment

demographic, clinical, and other data collected for each participant at the beginning of the trial before the intervention is administered

statistical approach named for Thomas Bayes that has among its features giving a subjective interpretation to probability, accepting the idea that it is possible to talk about the probability of hypotheses being true and of parameters having particular values

probability of showing no significant difference when a true difference exists; a false acceptance of the null hypothesis

situation or condition that causes a result to depart from the true value in a consistent direction. Refers to defects in study design or measurement

rate and extent to which a drug is absorbed or is otherwise available to the treatment site in the body

scientific basis on which drugs with the same active ingredients are compared. To be considered bioequivalent, the bioavailability of two products must not differ significantly when the two products are given in studies at the same dosage under similar conditions

a characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention

branch of statistics applied to the analysis of biological phenomena

checking and assessing data prior to breaking the blind, for the purpose of finalizing the planned analysis

products that appear identical in size, shape, color, flavor and other attributes to make it very difficult for subjects and investigators to determine which medication is being administered

study in which the subject, the investigator or anyone assessing the outcome is unaware of the treatment assignments. Used to reduce potential for bias

procedure to limit bias by preventing subjects and/or study personnel from identifying which treatments or procedures are administered, or from learning the results of tests and measures undertaken as part of a clinical investigation

effects of treatment that persist after treatment has been stopped, sometimes beyond the time of a medication’s known biological activity

a printed, optical, or electronic document designed to record all of the protocol-required information to be reported to the sponsor for each trial subject; a record of clinical study observations and other information that a study protocol designates must be completed for each subject

data evaluated by sorting values into various categories

an evaluation performed by a medical professional concerning the likelihood that a therapy or product under study caused or contributed to an adverse event

a therapeutic intervention may be said to confer clinical benefit if it prolongs life, improves function, and/or improves the way a subject feels

data pertaining to the medical well-being or status of a patient or subject

document that describes the collection of clinical studies that are to be performed in sequence, or in parallel, with a particular active substance, device, procedure or treatment strategy, typically with the intention of submitting them as part of an application for a marketing authorization

power or capacity to produce a desired effect

science that deals with the characteristics, effects, properties, reactions and uses of drugs, particularly their therapeutic value in humans, including their toxicology, safety, pharmacodynamics and pharmacokinetics

the testing of a drug compound in humans primarily done to determine its safety and pharmacological effectiveness. Clinical development is done in phases, which progress from very tightly controlled dosing of a small number of subjects to less tightly controlled studies involving large numbers of patients

person employed by a sponsor or by a contract research organization acting on a sponsor’s behalf, who monitors the progress of investigator sites participating in a clinical study.

person who handles most of the administrative responsibilities of a clinical trial on behalf of a site investigator, acts as liaison between investigative site and sponsor, and reviews all data and records before a monitor’s visit

change in a subject’s clinical condition regarded as important whether or not due to the test information

a written description of a study of any therapeutic, prophylactic, or diagnostic agent conducted in human subjects, in which the clinical and statistical description, presentations and analysis are fully integrated into a single report

a research investigation involving human subjects that is designed to answer specific questions about the safety and efficacy of a biomedical intervention

complete set of supplies provided to an investigator by the trial sponsor

a group of individuals who share a common exposure, experience or characteristic

study of a group of individuals, some of whom are exposed to a variable of interest, in which subjects are followed over time.

one in which the investigative drug is compared against another product, either active drug or placebo

an investigational or marketed product or placebo used as a reference in a clinical trial

a measure of the precision of an estimated value. The interval represents the range of values, consistent with the data, that is believed to encompass the “true” value with high probability (usually 95%). The confidence interval is expressed in the same units as the estimate. Wider intervals indicate lower precision; narrow intervals, greater precision

phase 3 trial during which the previously revealed actions of a therapeutic intervention are confirmed. NOTE: procedures in confirmatory trials should be set firmly in advance

document used during the informed consent process that is the basis for explaining to potential subjects the risks and potential benefits of a study and the rights and responsibilities of the parties involved

a person or an organization contracted by the sponsor to perform one or more of a sponsor’s trial-related duties and functions

the group of subjects in a controlled study that receives no treatment, a standard treatment or a placebo

a comparator against which the study treatment is evaluated; computer: processes or operations intended to ensure authenticity, integrity and confidentiality of electronic records

a study in which a test article is compared with a treatment that has known effects. The control group may receive no treatment, active treatment, placebo or dose comparison concurrent control.

