Exam 1 (First Two Handouts) Flashcards by Cassie Kellogg

The degree of confidence a researcher has that the changes observed in the dependent variable (clinical outcome) are because of the independent variable (treatment)

Internal Validity

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The extent to which the results of a study can be generalized to other settings

External Validity

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The outcome is a result of the treatment

Causality

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Cause occurs before the outcome

Temporality

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Plausibility of causation increases with the strength of the relationship

Strength

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A linear relationship like a dose-response is observed

Biological Gradient

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One-to-one relationship between cause and effect

Specificity

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There should be a reasonable biological explanation/mechanism for the effect

Plausibility

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The relationship should be explained by existing knowledge

Coherence

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The effect is similar to those accepted phenomena as observed in preclinical trials

Analogy

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It is the strongest evidence when the cause is controlled by the researcher and the effect is observed in randomly assigned subjects as in RCTs

Experiment

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To describe a phenomenon

Descriptive

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Provide casual interpretation of an existing phenomenon

Analytical

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What are the five important elements (5 W’s) pertaining to a new disease or event:

-Who
-What
-Why
-When
-Where
-So What (later added as a 6th element)

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Analytical studies typically involve this word (hint)

Causal

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Prospective Studies

-data are collected after the study as individuals are followed
-researchers get to determine the variables and collect relevant data
-they are resource (time and cost) intensive

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Retrospective Studies

-researchers go backward in time to determine the relationship between cause and outcome that has already occurred
-analyze existing data so they relatively require minimal resources
-researchers have no control over the variables needed and how they are defined and collected

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What three things play an important role in choosing an appropriate research design?

-objective of research
-topic of research
-practical considerations

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Experimental designs to test their research hypotheses are common in what type of research?

Basic

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We see both experimental and observational designs in what type of research?

Clinical Research

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What is the most commonly used experimental design?

RCTs

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What are the commonly used observational designs are..

-cohort
-case-control
-cross-sectional
-case series
-case reports

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What is the gold standard in evaluating the safety and efficacy of an intervention?

Randomized Controlled Trials

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What are the TWO essential elements of RCTS?

-randomization
-prospective

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However the elements of RCTs that increase the internal validity of study results also contribute towards restricting...

their external validity (generalizability)

The key element in observational studies is..

non-randomization of the independent variable of interest

Primary data technique examples

-interviews -surveys

Secondary data technique examples

-medical charts -medical claims

Involve a study of a single case of a new disease or manifestation

Case Report

Exposure and outcome are measured at the same point in time

Cross-sectional

Involve comparison of exposure status among individuals with the disease or outcome of interest (cases) and those without the disease or outcome (controls)

Case-control

Cases and controls are both identified from the same..

source population

Follow exposed and unexposed over a period until the development of outcome

Cohort

Cohort studies determine the incidence of the outcome among exposed and unexposed groups and therefore provide a measure of..

relative risk

There is no randomization involved in Cohort studies which makes it vulnerable to..

selection bias

Cohort studies can be divided into two types:

retrospective and prospective

Involve numerical or countable information to study research phenomenon

Quantitative Data

Involve words or textual information

Qualitative Data

The consistency and reproducibility of results

Reliability

Consistent when the same measurement instrument is used by multiple investigators

Reproducibility

Measures what is it intended

Validity

Participant is aware that he/she is being observed

Obtrusive Observation

Participant is unaware of the observer who may be either hidden or under disguise

Unobtrusive Observation

Strongest type of primary study design and most common type of trial

RCTs

RCTs are required by the FDA as part of a...

New Drug Application (NDA)

Phase 3 studies are also referred to as..

"pivotal trials"

The degree to which the findings represent the truth

Validity

How can you strengthen internal validity?

include a control group

What is the most effective way to reduce selection bias?

Randomization

Subjects modify their behavior because of the fact that they are being studied or observed

Hawthorne Effect

The external events that occur during the course of the study

History Bias

Normal changes in study participants over time

Maturation Bias

Refers to differential dropout of patients from the treatment or control groups

Attrition Bias

The mean refers to cases where initial measurements of a variable are extremely different from the population mean, but then subsequent measurements are closer to the average

Regression (or reversion)

Systematic differences between groups in how outcomes are determined

Detection Bias

Random Number Generator

Simple Randomization

To ensure that treatment groups have an equal number of patients

Block Randomization

Ensuring certain baseline characteristics are equal between the groups of a study

Stratified Randomization

A specific group of subjects are selected for randomization such as those enrolled in a clinic or hospital

Cluster (Group) Randomization

Those involved in the trial are unaware of what treatment the patients are receiving

Blinding (masking)

