15 General Methodological Concepts of Research

Question 1

What does a research evidence pyramid show?

Answer 1

The increasing strength of evidence for various study designs.

Question 2

Which study design is described by the following?

Systemic review of all published literature of topic up to a specific point in time.

Answer 2

Systemic Review

Question 3

What is entailed with a meta-analysis?

Answer 3

It takes all studies and put them together so that it appears that there was one large, new study and build a consensus from the meta-analysis.

Question 4

Describe case reports and case series.

Answer 4

Case reports and case series are just reports of one or more individuals that have had an unique experience; usually hypothesis generated.

Question 5

List the criteria study design selection is based on.

Answer 5

The right study design comes from the type of question you are trying to answer.
Perspective of research question (hypothesis)
Ability/desire to force group allocation (randomization)
Ethics of methodology
Efficiency & practicality (time/resource commitment)
Costs
Validity of acquired information (internal & external)
• Internal validity = is the study design and the methods used valid?
• External validity = how well can I take the findings from the study and externalize that information to patients that are similar to the study?

Question 6

Define Null Hypothesis

Answer 6

A research perspective which states there will be no (true) difference between the groups being compared

Question 7

Define Alternate Hypothesis

Answer 7

A research perspective which states there will be a (true) difference between the groups being compared

Question 8

What are the possible statistical-perspectives that can be taken by a researcher?

Answer 8

Superiority
Noninferiority
Equivalency

Question 9

Define type I error and give an example.

Answer 9

False positive; Telling a man he is pregnant

Question 10

Define type II error and give an example.

Answer 10

False negative; Telling a pregnant woman she isn’t pregnant

Question 11

What is an observational study?

Answer 11

Study designs considered “natural”
Researchers “observe” subject elements occurring naturally or selected by individual (naturally or freely)
• Useful for unethical study design using forced interventions
Most observational study designs are not able to prove causation
• There is NO researcher-forced group allocation

Question 12

What is an interventional study?

Answer 12

Study designs are considered “experimental”
Investigator-selected interventions (exposure)
• There IS researcher-forced group allocation
Randomization process

Question 13

Define population.

Answer 13

All individuals making up a common group; from which a sample (smaller set) can be obtained, if desired
• Not to be confused with the “study population”, which is simply the final group of individuals selected for a study

Question 14

Define sample.

Answer 14

A subset or portion of the full, complete population (representatives)
• Useful when studying the complete population is not feasible
• Random processes commonly utilized to draw sample

Question 15

What is the study population selection based on?

Answer 15

Research Hypothesis/Question
Inclusion & Exclusion selection criteria
Ethics
Equipoise
Agreeing to Participate in Interventional Studies
Ethical conduct of research
Methodology of Study

Question 16

What is inclusions & exclusion criteria?

Answer 16

• Desired vs. Logical vs. Plausible selection criteria
• These absolutely impact generalizability
• Control group needs to be identical to the case group in every way except for the presence of disease

Question 17

List and describe the 4 key principles of bioethics.

Answer 17

Autonomy
• Self-rule/self-determination. Participants must…
o Have full and complete understanding of the risks and benefits
No misinformation, incomplete information, or ineffectively-conveyed information (language or education level)
o Decide for ones-self, without outside influences
No coercion, reprisal, financial manipulation
Beneficence
• To benefit, or do good for, the patient (not society)
Justice
• Equal and fair treatment regardless of patient characteristics
Nonmaleficence
• Do no harm. Researchers must not…
o Withhold information
o Provide false information
o Exhibit professional incompetence

Question 18

What is the Belmont Report?

Answer 18

A document created in 1978 by the National Commission for Protection of Human Subjects of Biomedical and Behavioral Research, that decides whether or not research conducted is ethical.

Question 19

What are the 3 guiding principles of the Belmont Report?

Answer 19

Respect for persons
• Research should be voluntary, subjects autonomous
Beneficence
• Research risks are justified by potential benefits
Justice
• Risks and benefits of the research are equally distributed

Question 20

What is equipoise?

Answer 20

• Genuine confidence that an intervention may be worthwhile (risk vs. benefit) in order to use it in humans

Question 21

Compare and contrast consent versus assent.

Answer 21

Consent
o Agreement to participate, based on being fully and completely informed
 Given by mentally-capable individuals of legal consenting age (i.e., adults; age 18 in most state)
Assent
o Agreement to participate, based on being fully and completely informed, given by mentally-capable individuals not able to give legal consent (i.e., children and adolescents)
 Children or adults not capable of giving consent requires the consent of the parent or legal guardian and the assent of the potential study subject

Question 22

True or False, all human subject studies MUST be reviewed by an IRB prior to study initiation.

Answer 22

True

Question 23

True or False, observational and interventional studies must be reviewed by an IRB prior to study initiation.

Answer 23

True, observational and interventional studies use human subjects.

Question 24

What are the levels of an IRB?

Answer 24

Full Board
• Used for ALL interventional trials with more than minimal/no risk to patients
o All medication-related studies
Expedited
• Minimal risk and/or no patient identifiers
Exempt
• No patient identifiers, low/no risk, de-identified dataset analysis, environmental studies, use of existing data/specimens (de-identified)

Question 25

What is the Data Safety and Monitoring Board (DSMB)?

Answer 25

o Semi-independent committee not involved with the conduct of the study but charged with reviewing study data as study progresses, to assess for undue risk or benefit
- Pre-determined review periods (interim analyses)
- Can stop study early, for either overly-positive or overly-negative findings
o Gets involved after research has been study

Question 26

What are the two types of outcomes?

Answer 26

o Patient-oriented best

o Individual vs. Combined

Question 27

Compare internal validity versus external validity.

Answer 27

Internal validity
o Assessments (measurements)
- Scientifically-rigorous and standardized
• Objective better than Subjective assessments
- Valid, accurate and reproducible
External validity
o Generalizability