Midterm

Question 1

What is a clinical trial?

Answer 1

• Prospective study comparing the effect and value of intervention(s) against a control in human beings.
• Intervention, prospective, experiment, comparison group.
• A prospective study comparing the effects and value of intervention(s) against a control in human beings.

Question 2

What is the vision and mission of a clinical trial?

Answer 2

Decrease Bias. Increase Precision. Unbiased inference with the best precision

Question 3

What is bias?

Answer 3

Systematic errors that occur in the design, conduct, analysis and/or reporting of a research study.

Question 4

Ways to decrease bias

Answer 4

–Rigorous study design
–Appropriate study population
–Appropriate comparison group
–Appropriate trial conduct
–Appropriate analytic methods

Question 5

Ways to decrease bias

Answer 5

–Rigorous study design
–Appropriate study population
–Appropriate comparison group
–Appropriate trial conduct
–Appropriate analytic methods

Question 6

Ways to decrease bias

Answer 6

–Rigorous study design
–Appropriate study population
–Appropriate comparison group
–Appropriate trial conduct
–Appropriate analytic methods

Question 7

Strength of Study

Answer 7

Clinical Trial
Cohort Study
Case-control study
Cross-sectional study
Case series

Question 8

Strength of Evidence

Answer 8

Temporality
Strength
Reversibility
Dose-response
Consistency
Biologic Plausibility
Analogy

Question 9

Cohort Study

Answer 9

Study where one ore more samples (cohorts) are followed prospectively and subsequent status evaluations with respect to a disease or outcome are conducted which initial participants exposure characteristics (risk factors) are associated with it.
•Advantages
•Subjects in cohorts can be matched (to reduce influence of confounding variables)
•Standardization of criteria/outcome is possible
•Easier and cheaper than a randomized controlled trial
•Disadvantages
•May be hard to identify cohorts due to confounding variables
•No randomization(imbalances in unmeasured patient characteristics could exist)
•Blinding/masking is difficult
•Outcome of interest could take a long time to occur

Question 10

Case-Control Study

Answer 10

Study that compares patients who have a disease or outcome of interest (cases) with patients who do not (controls), and looks back retrospectively to compare how frequently the exposure to a risk factor is present in each group to determine the relationship between the risk factor and the disease
•Advantages
•Good for studying rare conditions or diseases
•Less time needed to conduct the study because the condition or disease has already occurred.
•Useful as initial studies to establish an association
•When not ethical to randomize, can be useful
•Disadvantages
•Retrospective studies have more problems with data quality because they rely on memory and people with a condition will be more motivated to recall risk factors (recall bias)
•Finding a suitable control group can be challenging (to avoid confounding)

Question 11

Phase 1 Overview

Answer 11

• Primary concern: Safety
• Goal: Estimate tolerability
• Population: Small sample of healthy individuals
• Process: Determine the Max Targeted Dose for Dose Limiting Toxicity

Question 12

Phase 1 Overview

Answer 12

• Primary concern: Safety
• Goal: Estimate tolerability
• Population: Small sample of healthy individuals
• Process: Determine the Max Targeted Dose for Dose Limiting Toxicity