General methodological study

Question 1

What are the two types of study design category?

Answer 1

Observational: naturally observing lives of people. Are not researcher-forced group allocations
Interventional: experimentally driven, with a researcher-forced group allocation

Question 2

List the Observational categories.

Answer 2

1. cohort
2. case-control
3. cross-sectional

Question 3

What are the experimental.

Answer 3

1. randomized and non-randomized allocations

Question 4

Equipoise

Answer 4

Genuine confidence may be risk vs benefit in human trials

Question 5

4 principles of Bioethics

Answer 5

1. Autonomy: self-rule and determination
2. beneficence: idea to benefit Pt regardless of their assigned group
3. Justice: equal and fair treatment regardless of characteristics
4. Non-maleficence: do no harm. Can’t withold info, or present incompetence.

Question 6

Assent

Consent

Answer 6

A: agree to participate with full understanding and mental capability. Not of legal age.
C: agree to participate with full understanding and mental capability, and of legal age or by parent/guardian.

Question 7

IRB has what influence?

Answer 7

IRB: will protect the 4 bioethics of of people in the study BEFORE the study begins. and is broken into 3 different levels.

Question 8

What are the 3 different levels of IRB?

Answer 8

1. Full board: used for interventional trials with more than no-risk.
2. Expedited: Minimal risk and no patient identifiers.
3. Exempt: Lowest and easiest to get approval from.

Question 9

After the IRB, who monitors the Pt safety during a study?

Answer 9

The data safety and monitoring board. that assess for safety during the study.

Question 10

What is problematic withing certain studies that can occur?

Answer 10

Possible to have generalization which will not extend to the overall population of the community.

Question 11

Belmont Report contained what?

Answer 11

1. Respect for persons: autonomous subjects
2. Beneficence: risk justified by benefits
3. Justice: equal distribution of risk/benefits

Question 12

What is the key difference of an interventional study compared to an observational study?

Answer 12

1. investigator selects interventions and forced-group interventions
2. readily shows causation

Question 13

Phases of Interventional Study Design…

Answer 13

1. Pre-clinical

2. Phase 1-4

Question 14

Pre-clinical stage of study

Answer 14

This phase is before human trials and demonstrates some type of need for the intervention that is going to be made

Question 15

phase 1

Answer 15

Start human trial tests. Small groups, Sometimes have the infected population.
Focused on safety.
Analyzing pharmacokinetics.
Short time frame

Question 16

Phase 2

Answer 16

Focused on effectiveness and efficacy.
Medium time frame, 100-400 population.
Restricted selection to reduce chance of change by factor other than the drug, or intervention.
If too narrow of restriction; results are not generalized to large populations

Question 17

Phase 3

Answer 17

Longer time frame. More focus on efficacy for the intended target in the intended target group. Analyze the safety in diseased individuals.

Question 18

Phase 4

Answer 18

Safety is large concern.
Views long term effects after FDA approval. Must be named drug at this point.
hundreds of thousands of people involved.

Question 19

Describe advantages of Interventional Study Design

Answer 19

1. The cause precedes the effect and is used for FDA approval process.

Question 20

Why would you not want to perform Interventional study design?

Answer 20

High cost.
Time consuming
Risk vs Benefits (Equipoise) Ethics
Possibility of a too specific study group. Problem?

Question 21

Name the 4 types of interventional study designs

Answer 21

1. simple
2. factorial
3. parallel
4. cross-over (self-control) group

Question 22

Simple interverntional study design

Answer 22

Analyze one hypothesis with 2 or more groups

Question 23

Factorial Interventional study design

Answer 23

A set of groups is further divided into sub-groups. Benefit to analyze a multi-leveled hypothesis. (like a flow-sheet)

Question 24

Parallel Interventional study design

Answer 24

groups are simultaneously and exclusively managed.

no switching of intervention groups after initial randomization

Question 25

Cross-Over

Answer 25

Group serves by controlling intervention compared to another study.

Question 26

Hawthorne Effect

Answer 26

Show compliance before the study begins.
Run-in/Lead-in phase.
study population given 1 or more placebos for therapy to determine “new” baseline of disease