Chapter 11: Clinical Trials Flashcards
What is a clinical trial?
A study of human subjects that is intended to ascertain an investigational product’s safety and efficacy.
What are the seven ethical considerations of clinical trials?
1.) social value.
2.) scientific validity.
3.) fair subject selection.
4.) informed consent.
5.) favorable risk benefit ratio.
6.) independent review board/independent ethics committee (IRB/IRC)
7.) respect for human subjects.
What is Phase I of a clinical trial?
The first experiment in which a drug is tested on the human body.
What is Phase II of a clinical trial?
Examines the safety and effectiveness of the drug in the targeted disease group.
What is Phase III of a clinical trial?
This confirms the efficacy of the drug in a much larger patient group (several hundred to thousands).
What is Phase IV of a clinical trial?
The post marketing approval trials to monitor the efficacy and side effects of the drug in an uncontrolled real-life situation.
Who is the investigator?
The person who conducts the trial.
What is an investigators brochure?
A collection of information prepared and updated by the sponsor for the investigator. Consists of all the data relevant to the drug under investigation.
What is informed consent?
Subjects should be thoroughly educated about the study’s aims, methods, risks, benefits, and alternatives.
What is protocol?
This document set out how a trial is to be conducted.
What is inclusion and exclusion criteria?
These criteria set out the conditions, under which a person may or may not be included in the trial.
What a case report form?
Document or file in which all the information relating to a subject is recorded
What is randomization, placebo-controlled and double blinded?
Not knowing who receives the placebo and who receives the drug. Less biased and the groups are random.
What is monitoring?
All processes are recorded in accordance with GCP and standard operating procedures. Info is recorded in case report forms.
What are adverse events?
Unintended reactions of the subjects as a consequence of taking the drug or placebo.
What are statistics?
The groups or sub groups to be studied. The frequencies, dosages, and the markers to monitor drug advocacy are important factors to consider.
What is the sponsor?
This is the organization or individual that initiates the clinical trial and finances the study.
What is the clinical research organization?
The organization that is contracted by the sponsor to conduct and monitor the trial
What are surrogate markers?
Endpoints or surrogate markers are used to monitor the pharmacodynamics and pharmacokinetics of the drug
What is the abstract?
Provides enough information about a study’s objective, size, and key findings. Like the summary.
What is the introduction?
Provides relevant background information
Answers the question why was the study performed?
What are the methods?
How the study was designed to be conducted
What are the results?
How the study turned out
What is the discussion/conclusions?
Where the findings are explained
Three steps to turn study obstacles into opportunities to sell?
1.) closely examine your clinical study.
2.) anticipate questions.
3.) handle objections
Abstract concept
The starting point for measurement, its best understood as a general idea in linguistic form that helps us describe reality
Association
Immeasurable change in one variable that occurs concurrently with changes in another variable.
Positive association is change in the same direction
Negative association is concurrent changes in opposite directions
Constant
A constant is an attribute of a concept that does not vary
What is construct validity?
Defines how well a test or experiment measures up to it claims
What is content validity?
Extend to which a test adequately represents the subject matter content or behavior to be measured
What is a continuous variable?
This is a measure that can take on any value within a given interval, or set of intervals.
Dependent variable
The value of the dependent variable depends on variations in another variable
Discrete variable
This is a measure that is represented by a limited number of values
Face validity
This evaluates whether the indicator appears to measure the abstract concept
Independent variable
Variable that is manipulated to affect variations or responses in another variable
Interval data
Variables that classify objects, according to type and logical order but also require that differences between levels of a category are equal
Nominal data
Variables that classify objects, according to type or characteristic
Operationalize
To create a measure of an abstract concept
Ordinal data
Variables that classify objects, according to type or kind, but also have some logical order
Predictive validity
This is the ability of an indicator to correctly predict an outcome
Random variable
Any variable with a potential value that is influenced by chance
Ratio data
These variables have a zero starting point and classify objects according to type and logical order, but also require that differences between levels of a category be equal
Reliability
The consistency or repeatability of your measurements
Spurious association
An association between two variables that can be better explained by or depends greatly on a third variable
Statistical inference
The process whereby one reaches a conclusion about a population on the basis of information obtained from a sample drawn from that population
Validity
Extent to which a measurement is an accurate representation of the concepts
Variable
A measure of a concept that can take on more than one value from one observation to the next
Biostatistics
A contraction of biology and statistics
Clinical statistics
Assesses the effects of clinical variables on the diagnosis and treatment of human diseases and disorders.
Epidemiology
The study of how factors influence disease and health in human populations
Bioinformatics
A broad area of research concerned with the analysis of biological data on a molecular level
Clinical trial experiment
Study in which the exposure status is determined by the investigator
Randomized controlled trial (RCT)
A special type of clinical trials, in which assignment to an exposure is determined by sophisticated randomization methods
Longitudinal cohort study
Observational study in which subjects with an exposure of interest and subjects, without the exposure are identified, and then followed forward in time to determine outcomes
Case control study
Observational study that first identifies a group of subjects with a certain disease and a control group without the disease, and then looks to back in time to find exposure to risk factors for the disease.
Good for studying rare diseases
Cross-sectional study
Observational study that is done to examine presence or absence of disease or exposure at a particular “snapshot” in time.
Case report or case series
Descriptive study that reports on a single or series of patients with a certain disease
Bias
Any systematic error in the design or conduct of a study that results in a mistaken estimate of an exposure’s effect on disease risk.
Selection bias
Bias introduced by the way, in which participants are chosen for a study
Confounding
This occurs when an investigator falsely concludes that a particular exposure is casually related to a disease without adjusting for other factors that are known risk factors for the disease and are associated with the exposure
Mean
The sum of observed values divided by the number of observations
Median
The observation that ranks in the middle when all observations are ordered from smallest to largest
Mode
The most frequently occurring value among all observations
Percentile
The percentage of a distribution that is below a specific value
Interquartile range
Refers to the upper and lower boundary defining the middle 50% of observations. Upper is 75th percentile and lower is 25th percentile
Spread (or variability)
Describes the manner in which data are scattered around a specific value
Range
The difference between the largest observation and the smallest
Standard deviation
Measures the spread of data around the mean
Standard error of the mean
Describes the variability of the population mean that is measured in several different samples
Incidence
The number of new events that occur during a specified period of time in a population at risk for developing the events
Incidence rate
The number of new events that occur over the sum of time individuals in the population were at risk for having the event
Prevalence
The number of persons in the population affected by a disease at a specific time, divided by the number of persons in the population at the time
Measures of association
The types of measures used to defined the association between exposes and outcome
Relative risk and cohort studies
The relative risk (or risk ratio) is defined as the ratio of the incidence of disease in the exposed group divided by the corresponding incidence of disease in the unexposed group.
Odds ratio and case-control studies
The odds ratio is defined as the odds of exposure in the group with disease, divided by the odds of exposure in the control group
Absolute risk
The relative risk and odds ratio provide a measure of risk compared with a standard
