Interventional Studies

Question 1

4 general differentiating factors to interventional studies?

Answer 1

Purpose
Population studied
Sample size
Duration

Question 2

What do we mean when we say pre clinical?

Answer 2

Research was done in animals

Question 3

Purpose, patient population, sample size and duration of phase 0?

Answer 3

Assess if the drug does what it is supposed to do, aka its MOA.
Healthy, can be diseased
Very small
Very short

Question 4

Purpose, patient population, sample size and duration of phase 1?

Answer 4

Assess safety and tolerance is number one priority
Healthy or disease
Small
short

Question 5

Purpose, patient population, sample size and duration of phase 2?

Answer 5

EFFECTIVENESS, while monitoring safety and tolerance
Disease
Larger
Short to medium

Question 6

Purpose, patient population, sample size and duration of phase 3?

Answer 6

EFFECTIVENESS, while monitoring safety and tolerance
Disease
Larger
Longer
Difference between 2 and 3 is 3 is more people and longer

Question 7

Purpose, patient population, sample size and duration of phase 4?

Answer 7

Post FDA approved and now its in the market, assess long term safety, effectiveness in the indicated patient population
Disease
Larger
Longer

Question 8

What is a simple study?
What is a factorial study?
What is a Parallel study?
What is a Cross Over study?
What is wash out and lead in?

Answer 8

1 randomization of allocation
2 or more randomizations leading to sub groups
Patients stay in their same allocated group the entire study
Patients switch from their allocated group at some point to the other allocated group (go from taking drug a to drug b)
Wash out is the time before switching over to get the first drug out of their system so they are ready for the second drug
Lead in is wash out before the study begins.

Question 9

How do we best describe a pragmatic study and what type of study is it?

Answer 9

Let’s do an interventional study, but lets give the doctors real world flexibility in managing their patients during the study. Drug ain’t working, increase the dose, change the drug, act like a clinician as things happen. Don’t just follow the script of the study.

How do I best manage the disease using this drug.

Question 10

2 types of group allocation procedures?

Answer 10

Random, everyone has an equal chance to get into each group

Non random, investigator picks how to select, time of day, day, first 50 people regardless

Question 11

3 types of randomization?

Answer 11

Simple - everyone has the same opportunity to be in a group
Blocked - this is done to make sure that each group has an equal number of people, so they will say every 20 people we will make sure its equal.
Stratified - same as blocked but they make sure its equal for a certain condition like age or gender or disease severity.

Question 12

Explain placebo effect or Hawthorne effect?

Answer 12

Condition improves because they think they are being treated

Subjects act differently because they are being studied/observed

Question 13

3 key guiding principles of the Belmont report?

Answer 13

Respect for the subjects
Benefit
Justice

Question 14

Difference between consent and assent?

Answer 14

Consent: agreement to participate after being completely informed and having capacity to agree

Assent: same as above but these are people not able to give legal consent

Question 15

Define single blind, double blind, and open label?

Answer 15

Subjects don’t know which group they are in but investigators can know
Subjects and investigators don’t know which groups the subjects are in
Everybody knows

Question 16

What is the purpose of a Run in or lead in?

Answer 16

To assess placebo effect, Hawthorne effect and compliance before the study begins

Question 17

What is the role of the IRB?
Which subjects need to be reviewed before the study begins?
What does a full board review do?
Expedited review?
Exempt review?
What is Equipoise?

Answer 17

Protect human subjects from undue risk
All subjects
All interventional trials and more than low risk
Low risk, no patient identifiers
Chart review or existing data review
Genuine confidence that an intervention/treatment may be worthwhile in older to use it in humans. We think it will provide benefit.

Question 18

What is the role of the DSMB?

Answer 18

They take over for monitoring the study after the IRB approves the study to begin.

Question 19

2 ways to manage drop outs?

Answer 19

Include them with intent to treat: use last known assessment or baseline assessment

Exclude them per protocol: must meet some level of compliance/participation to be included. You are normally measuring efficacy here.

Question 20

2 advantages of intervention studies?

Answer 20

Causation

Only design to get FDA approval

Question 21

Disadvantages of interventional studies?

Answer 21

Cost, complex and time, ethical issues, can you generalize it