| Adverse Drug Reaction (ADR) |
An unintended reaction to a drug taken at doses normally used in man for prophylaxis, diagnosis, or therapy of disease, or for the
modification of physiological function. In clinical trials, an ADR would include any injuries by overdosing, abuse/dependence, and unintended
interactions with other medicinal products. |
| Adverse Event |
Any change in health that occurs in a person after he or she enrolls in a clinical trial. Not every adverse event is related to the treatment or
test being studied, but researchers must report all adverse events to the Food and Drug Administration (FDA). See also serious adverse event. |
| Approved Drugs |
In the U.S., the Food and Drug Administration (FDA) must
approve a substance as a drug before it can be marketed.
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 by the manufacturer of the drug, FDA review
of the application, and FDA approval/rejection of
application (See Food and Drug Administration). |
| Arm |
Any of the treatment groups in a randomized trial. Most
randomized trials have two arms, but some have three
arms, or even more (See Randomized Trial). |
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| 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 experimental treatment which is being
tested. At this reference point, measurable values such
as CD4 count are recorded. Safety and efficacy of a drug
are often determined by monitoring changes from the
baseline values. |
| Bias |
When a point of view prevents impartial judgment on
issues relating to the subject of that point of view. In
clinical studies, bias is controlled by blinding and randomization |
| Blind |
A randomized trial is Blind if the participant is not
told which arm of the trial he is on. A clinical trial
is Blind if participants are unaware on whether they
are in the experimental or control arm of the study;
also called masked. |
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| Case Report Form (CRF) |
A record of pertinent information collected on each
subject during a clinical trial, as outlined in the
study protocol. |
| Clinical Investigator |
A medical researcher in charge of carrying out a
clinical trial's protocol. |
| Clinical Research Associate (CRA) |
Person employed by the study sponsor or CRO to monitor a
clinical study at all participating sites. See also,
monitor. |
| Clinical Research Coordinator (CRC) |
Site administer for the clinical study. Duties are
delegated by the investigator. Also called research,
study or healthcare coordinator, and data manager,
research nurse or protocol nurse. |
| Clinical Study Materials |
Study supplies (i.e., study test article, laboratory
supplies, case report forms) provided by the study
sponsor to the investigator. |
| Clinical Trial |
A clinical trial is a research study to answer specific
questions about vaccines or new therapies or new ways of
using known treatments. Clinical trials (also called
medical research and research studies) are used to
determine whether new drugs or treatments are both safe
and effective. Carefully conducted clinical trials are
the fastest and safest way to find treatments that work
in people. Trials are in four phases: Phase I tests a
new drug or treatment in a small group; Phase II expands
the study to a larger group of people; Phase III expands
the study to an even larger group of people; and Phase
IV takes place after the drug or treatment has been
licensed and marketed. (See Phase I, II, III, IV trials) |
| Compassionate Use |
A method of providing experimental therapeutics prior to
final FDA 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 FDA for compassionate use of a drug
or therapy. |
| Confidentiality for Trial Participants |
Refers to maintaining the confidentiality of trial
participants including their personal identity and all
personal medical information. 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. |
| Consent Form |
A document explaining all relevant study information to
assist the study volunteer in understanding the
expectations and requirements of participation in a
clinical trial. This document is presented to and signed
by the study subject. |
| 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 study-related duties and functions. |
| Control |
A control is the nature of the intervention control. |
| Control Group |
The standard by which experimental observations are
evaluated. In many clinical trials, one group of
patients will be given an experimental drug or
treatment, while the control group is given either a
standard treatment for the illness or a placebo (See
Placebo and Standard Treatment |
| Control Group |
A comparison group of study subjects who are not treated
with the investigational agent. The subjects in this
group may receive no therapy, a different therapy, or a
placebo. |
| Controlled Trials |
Control is a standard against which experimental
observations may be evaluated. In clinical trials, one
group of participants is given an experimental drug,
while another group (i.e., the control group) is given
either a standard treatment for the disease or a
placebo. |
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| Data |
This term is legally defined according to the
institution. It generally refers to recorded information
regardless of form. Most institutions hold title to data
while researchers have rights to access the data.
