The following list of terms and descriptions is by no means a comprehensive list.

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TERMS	MEANING
AAPS	American Association of Pharmaceutical Scientists
ACRP	Association of Clinical Research Professionals
ADE	Adverse Drug Event; Adverse Drug Effect
ADME	Absorption, Distribution, Metabolism and Excretion (used to describe pharmacokinetic processes)
ADR	Adverse Drug Reaction
Adverse Event (AE)	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
AE	Adverse Event
Alpha error	The likelihood that a relationship observed between 2 variables is due to chance. The probability of a Type 1 error.
ANDA	Abbreviated New Drug Application (for a generic drug – FDA, US)
API	Active Pharmaceutical Ingredient
ARCS	Association of Regulatory and Clinical Scientists (Australia)
Arm	A planned sequence of elements typically equivalent to a treatment group.
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 were recorded, analyzed and accurately reported according to the protocol, sponsor’s standard operating procedures (SOPs), good clinical practice (GCP), and the applicable regulatory requirements.
Audit trail	1. Documentation that allows reconstruction of the course of events. 2. A secure, time-stamped record that allows reconstruction of the course of events relating to the creation, modification, and deletion of an electronic study record.
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BARQA	British Association of Research Quality Assurance
Baseline assessment	Assessment of subjects as they enter a trial and before they receive any treatment.
Baseline characteristics	Demographic, clinical and other data collected for each participant at the beginning of the trial before the intervention is administered.
Beta error	Probability of showing no significant difference when a true difference exists; a false acceptance of the null hypothesis.
BfArM	Bundesinstitut für Arzneimittel und Medizinprodukte (Federal Institute for Drugs and Medical Devices, Germany)
Bias	Situation or condition that causes a result to depart from the true value in a consistent direction. Bias refers to defects in study design or measurement.
Bioanalytical assays	Methods for quantitative measurement of a drug, drug metabolites, or chemicals in biological fluids.
Biochip	An electronic device that uses organic molecules to form a semiconductor.
Bioequivalence	Scientific basis on which drugs with the same active ingredient(s) are compared.
Biologic	A therapeutic or prophylactic derived from a living source (human, animal or unicellular). Most biologics are complex mixtures that are not easily identified or characterized, and many are manufactured using biotechnology.
Biologics Licensing Application (BLA)	An application to FDA for a license to market a new biologic product in the United States.
Biomarker	A characteristic that is objectively measured and evaluated as an indicator of normal biological processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention.
Biostatistics	Branch of statistics applied to the analysis of biological phenomena.
Blind review	Checking and assessing data prior to breaking the blind, for the purpose of finalizing the planned analysis.
BLA	Biologics Licensing Application
Blinded (masked) medications	Products that appear identical in size, shape, colour, flavour and other attributes to make it very difficult for subjects and investigators (or anyone assessing the outcome) to determine which medication is being administered.
Blinded study	A study in which the subject, the investigator, or anyone assessing the outcome is unaware of the treatment assignment(s). Blinding is used to reduce the potential for bias.
Blinding	Preventing identification of treatments/procedures/test to trial subjects or study personnel results in order to limit bias.
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CA	Competent Authority
CAPRA	Canadian Association of Professional Pharmaceutical Regulatory Affairs (also ACPR:  Association canadienne des professionnels en réglementation)
Carcinogen	Cancer-causing agent
Carry-over effect	Effects of treatment that persist after treatment has been stopped, sometimes beyond the time of a medication’s known biological activity.
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 for each trial subject.
Categorical data	Data evaluated by sorting values (for example, severe, moderate, and mild) into various categories.
Causality assessment	An evaluation performed by a medical professional concerning the likelihood that a therapy or product under study caused or contributed to an adverse event.
