Basic Research Designs

Dr. Tauseef Ismail

Associate Professor
Dept of Public Health KGMC
At the end of the topic, students should be able to:

• Describe the major epidemiological study

designs and their applications,
• Differentiate between the basic design of
descriptive and analytic studies
• Distinguish between observational studies and
experiments;

2
 Core Knowledge
• The evidence for evidence-based medicine is
all collected via research, which uses a variety
of study designs.
• Evidence-based medicine (EBM) is the process
of systematically reviewing, appraising and
using clinical research findings to aid the
delivery of optimum clinical care to patients.

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Different study designs provide information of different
quality. Of course, we always try to use the best
possible design, but sometimes this is not practical or
ethically acceptable (you cannot do an experiment to
expose some people to a harmful substance to see
what effect it has). Therefore, you need to
understand the strengths and limitations of each type
of study design, as applied to a particular research
purpose. The purposes we will consider include
(1) describing the prevalence of health problems;
(2) identifying causes of health problems,
(3)evaluating therapy, including treatment and
prevention.
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 Did investigator assign exposures?

Yes No

Experimental study Observational study

Random allocation? Comparison group?

Yes No Yes No

Randomized Non-randomized Descriptive

Analytical study
controlled trial controlled trial study

Exposure &
Outcome
Exposure to outcome Direction? at same time
Outcome to
exposure
Cohort study Case-control Cross-sectional
study study

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Classification of types of Epidemiological research
 Investigation Sequences

Case Case Descriptive

Reports Series Epidemiology

Analytic
Clinical trials
Epidemiology

Preventive Therapeutic
Case Cross
Cohort
Control Sectional

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 Case Reports
• Detailed presentations of a single case

• Reports a new finding or is uniquely educational

• Initial step in recognizing new disease entity

• Useful in bringing attention to a new area of

concern for further investigation

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 Types of case Reports
• Previously undescribed syndrome or
disease

• Unexpected association between 2 or

more diseases or manifestations

• Unexpected evolution suggesting

surprising new therapeutic effect or
adverse drug effect
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 Case Report -Examples
• Hepatocellular adenoma in young women

• Blindness in newborn infants

• Kaposi’s sarcoma in young men

• Angiosarcoma of liver in plastic industry

workers exposed to Vinyl Chloride
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 Case Reports
Advantages:
• Surveillance for rare clinical events
• Rich source of hypothesis about disease
frequency, risk, prognosis, treatment
• Useful in elucidating mechanisms of
disease and treatment
Disadvantages:
• Susceptible to bias
• Represent isolated events

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 Case series
• Prevalence survey of a group of
individuals with a particular disease or
characteristic, performed at a single point
in time.

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 Example: Case Series
First 1000 cases of AIDS in USA
Complications % Risk Group %
• Pneumocystis 49.7 Homo- 72. 7
carini or bisexual
• Kaposi’s s 28.4 IV drug users
15.5
• PCP and KS 8.3 Haitian natives 5
• Other opport 13.6 Haemophilics
0.7
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• In observational studies, the researcher observes and
systematically collects information, but does not try to
change the people (or animals, or reagents) being
observed.

• In an experiment, by contrast, the researcher intervenes

to change something (e.g., gives some patients a drug)
and then observes what happens.

In an observational study there is NO intervention.

16
Examples of observational studies:

• A survey of drinking habits among students;

• A researcher who joins a biker gang to study their

lifestyle (note, as long as the researcher does not try to
change their behavior, it's an observational study);

• Taking blood samples to measure blood alcohol levels

during Monday morning lectures (yes, you are
intervening to take the blood, but you are not trying to
change the blood alcohol level: it's just a measurement).

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Examples of experiments:

• plying a law student with beer to see whether lawyers

argue better when drunk;

• Prophylaxis with drugs in preventing disease (i.e.,

penicillin to prevent rheumatic fever)

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When do you do an observational study?

• When you merely want to collect descriptive

information: "Is the incidence of diabetes rising?"
• When you want to report on the causes of a problem
without disturbing the natural setting “I want to find
out why students do not attend lectures”
• When you can't do an experiment: "How fast does the
earth move around the sun?"
• When it's not acceptable to do an experiment: "How
much does not wearing a condom increase the
likelihood of HIV infection?"