|
| Device |
An instrument, apparatus, implement, machine,
contrivance, implant, in vitro reagent, or other similar
or related article, including any component, part or
accessory, which is intended for use in the diagnosis,
cure, treatment or prevention of disease. A device does
not achieve its intended purpose through chemical action
in the body and is not dependent upon being metabolized
to achieve its purpose. |
| Diagnostic Trials |
Refers to trials that are conducted to find better tests
or procedures for diagnosing a particular disease or
condition. Diagnostic trials usually include people who
have signs or symptoms of the disease or condition being
studied. |
| Documentation |
All forms of records that describe or document study
methods, conduct and results, including any adverse
events and actions taken. |
| 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 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. |
| Drug |
As defined by the Food, Drug and Cosmetic Act, drugs are
articles (other than food) intended for the use in the
diagnosis, cure, mitigation, treatment, or prevention of
disease in man or other animals, or to affect the
structure or any function of the body of man or other
animals. |
| Drug or Device Accountability Records (DAR) |
Required documentation for material accountability,
quantity used and left over, and date of disposal. |
| Drug Product |
A finished dosage form (e.g. tablet, capsule, or
solution) that contains the active drug ingredient
usually combined with inactive ingredients. |
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| 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 |
A product's ability to produce beneficial effects on the
duration or course of a disease. Efficacy is measured by
evaluating the clinical and statistical results of
clinical tests. |
| Endpoint |
Overall outcome that the protocol is designed to
evaluate. Common endpoints are severe toxicity, disease
progression, or death. |
| 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. |
| Exclusion Criteria |
Refers to the characteristics that would prevent a
subject from participating in a clinical trial, as
outlined in the study protocol. |
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| FDA Form 1572
| A list of commitments and requirements by the FDA for
each investigator performing drug/biologics studies.
Also referred to as a statement of the investigator. |
| FOOD AND DRUG ADMINISTRATION (FDA): |
The U.S. Department
of Health and Human Services agency responsible for
ensuring the safety and effectiveness of all drugs,
biologics, vaccines, and medical devices, including
those used in the diagnosis, treatment, and prevention
of HIV infection, AIDS, and AIDS-related opportunistic
infections. The FDA also works with the blood banking
industry to safeguard the nation's blood supply. |
| Formulation |
The mixture of chemicals and/or biological substances
and excipients used to prepare dosage forms. |
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| 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. |
| Good Clinical Practice (GCP) |
International ethical and scientific quality standard
for designing, conducting, monitoring, recording,
auditing, analyzing and reporting studies. Insures that
the data reported is credible and accurate, and that
subject's rights and confidentiality are protected. |
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| Human Subject
| A patient or healthy individual participating in a
research study. A living individual about whom an
investigator obtains private information or data through
intervention or interaction. |
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| In Vitro Testing |
Non-clinical testing conducted in an artificial
environment such as a test tube or culture medium. |
| Inclusion Criteria |
A list of criteria that must be met by all study
subjects. |
| Informed Consent |
A document that describes the rights of the study
participants, and includes details about the study, such
as its purpose, duration, required procedures, and key
contacts. Risks and potential benefits are explained in
the informed consent document. The participant then
decides whether or not to sign the document. Informed
consent is not a contract, and the participant may
withdraw from the trial at any time. |
| Institution |
Location of research. Retains ultimate responsibility
for human subject regulation compliance. |
| Institutional Review Board (IRB) |
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 IRB's
responsibility to ensure that the study adheres to the
FDA's regulations. |
| Intervention Name |
The generic name of the precise intervention being
studied. |
| Interventions |
Primary interventions being studied: types of
interventions are Drug, Gene Transfer, Vaccine,
Behavior, Device, or Procedure. |
| Investigational Device Exemption (IDE) |
Exemption from FD & C Act to study investigational
medical devices. |
| Investigational New Drug Application (IND) |
The petition through which a drug sponsor requests the
FDA to allow human testing of its drug product. |
| Investigator |
A medical professional, usually a physician but may also
be a nurse, pharmacist or other health care
professional, under whose direction an investigational
drug is administered or dispensed. A principal
investigator is responsible for the overall conduct of
the clinical trial at his/her site. |
| Investigator's Brochure |
Relevant clinical and non-clinical data compiled on the
investigational drug, biologic or device being studied. |
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| Longitudinal Study |
A study conducted over a long period of time. |
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| MedWatch Program |
An FDA program designed to monitor adverse events (AE)
from drugs marketed in the U.S. Through the MedWatch
program, health professionals may report AEs voluntarily
to the FDA. Drug manufacturers are required to report
all AEs brought to their attention. |
| Monitor |
Person employed by the sponsor or CRO who reviews study
records to determine that a study is being conducted in
accordance with the protocol. A monitor's duties may
include, but are not limited to, helping to plan and
initiate a study, and assessing the conduct of studies.
