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Glossary
510(K) Device - A medical device that is considered substantially equivalent to a device that
was or is being legally marketed. A sponsor planning to market such a device must submit
notification to the FDA 90 days in advance of placing the device on the market. If the FDA
concurs with the sponsor, the device may then be marketed. 510(k) is the section of the Food,
Drug and Cosmetic Act that describes premarket notification; hence the designation "510(k)
device."
Abuse-Liable - Pharmacological substances that have the potential for creating abusive
dependency. Abuse-liable substances can include both illicit drugs (e.g., heroine) and licit
drugs (e.g., methamphetamines).
Accepts Healthy Volunteers - Indicates whether a clinical study allows people who do not
have the condition or related conditions or symptoms being studied to participate in that
study.
Active Comparator Arm - A group of participants that receives an intervention that is
considered to be effective. One of several Arm Types.
Active, Not Recruiting - The clinical study is ongoing (that is, participants are receiving an
intervention or being examined), but potential participants are not currently being recruited
or enrolled. A type of Recruitment Status.
ADAMHA - Alcohol, Drug Abuse, and Mental Health Administration; reorganized in October
1992 as the Substance Abuse and Mental Health Services Administration (SAMHSA).
ADAMHA included the National Institute of Mental Health (NIMH), the National Institute
on Alcohol Abuse and Alcoholism (NIAAA), the National Institute on Drug Abuse (NIDA),
the Office for Substance Abuse Prevention (OSAP), and the Office for Treatment Intervention
(OTI). NIMH, NIAAA, and NIDA are now part of the National Institutes of Health (NIH).
(See also: SAMHSA.)
Adjuvant Therapy - Therapy provided to enhance the effect of a primary therapy; auxiliary
therapy.
Adverse Drug Reaction - 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
phys-iological function. In clinical trials, an ADR would include any injuries by overdosing,
abuse/dependence, and unintended interactions with other medicinal products.
Adverse Effect - An undesirable and unintended, although not necessarily unexpected, result
of therapy or other intervention (e.g., headache following spinal tap or intestinal bleeding
associated with aspirin therapy).
Adverse Event (AE) - A negative experience encountered by an individual during the course
of a clinical trial that is associated with the drug. An AE can include previously undetected
symptoms, or the exacerbation of a pre-existing condition. When an AE has been determined
to be related to the investigational product, it is considered an Adverse Drug Reaction.
Adverse Event Reports - Investigator reports of all serious and adverse events, injury and
deaths given to the sponsor, the IRS and the FDA.
Advertising - One mechanism or method used by researchers to recruit subjects for research
studies.
Allocation - A clinical trial design strategy used to assign participants to an arm of a study.
Types of Allocation include Randomized, and Nonrandomized
Alternatives - Options that exist for a subject who is thinking about participating in research.
Arena Applied Research Ethics National Association - A membership organization for
individuals interested in ethical issues relating to medicine and research.
Arm - A group or subgroup of participants in a clinical trial who receives specific
interventions, or no intervention, according to the study protocol. This is decided before the
trial begins.
Arm Type - A general description of the clinical study arm. It identifies the role of the
intervention that participants will receive. Types of arms include Experimental, Active
comparator, Placebo comparator, Sham comparator, and No intervention.
Assent - Agreement by an individual not competent to give legally valid informed consent to
participate in research (e.g., a child).
Assurance - A formal written, binding commitment that is submitted to a federal agency in
which an institution promises to comply with regulations governing the protection of human
subjects in research. Assurance is the word used in the Federal Policy (Common Rule).
Authorized Institutional Official - An officer of an institution with the authority to speak for
and legally commit the institution to adherence to the requirements of the federal regulations
regarding the involvement of human subjects in biomedical and behavioral research.
Autonomy Personal - Capacities to consider alternatives, make choices, and act without
undue influence or interference of others.
Autopsy - Examination by dissection of the body of an individual to determine cause of death
and other medically relevant facts.
Baseline Characteristics - Data collected at the beginning of a clinical study for all
participants and for each arm or comparison group.
Belmont Report - A statement of basic ethical principles governing research involving human
subjects issued in 1978 by the National Commission for the Protection of Human Subjects of
Biomedical and Behavioral Research.
Beneficence - An ethical principle discussed in the Belmont Report that entails an obligation
to protect persons from harm. The principle of beneficence can be expressed in two general
rules: (1) do not harm; and (2) protect from harm by maximizing possible benefits and
minimizing possible risks of harm.
Benefit - A valued or desired outcome; an advantage.
Biologic - A virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or
derivative, allergenic product, or analogous product applicable to the prevention, treatment
or cure of diseases or injuries of man.
Biotechnology - Any technique that uses living organisms, or substances from organisms,
bio-logical systems, or processes to make or modify a product or process, to change plants or
animals, or to develop micro-organisms for specific uses.
Blind Study Designs - See: Masked Study Designs; Double-Masked Design; and SingleMasked Design.
Cadaver - The body of a deceased person.
Case Report Forms - Study document of all data required by protocol on each subject.
Case-Control Study - A study comparing persons with a given condition or disease (the
cases) and persons without the condition or disease (the controls) with respect to antecedent
factors. (See also: Retrospective Studies.)
Cat Scan - Abbreviation for Computerized Axial Tomography, an X-ray technique for
producing images of internal bodily structures through the assistance of a computer.
CDC Centers for Disease Control and Prevention; an agency within the Public Health Service,
Department of Health and Human Services.
Certain Agreements - As required by Section 801 of the Food and Drug Administration
Amendments Act, in general, a description of any agreement between the sponsor of a
clinical study and the principal investigator (PI) that does not allow the PI to discuss the
results of the study or to publish the study results in a scientific or academic journal after the
trial is completed.
Certificate of Confidentiality - A Certificate of Confidentiality protects the compelled release
of identifiable information about research subjects in any legal proceeding. These documents
are issued by the DHHS and can be requested for all research, regardless of funding source.
Certification - The human subject regulations, in certain parts require the Institutional
Review Board (IRB) to provide a "certification" to the government.
Certified Clinical Research Coordinator - (CCRC) CRC with more than two years’ experience
and with certification earned by passing required program and exam.
Certified IRE Professional - (CIP) IRS administrative personnel with more than two years’
experience and with certification earned by passing required exam.
Chair - The person who leads the activities of the IRB.
Children Persons - who have not attained the legal age for consent to treatment or procedures
involved in the research, as determined under the applicable law of the jurisdiction in which
the research will be conducted [45 CFR 46.401(a)].
Class I, II, III Devices - Classification by the Food and Drug Administration of medical
devices according to potential risks or hazards.
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 Food and Drug Administration (FDA).
Clinical Research Associate (CRA) - Person employed by the study sponsor or CRO to
monitor a clinical study at all participating sites. See also, moni-tor.
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 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. Note that in this manual, this term is used in its
narrow sense as used by the FDA. Thus, it does not encompass all the research that is carried
out in the clini-cal setting (e.g., health services research). See also, patient-oriented research.