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 Observational Study Designs
By the end of this lesson the students of 4th year
MBBS should be able to:
• Describe the key characteristics of cross-sectional,
case-control and cohort studies regarding subject
selection, data collection and analysis.
• Identify the design of a particular study.
• Discuss the factors that determine when a
particular design is indicated.
• Discuss the strengths and limitations of each study
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Lots of Observational study designs are there but you
need to know about 3 main ones:

Cross- sectional
surveys

Observational studies

Case-control studies

Cohort studies

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 Defined population

Information on both exposure & disease

Exposed & Exposed, Not Not

have the don’t have exposed, exposed, do
disease the disease have the not have
E+ E+ disease the disease
D+ D- E– E-
D+ D-

Sketch of a cross- sectional study design

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 Features of cross-sectional studies
1. Cross-sectional design is referred to as non-directional or
one point in time survey, where data is collected on both
outcome and exposure status of the individuals under
study (Provide snap shot picture).
2. Exposure & disease assessed simultaneously.
3. Such studies are useful to describe characteristics of study
population and can generate new etiological hypothesis.
4. This study design involves disease prevalence.
5. Cross-sectional studies can evaluate the impact of changes
in health services during an intervening period. This can
be accomplished by conducting a cross-sectional study
twice: before and after the intervention.

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Example:
What is the prevalence of diabetes in this community?
Here, you draw a random sample of people and record
information about their health in a systematic manner.
You can also compare people with, and without, diabetes
in terms of characteristics (such as being overweight) that
may be associated with the disease. The problem is that
you cannot be sure which came first: the diabetes or the
weight problem, so this is a very weak design for drawing
conclusions about causes.

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 Analysis of Cross Sectional Studies

• Distribution of characteristics
• Frequency
• Means
• Proportions
• Prevalence

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Main outcome is Prevalence. Using a 2 x2 table:
Diseas Non-
e diseased
Exposed
a b a+b
Non-
exposed
c d c+d
a+c b+d

Diseas Non- Diseas Non-

e diseased e diseased
Exposed
Exposed
a b a b
Non-
Non-
exposed
c d exposed
c d
a+c b+d a+c b+d
Disease status in exposed Exposure status in diseased
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 Diseas Non- Diseas Non-
e diseased e diseased
Exposed
Exposed
a b a b
Non-
Non-
c d exposed
c d
exposed
a+c b+d a+c b+d
Prevalence of disease Prevalence of exposure
compared in exposed & compared in diseased &
non-exposed non-diseased
a_ Vs. __c_ _a__ Vs. _ b_
a+b c+d a+c b+d

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 Cross-Sectional Study

Disease Status
Yes No Total
Exposed A B A+B
Not Exposed C D C+D
A+C B+D N

Prevalence Ratio (PR) = a/ (a

+ b)

c/ (c +
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d)
 Cross-Sectional Study
Example:

Hypothesis:
• Obesity is a risk
factor for knee
osteoarthritis.

Sample:
• 100 retirees living
at “University
Village”

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 Cross-Sectional Study
Medical exam + X-rays to diagnose
osteoarthritis of the knee

Osteoarthritis
Yes No Total
Yes 40 10 50
Obesity
No 20 30 50
60 40 100
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 Cross-Sectional Study
Prevalence of osteoarthritis among
obese subjects:
40/50 = 0.8

Prevalence of osteoarthritis among non-

obese subjects: 20/50 = 0.4

Prevalence ratio = 0.8/0.4 = 2.0

Obese subjects are 2 times more likely

to have osteoarthritis of the knee than
non-obese subjects.

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Cross-Sectional Study

Chicken or egg dilemma

What came first?

Obesity or Osteoarthritis

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Advantages of cross-sectional studies
1. Easy to carry out & economical to conduct.
2. Describe the distribution of both exposure and outcome in a
population.

3. Provide estimates of the magnitude of a disease problem in a

community, which might be very important for planning of
health services.

4. Compared with other studies are relatively quick and

inexpensive. Often, involve only one-time survey.

5. Largely applicable: provision of health care services as well as

generation of etiological hypothesis.

6. Provides Base line information

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Disadvantages of cross-sectional studies
1. Do not measure risk, because this would require
incidence data.

2. Can not determine cause-effect relationship.

3. Diseases of short duration may be missed. Thus, cross-

sectional studies are best applied to the study of chronic
or persistent conditions.