CBER	Centre for Biologics Evaluation and Research (FDA US)
CCRA	Certified Clinical Research Associate (certification issued to clinical coordinators by ACRP)
CCRC	Certified Clinical Research Coordinator (certification issued to clinical coordinators by ACRP)
CCRP	Certified Clinical Research Professional (SoCRA certification of coordinators, monitors, and other research professionals)
CDE	Centre for Drug Evaluation, Taiwan
CDER	Centre for Drug Evaluation and Research (FDA US)
CDISC	Clinical Data Interchange Standards Consortium
CDRH	Centre for Devices and Radiological Health (FDA US)
CFR	Code of Federal Regulations (usually cited by title and part; for example, Title 21, Part 211 is shown as 21 CFR 211) (FDA US)
CGMP	Current Good Manufacturing Practices
CIOMS	Council for International Organisations of Medical Sciences
Clean database	A set of reviewed data in which errors have been resolved to meet quality assurance requirements for error rate and in which measurements and other values are provided in acceptable units.
Clinical data	Data pertaining to the medical well-being or status of a patient or subject.
Clinical Development Plan	A 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.
Clinical efficacy	Power or capacity to produce a desired effect (ie. appropriate pharmacological activity in a specified indication/disorder) in humans.
Clinical investigation	A systematic study designed to evaluate a product (drug, device, or biologic) using human subjects, in the treatment, prevention, or diagnosis of a disease or condition, as determined by the product's benefits relative to its risks. Clinical investigations can only be conducted with the approval of the TGA.
Clinical research	Study of drug, biologic or device in human subjects with the intent to discover potential beneficial effects and/or determine its safety and efficacy. Also called clinical study and clinical investigation. Thus, it does not encompass all the research that is carried out in the clinical setting (e.g. health services research).
Clinical pharmacology	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 (ADME).
Clinical Research Associate (CRA)	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. At some sites (primarily in academic settings), clinical research coordinators are called CRAs.
Clinical Research Coordinator (CRC)	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.
Clinical significance	Change in a subject’s clinical condition regarded as important whether or not due to the test intervention/treatment.
Clinical Study (trial) Report	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.
Clinical trial	Any investigation in human subjects intended to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of one or more investigational medicinal product(s), and/or to identify any adverse reactions to one or more investigational medicinal product(s), and/or to study absorption, distribution, metabolism and excretion of one or more investigational medicinal product(s) with the object of ascertaining its (their) safety and/or efficacy.
Clinical Trial Exemption (CTX)	A scheme that allows sponsors to apply for approval for each clinical study in turn, submitting supporting data to the Therapeutic Goods Administration (TGA Australia) which approves or rejects the application (generally within 50 working days).
Clinical trial materials	Complete set of supplies provided to an investigator by the trial sponsor.
Clinical Trial Notification (CTN)	A scheme that allows notification to the TGA (Australia) of the intention to start a trial once ethical-scientific approval is obtained.
CMC	Chemistry, Manufacturing and Control
Codelist	Finite list of codes and their meanings that represent the only allowed values for a data item.
Coding	In clinical trials, the process of assigning data to categories for analysis (adverse events, for example, may be coded using MedDRA).
Cohort study	Study of a group of individuals, some of whom are exposed to a variable of interest, in which subjects are followed over time. Cohort studies can be prospective or retrospective.
Common Technical Document (CTD)	A format agreed upon by ICH to organize applications to regulatory authorities for registration of pharmaceuticals for human use.
Comparative Study	One in which the investigative drug is compared against another product, either active drug or placebo.
Comparator (product)	An investigational or marketed product (ie. active control), or placebo, used as a reference in a clinical trial.
Compassionate Use	A method of providing experimental therapeutics prior to Regulatory approval 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 from the Regulator for “compassionate use” of a drug or therapy.
Competent Authority (CA)	The regulatory body charged with monitoring compliance with the national statutes and regulations of European Member States.
Compliance (in relation to trials)	Adherence to trial-related requirements, good clinical practice (GCP) requirements, and the applicable regulatory requirements.
Confidence interval	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.
Confirmatory trial	Phase 3 trial during which the previously revealed actions of a therapeutic intervention are confirmed.
Consent form	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 involves.
Contract Research Organization (CRO)	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 group	The group of subjects in a controlled study that receives no treatment, a standard treatment, or a placebo.
Control(s)	Comparator against which the study treatment is evaluated [eg. concurrent (placebo, no treatment, dose-response, active) and external (historical, published literature)]
Controlled study	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.
