Index
A
Accidental bias.
SeeBias,
accidental
Adaptive design.
SeeDesign, adaptive
Adaptive randomization.
SeeRandomization, adaptive
Adherence
effects on adverse events
definition
design factors
discussing with participants
maintaining
monitoring
planning
protocol adherence
run-in
withdrawal
Adverse effects/adverse events
analysis
ascertainment
assessment and reporting
avoiding
classification
CONSORT statement
data collection
definition
design issues
effects on adherence
factorial designs
genetic subgroups
identification
meta-analysis
monitoring
missing data
post-marketing
predicted by baseline measures
regulatory
reporting
serious
unexpected
withdrawal studies
Alpha spending functions.
SeeSequential testing
Alternative hypothesis.
SeeHypotheses
Analysis
intention-to-treat
on-treatment
per protocol
trend adaptive designs
Ancillary
Ascertainment
adverse events
bias
health related quality of life
low adherence effects on
outcomes
post-study
Audits
B
Baseline data
comparability
definition
measurement
testing for imbalance
uses
Bayesian
data monitoring
methods
Belmont Report.
SeeEthics
Bias
accidental
allocation
ascertainment
assessment
estimation
experimental
interviewer
investigator
publication
selection
Biased coin randomization.
SeeRandomization, adaptive
Biomarkers
genetic
outcomes
personalized medicine
run-in period
screening for
subgroup
surrogate outcomes
Blinding
assessment
double-blind design
double-dummy
open trial
protecting
reporting
single-blind design
triple-blind design
unblinded (open) design
unblinding
Blindness
Blocked randomization.
SeeRandomization, blocked
Bonferroni.
SeeMultiple testing
C
Case-control studies.
SeeDesign
Censoring.
See
also Under
survival
bias
comparing survival curves
definition
information loss
informative
Kaplan-Meier
nonadherence
sequential methods
Classical sequential monitoring.
SeeSequential testing
Clinical equipoise.
SeeEquipoise
Close-out
transfer of care
visit
Common Rule.
SeeEthics
Comparison of survival curves.
SeeSurvival
Compensatory treatment
Competing event issues
Compliance.
SeeAdherence
Concomitant treatment
Concurrent control studies.
SeeDesign
Conditional power.
SeeSequential testing
Conflict of interest
CONSORT statement
Continuous response.
SeeResponse variables
Control group
concurrent, non-randomized
historical
placebo
randomized
Covariate adjustment
using baseline data
using surrogates
Covariate-intervention interaction
Cross-over.
SeeDesign
Cutler-Ederer.
SeeSurvival, Cutler-Ederer
D
Data analysis
Conflicts of interest
Issues
Presentation
Randomization process
Data and safety monitoring board.
SeeData
monitoring committee
Databases
dissemination
electronic health
finalization
historical controls
Data collection
data entry
enhancing quality
essential data
forms
See(Forms)
guidelines
problems
verification
Data, missing.
SeeMissing data
Data monitoring committee
decision process
meetings
membership
Data sharing
See
alsoDissemination
Declaration of Helsinki.
SeeEthics
Design
adaptive
response adaptive
sample size
trend adaptive
case-control studies
concurrent control studies
cross-over
double-blind
factorial
hybrid
large simple trials
Dichotomous response.
SeeResponse
Dissemination
Dose-escalation studies
Double-blind design.
SeeDesign
Drop-in
Drop-out
Drugs
coding
matching
quality control
E
Effectiveness trial
Efficacy trial
Equipoise
clinical
Equivalence study
Ethics
Belmont Report
clinical equipoise
common rule
conflict of interest
data monitoring
Declaration of Helsinki
developing countries
emergency settings
financial incentives
guidelines
informed consent
Nuremberg Code
privacy and confidentiality
publication bias
surrogate consent
Ethics review committee
Exclusion
from analysis
from follow-up
ineligibility
missing data
nonadherence
Expected number of events
Exponential distribution
See
alsoSurvival
Extension of follow-up
F
Factorial design.
SeeDesign, factorial
Falsification of data
Fixed allocation
Follow-up, poststudy
Forms
development
monitoring
pretesting
G
Ghost authorship
Greenwood’s variance formula
Group allocation
Group sequential
alpha spending functions
asymmetric boundaries
Haybittle–Peto
Lan-DeMets alpha spending functions
methods for monitoring
monitoring applications
O’Brien–Fleming
Pocock
Guest authorship
H
Haybittle–Peto group sequential
method.
SeeGroup
sequential, Haybittle–Peto
Health-related quality of life
administration
clinical significance
components
definition
design
interpretation
methodology
outcomes
pretesting
primary dimension
selection of instruments
uses
utility measures
Historical control studies
Hochberg procedure for multiple
testing.
SeeMultiple testing
Holm procedure for multiple testing.
SeeMultiple testing
Hybrid design.
SeeDesign, hybrid
Hypotheses
alternative
null
one-sided vs. two-sided
I
Imbalance
baseline
covariate adjustment
randomization
safety events
subgroups
Imputation.
SeeMissing data
Industry modified NIH clinical trials
model
Informative censoring
Informed consent
changes
closeout
ethics
genetics
privacy and confidentiality
quality monitoring
regulatory issues
study population
Institutional Review Board (IRB)
Intention-to-treat (ITT)
Interim events
IRB.
SeeInstitutional Review Board
(IRB)
IVRS, IWRS.