4. Chicken or egg dilemma

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35
 Case-Control Studies

The case-control study begins with a group of

cases of a specific disease. This (the disease) is
the starting point of the study.
So, the case-control approach is directed at the
prior exposures, which caused the disease and
thus proceeds from effect (outcome) to cause
(exposure).

36
 Case-Control Studies (CCS)
• Question:
– How do diseased (cases) differ from non-diseased
(controls) with respect to prior exposure history?

– Compare frequency of exposure among cases and

controls

– Effect cause.

– Cannot calculate disease incidence rates because the

CCS does not follow a disease free- population over
time

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Schulz KF and Grimes DA 2002. Case-control studies. Lancet 359:431-34. 38
 Selecting cases
• Select cases after the diagnostic criteria and
definition of the disease is clearly established
• Study cases should be representative of all
cases
• Cases may be located from hospitals, clinics,
disease registries, screenings etc

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 Selecting Controls
• Conceptually, controls should come from the
same population at risk of disease from which
cases develop
• But practically, controls are often selected to
be similar to cases on key factors but without
the disease—because it is difficult to define
the population at risk of disease
• Different types of controls may be used, and
they have different limitations

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 Types of Controls
• Hospital controls
− Have similar quality of information and are convenient to select,
but they may have characteristics or diseases that led to
hospitalization
• Dead controls
− If cases are dead, information of past exposures will be given by
surrogates, such as spouse or children. Dead controls share the
same limitation
• Best friend or neighbor controls
− May share similar characteristics (too similar?)
• Population controls
− Random digit dialing (RDD) is often used
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 Three Qualities Needed In Controls

• Comparability is more important than

representativeness in the selection of controls
• The control must be at risk of getting the
disease.
• The control should resemble the case in all
respects except for the presence of disease

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 COMPARABILITY VS. REPRESENTATIVENESS

Usually, study cases are not a random sample of all

cases in the population, and therefore controls must be
selected so as to mirror the same biases that entered
into the selection of cases

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 Case Definition (example)
• Incident(=newly diagnosed case of breast
cancer)
• Between 1/1/92 and 31/12/93
• Resident of Geneva
• Aged < 75 yrs
• Identified: All pathology labs of Geneva

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 Control Definition
• Never diagnosed with breast cancer
• Between 1/1/92 and 31/12/93
• Resident of Geneva
• Aged <75 yrs
• Randomly from Geneva population

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 First, select

Cases (with Controls

disease) (without
disease)
Then, Were exposed a b
measure Were not c d
past exposed
exposure Totals a+c b+d

Proportions exposed: __a__ __b__

a+c b+d

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Analysis:
Case-Control Study: Example
Cases (CHD) Controls
(without CHD)
Smoked cigarettes 112 176
Did not smoke 224
cigarettes 88

totals 200 400

% smoking cigarettes 112 = 56% 176 = 44 %

200 400

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 Odds Ratio
In a CCS we cannot calculate the Relative Risk (RR) directly
to determine whether there is an association between the
exposure & the disease, coz having started with cases &
controls rather than with exposed & non-exposed
persons, we do not hv info about the incidence of disease
in exposed & non-exposed persons.

Therefore, we calculate the OR, which is defined as the

ratio of the odds that the cases were exposed to the odds
that the controls were exposed.

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 The OR as a measure of association

• The only valid measure of association for the CCS

is the Odds Ratio (OR)

OR = Odds of exposure among cases (diseased)

Odds of exposure among controls (non-dis)

• Odds of exposure among cases = a / c

• Odds of exposure among controls = b / d
• Odds ratio = a/c = a.d [ cross-product ratio]
b/d b.c

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 Referring to e.g. of CHD & smoking on slide 29

OR = 112x224/176x88 = 1.6
Interpretation:
1. OR = 1.0 (implies equal odds of exposure - no effect)
2. If the exposure is +vely related to the effect or
disease, the OR will be > 1
3. If the exposure is –vely related to the disease, the
OR will be < 1
OR of 1.6 means???