Coordinating committee	A committee that a sponsor may organize to coordinate the conduct of a multicentre trial.
Correlation	The degree to which two or more variables are related.
COSA	Clinical Oncological Society of Australia
COSTART	Coding Symbols for a Thesaurus of Adverse
Covariate (prognostic)	Factor or condition that influences outcome of a trial.
CRA	Clinical Research Associate
CRC	Clinical Research Coordinator
CRF	Case Report form
CRO	Contract Research Organization
Crossover trial	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 wash-out period between the two products.
CSM	Committee on Safety of Medicines (UK)
CSR	Clinical Study Report
CT	Clinical Trial
CTA	Clinical Trial Agreement
CTD	Common Technical Document
CTM	Clinical Trials Materials
CTN	Clinical Trial Notification
CTX	Clinical Trial Exemption
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Data clarification	Answer supplied by the investigator in response to a query.
Data clarification form	A form used to query an investigator and collect feedback to resolve questions regarding data.
Data management	Tasks associated with the entry, transfer and/or preparation of source data and derived items for entry in a clinical trial database.
Data monitoring	Process by which clinical data are examined for completeness, consistency and accuracy.
Data quality	Describes the characteristics that confirm “fitness for use” – that is, ability to support meaningful and trustworthy conclusions and interpretations. Quality is established through formal assessment, quality control and auditing.
Data transformations	Algorithmic operations on data or data sets to achieve a meaningful set of derived data for analysis.
Dataset	An organized collection of data or information with a common theme arranged in rows and columns represented as a single file; comparable to a database table.
DD	Department of Drugs (Swedish Regulatory Agency)
Declaration of Helsinki	A set of recommendations or basis principles that guide medical doctors in the conduct of biomedical research involving human subjects.
Demographic data	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.
Derived variable	New variable created as a function of existing variables and/or application of mathematical functions.
Development plan	An ordered program of clinical trials, each with specific objectives.
DIA	Drug Information Association
Diagnostic	A product used for the diagnosis of disease or medical condition.
Discontinuation	The act of concluding participation, prior to completion of all protocol-required elements, in a trial by an enrolled subject.
Discrepancy	The failure of a data point to pass a validation check.
DLT	Dose-limiting toxicity
DNA chip	A small piece of glass or silicon that has small pieces of DNA arrayed on its surface.
Dosage	The amount of drug administered to a patient or test subject over the course of the clinical study; a regulated administration of individual doses.
Dosage regimen	The number of doses per given time period; the elapsed time between doses (for example, every six hours) or the time that the doses are to be given (for example, at 8 am and 4 pm daily); and/or the amount of a medicine (the number of capsules, for example) to be given at each specific dosing.
Dose	The amount of drug administered to a patient or test subject at one time or the total quantity administered.
Dose-ranging Study	A clinical trial in which two or more doses of an agent (such as a drug) are tested against each other to determine which dose works best and is least harmful.
Double-blind study	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.
Double-dummy	A technique for retaining the blind when administering supplies in a clinical trial, when the two treatments cannot be made identical.
Dropout	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.
Drug	Article other than food intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease; or intended to affect the structure or any function of the body.
Drug delivery	The process by which a formulated drug is administered to the patient.
Drug development process	The program for advancing an investigational product from preclinical studies through approval for marketing following review by regulatory agencies.
Drug-Drug interaction	A modification of the effect of a drug when administered with another drug. The effect may be an increase or a decrease in the action of either substance, or it may be an adverse effect that is not normally associated with either drug.
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EC	European Commission
eCRF	Electronic case report form
eCTD	Electronic Common Technical Document
EDC	Electronic data capture/collection
Edit check	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.
Effective dose	The dose of an investigational agent that produces the outcome considered "effective", as defined in the study protocol. This could mean a cure of the disease in question or simply the mitigation of symptoms.
Efficacy	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 (and patient population) for which it is designed.
EFGCP	European Forum for Good Clinical Practice
EFPIA	European Federation of Pharmaceutical Industries and Associations
Electronic data capture (EDC)	The process of collecting clinical trial data into a permanent electronic form.
EMEA	European Agency for the Evaluation of Medicinal Products
Endpoint	Variable that pertains to the efficacy or safety evaluations of a trial.