Monitors work with the clinical research coordinator to
check all data and documentation from the study. See
also CRA. |
| Monitoring |
Reviewing a clinical study, ensuring conduct, proper
records and reports are performed as stated in the
clinical protocol, standard operating procedures, GCP
and by regulatory requirements. |
| Multiple Project Assurance |
Permit given to institution for multiple federally
funded research grants for a specified period of time.
States institution retains responsibility for all
research involving humans and that the institution must
have an established IRB. |
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| National Institutes of Health (NIH) |
Agency within DHHS that provides funding for research,
conducts studies and funds multi-site national studies. |
| National Research Act |
Act created by the National Commission for Protection of
Human Subjects of Biomedical and Behavioral Research in
1974 and mandated review of studies by institutional
review boards and subject protection by informed
consent. |
| New Drug Application (NDA) |
The compilation of all non-clinical, clinical,
pharmacological, pharmacokinetic and stability
information required about a drug by the FDA in order to
approve the drug for marketing in the U.S. |
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| Off Label |
The unauthorized use of a drug for a purpose other than
that approved of by the FDA. |
| Office for Human Research Protection (OHRP) |
A federal government agency that issues Assurances and
overseas compliance of regulatory guidelines by research
institutions. |
| Open-Label Study |
A study in which all parties, (patient, physician and
study coordinator) are informed of the drug and dose
being administered. In an open-label study, none of the
participants are given placebos. These are usually
conducted with Phase I & II studies. |
| Orphan Drug |
A designation of the FDA to indicate a therapy developed
to treat a rare disease (one which afflicts a U.S.
population of less than 200,000 people). Because there
are few financial incentives for drug companies to
develop therapies for diseases that afflict so few
people, the U.S. government offers additional incentives
to drug companies (i.e. tax advantages and extended
marketing exclusivity) that develop these drugs. |
| Over-the-Counter (OTC) |
Drugs available for purchase without a physician's
prescription. |
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| Patient |
Individual seeking medical care. |
| Pharmacoeconomics |
The study of cost-benefit ratios of drugs with other
therapies or with similar drugs. Pharmacoeconomic
studies compare various treatment options in terms of
their cost, both financial and quality-of-life. Also
referred to as outcomes research. |
| Phase I Study |
The first of four phases of clinical trials, Phase I
studies are designed to establish the effects of a new
drug in humans. These studies are usually conducted on
small populations of healthy humans to specifically
determine a drug's toxicity, absorption, distribution
and metabolism. |
| Phase II Study |
After the successful completion of phase I trials, a
drug is then tested for safety and efficacy in a
slightly larger population of individuals who are
afflicted with the disease or condition for which the
drug was developed. |
| Phase III Study |
The third and last pre-approval round of testing of a
drug is conducted on large populations of afflicted
patients. Phase III studies usually test the new drug in
comparison with the standard therapy currently being
used for the disease in question. The results of these
trials usually provide the information that is included
in the package insert and labeling. |
| Phase IV Study |
After a drug has been approved by the FDA, phase IV
studies are conducted to compare the drug to a
competitor, explore additional patient populations, or
to further study any adverse events. |
| Pivotal Study |
Usually a phase III study which presents the data that
the FDA uses to decide whether or not to approve a drug.