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 An odds ratio of
• 1.0 (or close to 1.0) indicates that the odds of exposure
among case-patients are the same as, or similar to, the odds
of exposure among controls. The exposure is not associated
with the disease.
• Greater than 1.0 indicates that the odds of exposure among
case-patients are greater than the odds of exposure among
controls. The exposure might be a risk factor for the disease.
• Less than 1.0 indicates that the odds of exposure among
case-patients are lower than the odds of exposure among
controls. The exposure might be a protective factor against
the disease.

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 Strength of the Association
The magnitude of the odds ratio is called the
“strength of the association.” The further away
an odds ratio is from 1.0, the more likely it is
that the relationship between the exposure and
the disease is causal. For example, an odds ratio
of 1.2 is above 1.0, but is not a strong
association. An odds ratio of 10 suggests a
stronger association.

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Can Prevalence be estimated from a case control study?
Example:

Cases (CHD) Controls

(without CHD)
Smoked cigarettes 112 a 176 b
Did not smoke
cigarettes 88 c 224 d

totals 200 400

Prevalence of CHD among the study population= ?

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 CCS - Advantages
• Quick and cheap (relatively)
– so ideal for outbreaks
http://www.cdc.gov/eis/casestudies/casestudies.htm

• Can study rare diseases (or new)

• Can evaluate multiple exposures or wide

range of risk factors

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Case-control Studies - Disadvantages
• uncertain of E D relationship (esp.
timing)
• cannot estimate disease incidence
• worry about representativeness of controls
• inefficient if exposures are rare
• Bias:
– Selection
– Confounding
– Measurement (especially recall bias)

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 Problems in CCS
1. Limitations of Recall: much of the info relating to
exposure often involves collecting data from subjects
by interviews. Because virtually all human beings are
limited to varying degrees in their ability to recall
information, limitations in recall are an important issue
in such studies.
2. BIAS: “Any systematic error in the design, conduct or
analysis of a study that results in a mistaken estimate
of an exposure’s effect on the risk of disease.”

Cont’d
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 Recall Bias
Recall Bias occurs when the recall is better
among cases than controls because of the
presence of the disease – consequently, a
false association may be found between
exposure & disease.

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Recall Bias (e.g.): suppose we are studying the possible
relationship of congenital malformations to prenatal infections.
We conduct a CCS & interview mothers of children with
congenital malformations (Cases) & mothers of children
without malformations (Controls).
Each mother is questioned regarding infections she may have had
during the pregnancy.
A mother who has had a child with a birth defect often tries to
identify some unusual event that occurred during her
pregnancy. She wants to know whether the abnormality was
caused by something she did. Such a mother may even recall an
event, such as a mild respiratory infection, that a mother of a
child without a birth defect may not even notice or may have
forgotten entirely. This type of bias is known as RECALL BIAS.

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Selection Bias: Cases and Controls may not be
representative – (prevented by matching), which is the
process of selecting controls so that they are similar to
the cases with regard to certain key characteristics –
such as age, sex & race.

Interviewer’s bias – when the interviewer knows the

hypothesis and also knows who the cases are (Can be
eliminated by double blinding).

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 Key Facts About Observation Bias
Observation error is an error that arises from systematic differences in the
way information on exposure & disease is obtained from study gps.
This type of bias results in participants who are incorrectly classified as either
exposed or unexposed or as diseased or not diseased.
It occurs after subjects have entered the study

It can occur in both retrospective & prospective studies

Specific types include recall bias, interviewer bias, & differential &
nondifferential misclassification
Methods for avoiding observation bias include masking interviewers and
subjects to the study hypothesis (interviewer & recall); using a control gp that
is composed of diseased individuals (recall bias); carefully designing the study
questionnaire (interviewer & recall); relying on noninterview data
(interviewer & recall); using multiple measurements, the most accurate
information source, & sensitive & specific criteria to define the exposure &
disease (misclassification)
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 Confounding
• Confounding comes from the Latin confundere- (to
mix together)
• Imagine you notice that some of your patients who
drink coffee get heart disease. This is worrying: could
coffee be causing the heart disease?
Beware the issue of confounding.

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Confounding occurs when a factor other than the one you are
studying is associated both with the disease and the factor
you are studying (drinking coffee). This can make it seem as
though the factor you are studying causes the disease even if,
in reality, it does not.
For example, if drinking coffee is associated with smoking
(perhaps people who drink coffee also tend to smoke) the
smoking forms a confounding variable that can make it falsely
appear as though coffee drinking leads to heart disease.
The result is that there appears to be an association between
coffee drinking and heart disease when, in reality, this may be
occurring just because the people who drink coffee also
smoke, and it is their smoking (not the coffee drinking) which
actually causes the heart disease.

