Interventional Studies

Question 1

Pre-Clinical

Answer 1

Non-human subjects (tissue/animals)

Question 2

Phase 0

Answer 2

“Exploratory or Investigational”
Healthy and/or diseased
Very small sample
Short duration (single dose or few days)

Question 3

Phase 1

Answer 3

Assess safety/tolerance and Pharmacokinetics
Healthy or diseased patients
20-80 subjects
Few weeks duration

Question 4

Phase 2

Answer 4

Assess effectiveness
Diseased Only
100-300
Few weeks to months duration

Question 5

Phase 3

Answer 5

Final Pre-FDA stage
Disease Volunteers
100-3000 participants
Time Frame: few months to years
Introduction of statistical perspectives

Question 6

Phase 4

Answer 6

Diseased Volunteers
Assess long term safety, effectiveness and optimal use
Duration: Few weeks, several years. “Ongoing”

Question 7

What are the advantages of Interventional trials?

Answer 7

Demonstrates causation

Only design used for FDA approval

Question 8

What are the disadvantages of Interventional trials?

Answer 8

Cost
Complexity/time
Ethical considerations
Generalizability or external validity

Question 9

Explain the difference between exploratory and explanatory studies?

Answer 9

Exploratory is the default, pre-set and non-clinical study design looking at effectiveness, safety and toxicity.

Pragmatic studies are more flexible and lifelike that explains qualities but can allow confounders and loss of precision of control.

Question 10

All studies can be _____ or _____ .

Answer 10

Simple or factorial

Question 11

Further, all studies are ____ or ____.

Answer 11

cross-over and parallel

Question 12

Unlike Pragmatic trials, Simple tries use a ____ hypothesis at a time.

Answer 12

Single

Question 13

What are the effects of using a factorial study design? (5)

Answer 13

Improves efficiency for answering clinical questions
Increases study population sample size
Increases complexity
Increases risk of drop outs
May restrict generalizability of results

Question 14

After randomization, ____ does no allow switching vs _____ does allow switching.

Answer 14

parallel

cross-over

Question 15

What key words can help us determine if a study is interventional? Use of ____ and _____ when describing tiers of study

Answer 15

Randomization and Phases

Question 16

What is the difference between wash-out and lead-in?

Answer 16

Wash-out is a period of time when no treatment is given so that the treatment before cross over can clean the system to remove chance of effect contamination. Lead-in is the same situation but occurs before a study.

Question 17

What is the benefit of lead-in?

Answer 17

Test patient’s compliance by practicing study protocol with study subjects
Also removes drugs that could interact with drug treatment in clinical trial.
Can be accomplished by given placebo for “standardization”

Question 18

What are the disadvantages of cross-over design?

Answer 18

Only for LT conditions that are not curable or for ST relief
Duration of study is longer including washout
Data analysis complexity

Question 19

What are the three forms of randomization?

Answer 19

Simple, Blocked and Stratified

Question 20

What is blocked randomization?

Answer 20

A type of modern randomization that guarantees relative equality. When using blocks, towards the end of the block we as researchers will analyze the groups and move around remaining #s in the group that have not been assigned in groups to allow balance.

Question 21

____ is used to assess adequacy of blinding.

Answer 21

Post-hoc survey’s

Question 22

What are ways that placebo therapy is used to look similar?

Answer 22

In terms of dosage form, frequency, therapy requirements and monitoring

Question 23

Double Dummy is when ____

Answer 23

More than 1 placebo is used