1

DESCRIPTIVE STUDY DESIGNS

The basic design strategies in epidemiologic

research are categorized into two according to
their focus of investigation. Descriptive studies
focus on the distribution of disease and analytic
studies focus in elucidating the determinants of
disease.

2
3
Purpose of descriptive studies

Some of the important features described below shows

the commonness and usefulness of descriptive studies in
improving health services and promoting health
research. Descriptive studies:

⎯ are mainly concerned with the distribution of diseases

with respect to time, place and person.

⎯ provide useful information for health managers to

allocate resource and to plan effective prevention
programmes. 4
⎯ generate epidemiological hypothesis, an important first
step in the search for disease determinants or risk
factors.

⎯ can use information collected routinely which are

readily available in many places. So generally
descriptive studies are less expensive and less time-
consuming than analytic studies.

⎯ are the most common type of epidemiological design

strategies in medical literature.
5
There are three main types of descriptive tudies, which are
discussed in detail below:

• Correlational/ecological

• Case report or case series

• Cross-sectional

6
Correlational or Ecological
 Uses data from entire population to compare disease
frequencies - between different groups during the same
period of time, or in the same population at different
points in time.
 Does not provide individual data, rather presents
average exposure level in the community.
 Cause could not be ascertained.

7
• Correlation coefficient (r) is the measure of association
in Correlational studies. It is important to note that
positive association does not necessarily imply a valid
statistical association.

8
Strength: can be done quickly and inexpensively, often
using available data.

Limitation:
 Inability to link exposure with disease. Data on exposure
and outcome are not linked at the individual level;
association found with aggregate data may not apply to
individuals (this is referred as ecological fallacy).
 Lack of ability to control for effects of potential
confounding factors.
9
 It may mask a non-linear relationship between
exposure and disease.

For example alcohol consumption and mortality

from CHD have a non-linear relationship (the
curve is "J" shaped), but this type of relationship
is impossible to demonstrate in Correlational
studies.

10
Case Report and Case Series

Describes the experience of a single or a group of patients with

similar diagnosis or health problem derived from either the
practice of one or more health care professionals or a
defined health care setting such as hospital, health centre or
specialized clinic. The following information/data are
important inputs in making better use of the case series
study:

1. Defining the disease or health problem clearly

2. Recording the date when the disease/death occurred (Time)

11
3.Recording where the person lived, worked,… (Place,
information relevant to the study with regard to place)

4. Recording personal characteristics of the person such as

age and sex (Person)

5. Explore the opportunities for collecting additional data

from records or the person directly.

6. Estimating the size and the characteristics of the

population at risk.

12
Strength:
 useful for studying signs and symptoms and creating case
definitions for epidemiological studies
 case-series that include cases at various stages of an illness
from mild cases to dead supplemented by investigation of
the past medical history of these cases and observing
them to death (doing autopsy as appropriate) can help
build up a picture of the natural history of a disease.
 very useful in providing critical information, for hypothesis
generation, for sound analytical studies. 13
Limitations:
 Report is based on single or few patients, which could
happen just by coincidence.
 Lack of an appropriate comparison group.

 Rates can not be calculated since the population

corresponding to the source of cases can not be defined well.
 Detailed and complete risk factor information is difficult to
obtain for all cases from records.
 Studies are prone to atomistic fallacy (the opposite of
ecological fallacy)
14
Cross Sectional Studies (Survey)
Cross sectional study investigate disease and risk factor
(exposure) patterns in a representative sample of a
population in a narrowly defined time period. An ideal
cross-sectional study is done on a geographically
defined population. It can be useful to identify
associations, generate and test hypothesis and, by
repeating at different time periods, measure change and
hence evaluate interventions.

15
Strength:
 Easy to conduct

 Not time consuming

 Can be used to compare population with

different characteristics as in comparative cross
sectional studies

Limitation:
 "chicken or egg" dilemma

 Survivor bias 16
 ANALYTICAL EPIDEMIOLOGY

Purpose of Analytic Studies

1. To search for cause and effect.
Why?? How??

2. To test hypothesis about causal relationship

Proof Vs Sufficient evidence

3. To quantify the association between exposure and

outcome
Measure of association
17
Broadly analytic studies classified into two:
– Observational and

– Interventional (Experimental)

Observational vs. Experimental analytic studies

Both types use “controls”. The use of controls is the

main distinguishing feature of analytic studies.

Observational :-Information is obtained by simple

observation of the event. Two basic types:
18
A. Case control studies :-
 Cases (subjects having a specific disease) and
controls (subjects not having the disease) are
compared for their exposure status.
 Assess retrospectively on exposure status

 Relatively cheaper, (Time and Cost)

 Measure of association is using Odds ratio

19
 Subjects are selected with respect to presence or
absence of disease, or outcome of interest, and
then inquiries are made about past exposure to
the factor(s) of interest.

E.g. Take people with and without TB, ask them if

they ever had BCG vaccination.