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W
Y

X
64
 Confounder
(e.g. smoking )
Risk Factor
Association

Exposure (Coffee Disease

Drinking) Outcome (CVD)

Relationship of Interest
The solution?
You have to do an analysis that statistically adjusts (or
controls) for the smoking. For example, you could
examine the association between coffee drinking and
heart disease at each level of smoking: first, among non-
smokers, is there an association between coffee and
heart disease? Next, among moderate smokers? And
among heavy smokers? If the original association
between coffee and heart disease was due to
confounding by smoking, you would no longer see an
association when you sub-divide people according to
their smoking level.

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A definition... For those who want a more formal
definition:
"In a study of the effect of variable X on disease Y,
a third variable, W, may be a confounder if
(a) it is a risk factor for disease Y, and
(b) it is associated with (but does not cause) variable X."

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 Assignment
What are the key characteristics of a
confounding variable?

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 Consider each of the following scenarios & state
whether the variable in question is a confounder:
A. A study of the relationship between contact lens use &
the risk of eye ulcers. The crude RR is 3 & the age-
adjusted RR is 1.5. Is age a confounder in this study?
YES
B. A case-control study of the relationship between cigarette
smoking & pancreatic cancer. In this study, coffee drinking
is associated with smoking and is a risk factor for
pancreatic cancer among both smokers & nonsmokers. Is
coffee drinking a confounder in this study?
YES

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C. A study of the relationship between exercise & heart
attacks that is conducted among men who do not
smoke. Is gender a confounder in this study?
NO

D. A cohort study of the risk of liver cirrhosis among

female alcoholics. Incidence rates of cirrhosis among
alcoholic women are compared to those among
nonalcoholic women. Nonalcoholics are individually
matched to alcoholics on month & year of birth. Is age
a confounder in this study?
NO

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An association between exposure to welding fumes and
COPD was explored in a case-control study. The
following data were reported for 399 COPD patients: 37
currently employed as welders; the remainder had no
occupational exposure. Among 800 controls. 48 were
employed as welders. Set up a 2x2 table, calculate the
OR and interpret it.
COHORT STUDIES

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 Cohort Studies
• Cohort studies begin with a group of people (a
cohort) free of disease.
• The people in the cohort are grouped by
whether or not they are exposed to a
potential cause of disease.
• The whole cohort is followed over time to see
if the development of new cases of the
disease (or other outcome) differs between
the groups with and without exposure.
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Example-1: you could do a cohort study if you suspect there
might be a causal relationship between the use of a certain
water source and the incidence of diarrhea among children
under five in a village with different water sources.
You select a group of children under five years, either all
children of that age in the village, a random sample taken from
the population register, or e.g. children living in the same area,
or attending the same clinic. Then you classify them as either
using the suspected water source or other water sources. You
check e.g. after two weeks whether the children have had
diarrhea.
You can then calculate the incidence of diarrhea among those
children using the suspected water source and those using
other sources of water supply

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Example-2: You want to see whether using a cell phone leads to
brain cancer.
1. Draw a sample of people (medical students, for e.g.) who do
not have the disease you are interested in, and collect
information on the factor you have hypothesized to be a cause
of the disease.
2. Collect information on how many minutes each student uses
their phone each week (you might get permission to obtain
this from their phone company bills), and collect this
information over a long time, and then eventually collect
information on who gets brain cancer. You could then see
whether the cases of brain cancer arose among the people
who used their cell phones most often.
3. In technical terms, you record the incidence of cancer among
those who use their phones more than a pre-determined
amount and compare this to the incidence in the non-users.
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1. Type of Analytic study
2. Unit of observation and analysis: Individual (not groups)
3. Also called follow-up studies, incidence studies,
longitudinal studies, or prospective studies

Some other terms need clarification:

• Fixed cohort
• Closed cohort Assignment
• Open population

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Steps in designing cohort studies
1. Identify group of exposed subjects
2. Identify group of non-exposed subjects
3. Follow-up both groups for disease
4. Measure frequency of disease occurrence in both
groups.
5. Compare risk between exposed and non-exposed
group

