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Epidemiology > [2] Class 16-19:Interventional studies > Flashcards

[2] Class 16-19:Interventional studies Flashcards

1
Q

What form of study allows for causation?

A

Interventional study

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2
Q

Type of study in which Researchers intervene and force ppl into groups allocating Tx

A

Interventional study

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3
Q

Which process commonly utilized to accomplish forced allocation in interventional studies?

A

Randomization

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4
Q

Which study is considered natural?

A

Observational

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5
Q

Type of study where researchers observe subject elements occurring that’re selected by the individual

A

Observational

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6
Q

What study type is not able to prove causation:

A

Observational

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7
Q

Form of researcher forced allocation in observational studies

A

There is no researcher forced group allocation

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8
Q

Human study,clinical trial/study,experimental, investigational study generally means

A

interventional study

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9
Q

Name the key difference in Interventional vs. observational studies:

A

Investigator selects “interventions” and allocates study subjects to intervention groups

It is more rigorous in ability to show cause-and-effect: IT CAN PROVE

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10
Q

Phases of interventional studies:

A

Pre-clinical-Phase 0-phase 4

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11
Q

Phase of the study at the lab ‘bench’ or animal investigation

A

Pre-clinical

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12
Q

Exploratory investigational new drug

A

Phase 0

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13
Q

3 factors to consider when looking at phases of interventional studies:

A

Sample size,
Length of study,
Purpose-of phase

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14
Q

Very small

A

Phase 0

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15
Q

Pharmacokinetics:

A

ADME

Absorption
Distribution
Metabolism
Excretion

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16
Q

Primary purpose is kinetics of the drug:

A

Phase 0

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17
Q

small 20-80 N- short duration-few weeks,
Healthy or diseased volunteers
Possible first time use in ppl,

A

Phase 1

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18
Q

Primary purpose: side effects

Secondary: does it actually do what it was set out to do [lower BP/BG]

A

Phase 1

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19
Q

Investigational New drug

A

Phase 1

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20
Q

Does safety remain a purpose after it is the primary purpose in phase 1?

A

Yes, it just is not be the primary purpose

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21
Q

Investigational new drug;indication/population

A

Phase 2

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22
Q

Medium N-100-300 and medium duration-few weeks to few months;
Commonly utilize patients w/ Dz of interest…begins to assess efficacy in diseased

A

Phase 2

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23
Q

Randomization think->

A

Interventional

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24
Q

First phase that begins to look at efficacy:

A

Phase 2

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25
Q

Which phase is likely to have narrow inclusion criteria?

A

Phase 2

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26
Q 
Large N 500-3,000
Longer duration:months-yrs
Ppl have dz of interest
Assessing for short-intermediate safety 
Broader inclusion criteria-more clinically relevant
A

Phase 3

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27
Q

Primary purpose is to assess for efficacy:

A

Phase 3

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28
Q

The concern w/ more restrictions on exclusions:

A

More restrictions = more specific and less generalizability

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29
Q

Post-marketing post-FDA approval phase:

A

Phase 4

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30
Q

What phase is req’d for FDA approval:

A

Phase 3

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31
Q

Larger N -1000s
Long term effects
Dz population
CAN be interventional or observational design

A

Phase 4

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32
Q

Is phase 0-4 sequential and req’d?

A

No can go in any order but phase 3 must be approved by FDA

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33
Q

FDAs medwatch is an example of what phase :

A

Phase 4

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34
Q

Which study is perfect?

A

NONE

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35
Q

Advantages of interventional trials:

A

Can prove causation

Only design that can be used for FDA approval

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36
Q

Disadvantages of Interventional trial:

A

Cost
Complexity/time
Ethical considerations
Generalizability-External valid.

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37
Q

What is the number one disadvantage to interventional trials?