Clinical Study - A research study using human subjects to evaluate the effect of interventions
or exposures on biomedical or health-related outcomes.
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 - Any investigation in human subjects intended to determine the clinical
phar-macological, pharmacokinetic, and/or other pharmacodynamics effects of an
investigational agent, and/or to identify any adverse reactions to an investiga-tional agent to
assess the agents’ safety and efficacy.
Closed Studies - Clinical studies that are no longer recruiting participants because they have
enough participants already, because they are completed, or because they have been stopped
for some reason.
Coercion - The act of inducing or pressuring an individual to consent to participate in
research or to stay in research.
Cognitive Impairment - Some disorder that affects cognitive or emotional functions to the
extent that capacity for judgment and reasoning is significantly diminished.
Cognitively Impaired - Having either a psychiatric disorder (e.g., psychosis, neurosis,
personality or behavior disorders, or dementia) or a developmental disorder (e.g., mental
retardation) that affects cognitive or emotional functions to the extent that capacity for
judgment and reasoning is significantly diminished. Others, including persons under the
influence of or dependent on drugs or alcohol, those suffering from degenerative diseases
affecting the brain, terminally ill patients, and persons with severely disabling physical
handicaps, may also be compromised in their ability to make decisions in their best interests.
Cohort - A group of subjects initially identified as having one or more characteristics in
common who are followed over time. In social science research, this term may refer to any
group of persons who are born at about the same time and share common historical or
cultural experiences.
Collaborator - A collaborator is an organization other than the sponsor that provides support
for a clinical study. This may include funding, design, implementation, data analysis, or
reporting.
Common Rule - The short description of the Federal Policy for the Protection of Human
Subjects in Research.
Compensation - Payment or medical care provided to subjects injured in research; does not
refer to payment (remuneration) for participation in research. (Compare: Remuneration.)
Competence - The capacity to act on one's own behalf; the ability to understand information
presented; to appreciate the consequences of acting or not acting on that information, and to
make a choice.
Condition - A disease, disorder, syndrome, illness, or injury that is being studied.
Confidentiality - Pertains to the treatment of information that an individual has disclosed in a
relationship of trust and with the expectation that it will not be divulged to others without
permission in ways that are inconsistent with the understanding of the original disclosure.
Consent - Agreement to do something. Informed consent is agreement to do something based
upon a complete understanding of that task.
Consent Form - Contains all relevant study information explained in lay terms and
documents voluntary participation of subject. Presented to and signed by subject.
Continuing Review - The regulatory requirement that the Institutional Review Board (IRB)
review research at intervals not greater than one year. The IRB may review research at more
frequent intervals.
Contract - An agreement; as used here, an agreement that a specific research activity will be
performed at the request, and under the direction, of the agency providing the funds.
Research performed under contract is more closely controlled by the agency than research
performed under a grant. (Compare: Grant.)
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.
Contraindicated - Disadvantageous, perhaps dangerous; a treatment that should not be used
in certain individuals or conditions due to risks (e.g., a drug may be contraindicated for
pregnant women and persons with high blood pressure).
Control - Subject(s) used for comparison who are not given a treatment under study or who
do not have a given condition, background, or risk factor that is the object of the study.
Control Group - The comparison group of subjects who are not treated with the
investigational agent. The subjects in this group may receive no therapy, a different therapy,
or a placebo.
Correlation Coefficient - A statistical index of the degree of relationship between two
variables. Values of correlation coefficients range from -1.00 through zero to +1.00. A
correlation coefficient of 0.00 indicates no relationship between the variables. Correlations
approaching -1.00 or +1.00 indicate strong relationships between the variables. However,
causal inferences about the relationship between two variables can never be made on the
basis of correlation coefficients, no matter how strong a relationship is indicated.
Crossover Design - Describes a clinical trial in which groups of participants receive two or
more interventions in a particular order.
Data and Safety Monitoring Board (DSMB) - A group of people who monitor a clinical trial
for adverse events and other trends. The Data and Safety Monitoring Board looks for any
information that might warrant modification or termination of the trial or notification of
subjects about new information that might affect their willingness to continue in the trial.
Data - Legally defined according to institution. Generally refers to recorded information
regardless of form. Most institutions hold title to data while researchers have rights to access
data.
Data Management - The process of handling the data gathered during a clinical trial. May
also refer to the department responsible for managing data entry and database generation
and/or maintenance.
Dead Fetus - An expelled or delivered fetus that exhibits no heartbeat, spontaneous
respiratory activity, spontaneous movement of voluntary muscles, or pulsation of the
umbilical cord (if still attached) [45 CFR 46.203(f)]. Generally, some organs, tissues, and cells
(referred to collectively as fetal tissue) remain alive for varying periods of time after the total
organism is dead.
Debriefing - Giving subjects previously undisclosed information about the research project
following completion of their participation in research. (Note that this usage, which occurs
within the behavioral sciences, departs from standard English, in which debriefing is
obtaining rather than imparting information.)
Deception - Intentionally misleading with respect to a research protocol.
Declaration of Helsinki - A code of ethics for clinical research approved by the World
Medical Association in 1964 and widely adopted by medical associations in various countries.
It was revised in 1975 and 1989.
Demographic Data - Refers to the characteristics of study participants, including sex, age,
family medical history, and other characteristics relevant to the study in which they are
enrolled.
Dependent Variable - The outcomes that are measured in an experiment. Dependent variables
are expected to change as a result of an experimental manipulation of the independent
variable(s).
Descriptive Study - Any study that is not truly experimental (e.g., quasi-experimental studies,
correlational studies, record reviews, case histories, and observational studies).
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.
DHEW - A federal agency: U.S. Department of Health, Education and Welfare; reorganized in
1980 as the Department of Health and Human Services (DHHS) and the Department of
Education.
DHHS - A federal agency: U.S. Department of Health and Human Services; formerly the
Department of Health, Education and Welfare (DHEW).
Diagnostic (Procedure) - Tests used to identify a disorder or disease in a living person.
Documentation - All forms of records that describe or document study methods, conduct and
results, including any adverse events and actions taken.
Double-Blind - The design of a study in which neither the investigator nor the subject know
which medication (or placebo) the subject is receiving. Sometimes referred to as DoubleMasked Design
Drug - As defined by the Food, Drug and Cosmetic Act, drugs are "articles intended for the
use in the diagnosis, cure, mitigation, treatment or prevention of disease in man or other
animals, or to affect the structure t 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. table, capsule, or solution) that contains active
drug ingredient usually combined with inactive ingredients.
Effective Dose - The dose of an investigational agent that produces the outcome consider
("effective as defined in the study protocol. This could mean a cure of the di F-Ase in question
or simply the mitigation of symptoms.
Efficacy - A product's ability to produce beneficial effects on the duration or course a disease.
Efficacy is measured by evaluating the clinical and statistical result of clinical tests.
Eligibility Criteria The key standards that people who want to participate in a clinical study
must meet or the characteristics that they must have.
Emancipated Minor - Defined by state law, this refers to the legal status of a person who has
not yet attained the age of legal competency but who is entitled to adult status for certain
matters.