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 Cohort study design

Diseased
Exposed
Not diseased
Target
Disease free
Population Cohort Diseased

Unexposed
Not diseased

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Framingham Study

• Select the cohort (all residents of Framingham)

• All the members are given questionnaire/clinical exam
to determine exposure status
• Exposures included: smoking, alcohol use, obesity,
diabetes, Elevated BP, Elevated Cholesterol levels etc
• The cohort is divided based on results
• The non exposed become controls
• Examined after every 2 years
• Outcome (CHD)—already defined and confirmed

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Cohort study: Calculate
Then, follow to see whether & compare
Disease Disease Incidence
First, develops does not of Disease
develop Totals
identify
Exposed a b a+b a/a + b
Not exposed c d c+d c/c + d

Incidence in exposed = a/a + b

Incidence in not exposed = c/c + d

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Analysis: Calculate
Then, follow to see whether & compare
Disease Disease Incidence
First, develops does not of Disease
develop Totals
identify
Exposed 84 2916 3000 84/3000
Not exposed 87 4913 5000 87/5000

Incidence in smokers = 84/3000 = 0.028

Incidence in not exposed = 87/5000 = 0.0174

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 Relative Risk
= Risk among exposed = a / a + b = 0.028 = 1.61
Risk among non-exposed c /c + d 0.0174
Interpretation:
• If RR is more than 1 exposure is causative
• If less than 1 exposure is protective.
• If equal to 1 means that the two incidence rates are equal
so the factor has no effect.
RELATIVE RISK is the ratio of the risk of disease (or death)
among people who are exposed to the risk factor, to the risk
among people who are unexposed.

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 Attributable risk
• the amount of disease incidence that can be
attributed to a specific exposure
– Difference in incidence of disease between exposed
and non-exposed individuals
= a/a+b – c/c+d = 0.028 – 0.0174 = 0.0106
• In general, all individuals, whether they have or have not
been exposed to a risk factor, have some chance of
developing a disease if no prevention measures have been
taken. The AR estimates the risk above and beyond this
baseline risk that all people have.
• AR can be used to indicate the health benefit of removing
the exposure.
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 Attributable Risk
Excess Risk

Risk
100

80 Risk among risk

AR = factor positives
60

40
Risk among risk
20 factor negatives
0
+ -
Risk Factor
85
 Advantages of cohort studies
• Measure incidence and thus permit direct
estimation of risk of disease.
• Do not rely on memory for information about
exposure status, hence avoid bias due to selective
recall.
• Cohort studies provide a logical approach to studies
of causation or effects of treatment.
• Cohort approach can yield information on
associations of exposure with several diseases
• Efficient for studying rare exposures
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 Disadvantages of cohort studies
• Require large samples to yield the same number of cases
that could be studied more efficiently in a case-control
study.
• Particularly inefficient for studies of rare diseases.
• Logistically difficult – long follow-up period, often serious
attrition to study subjects.
• Direct observation of participants may cause changes in
health behavior.
• Possible bias in ascertainment of disease due to changes
over time in criteria and methods.
• Very costly.
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 Types of Cohort studies
• Prospective Cohort study
– Concurrent Cohort study or Longitudinal study
• Retrospective Cohort study
– Non-concurrent Cohort or historical Cohort study
• Ambidirectional Cohort study
– To look both ways

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Differentiating b/w Prospective & Retrospective

Prospective cohort study

− Investigator
 Starts the study (from the beginning) with the
identification of the population and the exposure
status (exposed/not exposed groups)
 Follows them (over time) for the development of
disease
 Takes a relatively long time to complete the study
(as long as the length of the study)
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Differentiating b/w Prospective & Retrospective

 Retrospective cohort study

− Investigator
 Uses existing data collected in the past to identify the population
and the exposure status (exposed/not exposed groups)
 Determines at present the (development) status of disease
− Investigator spends a relatively short time to:
 Assemble study population (and the exposed/not exposed
groups) from past data
 Determine disease status at the present time (no future follow-
up)
Cont’d

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91
 Timing of Cohort Studies
Prospective Cohort Study
* Exposure *
* Disease *
Study starts

Retrospective Cohort Study

* Exposure *
* Disease *

Study starts
Ambidirectional Cohort Study
* Exposure *
* Disease *

Study Starts
Time 92
What are the factors that influence the
decision of the researcher to carry out a
particular type of Cohort study?