the degree to which two or more variables are related. Typically the linear relationship is measured with either Pearson’s correlation or Spearman’s rho

factor or condition that influences outcome of a trial

case report form in which the data items are linked by the physical properties of paper to particular pages. NOTE: data are captured manually and any comments, notes, and signatures are also linked to those data items by writing or typescript on the paper pages

a trial design for which subjects function as their own control and are assigned to receive investigational product and controls in an order determined by randomizations, typically with a washout period between the two products

group of individuals with pertinent expertise that reviews on a regular basis accumulating data from an ongoing clinical trial. The DMC advises the sponsor regarding the continuing safety of current participants and those yet to be recruited, as well as the continuing validity and scientific merit of the trial.

tasks associated with the entry, transfer and/or preparation of source data and derived items for entry into a clinical trial database

process by which clinical data are examined for completeness, consistency and accuracy

(1) checking data for correctness and/or compliance with applicable standards, rules, and conventions. (2) process used to determine if data are inaccurate, incomplete or unreasonable. The process may include format checks, completeness checks, check key tests, reasonableness checks, and limit checks

a collection of data or information, typically organized for ease and speed of search and retrieval

action taken to prevent further changes to a clinical trial database

a set of recommendations or basic principles that guide medical doctors in the conduct of biomedical research involving human subjects. It was originally adopted by the 18th World Medical Assembly and recently revised

removal of elements connected with data which might aid in associating those data with an individual. Examples include name, birth date, social security number, home address, telephone number, email address, medical record numbers, health plan beneficiary numbers, full-face photographic images, etc.

characteristics of subjects or study populations, which include such information as age, sex, family history of the disease or condition for which they are being treated, and other characteristics relevant to the study in which they are participating.

outcomes that are measured in an experiment and that are expected to change as a result of an experimental manipulation of the independent variables

(1) in statistics, a group of ordered variables; the frequencies or relative frequencies of all possible values of a characteristic. (2) in pharmacokinetics, the processes that control transfer of a drug from the site of measurement to its target and other tissues

a study in which neither the subject nor the investigator nor the research team interacting with the subject or data during the trial knows what treatment a subject is receiving

a technique for retaining the blind when administering supplies in a clinical trial, when the two treatments cannot be identical.

a subject in a clinical trial who for any reason fails to continue in the trial until the last visit or observation required of him/her by the study protocol

(1) auditable electronic record designed to capture information required by the clinical trial protocol to be reported to the sponsor on each trial subject. (2) a CRF in which related data items and their associated comments, notes and signatures are linked electronically

an auditable process, usually automated, of assessing the content of a data field against its expected logical, format, range, or other properties that is intended to reduce error

an effect attributed to a treatment in a clinical trial. In most clinical trials, the treatment effect of interest is a comparison of two or more treatments

the desired measure of a drug’s influence on a disease or condition as demonstrated by substantial evidence from adequate and well-controlled investigaitons

: the capacity of a drug or treatment to produce beneficial effects on the course or duration of a disease at the dose tested and against the illness for which it is designed

the process of collecting clinical trial data into a permanent electronic form

variable that pertains to the efficacy or safety evaluations of a trial

(1) the act of enrolling one or more subjects. (2) the class of enrolled subjects in a clinical trial

a state in which an investigator is uncertain about which arm of a clinical trial would be therapeutically superior for a patient

a trial with the primary objective of showing that the response to two or more treatments differs by an amount that is clinically unimportant

documents that individually and collectively permit evaluation of the conduct of a study and the quality of the study produced

list of characteristics in a protocol, any one of which may exclude a potential subject from participation in a study

a written description of a trial/study of any therapeutic, prophylactic, or diagnostic agent conducted in human subjects, in which the clinical and statistical description, presentations and analyses are fully integrated into a single report

the date and time the first subject is enrolled and randomized into a study. The subject will have met the inclusion/exclusion criteria to participate in the trial and will have signed an informed consent form

first subject who signs the informed consent form and is screened for potential enrollment and randomization into a study but has not yet been determined to meet the inclusion/exclusion criteria for the trial

the first phase 1 study in which the test product is administered to human subjects

the US regulatory authority charged with, among other responsibilities, granting IND and NDA approvals

status of a database, file, or element that has been presumed to be in its final state pending “lock” and where further editing is preventing without “unfreezing” ; freezing and unfreezing are usually formalized in audit trials and differ from “locking” and “unlocking” only in the degree of approval required

the extent to which the findings of a clinical trial can be reliably extrapolated from the subjects who participated in the trial to a boarder patient population and a boarder range of clinical settings

