Clinical Trial Design

Question 1

What’s the recommended dose for Phase 2 Trial?

Answer 1

5.
The maximum administered dose = 7 (>/= 1 with DLT). The MTD should have

Question 2

What are some characteristics of Phase 0 trial?

Answer 2

• proof of concept, first in species
• find biomarkers
• utilize lower and likely subtherapeutic drug doses
• PK and PD parameters

Question 3

Define pharmacokinetics.

Answer 3

what the body does to the drug
- ex. absorption, bioavailability, distribution, metabolism, excretion, clearance

Question 4

Define pharmacodynamics.

Answer 4

what the drug does to the body.
- side effects, drug interactions

Question 5

Label the following with Type I error, Type II error, True Positive, True Negative, False Positive, False Negative

Answer 5

Question 6

Question 3: In a hypothetical study design, the principle investigator will accept a 10% chance of incorrectly rejecting a null hypothesis when it’s actually true, and a 20% risk of not finding an association when it actually exists. What’s the “power” of this study?

Answer 6

Power = 1-beta, = 0.8

Question 7

What’s the difference between efficacy and effective?

Answer 7

Efficacy = does the drug work?
Effective = does the drug help the targeted population?