Embryo - Early stages of a developing organism broadly used to refer to stages immediately
following fertilization of an egg through implantation and very early pregnancy.
Enrolling by Invitation - A clinical study that selects its participants from a population, or
group of people, decided on in advance by the researchers.
Enrollment - The number of participants in a clinical study. The "estimated enrollment" is the
number of participants that the researchers need for the study.
Epidemiology - A scientific discipline that studies the factors determining the causes,
frequency, and distribution of diseases in a community or given population.
Equitable - Fair or just; used in the context of selection of subjects to indicate that the benefits
and burdens of research are fairly distributed [Federal Policy §___.111 (a) (3)].
Ethical Principles and Guidelines for the Protection of Human Subjects of Research - See
Belmont Report.
Ethics Advisory Board - An interdisciplinary group that advises the Secretary, HHS, on
general policy matters and on research proposals (or classes of proposals) that pose ethical
problems.
Ethics Committee - An independent group of both medical and non-medical professionals
ware responsible for verifying the integrity of a study and ensuring the safe integrity, and
human rights of the study participants.
Ethnographic Research - Ethnography is the study of people and their culture. Ethnographic
research, also called fieldwork, involves observation of and interaction with the persons or
group being studied in the group's own environment, often for long periods of time. (See
also: Fieldwork.)
Exclusion Criteria - Refers to the characteristics that would prevent a subject from
participation in a clinical trial, as outlined in the study protocol.
Exemptions - The Federal Policy for the Protection of Human Subjects contains six
exemptions. Research falling under one of these exemptions is not required to undergo IRB
review and the investigator is not required to abide by the requirements for obtaining
information consent. FDA regulations contain an exemption from IRB review requirements
for the emergency use of a test article and for certain taste and food quality evaluations and
consumer acceptance studies.
Expanded Access - A process regulated by the Food and Drug Administration (FDA) that
allows manufacturers to provide investigational new drugs to patients with serious diseases
or conditions who cannot participate in a clinical trial.
Expanded Availability - Policy and procedure that permits individuals who have serious or
life-threatening diseases for which there are no alternative therapies to have access to
investigational drugs and devices that may be beneficial to them. Examples of expanded
availability mechanisms include Treatment INDs, Parallel Track, and open study protocols.
Expedited Review - Review of proposed research by the IRB chair or a designated voting
member or group of voting members rather than by the entire convened
Experiment - Generally, this refers to an intervention or interaction that is unproven and not
yet scientifically validated.
Experimental Arm - A group of participants that receives the intervention that is the focus of
the study.
Experimental Study - A true experimental study is one in which subjects are randomly
assigned to groups that experience carefully controlled interventions manipulated by the
experimenter according to a strict logic allowing causal inference about the effects of the
interventions under investigation. (See also: Quasi-Experimental Study).
Experimental - Term often used to denote a therapy (drug, device, procedure) that is
unproven or not yet scientifically validated with respect to safety and efficacy. A procedure
may be considered "experimental" without necessarily being part of a formal study (research)
to evaluate its usefulness. (See also: Research.)
Factorial Design - Describes a clinical study in which groups of participants receive one of
several combinations of interventions
False Negative - When a test wrongly shows an effect or condition to be absent (e.g., that a
woman is not pregnant when, in fact, she is).
False Positive - When a test wrongly shows an effect or condition to be present (e.g. that is
woman is pregnant when, in fact, she is not).
Family Educational Rights and Privacy Act (FERPA) - Covers rights of par-ents of school
children regarding reviewing, amending and disclosing educational records.
FDA Food and Drug Administration - Agency of the federal government established by
Congress in 1912 and presently part of the Department of Health and Human Services. FDA
is responsible for protecting the public health by making sure that human and veterinary
drugs, vaccines and other biological products, medical devices, the nation's food supply,
cosmetics, dietary supplements, and products that give off radiation are safe, effective, and
secure.
Federal Policy - The federal policy that provides regulations for the involvement of human
subjects in research. The Policy applies to all research involving human subjects conducted,
supported, or otherwise subject to regulation by any federal department or agency that takes
appropriate administrative action to make the Policy applicable to such research. Currently,
sixteen federal agencies have adopted the Federal Policy. (Also known as the "Common
Rule.")
Federal Register - The government's publication in which final and proposed rules or notices
are published.
Federal-Wide Assurance (FWA) - Permit given to institution for multiple federally funded
research grants for a specified period of time. States insti-tution-retains responsibility for all
research involving humans and that the institution must have an established IRE.
Fetal Material - The placenta, amniotic fluid, fetal membranes, and umbilical cord.
Fetus - The product of conception from the time of implantation until delivery.
Fieldwork - Behavioral, social, or anthropological research involving the study of persons or
groups in their own environment and without manipulation for research purposes
(distinguished from laboratory or controlled settings). (See also: Ethnographic Research.)
Formulation - The mixture of chemicals and/or biological substances and excipients used to
prepare dosage forms.
Full Board Review - Review of proposed research at a convened meeting of the IRB, at which
a majority of the membership of the IRB is present, including at least one member whose
primary concerns are in a nonscientific area.
Funder Type - Describes the organization that provides funding or support for the clinical
study.
Genders Eligible for Study - The physical gender of persons who may participate in the
clinical study (female, male, or both).
Gene Therapy - The treatment of genetic disease accomplished by altering the genetic
structure of either somatic (non-reproductive) or germ-line (reproductive) cells.
General Assurance - Obsolete term, previously used to denote an institutional assurance
covering multiple research projects. (See also: Assurance.)
General Controls - Certain FDA statutory provisions designed to control the safety of
marketed drugs and devices. The general controls include provisions on adulteration,
misbranding, banned devices, good manufacturing practices, notification and record keeping,
and other sections of the Medical Device Amendments to the Food, Drug and Cosmetic Act
[21 U.S. Code §360(c) (Food, Drug and Cosmetic Act §513)].
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.
Genetic Screening - Tests to identify persons who have an inherited predisposition to a certain
phenotype or who are at risk of producing offspring with inherited diseases or disorders.
Genotype - The genetic constitution of an individual.
Good Clinical Practice (GCP) - The FDA has established regulations and guidelines that
specify the responsibilities of sponsors, investigators, monitors, and IRBs involved in clinical
drug testing. These regulations are meant to protect the safety, rights and welfare of the
patients in addition to ensuring the accuracy of the collected study data.
Grant - Financial support provided for a research study designed and proposed by the
principal investigator.
Guardian - An individual who is authorized under applicable state or local law to give
permission on behalf of another person to participate in research.
Health Authority - A national or international health organization that has authority over the
clinical study.
Helsinki Declaration - See: Declaration of Helsinki.
Historical Controls - Control subjects (followed at some time in the past or for whom data
are available through records) who are used for comparison with subjects being treated
concurrently. The study is considered historically controlled when the present condition of
subjects is compared with their own condition on a prior regimen or treatment.
Human in Vitro Fertilization - Any fertilization involving human sperm and ova that occurs
outside the human body.