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

subject (not a patient) in a clinical trial; usually serve as subjects in phase 1 trials

individual who is or becomes a participant in research, either as a recipient of the test article or as a control. A subject may be either a healthy human or a patient

the criteria in a protocol that prospective subjects must meet to be eligible for participation in a study

a committee established by the sponsor to assess at intervals the progress of a clinical trial, safety data, and critical efficacy variables and recommend to the sponsor whether to continue, modify, or terminate the trial

a health problem or disease that is identified as likely to be benefited by a therapy being studied in clinical trials

an ongoing process that provides the subject with explanations that will help in making educated decisions about whether to begin or continue participating in a trial. Informed consent is an ongoing, interactive process rather than a one-time information session

an independent body constituted of medical, scientific, and non-scientific members, whose responsibility is to ensure the protection of the rights, safety and well-being of human subjects involved in a trial by, among other things, reviewing, approving, and providing continuing review of trial protocol and of the methods and material to be used in obtaining and documenting informed consent of the trial subjects

the principle that asserts that the effect of a treatment policy can be best assessed by evaluating the basis of the intention to treat a subject rather than the actual treatment given. NOTE: this has the consequence that subjects allocated to a treatment group should be followed up, assessed, and analyzed as members of that group irrespective of their compliance with the planned course of treatment. The principle is intended to prevent bias caused by loss of participants that may reflect non-adherence to the protocol and disrupt baseline equivalence established by random assignment

analysis comparing intervention groups at any time before the formal completion of the trial, usually before recruitment is complete

the property of scales yielding equivalent results when used by different raters on different occasions

an individual who actually conducts a clinical investigation (under whose immediate direction the test article is administered or dispensed to, or used involving a subject, or, in the event of an investigation conducted by a team of individuals, is the responsible leader of that team)

: a compilation of the clinical and nonclinical data on the investigational products that is relevant to the study of the investigational products in human subjects

(1) the date and time when the last subject has reached a planned or achieved milestone representing the completion of the trial. (2) the last subject to complete a trial

any adverse drug experience that places the patient or subject, in the view of the investigator, at immediate risk of death from the reaction as it occurred

the sum of the values of all observations or data points divided by the number of observations; an arithmetical average

the middle value in a data set; that is, just as many values are greater than the median and lower than the median value

a sponsor representative who has medical authority for the evaluation of the safety aspects of a clinical trial

the formal evaluation of the quantitative evidence from two or more trials bearing on the same question

the biochemical alteration of substances introduced into the body

most frequently occurring value in a data set

person employed by the sponsor or CRO who is responsible for determining that a trial is being conducted in accordance with the protocol and GCP guidance

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, good clinical practice and the applicable regulatory requirements

a written report from the monitor to the sponsor after each site visit and/or other trial-related communication according to the sponsors SOPs.

a visit to a study site to review the progress of a clinical study and to ensure protocol adherence, accuracy of data, safety of subjects, and compliance with regulatory requirements and good clinical practice guidelines

clinical trial conducted according to a single protocol but at more than one site and therefore carried out by more than one investigator

the assertion that no true association or difference in the study outcome or comparison of interest between comparison groups exists in the larger population from which the study samples are obtained

the reason for performing a trial in terms of the scientific questions to be answered by the analysis of data collected during the trial

(1) an assessment of patient condition or analysis of data collected on an individual patient or a group of patients; (2) a discrete piece of information collected during a study

a trial in which subjects and investigators know which product each subject is receiving; opposite of a blinded or double blind study

events or experiences that clinicians or investigators examining the impact of an intervention or exposure measure because they believe such events or experiences may be influenced by the intervention or exposure

research concerned with benefits, financial costs, healthcare system usage, risks, and quality of life as well as their relation to therapeutic interventions

subjects are randomized to one of two or more differing treatment groups and usually receive the assigned treatment during the entire trial

person under a physicians care for a particular disease or condition

information coming directly from patients or subjects through interviews or self-completed questionnaires or other data capture tools such as diaries about their life, health conditions and treatment

the set of data generated by the subset of subjects who complied with the protocol sufficiently to ensure that these data would be likely to exhibit the effects of treatment according to the underlying scientific model

an effect occurring during a period of a trial in which subjects are observed and no treatment is administered

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

study of the processes of bodily absorption, distribution, metabolism and excretion of medicinal products

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

term used for adverse event monitoring and reporting

initial introduction of an investigational new drug into humans. Phase 1 studies are typically closely monitored and may be conducted in patients or normal volunteer subjects

Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition under study and to determine the common short-term side effects and risks associated with the drug

Studies are expanded controlled and uncontrolled trials. They are performed after preliminary evidence suggesting effectiveness of the drug has been obtained and are intended to gather the additional information about effectiveness and safety that is needed to confirm efficacy and evaluate the overall benefit-risk relationship of the drug and to provide an adequate basis for physician labeling

Postmarketing studies to delineate additional information about the drugs risks, benefits, and optimal use that may be requested by regulatory authorities in conjunction with marketing approval