-Least object type of design -All individuals in the study are aware of what treatment the patients are receiving -Restricted to early pharmacokinetic studies or phase 1 trials

Open Label

Only one set of individuals is unaware

Single Blind

Two sets of individuals are unaware

Double Blind

All individuals involved in the study are unaware of what the patients were receiving (including investigators)

Triple Blind

More than one placebo is used

Double Dummy

Measures the capacity to detect a difference in the study groups if a true difference exists

Power

Standardized document that provides instructions to the investigators on all aspects of carrying out the study

Clinical Research Protocol

Specific characteristics that the investigator is most interested in studying

Inclusion Criteria

Factors that would confound or impair the ability not interpret the study results or eliminate patients that should not be receiving the intervention or an active control

Exclusion Criteria

Each subject is only randomized to either to a treatment group or a control group

Parallel Study Design

Receives all of the interventions based on a specified sequence of events

Crossover

Each subject receives all of the interventions based on a specified sequence of events

Crossover

What is the most common type of RCT and what is its purpose?

-Superiority trial -Which aims to determine whether or not one treatment is better than another

Aims to determine whether or not two interventions are equivalent or nearly equivalent based on a set of acceptance criteria

Noninferiority Trial

Systematic study to gain knowledge or understanding necessary to determine the means by which a recognized and specific need may be met

Applied Research

Refers to the collection of information based on human experience. All aspects of science should be observed and measured to be considered as scientific evidence

Empiricism

Means that there is no subjectivity or bias in any aspect of research including definition, measurement, design, and analysis

Objectivity

Provides an understanding or explanation of a natural phenomenon. In pharmaceutical research, theories are often based on pathophysiology of a disease and pharmacology of a medication to investigate medication effects

Theory

Provide the moral societal standards for responsible research conduct

Ethics

What is the framework often used to develop a good clinical question?

PICOTS

What does PICOT stand for

P- Population to be studied I- Intervention to be applied C- Comparator to be used O- Outcomes to be evaluated T- Timeline to evaluate the outcomes and healthcare S- Setting of interest

Specifies an expected relationship between intervention and outcome, or two or more variables.

Hypothesis

involve testing a drug in humans with the intent of establishing the initial toxicity profile of the substance ("tolerability")

Phase 1 Trials

Are designed to provide preliminary evidence of efficacy and safety

Phase 2 Trials

-Generate efficacy and safety data to allow evaluation of the overall risk to benefit relationship of the drug -Are usually set up as a Randomized Controlled Trial (RCT) often involving blinding and placebo controls.

Phase 3 Trials

Studies may be required by FDA to generate longerterm safety/toxicity data.

Phase 4 Trials or Post-Marketing

Is intended to allow patients to obtain investigational medications outside of clinical trials or current expanded access programs

Right-To-Try Act

-Individuals should be treated as autonomous agents -Persons with diminished autonomy are entitled to protection -The informed consent process ensures the respect to person

Respect for Persons

-Maximizing possible benefits. -Minimizing possible harm. -Never knowingly cause (permanent) injury

Beneficence

-Sense of fairness in distributing the burdens and benefits of research. -Researchers are obligated to ensure that no group inappropriately bears the burdens of research for others' benefit.

Justice

What are the three basic principles of the Belmont Report?

-Respect for persons -Beneficence -Justice

National Commission for Protection of Human Subjects of Biomedical and Behavioral Research, which was set up by the National Research Act, published Ethical Principles and Guidelines for the Protection of Human Subjects of Research known as the

Belmont Report

There are three types of IRB review:

-Exemption *Surveys/interviews, use of existing data, or specimens without identifiers -Expedited review by the chair or designee *minimal risk protocols that do not meet one of the exemptions -Full review by the whole board *all protocols involving greater than minimal risk

Charged with protecting the rights and welfare of human subjects of research and ensuring that research is conducted in accordance with accepted ethical standards.

Institutional Review Board

Used to estimate the effect of an exposure on a disease outcome (measures of effect such as causal RRs and causal rate ratios, respectively).

Risk ratios (RRs) and rate ratios

The most basic form of medical evidence that often provides the first suspicion that an issue exists

Case Report

Case reports only serve to provide a description of a situation and is not...

intended to lead to a conclusion or answer a hypothesis

The removal of most medications from the market due to adverse reactions are due to...

case report evidence

Report adverse drug reactions to...

MedWatch

Why are adverse drug reactions in special populations not frequently identified?

these patients are often excluded from research

Due to case reports describing novel occurrences, it has a high _______

sensitivity; however it has a chance of reporting false results and therefore a lower level of specificity

How are sensitivity and specificity related?

Inversely

Case Series

Descriptive