Human Protections Administrator - An individual who has responsibility for day-to- day
operation and implementation of the institution's program for protecting human subjects.
Human Subject - A patient or healthy individual participating in a research study. A living
individual about whom an investigator obtains private infor-mation or data through
intervention or interaction.
Human Subjects Review Board - A group of people who review, approve, and monitor the
clinical study protocol.
IDE - See: Investigational Device Exemptions.
In Vitro - Literally, "in glass" or "test tube;" used to refer to processes that are carried out
outside the living body, usually in the laboratory, as distinguished from in vivo.
In Vitro Testing - Non-clinical testing conducted in an artificial environment such as a test
tube or culture medium.
In Vivo - Literally, "in the living body;" processes, such as the absorption of a drug by the
human body, carried out in the living body rather than in a laboratory (in vitro).
In Vivo Testing - Testing conducted in living animal and human systems.
Incapacity - Refers to a person's mental status and means inability to understand information
presented, to appreciate the consequences of acting (or not acting) on that information, and to
make a choice.
Inclusion Criteria - The criteria that establish whether a person is eligible to participate in a
clinical trial.
Incompetence - A legal term meaning inability to manage one's own affairs.
IND - See: Investigational New Drug.
Independent Ethics Committee (IEC) - The equivalent of an IRB under the International
Conference on Harmonization Guidelines for Good Clinical Practice.
Independent Variables - The conditions of an experiment that are systematically manipulated
by the investigator.
Informed Consent - A person's voluntary agreement, based upon adequate knowledge and
understanding of relevant information, to participate in research or to undergo a diagnostic,
therapeutic, or preventive procedure.
Institution Location of research. Retains ultimate responsibility for human subject regulation
compliance.
Institutional Official - The individual at an institution who is responsible for ensuring the
effective administration and implementation of the institution's system for the protection of
human subjects.
Institutional Review Board (IRB) - A review body established by regulation to protect the
welfare of human subjects recruited to participate in research.
Institutionalized Cognitively Impaired - Persons who are confined, either voluntarily or
involuntarily, in a facility for the care of the mentally or otherwise disabled (e.g., a psychiatric
hospital, home, or school for the retarded).
Institutionalized - Confined, either voluntarily or involuntarily (e.g., a hospital, prison, or
nursing home).
Intervention - A process or action that is the focus of a clinical study.
Intervention Model (Design) - The general design describing the strategy in which
interventions will be assigned to participants in a clinical study.
Intervention Name The intervention being studied.
Intervention Type - The general category for the intervention being studied. Intervention
types include Drug, Device, Biological, and Procedure.
Interventional Study (or Clinical Trial) - A clinical study in which participants are assigned
to receive one or more interventions (or no intervention) so that researchers can evaluate the
effects of the interventions on biomedical or health-related outcomes
Investigational Device Exemption (IDE) - Exemptions from certain regulations found in the
FDA, Medical Device Amendments that allow shipment of unapproved devices for use in
clinical investigations.
Investigational Materials -Test articles under clinical investigation.
Investigational New Drug Application (IND) - An application to conduct a clinical
investigation involving a drug not yet determined by the Food and Drug Administration to
be safe and effective for a particular use in the general population and not yet licensed for
marketing.
Investigational New Drug or Device - A drug or device permitted by FDA to be tested in
humans but not yet determined to be safe and effective for a particular use in the general
population and not yet licensed for marketing.
Investigator - A medical professional, usually a physician but may also be a nurse,
pharmacist or other health care professional, under whose direction an investiga-tional drug
is administered or dispensed. A principal investigator is respon-sible for the overall conduct
of the clinical trial at his/her site.
Investigator Sponsor - Individual with both responsibilities of initiating and conducting a
clinical study.
Investigator's Brochure Relevant clinical and non-clinical data compiled on the
investigational drug, biologic or device being studied.
IRB - See: Institutional Review Board.
Justice - An ethical principle discussed in the Belmont Report requiring fairness in
distribution of burdens and benefits; often expressed in terms of treating persons of similar
circumstances or characteristics similarly.
Kefauver-Harris Amendments Amendment to FD&C - Act that requires informed consent for
experimental drugs.
Lactation - The period of time during which a woman is providing her breast milk to an
infant or child.
Legally Authorized Representative (LAR) - The person authorized by state law to consent to
something on behalf of another person. For research purposes, only select states permit a
LAR to consent for research participation.
Legally Authorized Representative - A person authorized either by statute or by court
appointment to make decisions on behalf of another person. In human subjects research, an
individual or judicial or other body authorized under applicable law to consent on behalf of a
prospective subject to the subject's participation in the procedure(s) involved in the research
[Federal Policy §___.102(c)].
Lod Score - An expression of the probability that a gene and a marker are linked.
Longitudinal Study - A study conducted over a long period of time.
Masked Study Design - Study designs comparing two or more interventions in which either
the investigators, the subjects, or some combination thereof do not know the treatment group
assignments of individual subjects. Sometimes called "blind" study designs. (See also: DoubleMasked Design; Single-Masked Design.)
Masking (or Blinding) - A clinical trial design strategy in which one or more parties involved
with the trial, such as the investigator or participant, do not know which participants have
been assigned which interventions.
Mature Minor - Someone who has not reached adulthood (as defined by state law) but who
may be treated as an adult for certain purposes (e.g., consenting to medical care). Note that a
mature minor is not necessarily an emancipated minor. (See also: Emancipated Minor.)
Medical Device - A diagnostic or therapeutic article that does not achieve any of its principal
intended purpose through chemical action within or on the body. Such devices include
diagnostic test kits, crutches, electrodes, pacemakers, arterial grafts, intraocular lenses, and
orthopedic pins or other orthopedic equipment.
Medical Device Amendment (MDA) - Amendments to the Federal Food, Drug and Cosmetic
Act passed in 1976 to regulate the distribution of medical devices and diagnostic products.
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.
Member - A person who is listed on the roster of an IRB as a voting participant in IRB
deliberations and actions.
Mentally Disabled - See: Cognitively Impaired.
Metabolism (of a drug) - The manner in which a drug is acted upon (taken up, converted to
other substances, and excreted) by various organs of the body.
Minimal Risk - The probability and magnitude of harm or discomfort anticipated in the
research are not greater in and of themselves than those ordinarily encountered in daily life
or during the performance of routine physical or psychological examinations or tests. The
definition of minimal risk for research involving prisoners differs somewhat from that given
for no institutionalized adults. [See 45 CFR 46.303(d) and Guidebook Chapter 6, Section E,
"Prisoners."]
Misconduct - Fabrication, falsification, plagiarism or other practices that seriously deviate
from those accepted within scientific community for con-ducting and reporting research.
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
documen-tation from the study. See also CRA.
Monitoring - A mechanism for keeping track of any part of the research process: data
analysis, recruitment of subjects, informed consent process, to ensure its compliance with
Institutional Review Board dictates and the federal regulations.
National Bioethics Advisory Commission (NBAC) - A Presidentially appointed commission
that issues reports and makes recommendations relating to the protection of human subjects
in research.