A

Ethical considerations are the # 1 reasons why they’re not completed

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38
Q

Loosens inclusion criteria/ controlling of intervention

More real life/more variable

Allows for others to be doctor of study

A

Pragmatic interventional study

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39
Q

An interventional study w/ a single randomization process and no more subsequently

A

Simple interventional study

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40
Q

Interventional study with 2 or more groups that are randomized into 2 or more sub-groups

A

Factorial interventional study

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41
Q

Ex of factorial study

A

Fish oil vs. placebo->

Fish oil and niacin vs. placebo and niacin.

42
Q

Types of factorial:

A

2x2; 2x2x2

43
Q

Can factorial designs be used to test multiple hypotheses/questions at the same time

A

Yes

44
Q

BEnefits of factorial design

A
  • Improved efficiency for answering clinical questions

- req larger study population

45
Q

Disadvantages to factorial interventional study

A

Increased complexity-barrier for recruitment

Increased risk of drop out[d/t complexity]

May restrict generalizability of results

46
Q

Interventional study where groups simultaneously and exclusively managed

No switching of intervention groups after initial randomization

A

Parallel interventional study

47
Q

All simple and factorial designs can be

A

Parallel

48
Q

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

A

Cross-over

49
Q

This study type allows for a smaller total n population -sample size

A

Cross-over study

50
Q

Study that self-controls for cofounders b/c same individual is receiving both Tx / interventions

A

Cross-over

51
Q

This time period is when the person is cleansing drug/Tx from body prior to receiving other drug/Tx

A

Wash-out period

52
Q

Cross-over study is typically for what type of disease/condition?

A

Chronic disease- long standing b/c it it wouldn’t be helpful to cure then taker another Tx regime.

53
Q

This can be used to assess placebo-effects, hawthorne-effects and compliance before the study begins:

A

Run-in; lead-in phase

54
Q

All study subjects blindly given placebo(s) for initial therapy [defined time period] to determine a “new” base-line of disease [satndardization]

Test run= complaisance assessment

Washout period p/t study or can wash-out existing Rx

A

Run-in / lead-in phase

55
Q

Disadvantages for cross-over study designs:

A
  • Only suitable for long-term conditions- not curable [short term relief]
  • duration of study for each subject is longer
  • carryover effect during washout
  • Tx by time period interaction
  • complexity in data analysis
  • smaller N req’ment applies only sometimes
56
Q

Most important key outcome

It is the main research question/hypothesis for conducting study

A

Primary outcomes

57
Q

Lesser importance yet still valuable outcome

Possible for future hypothesis generation

A

Secondary/tertiary/etc. outcome

58
Q

Combines multiple endpoints into a single outcome

Can be considered primary outcome and secondary outcomes can be individual outcome elements

A

Composite outcome

59
Q

Ex of POEMs (patient oriented endpoints

A

Death, stroke, MI, hospitalization, preventing need for dialysis

60
Q

Ex of DOEs Disease oriented endpoints

A

Risk for stroke
Risk for MI
Worsening in renal function

61
Q

Sample selection in which subjects don’t have equal probability of being selected or assigned to each intervention group

“First couple of people that happen to walk by”

A

Non-random sample selection

62
Q

Sample selection in which Subjects do have an equal probability of being assigned to each intervention group

Created by random number generators

A

Random sample selection(most commonly utilized

63
Q

Random sampling hopefully removes:

A

Bias

64
Q

What is the purpose to randomization:

A

To make groups as equal as possible

65
Q

Attempts to reduce systematic diffferences [bias] between groups that could impact results

Based on known and unknown factors [confounders]

A

Randomization

66
Q

W/ randomization is equality guaranteed?