Enroll	To register or enter into a clinical trial; transitive and intransitive.
EORTC	European Organisation for Research and Treatment of Cancer
ePRO	Patient Research Outcome data initially captured electronically.
Equivalence trial	A trial with the primary objective of showing that the response to two or more treatments differs by an amount that is clinically unimportant.
ERSR	Electronic regulatory submissions and review (FDA’s e-Submissions processing group)
Essential documents	Documents that individually and collectively permit evaluation of the conduct of a study and the quality of the data produced.
Ethics Committee	An independent group of both medical and non-medical professionals who are responsible for verifying the integrity of a study and ensuring the safety, integrity, and human rights of the study participants.
EU	European Union
EUDRACT	European Union clinical trials database
Evaluable (for efficacy and safety)	Pertains to data or subjects that meet Statistical Analysis Plan criteria for inclusion in Efficacy/Safety datasets.
EWG	Expert Working Group (EU)
Exclusion criteria	List of characteristics in a protocol, any one of which may exclude a potential subject from participation in a study.
Exploratory trial	Phase 1 or 2 trial during which the actions of a therapeutic intervention are assessed and measured.
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FDA	Food and Drug Administration (US)
First subject (patient) in (FSI, FPI)	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 screened	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.
First-in-humans study (first-in-man study)	The first Phase 1 study in which the test product is administered to human beings.
FPI	First patient in
FSI	First subject in
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GCP	Good Clinical Practice
Gene therapy	The replacement of a defective gene in an organism suffering from a genetic disease. Recombinant DNA techniques are used to isolate the functioning gene and insert it into cells.
Generic drug	A medicinal product with the same active ingredient, but not necessarily the same inactive ingredients as a brand-name drug. A generic drug may only be marketed after the original drug's patent has expired.
Generic name	The drug identifying name to which all branded (proprietary) names for that indication are associated.
Genotype	Genetic makeup of an individual or group.
Glossary	A collection of specialized words or terms with their meanings.
GLP	Good Laboratory Practice
GMP	Good Manufacturing Practices
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.
GPMS	Good Postmarketing Surveillance Practice (Japan)
Group sequential design	A trial design that allows a look at the data at particular time points or after a defined number of patients have been entered and followed up based on formulating a stopping rule derived from repeated significance tests.
GTRAP	Gene and Related Therapies Research Advisory Panel (Australia)
GxP	Good [pharmaceutical] practice
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Healthy volunteer	Subject (not a patient) in a clinical trial.
HIPAA	Health Insurance Portability and Accountability Act
HL7	Health Level 7 (a not-for-profit ANSI-accredited standards developing/development organization (SD))
HREC	Human Research and Ethics Committee (Australia)
Human Research Ethics Committee (HREC)	An independent group of professionals designated to review and approve the clinical protocol, informed consent forms, study advertisements, and patient brochures, to ensure that the study is safe and effective for human participation. It is also the HREC's responsibility to ensure that the study adheres to the TGA's regulations.
Human subject	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.
Hypothesis to test	In a trial, a statement relating to the possible different effect of the interventions on an outcome. The null hypothesis of no such effect is amenable to explicit statistical evaluation by a hypothesis test, which generates a P value.
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IB	Investigator’s brochure
IBC	Institutional Biosafety Committee
IC	Informed Consent
ICD9	International Classification of Diseases, 9th revision. (See also MedDRA)
ICF	Informed Consent Form
ICH	International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use
IDMC	Independent Data Monitoring Committee
IEC	Independent Ethics Committee
IFAPP	International Federation of Associations of Pharmaceutical Physicians
IFPMA	International Federation of Pharmaceutical Manufacturers and Associations
Inclusion criteria	The criteria in a protocol that prospective subjects must meet to be eligible for participation in a study.
IND	Investigational New Drug application (FDA).
Independent Data Monitoring Committee (IDMC)	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.
Independent Ethics Committee (IEC)	An independent body (a review board or a committee, institutional, regional, national, or supranational) constituted of medical/scientific professionals and 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.
Indication	A health problem or disease that is identified as likely to be benefited by a therapy being studied in clinical trials.