National Commission National Commission for the Protection of Human Subjects of
Biomedical and Behavioral Research - An interdisciplinary advisory body, established by
Congressional legislation in 1974, which was in existence until 1978, and which issued a series
of reports and recommendations on ethical issues in research and medicine, many of which
are now embodied in federal regulations.
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 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 sub-ject protection by informed consent.
NDA - See: New Drug Application.
New Drug Application (NDA) - The compilation of all nonclinical, 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.
NIAAA - National Institute on Alcohol Abuse and Alcoholism; an institute in NIH.
NIDA - National Institute on Drug Abuse; an institute in NIH.
NIH National Institutes of Health - A federal agency within the Public Health Service,
DHHS, comprising 21 institutes and centers. It is responsible for carrying out and supporting
biomedical and behavioral research.
NIMH - National Institute of Mental Health; an institute in NIH.
No Intervention Arm - A group of participants that does not receive any interventions during
a clinical study.
Non-Affiliated Member - Member of an IRB who has no ties (and whose immediate family
members have no ties) to the parent institution, its staff, or faculty. This individual is usually
from the local community.
Non-Scientist - Member of an IRB who does not have a scientific background, but may be
affiliated with the institution. At least one non-scientist member must be present at convened
meetings to approve research.
Non-significant Risk Device - An investigational medical device that does not present
significant risk to the patient. (See also: Significant Risk Device.)
Non-therapeutic Research - Research that has no likelihood or intent of producing a
diagnostic, preventive, or therapeutic benefit to the current subjects, although it may benefit
subjects with a similar condition in the future.
Nonviable Fetus - An expelled or delivered fetus which, although it is living, cannot possibly
survive to the point of sustaining life independently, even with the support of available
medical therapy [45 CFR 46.203 (d) and (e)]. Although it may be presumed that an expelled or
delivered fetus is nonviable at a gestational age less than 20 weeks and weight less than 500
grams [Federal Register 40 (August 8, 1975): 33552], a specific determination as to viability
must be made by a physician in each instance. (See also: Viable Infant.)
Normal Volunteers - Volunteer subjects used to study normal physiology and behavior or
who do not have the condition under study in a particular protocol, used as comparisons
with subjects who do have the condition. "Normal" may not mean normal in all respects. For
example, patients with broken legs (if not on medication that will affect the results) may serve
as normal volunteers in studies of metabolism, cognitive development, and the like. Similarly,
patients with heart disease but without diabetes may be the "normals" in a study of diabetes
complicated by heart disease.
Notice of Proposed Rule-Making (NPRM) - Pursuant to the Administrative Procedure Act,
the government must typically issue a notice of a proposed rule before it issues the final rule.
This affords the public the opportunity to comment on contemplated government action.
Null Hypothesis - The proposition, to be tested statistically, that the experimental
intervention has "no effect," meaning that the treatment and control groups will not differ as a
result of the intervention. Investigators usually hope that the data will demonstrate some
effect from the intervention, thereby allowing the investigator to reject the null hypothesis.
Nuremberg Code - As a result of the medical experimentation conducted by Nazis during
World War II, the U.S. Military Tribunal in Nuremberg in 1947 set forth a code of medical
ethics for researchers conducting clinical trials. The code is designed to protect the safety and
integrity of study participants.
Observational Study - A clinical study in which participants identified as belonging to study
groups are assessed for biomedical or health outcomes. Participants may receive diagnostic,
therapeutic, or other types of interventions, but the investigator does not assign participants
to specific interventions (as in an interventional study).
Observational Study Model (Design) - The general design, describing the strategy for
identifying and following up with participants during observational studies.
Off Label - The unauthorized use of a drug for a purpose other than that approved of by the
FDA.
Office for Human Research Protection (OHRF) - Federal government office that issues
Assurances and overseas compliance.
Office for Protection from Research Risks (OPRR) - The office within the National Institutes
of Health, an agency of the Public Health Service, Department of Health and Human Services,
responsible for implementing DHHS regulations (45 CFR Part 46) governing research
involving human subjects.
Open Design - An experimental design in which both the investigator(s) and the subjects
know the treatment group(s) to which subjects are assigned.
Open Studies - Studies that are currently recruiting participants, will be recruiting
participants in the future, or involve drugs that are available for expanded access.
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.
OPRR - See: Office for Protection from Research Risks.
Oral Consent - Typically refers to informed consent that is obtained from a subject without
use of a written informed consent document.
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
Outcome Measure - A planned measurement described in the protocol that is used to
determine the effect of interventions on participants in a clinical trial. For observational
studies, a measurement or observation that is used to describe patterns of diseases or traits, or
associations with exposures, risk factors, or treatment..
Over-the-Counter (OTC) - Drugs available for purchase without a physician's prescription.
Parallel Design - Describes a clinical trial in which two or more groups of participants receive
different interventions.
Parental Permission - The agreement of one or both parents or a guardian to research
involving a minor.
Paternalism - Making decisions for others against or apart from their wishes with the intent
of doing them good.
Patient - Individual seeking medical care.
Patient Oriented Research - Research conducted with human subjects (or on material of
human origin) in which an investigator or colleague directly interacts with human subjects.
(Nili definition of clinical research)
Permission - The agreement of parent(s) or guardian to the participation of their child or
ward in research [45 CFR 46.402(c)].
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."
Pharmacology - The scientific discipline that studies the action of drugs on living systems
(animals or human beings).
Phase 1 Clinical Trials - Phase 1 trials include the initial introduction of an investigational
new drug into humans. These studies are typically conducted with healthy volunteers;
however, where the drug is intended for use in patients with a particular disease, such
patients may participate as subjects. Phase 1 trials are designed to determine the metabolic
and pharmacological actions of the drug in humans, the side effects associated with
increasing doses (to establish a safe dose range), and, if possible, to gain early evidence of
effectiveness. They are typically closely monitored. The ultimate goal of Phase 1 trials is to
obtain sufficient information about the drug's pharmacokinetics and pharmacological effects
to permit the design of well-controlled, sufficiently valid Phase 2 studies. Other examples of
Phase I studies include studies of drug metabolism, structure-activity relationships, and
mechanisms of actions in humans, as well as studies in which investigational drugs are used
as research tools to explore biological phenomena or disease processes. Typically, Phase 1
investigations involve anywhere from 20¬80 subjects.
Phase 1 Drug Trial - Phase 1 trials include the initial introduction of an investigational new
drug into humans. These studies are typically conducted with healthy volunteers; sometimes,
where the drug is intended for use in patients with a particular disease, however, such
patients may participate as subjects. Phase 1 trials are designed to determine the metabolic
and pharmacological actions of the drug in humans, the side effects associated with
increasing doses (to establish a safe dose range), and, if possible, to gain early evidence of
effectiveness; they are typically closely monitored. The ultimate goal of Phase 1 trials is to
obtain sufficient information about the drug's pharmacokinetics and pharmacological effects
to permit the design of well-controlled, sufficiently valid Phase 2 studies. Other examples of
Phase 1 studies include studies of drug metabolism, structure-activity relationships, and
mechanisms of actions in humans, as well as studies in which investigational drugs are used
as research tools to explore biological phenomena or disease processes. The total number of
subjects involved in Phase 1 investigations is generally in the range of 20-80.
