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Epidemiology Exam 2 > Interventional Study Designs > Flashcards

Interventional Study Designs Flashcards

1
Q

What is the key difference between interventional study designs and observational?

A

investigators select interventions and allocates study subjects to force intervention groups

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2
Q

Interventional study designs can demonstrate______

A

causation

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3
Q

What is the preclinical phase?

A

prior to human investigation

‘bench’ or animal research

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4
Q

What is Phase 0?

A

exploratory, investigational new drug

  1. assess drug taking actions and possibly pharmacokinetics in single or a few doses
  2. healthy volunteers
  3. very small N <20
  4. very short duration
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5
Q

What is Phase 1?

A

investigational new drug

  1. assess safety/tolerance and pharmacokinetics of one or more dosages
  2. healthy or disease volunteers
  3. small N (eg 20-80)
  4. short duration
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6
Q

What is phase 2?

A
  1. assess effectiveness
  2. diseased volunteers
  3. larger N (100-300)
  4. Short to medium duration
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7
Q

What is phase 3?

A

investigational new drug, indication/population

  1. assess effectiveness
  2. diseased volunteers
  3. larger N (500-3000)
  4. longer duration
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8
Q

What is phase 4?

A

post FDA approval

  1. assess long term safety, effectiveness, optimal use
  2. diseased volunteers
  3. population N (few hundred to few hundred thousand)
  4. wide range of durations
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9
Q

What are the advantages of interventional trials?

A

cause precedes effect (can demonstrate causation)

only designs used by FDA for approval process

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10
Q

What are disadvantages of interventional trials?

A

cost
complexity/time
ethical considerations
generalizability (external validity)

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11
Q

What is a exploratory study?

A

explores the ethics, causes, etc. of a drug in a non clinical setting. Such as if the drug is useful, or what dosage of the drug is needed

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12
Q

What is a pragmatic study?

A

Explains how we treat the diseases part of a clinical study, more flexible (can switch drug that subject is using if needed)

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13
Q

What are the two types of interventional study designs?

A

simple

factorial

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14
Q

What is a simple study design?

A

divides (randomizes) subjects exclusively into greater than or equal to two groups
tests a single hypothesis

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15
Q

What is a factorial study design?

A

Dives (randomizes) subjects into greater than or equal to two groups and then further sub divides (randomizes each of the groups into more sub groups
test multiple hypotheses

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16
Q

What are the benefits of a factorial study design?

A 
improves efficiency for answering clinical questions 
increases study population sample size 
increase complexity 
increases risk of drop outs
may restrict generalizability of results
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17
Q

In what ways can simple or factorial study designs be managed?

A

parallel

cross over

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18
Q

What is parallel study design?

A

groups simultaneously and exclusively managed

no switching of intervention groups after initial randomization

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19
Q

What is cross over design?

A

groups serve as their own control by crossing over from one intervention to another during the study

20
Q

What is the wash out phase?

A

part of cross over design, when you wait until everything is cleared out of a patient before starting second group

21
Q

What is the lead in phase?

A

all study subjects blindly given one or more placebos for initial therapy to ermine a new baseline of diseases
can wash out existing medication
can assess compliance of subjects

22
Q

What are the disadvantages to cross over design?

A

only suitable for long term conditions which are not curable
duration of study for each subject is longer
carry over effects during cross over
treatment by period interaction
smaller N requirement only applicable if within subjects variation less than between subjects variation
complexity in data analysis

23
Q

What are the outcomes/endpoints in interventional study design?

A

primary
secondary/tertiary/ etc.
composite

24
Q

What is a primary outcome?

A

most important, key outcome

main research question used for developing study

25
Q

What is a secondary/tertiary/ etc. outcome?

A

lesser importance yet still valuable

possible for future hypothesis generation

26
Q

What is a composite outcome?

A

combines multiple endpoints into a single outcome

27
Q

What are examples of patient oriented endpoints (POEMs)

A

death
stroke or myocardial infarction
hospitalization
preventing need for dialysis

28
Q

What are examples of disease orientated endpoints (DOEs)?

A

elements used in place of evaluating patient orientated endpoints
blood pressure
cholesterol
change in SCr

29
Q

What are the two types of group allocation?

A

non random

random

30
Q

What is the most common utilized group allocation?

A

random

31
Q

What is the purpose of randomization?

A

to make groups as equal as possible

equality of groups not guaranteed

32
Q

What are examples of forms of randomization?

A

simple
blocked
stratified

33
Q

What is simple randomization?

A

equal probability for allocation within one of the study groups

34
Q

What is blocked randomization?

A

ensures balance within each intervention group

35
Q

What is stratified randomization?

A

ensures balance with known confounding variables

36
Q

What is a single blind masking?

A

study subjects not informed which treatment they are in, but investigators know

37
Q

What is double blind masking?

A

study subjects and investigators dont know who has what treatment

38
Q

What is open label masking?

A

study subjects and researchers known what treatment is being received

39
Q

What is placebo (dummy) therapy?

A

inert treatments made to look identical in all aspects to the active treatment

40
Q

What is double dummy therapy?

A

more than one placebo used

41
Q

What is the placebo effect?

A

improvement in condition by power of suggestion of being treated

42
Q

What is the Hawthorne effect?

A

study subjects change their behavior solely due to awareness of being studied/observed

43
Q

How can you manage drop outs?

A

include them anyway
ignore them
treating them as treated

44
Q

What is the intent(ion) to treat?

A

When including drop outs anyway, most conservative decision

45
Q

What does intent(ion) to treat result in?

A

preserves randomization process
preserves baseline characteristics and group balance at baseline which controls for known and unknown confounders
maintains statistical power

46
Q

What is it called when you ignore drop outs?

A

per protocol or efficacy analysis

compliance is pre defined

47
Q

What happens when you treat drop outs as “as treated”?

A

ignores group assignments

allows subjects to switch groups and be evaluated in group the moved to, end in, or stay in most

Epidemiology Exam 2 (6 decks)

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