• Your research question

• Practical constraints such as time &
money and the
• Availability of suitable study populations
and records

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Hierarchy of Study Designs

Validity ranking Types of study

design
Highest
Experimental study

Controlled exp/clinical trial

Prospective cohort study

Retrospective cohort study

Nested case-control study

Time-series analysis
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Points Regarding Study
Designs
 Do not confuse scientific accuracy
with clinical relevance

 Well done cohort and case-control

studies can be much more valuable
than irrelevant clinical trials

 The question being asked (usually)

determines the appropriate research
strategy.

95
 Study Designs
M R
• Observational No No
• Quasi-experimental Yes No
• Experimental Yes Yes

– Manipulation of Study Factor

– Randomization of Study Subjects

96
Spotting The Study Design – General
Rules
When were the outcomes determined?
• Some time after the exposure?
– Yes… cohort study (‘prospective study’)
• At the same time as the exposure or
intervention?
– Yes… cross sectional study or survey
• Before the exposure was determined?
– Yes… case-control study (‘retrospective
study’)

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 Case-Control Studies:
Question: What Happened?

Direction of inquiry is backward: from outcome to 98

 Cohort Studies: Question:
What Will Happen?

Direction of inquiry is forward: from risk factor to 99

RCT: Question: What Are The
Effects Of This Intervention?

100
 Did investigator assign exposures?

Yes No

Experimental study Observational study

Random allocation? Comparison group?

Yes No Yes No

Randomized Non-randomized Descriptive

Analytical study
controlled trial controlled trial study

Exposure &
Outcome
Exposure to outcome Direction? at same time
Outcome to
exposure
Cohort study Case-control Cross-sectional
study study

101
Classification of types of Epidemiological research
What do Cohort Studies Tell You:

1. Theuse of cohorts is often mandatory because a randomized control

study may be unethical. For example, you cannot deliberately expose
people to asbestos, you can only study its effects on those who have
already been exposed. Research that measures risk factors often relies
upon cohort designs.
2. Because cohort studies measure potential causes before the outcome
has occurred, they can demonstrate that these “causes” preceded the
outcome, thereby avoiding the debate as to which is the cause and
which is the effect.

3. Cohort analysis is highly flexible and can provide insight into

effects over time, and related to a variety of different types of changes
[e.g., social, cultural, political, economic, etc.

4. Either original data or secondary data can be used in this design .

102
 When Is Odds Ratio a Good Estimate of
Relative Risk?
• When the “cases” studied are representative of all
people with the disease in the population from which
the cases were drawn, with regards to history of the
exposure
• When the “controls” studied are representative of all
people without the disease in the population from
which the cases were drawn, with regards to history
of exposure
• When the disease being studied is not a frequent one

103
Comparison of Cohort & Case-Control study designs

104
Retrospective cohort studies are characterized by all of
the following except:

A. The study groups are exposed and unexposed

B. Incidence rates may be computed
C. The required sample size is smaller than that
needed for a prospective cohort study
D. The required sample size is similar to that
needed for a prospective cohort study
E. They are useful for rare exposures

105
In cohort studies of the role of a suspected factor in the
etiology of a disease, it is essential that:
A.There be equal numbers of persons in both study groups
B. At the beginning of the study, those with the disease and
those without the disease have equal risks of having the
factor
C. The study group with the factor and the study group
without the factor be representative of the general
population
D. The exposed and unexposed groups under study be as
similar as possible with regard to possible confounding
factors
E. Both B and C
D
106
 References & preparing for exams

1. http://www.med.uottawa.ca/sim/data/Study_Designs_e.htm
2. http://www.snz.unizg.hr/ph-see/full/u3-t2-roshi-burazeri.pd
f
3. Gordis L. Epidemiology. 5th Edition. Canada: Elsevier
Saunders; 2014.
4. Aschengrau A, George R, Seage III. Essentials of Epidemiology
in Public Health. 2nd Edition. USA: Jones and Bartlett
Publishers; 2008.
5. Park K. Preventive and Social Medicine. 20th Edition.
India: M/s Banarsidas Bhanot Publishers; 2009.
6. http://ocw.jhsph.edu/courses/FundEpiII/PDFs/Lectur
e13.pdf
107