Informed consent	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.
INN	International Nonproprietary Name
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 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. (US)
Intention to treat	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 (ie. the planned treatment regimen) rather than the actual treatment given. 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.
Interaction (qualitative and quantitative)	The situation in which a treatment contract (eg. difference between investigational product and control) is dependent on another factor (for example, the centre). A quantitative interaction refers to the case where the magnitude of the contrast differs at the different levels of the factor; for a qualitative interaction, the direction of the contrast differs for at least one level of the factor.
Interim analysis(es)	Analysis comparing intervention groups at any time before the formal completion of the trial, usually before recruitment is complete.
Investigational New Drug (IND)	A new drug, antibiotic drug, or biological drug that is used in a clinical investigation. It also includes a biological product used in vitro for diagnostic purposes. (FDA, US)
Investigational treatment	An intervention under investigation in a clinical trial.
Investigator	1. 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. 2. The individual “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”.
Investigator’s Brochure (IB)	A compilation of the clinical and nonclinical data on the investigational product(s) which is relevant to the study of the investigational product(s) in human subjects.
In Vitro Testing	Non-clinical testing conducted in an artificial environment such as a test tube or culture medium.
In Vivo Testing	Testing conducted in living animal and human systems.
IRB	Institutional Review Board; Independent Review Board (US)
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KFDA	Korean Food and Drug Administration
Korean Food and Drug Administration (KFDA)	The South Korean agency responsible for ensuring that medicines and medical devices work, and are acceptably safe.
Label	Description of a drug product/device that includes: the indication, who should use it, adverse events, instructions for use, and safety information.
Last subject (patient) out/complete (LSC/LPC or LSO/LPO)	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
Longitudinal study	Investigation in which data is collected from a number of subjects over a long period of time.
LPC	Last Patient Complete
LPO	Last Patient Out
LSC	Last Subject Complete
LSO	Last Subject Out
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MA	Marketing Authorization
MAA	Marketing Authorization Application (EU)
MABEL	Minimum Anticipated Biological Effect Level
MAH	Marketing Authorization Holder (EU)
Marketing Support Trials	Clinical studies that are designed to clarify therapeutic benefits of a marketed product or to show potential decision-makers the rationale for preferring one therapy over another.
Masking	See blinding
Mean	The sum of the values of all observations or data points divided by the number of observations; an arithmetical average.
MedDRA	Medical Dictionary for Regulatory Activities (global standard medical terminology designed to supersede other terminologies used in the medical product development process, including COSTART, ICD9, and others)
Median	The middle value in a data set; that is, just as many values are greater than the median and lower than the median value. (With an even number of values, the conventional median is halfway between the two middle values.)
Medical monitor	A sponsor representative who has medical authority for the evaluation of the safety aspects of a clinical trial.
Medicines and Healthcare products Regulatory Agency (MHRA)	The UK Government agency responsible for ensuring that medicines and medical devices work, and are acceptably safe.
Medsafe	New Zealand Medicines and Medical Devices Safety Authority
Meta-analysis	The formal evaluation of the quantitative evidence from two or more trials bearing on the same question.
Metadata	Data that describe other data, particularly XML tags characterizing attributes of values in clinical data fields.
MHRA	Medicines and Healthcare products Regulatory Agency
Mode	The most frequently occurring value in a data set.
Monitor	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.
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 (SOPs), good clinical practice (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 SOPs.
MOU	Memorandum of Understanding
MRA	Medical Research Associate
MREC	Multicentre Research Ethics Committee (UK)
MTD	Maximum Tolerated Dose
Multicentre trial	Clinical trial conducted according to a single protocol but at more than one site, and, therefore, carried out by more than one investigator.
MVP	Master Validation Plan
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NAI	No Action indicated (most favourable FDA post-inspection classification)
NCE	New Chemical Entity
NCR	No Carbon [paper] Required
NDA	New Drug Application (FDA US)
NDS	New Drug Submission (Canada’s new drug application or marketing authorisation application)
New Drug Application (NDA)	An application to FDA for a license to market a new drug in the United States.