Phase 1, 2, 3, 4 Drug Trials - Different stages of testing drugs in humans, from first
application in humans (Phase 1) through limited and broad clinical tests (Phase 3), to post
marketing studies (Phase 4).
Phase 1,2,3,4, Clinical Trials - Different stages of testing drugs in humans, from first
application in humans (Phase 1) through limited and broad clinical tests (Phase 3), to post
marketing studies (Phase 4).
Phase 2 Clinical Trials - Phase 2 trials include controlled clinical studies conducted to
evaluate the drug's effectiveness for a particular indication in patients with the disease or
condition under study, and to determine the common short-term side effects and risks
associated with the drug. These studies are typically well-controlled, closely monitored, and
conducted with a relatively small number of patients, usually involving no more than several
hundred subjects.
Phase 2 Drug Trial - Phase 2 trials include controlled clinical studies conducted to evaluate
the drug's effectiveness for a particular indication in patients with the disease or condition
under study, and to determine the common short-term side effects and risks associated with
the drug. These studies are typically well-controlled, closely monitored, and conducted with
a relatively small number of patients, usually involving no more than several hundred
subjects.
Phase 3 Clinical Trials - Phase 3 trials involve the administration of a new drug to a larger
number of patients in different clinical settings to determine its safety, efficacy, and
appropriate dosage. They are performed after preliminary evidence of effectiveness has been
obtained, and are intended to gather necessary additional information about effectiveness
and safety for evaluating the overall benefit-risk relationship of the drug, and to provide an
adequate basis for physician labeling. In Phase 3 studies, the drug is used the way it would be
administered when marketed. When these studies are completed and the sponsor believes
that the drug is safe and effective under specific conditions, the sponsor applies to the FDA
for approval to market the drug. Phase 3 trials usually involve several hundred to several
thousand subjects.
Phase 3 Drug Trial - Phase 3 trials involve the administration of a new drug to a larger
number of patients in different clinical settings to determine its safety, efficacy, and
appropriate dosage. They are performed after preliminary evidence of effectiveness has been
obtained, and are intended to gather necessary additional information about effectiveness
and safety for evaluating the overall benefit-risk relationship of the drug, and to provide an
adequate basis for physician labeling. In Phase 3 studies, the drug is used the way it would be
administered when marketed. When these studies are completed and the sponsor believes
that the drug is safe and effective under specific conditions, the sponsor applies to the FDA
for approval to market the drug. Phase 3 trials usually involve several hundred to several
thousand patient-subjects.
Phase 4 Clinical Trials - The FDA, when it gives market approval, may seek an agreement
from the sponsor to conduct certain post marketing studies to ascertain additional
information about the drug's risks, benefits, and optimal use. These studies could include, but
would not be limited to, studying different doses or schedules of administration than were
used in Phase 2 studies, use of the drug in other patient populations or other stages of the
disease, or use of the drug over a longer period of time.
Phase 4 Drug Trial - Concurrent with marketing approval, FDA may seek agreement from
the sponsor to conduct certain post marketing (Phase 4) studies to delineate additional
information about the drug's risks, benefits, and optimal use. These studies could include, but
would not be limited to, studying different doses or schedules of administration than were
used in Phase 2 studies, use of the drug in other patient populations or other stages of the
disease, or use of the drug over a longer period of time [21 CFR §312.85].
Phenotype - The physical manifestation of a gene function.
PHS Public Health Service - Part of the U.S. Department of Health and Human Services, it
includes FDA, NIH, CDC, SAMHSA, and HRSA.
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 - An inactive substance designed to resemble the drug being tested. It is used as a
control to rule out any psychological effects testing may present. Most well-designed studies
include a control group which is unwittingly taking a placebo.
Placebo Comparator Arm - A group of participants that receives a placebo during a clinical
study.
Post amendment Devices - Medical devices marketed after enactment of the 1976 Medical
Device Amendments.
Preamendment Devices - Medical devices marketed before enactment of the 1976 Medical
Device Amendments.
Pre-Clinical Investigations - Laboratory and animal studies designed to test the mechanisms,
safety, and efficacy of an intervention prior to its applications to humans.
Pre-Clinical Testing - Before a drug may be tested on humans, pre-clinical studies must be
con-ducted either in vitro but usually in vivo on animals to determine that the drug is safe.
Predicate Devices - Currently legally marketed devices to which new devices may be found
substantially equivalent under the 510(k) process.
Pregnancy - The period of time from confirmation of implantation of a fertilized egg within
the uterus until the fetus has entirely left the uterus (i.e., has been delivered). Implantation is
confirmed through a presumptive sign of pregnancy such as missed menses or a positive
pregnancy test. This confirmation may be in error, but, for research purposes, investigators
must presume that a living fetus is present until evidence to the contrary is clear. Although
fertilization occurs a week or more before implantation, the current inability to detect the
fertilization event or the presence of a newly fertilized egg makes a definition of pregnancy
based on implantation necessary.
Premarket Approval - Process of scientific and regulatory review by the FDA to ensure the
safety and effectiveness of Class III devices.
President's Commission President's Commission for the Study of Ethical Problems in
Medicine and Biomedical and Behavioral Research - An interdisciplinary advisory group,
established by congressional legislation in 1978, which was in existence until 1983, and which
issued reports on ethical problems in health care and in research involving human subjects.
Primary Purpose - The one main reason for the clinical trial. Types of primary purposes
include treatment, prevention, diagnostic, supportive care, screening, health services
research, and basic science.
Principal Investigator (PI) - The person with primary responsibility for design and conduct
of a research project.
Prisoner - An individual involuntarily confined or detained in a penal institution, including
persons: (1) sentenced under a criminal or civil statute; (2) detained pending arraignment,
trial, or sentencing; and (3) detained in other facilities (e.g., for drug detoxification or
treatment of alcoholism) under statutes or commitment procedures providing such
alternatives to criminal prosecution; or (4) incarcerated in a penal institution.
Prisoner Representative - A member of an IRB who has appropriate background and
experience to represent the interests and concerns of an individual who is involuntarily
confined to an institution.
Privacy - Control over the extent, timing, and circumstances of sharing oneself (physically,
behaviorally, or intellectually) with others.
Proband - The person whose case serves as the stimulus for the study of other members of the
family to identify the possible genetic factors involved in a given disease, condition, or
characteristic.
Prophylactic - Preventive or protective; a drug, vaccine, regimen, or device designed to
prevent, or provide protection against, a given disease or disorder.
Prospective Studies - Studies designed to observe outcomes or events that occur subsequent
to the identification of the group of subjects to be studied. Prospective studies need not
involve manipulation or intervention but may be purely observational or involve only the
collection of data.