A pharmaceutical preparation that doesn’t contain the investigational agent. In blinded studies, it is generally prepared to be physically indistinguishable from the preparation containing the investigational product

Any finite or infinite collection of subjects from which a sample is drawn for a study to obtain estimates for values that would be obtained if the entire population were sampled

The primary objective(s) is the main question to be answered and drives any statistical planning for the trial (e.g.- calculation of the sample size to provide the appropriate power for statistical testing)

An outcome variable specified in the protocol to be of greatest importance to the primary objective of the trial, usually the one used in the sample size calculation

Investigation in which a group of subjects is recruited and monitored in accordance with criteria described in a protocol

A document that describes the objectives, design, methodology, statistical considerations, and organization of a trial. The protocol usually also gives the background and rationale for the trial, but these could be provided in other protocol referenced documents. Throughout the ICH GCP Guideline the term protocol refers to protocol and protocol amendments.

A written description of a change to or formal clarification of a protocol

A variation from processes or procedures defined in a protocol. Deviations usually do not preclude the overall evaluability of subject data for either efficacy or safety, and are often acknowledged and accepted in advance by the sponsor

A significant departure from processes or procedures that were required by the protocol. Violations often result in data that are not deemed evaluable for a per-protocol analysis, and may require that the subjects who violate the protocol be discontinued from the study.

Study findings can also be assessed in terms of their statistical significance. The p-value represents the probability that the observed data could have arisen by chance when the interventions did not differ

One that can’t be measured on a continuum and represented in quantitative relation to a scale. Data that fit into discrete categories according to their attributes.

A broad ranging concept that incorporates an individuals physical health, psychological state, level of independence, social relationships, personal beliefs, and their relationships to salient features of the environment

A request for clarification on a data item collected for a clinical trial; specifically a request from a sponsor or sponsor’s representative to an investigator to resolve an error or inconsistency discovered during data review.

The closure of a query usually based on information contained in a data clarification.

Assignment of subjects to treatment (or control) groups in an unpredictable way.

The process of assigning trial subjects to treatment or control groups using an element of chance to determine the assignments in order to reduce bias

Data as originally collected. Distinct from derived. Raw data includes records of original observations, measurements, and activities without conclusions or interpretations. Researcher’s records of subjects/patients, such as patient medical charts, hospital records, x-rays, and attending physician’s notes.

Process used by investigators to find and enroll appropriate subjects into a clinical study.

Time period during which subjects are or are planned to be enrolled in a clinical trial

The act of enrolling a clinical trial subject to compensate for the withdrawal of another

The proposition that a study sets out to support. For example: “blood pressure will be lowered by (specific endpoint) in subjects who receive the test product”

Capture of clinical trial data is retrospective when it is recalled from memory rather than captured contemporaneously in real time

Relative freedom from harm. In clinical trials, this refers to an absence of harmful side effects resulting from use of the product and may be assessed by laboratory testing of biological samples, special tests and procedures, psychiatric evaluation, and/or physical examination of subjects

The safety of a medical product concerns the medical risk to the subject, usually assessed in a clinical trial by laboratory tests, vital signs, clinical adverse events, and other special safety tests. The tolerability of the medical product represents the degree to which overt adverse effects can be tolerated by the subject

(1) A subset of a larger population, selected for investigation to draw conclusions or make estimates about the larger population. (2) The number of subjects in a clinical trial.

A standardized representation of planned clinical trial activities including interventions and study administrative activities as well as assessments

A tabular representation of planned protocol events and activities, in sequence.

Potential subject who did not meet one or more criteria required for participation in a clinical trial

The primary outcome is the outcome of greatest importance. Data on secondary outcomes are used to evaluate additional effects of the intervention

A data discrepancy that can be easily and obviously resolved on the basis of existing information on the CRF.

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.

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

Bodies having the power to regulate.

In clinical trials, the probability of harm or discomfort for subjects

(1) An individual, company, institution, or organization that takes responsibility for the initiation and management of a clinical trial, although may or may not be the main funding organization. If there is also a secondary sponsor, this entity would be considered the primary sponsor. (2) A corporation or agency whose employees conduct the investigation is considered a sponsor and the employees are considered investigators

A measurement of a drug’s biological activity that substitutes for a clinical endpoint such as death or pain relief

Brief overview prepared at the conclusion of a study as a routine part of a regulatory submission, summarizing the study plan and results; includes numerical summary of efficacy and safety results, study objective, criteria for inclusion, methodology, etc.

A written communication from FDA notifying an individual or firm that the agency considers one or more products, practices, processes, or other activities to be in violation of the Federal FD&C Act, or other acts and that failure of the responsible part to take appropriate and prompt action to correct and prevent any future repeat of the violation may result in administrative and/or regulatory enforcement action without further notice