NHMRC	National Health and Medical Research Council (Australia)
NIH	National Institutes of Health (US)
NME	New Molecular Entity
NOAEL	No-observed-adverse-effect level
NOEL	No observable effect level (dose of an experimental drug given preclinically that does not produce an observable toxicity)
Null hypothesis	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.
Nuremberg Code	Code of ethics, set forth in 1947, for conducting human medical research.
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ODBT	Office of Devices, Blood and Tissues (FDA, US)
Off-Label use	A drug prescribed for conditions other than those approved by the Regulator.
OGTR	Office of the Gene Technology Regulator (Australia)
Open-label study	A trial in which subjects and investigators know which product each subject is receiving; opposite of a blinded or double-blind study.
Original data	Those values that represent the first recording of study data.
Orphan drugs	An FDA category that refers to medications used to treat diseases and conditions that occur rarely. There is little financial incentive for the pharmaceutical industry to develop medications for these diseases or conditions. Orphan drug status, however, gives a manufacturer specific financial incentives to develop and provide such medications.
OTC	Over-the-counter (refers to nonprescription drugs)
Outcomes research	Research concerned with benefits, financial costs, healthcare system usage, risks, and quality of life as well as their relation to therapeutic interventions.
Outliers	Values outside of an expected range.
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Packaging	The material, both physical and informational, that contains or accompanies a marketed or investigational therapeutic agent once it is fully prepared for release to patients and/or subjects in clinical trials.
Pairing	A method by which subjects are selected so that two subjects with similar characteristics (for example, weight, smoking habits) are assigned to a set, but one receives Treatment A and the other receives Treatment B
Parallel trial	Subjects are randomized to one of two or more differing treatment groups (usually investigational product and placebo) and usually receive the assigned treatment during the entire trial.
Parameter	A variable in a model, or a variable that wholly or partially characterizes a probability distribution (mathematics and statistics). In clinical trials the term is often used synonymously with “variable” for factual information (age, date of recovery), measurements and clinical assessments.
Participant	A person or entity with a role in healthcare or a clinical study.
Patient	Person under a physician’s care for a particular disease or condition.
Patient file	One that contains demographic, medical and treatment information about a patient or subject. It may be paper- or computer-based or a mixture of computer and paper records
Patient-reported outcome (PRO)	Report coming directly from patients or subjects through interviews or self-completed questionnaires or other data capture tools such as diaries about their life, health condition(s) and treatment.
Period effect	An effect occurring during a period of a trial in which subjects are observed and no treatment is administered.
Per-protocol analysis set	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.
Pharmacodynamics	Branch of pharmacology that studies reactions between drugs and living structures, including the physiological responses to pharmacological, biochemical, physiological, and therapeutic agents.
Pharmacoeconomics	Branch of economics that applies cost-benefit, cost-utility, cost-minimization, and cost-effectiveness analyses to assess the utility of different pharmaceutical products or to compare drug therapy to other treatments.
Pharmacogenetics	Study of the way drugs interact with genetic makeup or the study of genetic response to a drug.
Pharmacogenomics	Science that examines inherited variations in genes that dictate drug response and explores the ways such variations can be used to predict whether a person will respond favourably, adversely, or not at all to an investigational product.
Pharmacokinetics	Study of the processes of bodily absorption, distribution, metabolism, and excretion (ADME) of medicinal products.
Pharmacology	Science that deals with the characteristics, effects and uses of drugs and their interactions with living organisms.
Pharmacovigilance	Term used for adverse event monitoring and reporting.
Phase 1	The initial introduction of an investigational new drug into humans. These studies are designed to determine the metabolism and pharmacologic actions of the drug in humans, the side effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness.
Phase 2	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.
Phase 3	Studies are expanded controlled and uncontrolled trials. They are performed after preliminary evidence suggesting the 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.
Phase 4	Postmarketing (Phase 4) studies to delineate additional information about the drug’s risks, benefits and optimal use that may be requested by regulatory authorities in conjunction with marketing approval.
Phenotype	Observable characteristics resulting from interaction between an organism’s genetic makeup and the environment.
PhRMA	Pharmaceutical Research and Manufacturers of America
PI	Principal Investigator
PK