Protection of Pupil Rights Amendment (PPRA) - Department of Education regulation that
states that surveys, questionnaires and instructional materi-als for school children must be
inspected by parents/guardians.
Protocol Amendment - Changes or clarifications made in writing to the original protocol.
Protocol - The formal design or plan of an experiment or research activity; specifically, the
plan submitted to an IRB for review and to an agency for research support. The protocol
includes a description of the research design or methodology to be employed, the eligibility
requirements for prospective subjects and controls, the treatment regimen(s), and the
proposed methods of analysis that will be performed on the collected data.
Public Health Service (PHS) - A division within the DHHS. PHS agencies include the
National Institutes of Health, Centers for Disease Control, the Indian Health Service, and the
Substance Abuse and Mental Health Services Administration.
Public Responsibility in Medicine and Research (PRIM&R) - A non-profit organization that
organizes conferences, workshops, and other activities to further the protection of human
subjects in research.
Publications - Published scientific articles or abstracts about a clinical study.
Purity - The relative absence of extraneous matter in a drug or vaccine that may or may not
be harmful to the recipient or deleterious to the product.
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.
Quasi-Experimental Study - A study that is similar to a true experimental study except that it
lacks random assignments of subjects to treatment groups. (See also: Experimental Study.)
Radioactive Drug - Any substance defined as a drug in §201(b)(1) of the Federal Food, Drug
and Cosmetic Act that exhibits spontaneous disintegration of unstable nuclei with the
emission of nuclear particles or photons [21 CFR 310.3(n)]. Included are any nonradioactive
reagent kit or nuclide generator that is intended to be used in the preparation of a radioactive
drug and "radioactive biological products," as defined in 21 CFR 600.3(ee). Drugs such as
carbon-containing compounds or potassium-containing salts containing trace quantities of
naturally occurring radionuclides are not considered radioactive drugs.
Radioactive Drug Research Committee (RDRC) - An institutional committee responsible for
the use of radioactive drugs in human subjects for research purposes. Research involving
human subjects that proposes to use radioactive drugs must meet various FDA requirements,
including limitations on the pharmacological dose and the radiation dose. Furthermore, the
exposure to radiation must be justified by the quality of the study and the importance of the
information it seeks to obtain. The committee is also responsible for continuing review of the
drug use to ensure that the research continues to comply with FDA requirements, including
reporting obligations. The committee must include experts in nuclear medicine and the use of
radioactive drugs, as well as other medical and scientific members [21 CFR 36.1].
Radiopaque Contrast Agents - Materials that stop or attenuate radiation that is passed
through the body, creating an outline on film of the organ(s) being examined. Contrast
agents, sometimes called "dyes," do not contain radioisotopes. When such agents are used,
exposure to radiation results only from the X-ray equipment used in the examination. The
chemical structure of radiopaque contrast agents can produce a variety of adverse reactions,
some of which may be severe — and possibly life-threatening — in certain individuals.
Radiopharmaceuticals - Drugs (compounds or materials) that may be labeled or tagged with
a radioisotope. These materials are largely physiological or sub pharmacological in action,
and, in many cases, function much like materials found in the body. The principal risk
associated with these materials is the consequent radiation exposure to the body or to specific
organ systems when they are injected into the body.
Random Assignment - Assignment of subjects to different treatments, interventions, or
conditions according to chance.
Randomization - Study participants are usually assigned to groups in such a way that each
par-ticipant has an equal chance of being assigned to each treatment (or control) group. Since
randomization ensures that no specific criteria are used to assign any patients to a particular
group, all the groups will be equally comparable.
Randomized Allocation - A strategy in which participants are assigned to arms of a clinical
trial by chance.
Recombinant - DNA Technology "The ability to chop up DNA, the stuff of which genes are
made, and move the pieces, [which] permits the direct examination of the human genome,"
and the identification of the genetic components of a wide variety of disorders [Holtzman
(1989), p. 1]. Recombinant DNA technology is also used to develop diagnostic screens and
tests, as well as drugs and biologics for treating diseases with genetic components. See
Guidebook Chapter 5, Section H, "Human Genetic Research."
Recruitment - The process of enrolling human subjects in research protocols.
Recruitment Period - Time allowed to recruit all subjects for a study.
Recruitment Status - Indicates the current stage of a clinical study and whether it is or will be
open for enrollment.
Registration - The process of submitting and updating summary information about a clinical
study protocol from its beginning to end, to a structured, Web-based registry that is
accessible to the public.
Registry - A structured online system that provides the public with access to summary
information about ongoing and completed clinical studies.
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 man-ages the entire drug
approval process.
REM - Acronym for Roentgen Equivalent in Man; the unit of measurement for a dose of an
ionizing radiation that produces the same biological effect as a unit of absorbed does (1 rad)
of ordinary X-rays. One millirem is equal to 1/1000 of a rem.
Remission - A period in which the signs and symptoms of a disease are diminished or in
abeyance. The term "remission" is used when one cannot say with confidence that the disease
has been cured.
Remuneration - Payment for participation in research. (NOTE: It is wise to confine use of the
term "compensation" to payment or provision of care for research-related injuries.) (Compare:
Compensation.)
Reporting (Or Comparison) Group - A grouping of participants in a clinical study that is used
in summarizing the data collected during the study. This grouping may be the same as or
different from a study arm.
Research - Systematic investigation designed to develop or contribute to generalizable
knowledge. Includes Clinical Research.
Research Team - Investigator, sub investigator and clinical research coordi-nator involved
with study.
Research - Under the Federal Policy and the DHHS Subpart A, research is a systematic
investigation designed to develop or contribute to generalizable knowledge.
Respect for Persons - A principle enunciated in the Belmont Report stating that (1)
individuals should be treated as autonomous agents, and, (2) persons with diminished
autonomy are entitled to protection.
Responsible Party - The sponsor, sponsor-investigator, or sponsor-designated principal
investigator who is responsible for submitting information and updating that information.
Results Database - A structured online system, that provides the public with access to
summary results and registration information for completed or terminated clinical studies.
Retrospective Studies - Research conducted by reviewing records from the past (e.g., birth
and death certificates, medical records, school records, or employment records) or by
obtaining information about past events elicited through interviews or surveys. Case control
studies are an example of this type of research.
Review (of Research) - The concurrent oversight of research on a periodic basis by an IRB. In
addition to the at least annual reviews mandated by the federal regulations, reviews may, if
deemed appropriate, also be conducted on a continuous or periodic basis [Federal Policy
§___.108(e)].
Risk - The probability of harm or injury (physical, psychological, social, or economic)
occurring as a result of participation in a research study. Both the probability and magnitude
of possible harm may vary from minimal to significant. Federal regulations define only
"minimal risk." (See also: Minimal Risk.)
Risk-Benefit Ratio - Risk to individual subject vs. potential benefits. Also called Risk-Benefit
Analysis.
Safety Reports - FDA report required by investigator for any serious and unexpected adverse
experience.