A pivotal study will generally be well-controlled,
randomized, of adequate size, and whenever possible,
double-blind. |
| 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. |
| Placebo Controlled Study |
A method of investigation of drugs in which an inactive
substance (the placebo) is given to one group of
participants, while the drug being tested is given to
another group. The results obtained in the two groups
are then compared to see if the investigational
treatment is more effective in treating the condition. |
| Placebo Effect |
A physical or emotional change, occurring after a
substance is taken or administered, that is not the
result of any special property of the substance. The
change may be beneficial, reflecting the expectations of
the participant and, often, the expectations of the
person giving the substance. |
| Pre-Clinical Testing |
Before a drug may be tested on humans, pre-clinical
studies must be conducted either in vitro but usually in
vivo on animals to determine that the drug is safe. |
| 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. |
| Protection of Pupil Rights Amendment (PPRA) |
Department of Education regulation that states that
surveys, questionnaires and instructional materials for
school children must be inspected by parents/guardians. |
| 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 |
Changes or clarifications made in writing to the
original protocol. |
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| Quality Assurance |
Systems and procedures designed to ensure that a study
is being performed in compliance with Good Clinical
Practice (GCP) guidelines and that the data being
generated is accurate. |
| Quality of Life Trials |
Refers to trials that explore ways to improve comfort
and quality of life for individuals with a chronic
illness. |
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| Randomization |
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 (See Arm). |
| Randomized Trials
| 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. |
| Recruitment |
Act of enrolling subjects with the proper inclusion
criteria. |
| Recruitment Period |
Time allowed to recruit all subjects for a study. |
| Regulatory Affairs |
In clinical trials, the department or function that is
responsible for ensuring compliance with government
regulations and interacts with the regulatory agencies.
Each drug sponsor has a regulatory affairs department
that manages the entire drug approval process. |
| Research Team |
Investigator, sub-investigator and clinical research
coordinator involved with study. |
| Risk-Benefit Ratio |
Risk to individual subject vs. potential benefits. Also
called Risk-Benefit Analysis. |
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| Safety Reports |
FDA report required by investigator for any serious and
unexpected adverse experience. |
| Serious Adverse Event (SAE) |
Any adverse event (AE) that is fatal, life-threatening,
permanently disabling, or which results in
hospitalization, initial or prolonged. |
| Side Effects |
Any undesired actions or effects of a drug or treatment.
Negative or adverse effects 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 (See Adverse
Reaction). |
| 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 Blind and Double-Blind Study). |
| Single Project Assurance |
Permit given to institution for single grant in
compliance with government standards. See assurance. |
| Source Data |
All information contained in original records and
certified copies of results, observations or other
facets required for the reconstruction and evaluation of
the study that is contained in source documents. |
| Source Documentation |
Location where information is first recorded including
original documents, data and records. |
| Sponsor |
Individual, company, institution or organization taking
responsibility for initiation, management and financing
of study. |
| Standard Operating Procedure (SOP) |
Official, detailed, written instructions for the
management of clinical trials. SOPs ensure that all the
functions and activities of a clinical trial are carried
out in a consistent and efficient manner. |
| Standard Treatment |
The currently accepted treatment or intervention
considered to be effective in the treatment of a
specific disease or condition. |
| Statistical Significance |
The probability that an event or difference occurred by
chance alone. In clinical trials, the level of
statistical significance depends on the number of
participants studied and the observations made, as well
as the magnitude of differences observed. |
| Study Coordinator |
The person at a medical center or research facility who
manages the daily activities of the study, including
coordinating the treatment or testing of participants. |
| Study Endpoint |
A primary or secondary outcome used to judge the
effectiveness of a treatment. |
| Study Type |
The primary investigative techniques used in an
observational protocol; types are Purpose, Duration,
Selection, and Timing. |
| Sub-investigator |
Helps design and conduct investigation at a study site. |
| Subject/Study Subject |
Participant in a study. See Human Subject. |
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| Telephone Report |
Notification via telephone to the FDA of unexpected
fatal or life threatening advent associated with a
clinical study. |
| Toxicity |
An adverse effect produced by a drug that is detrimental
to the participant's health. The level of toxicity
associated with a drug will vary depending on the
condition which the drug is used to treat. |
| Treatment Group |
The group of participants that receives an experimental
treatment. See also control group, standard treatment. |
| Treatment IND |
A method through which the FDA allows seriously ill
patients with no acceptable therapeutic alternative to
access promising investigational drugs still in clinical
development. The drug must show sufficient evidence of
safety and effectiveness. In recent decades many AIDs
patients have been able to access unapproved therapies
through this program. |
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| Unexpected Adverse Drug Reaction |
A reaction that is not consistent in nature or severity
with study application. |
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| Vulnerable Subjects |
Group/individual that cannot give informed consent
because of limited autonomy (e.g., children, mentally
ill and prisoners). Also refers to subjects who may be
unduly influenced to participate (e.g., students,
subordinates and patients). |
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| Well-being |
Subject's physical and mental soundness. |
| Withdrawal Application |
Investigator/sponsor letter to FDA requesting
application withdrawal when no additional work is
envisioned. |
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