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# Chapter 2:

Types of Studies

2: Types of Studies 1
In Chapter 2:

2.1 Surveys
2.2 Comparative Studies

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Types of Studies
Surveys describe population characteristics
Surveys:
2.1
Comparative studies: determine relationships
between variables 2.2

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2.1 Surveys
Goal: describe population characteristics
Normally based on a sample (or subset)
of the population
Types of samples
Probability sample (e.g., simple random
sample)
Non-probability sample (e.g., convenience
sample)

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Sampling
Probability samples use chance
mechanisms to select individuals
The most basic type of probability sample
is the simple random sample (SRS)
SRS a sample of size n selected so that
all possible combinations of n individuals
from the population are equally likely to
comprise the sample

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SRS Method
1. Identify each population member with the
numbers
1, 2, . . ., N
2. Pick a random spot to enter Table A
3. Select n tuples, discarding inappropriate tuples

## Alternatively, use a random number generator

(e.g., www.random.org) to generate n random
numbers between 1 and N

## Keep in mind: the objective of an SRS: every

possible subset is equally likely!
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Other Types of Probability
Stratified random sample: randomly
sample strata (subsets) with the population
Cluster sample: randomly sample
clusters comprising varying numbers of
observations
Multistage sampling: randomly sample
random samples in multiple stages

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Cautions
Undercoverage: groups in the source
population are left out or
underrepresented sampling frame
Volunteer bias: self-selected
participants (volunteers) are atypical
population members
Nonresponse bias: a large percentage
of individuals refuse to participate or
cannot be contacted

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2.2 Comparative Studies
Objective: to determine the relationship
between an explanatory and response
variable
Experimental designs assign subjects
to treatment or exposure groups and
compare outcomes
Nonexperimental design classified
subjects in exposure groups (no
intervention) and compare outcomes
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Study Design Outlines

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Womens Health Initiative (WHI)
Experimental Design
The WHI study randomly assigned about half its
subjects to a group that received hormone
replacement therapy (HRT). The other half
Subjects were followed for ~5 years to ascertain
various health outcomes, including heart attacks,
strokes, breast cancer and so on.
Example of publication: Writing Group for the Women's
Health Initiative Investigators. (2002). Risks and benefits of
estrogen plus progestin in healthy postmenopausal women:
principal results from the Women's Health Initiative
randomized controlled trial. JAMA, 288(3), 321-333.
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Nurses Health Study (NHS)
Nonexperimental Design
This study classified individuals according to
Subjects were followed (ongoing) for various
health outcomes, e.g., heart attacks, strokes,
breast cancer and so on.
Example of publication: Stampfer, M., Colditz, G.,
Willett, W., Manson, J., Rosner, B., Speizer, F., et
al. (1991). Postmenopausal estrogen therapy and
cardiovascular disease. Ten-year follow-up from
the nurses' health study. New England Journal of
Medicine, 325(11), 756-762.
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Comparison of WHI and NHS
Both the WHI and NHS analyzed
relationships between HRT (explanatory
variable) and various health outcomes
(response variables)
WHI investigators assigned the
exposure (HRT) experimental
NHS investigators measured the
exposure but did not assign it
nonexperimental (observational)
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Let us focus on selected
experimental design concepts
and techniques
Experimental designs provides a
designs.

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Terminology Used in Experiments
Subject an individual participating in the
experiment
Factor an explanatory variable being
studied in an experiment
Treatment a specific set of factors

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Subjects, Factors, Treatments
(Illustration)

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Subjects, Factors, Treatments,
Example, cont.
Subjects = 100 individuals who participated in the study
Factor A = Health education (active, passive)
Factor B = Medication (Rx A, Rx B, or placebo)
Treatments = the six specific combinations of factor A
and factor B

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Schematic Outline of Study Design

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Randomized Controlled Trials
(RCT)
Randomized: chance assigns the
exposure
Controlled: the exposed group is
compared to a non-exposed group
(comparative study design)
without knowledge subjects exposure
status
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Randomization
Randomization is the second principle of
experimentation
Randomization refers to the use of chance
mechanisms to assign exposures /
treatments
Randomization balances lurking variables
among comparison groups, mitigating
confounding effects of lurking variables

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Randomization - Example
Source: JAMA 1994;271:595-600 (link is active)
Explanatory variable: Nicotine or placebo patch
60 subjects (30 in each group)
Response variable / outcome: Cessation of
smoking (yes/no)

Group 1 Treatment 1
30 smokers Nicotine Patch

Random Compare
Assignment Cessation
Treatment 2 rates
Group 2
30 smokers Placebo Patch
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Randomization Example
Number subjects 01,,60
Use Table A (or a random number
generator) to select 30 two-tuples
between 01 and 60
If you use Table A, arbitrarily select a different
starting point each time
For example, if we start in line 19, we see
04247 38798 73286
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Randomization, cont.
We identify random two-tuples, e.g., 04, 24, 73, 87,
etc.
Random two-tuples greater than 60 are ignored
The first three individuals in the treatment group are
01, 24, and 29
Keep selecting random two-tuples until you identify
30 unique individuals
The remaining subjects are assigned to the control
group
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Controlled Trial
Controlled means there is non-exposed control
group
A concurrent control group is essential
You cannot judge the effects of an expose without
a concurrent control group because:
Many factors contribute to a health outcome
(so-called multi-causality; causal
interdependence)
Conditions change on their own over time
The placebo (and related) effects
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Blinding
Blinding: the measurement of the response made
without knowledge of treatment type
Works by balancing measurement error and
preventing observer bias
Can occur at several levels:
Single blinding: subjects blinded
Double blinding: subjects and measurement-
takers blinded
Triple blinding: subjects, measurement-takers,
analysts blinded
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Ethics of Experimental Studies
Equipoise - balanced doubt; cant
knowingly expose subjects to harm or
withhold benefit
Informed consent subjects must be
aware of study objectives
Beneficence must provide overall benefit
to society