SeeRandomization
K
Kaplan-Meier
See
also Under
survival
L
Lan-DeMets alpha spending functions.
SeeGroup
sequential, Lan-DeMets alpha spending functions
Large simple trials.
SeeDesign, Large simple trials
Last observation carried forward.
SeeMissing data, Last observation
carried forward
Levels of significance
M
Mantel Haenszel statistic
survival analysis
two by two tables
Manual of procedures
Margin of indifference
Margin of noninferiority
Masking.
SeeBlinding
Maximally tolerated dose
Measurement error
Medications.
SeeDrugs
Meta-analysis
issues
methods
Minimization allocation
Missing data
imputation
last observation carried forward
missing at random (MAR)
missing completely at random (MCAR)
Monitoring
Committee
interim
procedures
quality
response variables
sequential
Multicenter trials
conduct
globalization
organization
reasons for
recruitment
Multiple testing
Bonferroni
Hochberg
Holm
N
Natural history
NIH clinical trials model
Nonadherence
analysis
impact on sample size
Noninferiority
analysis
assay sensitivity
constancy assumption
control group
efficacy imputation
interpretation
margin of indifference
margins
Null hypothesis
Nuremberg Code.
SeeEthics
O
O’Brien–Fleming.
SeeGroup
sequential, O'Brien–Fleming
Observational studies
One-sided vs. two-sided hypothesis.
See
Hypotheses, one-sided vs.
two-sided
Open trial.
SeeBlinding
P
Per protocol analysis.
SeeAnalysis, Per protocol
Pharmacogenetics
Phases of trials
early phase
late phase
phase I
phase II
phase III
phase IV
Pilot study
Placebo control
Play-the-winner
Pocock group sequential method.
SeeGroup
sequential
Pocock–Simon randomization.
SeeRandomization
Population
definition
eligibility criteria
external validity
generalization
heterogeneity
homogeneity
large, simple trials
recruitment
representativeness
selection
Power.
SeeSample size
Pragmatic trial
Privacy
Protocol
adherence
SeeAdherence
adverse events prespecified
baseline factors prespecified
closeout
contents
defining study population
guidelines and registries
health related quality of life
modification
multicenter trial
Per protocol
SeeAnalysis, per protocol
pilot
regulatory
subgroups prespecified
violation
Publication bias
Q
Quality control
certification
monitoring
Quality of life.
SeeHealth-related quality of life
Question
adverse event
ancillary
natural history
primary
secondary
R
Randomization
adaptive
baseline adaptive
biased coin (adaptive?)
blocked
fixed allocation
group allocation
IVRS, IWRS
mechanics
Pocock–Simon method
response adaptive
simple
two-armed bandit
urn design
Randomized control trials
Recruitment
approaches to lagging recruitment
conduct
contingency plan
ethics
logs
monitoring
planning
problems
reasons for participating
recycling subjects
selection of study sample
sources
Registration of trials
Regression toward the mean
Repeated confidence intervals.
SeeSequential testing
Repeated testing for significance
See
alsoMultiple testing
Reporting results
analysis
guidelines
interpretation
publication bias
Response adaptive randomization.
SeeRandomization, response
adaptive
Response variables
assessment
combined (composite)
continuous
dichotomous
discrete
monitoring
surrogate
Results, dissemination
S
Safety
advantages
analysis
Committee
SeeMonitoring committee
early termination
eligibility
ethical issues
hampered by blinding
importance to primary question
meta-analysis
monitoring
regulatory considerations
reporting
Sample size
cluster designs
continuous outcome for repeated
measures
continuous outcome for two independent
samples
continuous outcome paired data
dichotomous outcome for paired data
dichotomous outcome for two independent
samples
multiple outcomes
nonadherence adjustment
noninferiority designs
parameter estimation
time to failure outcome
Screening
effect
eligibility
prescreening
recruitment
visit
Selection bias.
SeeBias,
selection
Sequential testing
alpha spending functions
classical
conditional power
repeated confidence intervals
statistical methods
Simple randomization.
SeeRandomization, simple
Single-blind design.
SeeBlinding
Statistical methods for data
monitoring.
SeeSequential testing, statistical
methods
Statistical power
Storage of study material
Stratification
of analysis
of randomization
Subgroup
analysis
definition
Superiority trials
Surrogate consent
Surrogate response variable
Survival
analysis methods
comparison of survival curves
Cox Proportional Hazards model
Mantel-Haenszel
medians
point by point
weighted rank tests
Wilcoxon–Gehan
curve estimation
Cutler-Ederer
exponential distribution
Greenwood’s variance formula
informative censoring
Kaplan-Meier
time to failure, time to event
T
Termination of trial
examples
planning
procedures
Therapeutic misconception
Time to failure response
See
alsoSurvival
Training
ethics
health related quality of life
interviewer
multicenter trial
pretesting
quality assurance
Triple-blind design.
SeeBlindness
Two-armed bandit randomization.
SeeRandomization
Type I error
Type II error
U
Unblinded design.
SeeBlinding
Unblinding,
ancillary studies
closeout
drug coding
emergency
inadvertent
monitoring committee
official
purposeful
reporting
serious adverse events
Urn design randomization.
SeeRandomization
V
Variability
cluster
estimates
measurement
quality control
reduction
sample size parameter
unequal allocation
W
Withdrawal of subjects
See
alsoExclusion
Withdrawal studies