SAMHSA - Substance Abuse and Mental Health Services Administration; includes the Center
for Substance Abuse Prevention, the Center for Substance Abuse Treatment and the Center
on Mental Health Services. Previously the Alcohol, Drug Abuse, and Mental Health
Administration (ADAMHA). (See also: ADAMHA.)
Scientific Review Group - A group of highly regarded experts in a given field, convened by
NIH to advise NIH on the scientific merit of applications for research grants and contracts.
Scientific review groups are also required to review the ethical aspects of proposed
involvement of human subjects. Various kinds of scientific review groups exist, and are
known by different names in different institutes of the NIH (e.g., Study Sections, Initial
Review Groups, Contract Review Committees, or Technical Evaluation Committees).
Secretary - In the context of the federal regulations pertaining to the protection of human
subjects in research, refers to the head of a federal agency.
Serious Adverse Event (SAE) - Any event that results in death, a life threat-ening situation,
hospitalization or prolonged hospitalization, disability, incapacity or a congenital
anomaly/birth defect. Also called serious adverse drug reaction (serious ADR).
Sham Comparator Arm - A group of participants that receives a procedure or device that is
made to be indistinguishable from the actual procedure or device being studied but does not
contain active processes or components.
Significant Risk Device - An investigational medical device that presents a potential for
serious risk to the health, safety, or welfare of the subject.
Single Blind Masking - A type of masking in which one party involved with the clinical trial,
either the investigator or participant, does not know which participants have been assigned
which interventions.
Single Group Design - Describes a clinical trial in which all participants receive the same
intervention.
Single-Masked Design - Typically, a study design in which the investigator, but not the
subject, knows the identity of the treatment assignment. Occasionally the subject, but not the
investigator, knows the assignment. Sometimes called "single-blind design."
Site Visit - A visit by agency officials, representatives, or consultants to the location of a
research activity to assess the adequacy of IRB protection of human subjects or the capability
of personnel to conduct the research.
Social Experimentation - Systematic manipulation of, or experimentation in, social or
economic systems; used in planning public policy.
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 that information is first recorded including original
documents, data and records.
Sponsor (of a Drug Trial) - A person or entity that initiates a clinical investigation of a drug —
usually the drug manufacturer or research institution that developed the drug. The sponsor
does not actually conduct the investigation, but rather distributes the new drug to
investigators and physicians for clinical trials. The drug is administered to subjects under the
immediate direction of an investigator who is not also a sponsor. A clinical investigator may,
however, serve as a sponsor-investigator. The sponsor assumes responsibility for
investigating the new drug, including responsibility for compliance with applicable laws and
regulations. The sponsor, for example, is responsible for obtaining FDA approval to conduct a
trial and for reporting the results of the trial to the FDA.
Sponsor - Individual, company, institution or organization taking responsi-bility for
initiation, management and financing of study.
Sponsor-Investigator - An individual who both initiates and actually conducts, alone or with
others, a clinical investigation. Corporations, agencies, or other institutions do not qualify as
sponsor-investigators.
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 - A determination of the probability of obtaining the particular
distribution of the data on the assumption that the null hypothesis is true. Or, more simply
put, the probability of coming to a false positive conclusion. [See McLarty (1987), p. 2.] If the
probability is less than or equal to a predetermined value (e.g., 0.05 or 0.01), then the null
hypothesis is rejected at that significance level (0.05 or 0.01).
Sterility (1) - The absence of viable contaminating microorganisms; aseptic state.
Sterility (2) - The inability to procreate; the inability to conceive or induce conception.
Sub investigator - Helps design and conduct investigation at a study site.
Surveys - Studies designed to obtain information from human subjects through written
questionnaires, telephone interviews, door-to-door canvassing, or similar procedures.
Suspension - Typically used in the context of a federal agency taking action against an
institution. For example, the Office for Human Research Protections can suspend an
Assurance, preventing the institution from continuing to conduct studies supported with
federal funds.
Telephone Report - Notification via telephone to the FDA of unexpected fatal or life
threatening advent associated with clinical study.
Terminated - The clinical study has stopped recruiting or enrolling participants early and will
not start again. Participants are no longer being examined or treated.
Test Article - Any drug, biological product for human use, medical device for human use,
human food additive, color additive, electronic product subject to FDA regulations.
Therapeutic Intent - The research physician's intent to provide some benefit to improving a
subject's condition (e.g., prolongation of life, shrinkage of tumor, or improved quality of life,
even though cure or dramatic improvement cannot necessarily be effected.) This term is
sometimes associated with Phase 1 drug studies in which potentially toxic drugs are given to
an individual with the hope of inducing some improvement in the patient's condition as well
as assessing the safety and pharmacology of a drug.
Therapy - Treatment intended and expected to alleviate a disease or disorder.
Treatment IND - A method through which the FDA allows seriously BI 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.
Unanticipated Problems Involving Risks to Subjects or Others - This is a regulatory phrase
which requires reporting of this event to the IRB and to the government.
Undue Influence - This refers to a prohibition in the Common Rule that investigators not use
unfair measures or influence to enroll persons in research.
Unexpected Adverse Drug Reaction - A reaction that is not consistent in nature or severity
with study application.
Unexpected Event - Any incident or reaction that puts subjects or others at risk and is not
described in the study plan and/or study application.
Uniform Anatomical Gift Act - Legislation adopted by all 50 States and the District of
Columbia that indicates procedures for donation of all or part of a decedent's body for such
activities as medical education, scientific research, and organ transplantation.
Vaccine - A biologic product generally made from an infectious agent or its components — a
virus, bacterium, or other microorganism — that is killed (inactive) or live-attenuated (active,
although weakened). Vaccines may also be biochemically synthesized or made through
recombinant DNA techniques.
Variable (NOUN) - An element or factor that the research is designed to study, either as an
experimental intervention or a possible outcome (or factor affecting the outcome) of that
intervention.
Viable Infant - When referring to a delivered or expelled fetus, the term "viable infant" means
likely to survive to the point of sustaining life independently, given the benefit of available
medical therapy [45 CFR 46.203(d)]. This judgment is made by a physician. In accordance
with DHHS regulations, the Secretary, HHS, may publish guidelines to assist in the
determination of viability. Such guidelines were published in 1975, and specify an estimated
gestational age of 20 weeks or more and a body weight of 500 grams or more as indices of
fetal viability [Federal Register 40 (August 8, 1975): 33552]. These indices depend on the state
of present technology and may be revised periodically. (See also: Nonviable Fetus.)
Voluntary - Free of coercion, duress, or undue influence. Used in the research context to refer
to a subject's decision to participate (or to continue to participate) in a research activity.
Vulnerable Population - This is a regulatory phrase which refers to a group of people who
have some condition or situation that makes them more susceptible to coercion or undue
influence.
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., stu-dents, subordinates and patients).
Waiver of Informed Consent - An action taken by the IRB permitting the investigator to
pursue research involving human subjects without obtaining informed consent.
Well-being - Subject's physical and mental soundness.
Withdrawal Application - Investigator/sponsor letter to FDA requesting application
withdrawal when no additional work is envisioned.
Withdrawn - The clinical study stopped before enrolling its first participant.