A

NO not guaranteed

67
Q

Which table typically shows group characteristics such as possible cofounders and p values:

A

Table 1

68
Q

Table 1

A

Documentation of equality of groups [effectiveness of randomization process] reported in 1 of several locales : p values

69
Q

3 types of randomization

A

Simple
Blocked
Stratified

70
Q

Equal probability for allocation within one of the study groups

Basic randomization- hopeful

A

Simple randomization

71
Q

Ensures balance within each intervention groups

When researchers want to assure that all groups are equal in size

More hands on approach for researcher-when study req’s exact #’s

A

Blocked randomization

72
Q

Ensures balance w/ known confounding variables

Ex: age, Dz, gender, comorbidities

Can also pre-select levels to be balanced w/in each interfering factor [cofounder]

More hands on approach for researchers

A

Stratified randomization

73
Q

3 types of masking:

A

Single-blind
Double-blind
Open-label

74
Q

Masking in which study subjects aren’t informed which intervention theyre receiving [but clinician/researchers know]

A

Single-blind masking

75
Q

Masking in which neither investigators nor study subjects are informed which intervention each subject is receiving

A

Double-blind masking

76
Q

Masking in which everyone knows which intervention each subject is receiving

A

Open-label

77
Q

How would you test a IM shot vs. PO tablet

A

Make each group receives both types of intervention, no matter which route is true Rx to prevent bias

78
Q

What can be used to asses adequacy of blinding?

A

Post hoc surveys

79
Q

Inert Tx made to look identical in all aspects to active Tx

A

Placebo / dummy therapy

80
Q

What is it called if more than 1 placebo / dummy therapy is used

A

Double-dummy

81
Q

Improvement in conditions by power of suggestion and due to the care provided

Can be as large as 30-50%

A

Placebo effect

82
Q

Desire of the study subject to “please” investigators by reporting positive results [improvement], regardless of Tx allocation

Desire for positive outcomes

A

Hawthorne effect

83
Q

Hawthorne effect can make up part of the rate of:

A

Placebo effect

84
Q

Define post hoc subgroups analysis

A

A stepwise multiple comparisons procedure used to identify sample means statistically different from each other

85
Q

When is post-hoc sub-group analysis accepted?

A

It is accepted as appropriate by most when it is prospectively planned for hypothesis generation

86
Q

When is post-hoc sub-group analysis not accepted?

A

Not accepted as appropriate when its not prospectively planed

87
Q

When post-hoc sub-group analysis is not accepted, what is the term used to define how the stat was found?

A

‘Fishing’ or ‘data-dredging’

88
Q

Why is fishing or data dredging not a good thing? 2 reasons

A

Reduces Power

And

Increases risk of type 2 error

89
Q

What should be included in the sample size determination?

A

Add in for anticipated drop-outs/ lost to F/u

90
Q

3 ways to manage drop-out/lost to f/u rates:

A

Intention to treat

Per-protocol or efficacy-analysis

As treated

91
Q

Which way of managing drop-outs is most conservative?

A

Intent(ion)-to-treat

92
Q

2 procedures for intent to treat:

A

Last known asssessment used for all subsequent, yet MISSED assessments

Convert subsequent missed assessments for a subject to null-effect (no benefit/change)

93
Q

Do authors tell you how they manage drop-out?

A

Yes, authors must disclose the form of drop-out management they will use

94
Q

Results of intent to treat [3]:

A
  • Preserves randomly selected process
  • preserves baseline characteristics and group balance at baseline which controls for known and unknown confounders
  • maintains statistical power
95
Q

Technique of ignoring drop-outs

Including only compliant subjects

A

Per-protocol or efficacy analysis

96
Q

What is the customarily set rate of compliance w/ per protocol or efficacy analysis?

A

80-90%

97
Q

Technique where drop-outs of study are treated

A

‘As-treated’

98
Q

Technique for managing drop-outs that allows ignoring group assignments-allows subjects to switch groups and be evaluated in group they moved to, end in, or stay in most

A

‘As-treated’

99
Q

Per-protocol dropout management technique results in:

A

Biases estimates of effect (commonly over-estimates effects)

Reduces generalizability

100
Q

Ways to assess compliance/adherence

A

Drug levels
Pill counts
Pill bottle counters [lids]

101
Q

Why is adherence used more than compliance now?

A

Compliance has a negative connotation to it by being forceful.
Adherence allows pt in decision making process…kind of

102
Q

Methods to improve adherence:

A

Frequent f/u visits [communication]

Tx alarms/ notifications

Rx blister packs or dosage containers

Epidemiology (12 decks)

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