- those with disease - CASES

- those without disease - CONTROLS 20

 This design strategy was developed in response to the
difficulty of studying diseases with very long latency
period
 Can evaluate the association of a disease to exposure
many years after the actual exposure.
 Allows examination of multiple risk factors(exposures)
for a single outcome(disease).
 It is also efficient in time and cost so now become the
most common analytic design seen in medical literature.
21
22
23
Selection of cases:
Setting clear definition of cases

 Choosing between Incident and Prevalent cases

 Representing spectrum of disease: mild, moderate

and severe groups

24
25
Selection of controls
Considerations:
 Avoiding selection bias
 Avoiding information (‘recall’) bias

26
Special controls
Special controls are individuals which are related to the cases
in some way. These are friends, household members
(siblings,...), neighbours,...

27
Advantages:
 they are healthy.
 more likely to be cooperative than members of the
general population, because of their interest in the cases.
 offer a degree of control over some confounding factors,
such as ethnicity, socioeconomic status, or environment.
Disadvantage:
 if the study factor is likely to be similar to the cases, an
underestimate of the true effect of the exposure of
interest may result.
E.g. if the study factor is diet, it will be similar for both
cases and controls, if controls are siblings.

28
 B. Cohort studies :-
Definition: An epidemiologic design that identifies
comparison groups according to their exposure status.
‘A cohort’ Vs ‘Cohort study’

• A ‘cohort’ is simply a group of persons with

common characteristics who are followed or
traced over a period of time.

• ‘Cohort design’ is an analytical epidemiologic

study that compares exposed and non-exposed
groups.
29
Healthy subjects are classified on the basis of their
specific exposure status then are followed for a
specified time to determine the development of
disease.
 There is usually a follow up.

 Relatively expensive (time, cost).

 Measure of association is using Relative risk

30
31
Types: There are two types of cohort studies, prospective and
retrospective, depending on the temporal relationship
between the initiation of the study and the occurrence of the
disease.

1. Prospective - At the beginning of the study the outcome has

not yet occurred. Regarded as more reliable than the
retrospective, if the sample size is large and follow-up
complete.

* The outcome has not occurred at the beginning of the study

2. Retrospective - Both exposure and outcome status have

occurred at the beginning of the study.
32
33
34
Selection of Exposed Group
Selection of exposed group should consider scientific and
feasibility issues which include:
- the frequency of the exposure of interest in the study
population.
- the need to obtain complete and accurate exposure and
outcome information on all study subjects. Example: the use
of physicians or nurses permits longer and fairly complete
follow up.
- the ability of obtaining sufficient exposed individuals in a
reasonable period of time - identify high risk population
35
(special group) to the exposure of interest.
Selection of controls
Always attempt to select a control group which is
comparable to the characteristics of the exposed
population. There is no single optimal control group that
can be used for any circumstance.

Challenges of Cohort Study

• Resources: high cost, long time

• Obtaining complete information for all comparison groups

• Loss to follow-up
36
37
C. Interventional/ Experimental

The main distinction from other types of analytic

studies is that individuals are allocated into
experiment or control group by the investigators. It
has essentially the same design as prospective
cohort study with one very key difference, exposure
status of the study population is deliberately
changed by the investigator to observe how this
alters the incidence of disease or other features of
the natural history. 38
 Investigator assigns subjects to exposure and
non-exposure and makes follow up to measures
the occurrence of disease.
 It is usually prospective.

 Very expensive, difficult to overcome ethical

issue.

39
40
Intervention trails could be done for various purposes:

- Proof of concept trail: designed solely to produce knowledge about

cause and effect, does not test the efficacy of the intervention in
actual practice.

- Prevention trail: interventions are to prevent disease and study

participants are persons without disease.

- Clinical trail: interventions are treatment based on drugs and study

participants are persons with disease.

41
The comparison groups in intervention study are
known as the intervention group and the control
group. The intervention group receives the test
drug (the preventive activity such as health
education, diet and exercise). The control group
shall be offered the best known alternative or a
placebo activity with no known effect on the
outcome.
42
It is very important that the two groups gain
equal amount of attention in the study.
Unequal attention leads to differences
attributed to the amount of attention each
group receives, not to the intervention-
known as a Hawthorne effect (bias).

43
Classification

1. Based on population

A. clinical trial - usually performed in clinical setting and the

subjects are patients.

B. Field trial - used in testing medicine for preventive

purpose and the subjects are healthy people. E.g. vaccine
trial

C. Community trial- unit of the study is group of

people/community. E.g. fluoridation of water to prevent
dental caries. 44
2. Based on design

A. Uncontrolled trial - no control group. control will

be past experience (history).

B. Non-randomized controlled- there is control

group but allocation into either group is not
randomized.

C. Randomized controlled - there is control group

and allocation into either group is randomized.
45
3. Based on objective for new drugs

A. Phase I - trail on small subjects to test a new

drug with small dosage to determine the toxic
effect.

B. Phase II - trial on small group to determine the

therapeutic effect.

C. Phase III- study on large population - usually a

randomized control trial.
46
Problems Related to Intervention Studies

1. Ethical considerations prevent evaluation of many

treatments or procedures using an intervention design
strategy.

Some of the ethical issues are:

• Practices or substances already known to be harmful

should not be used in this study.

• Therapies known to be beneficial should not be withheld

from any affected individuals in the study population.
47
• Investigators have to have a complete knowledge of the
subject under study.
• The researcher must have at least informed consent from
each study participant and subjects should be left free to
withdraw from the study at anytime.

• A written research protocol is a must.

2. Feasibility/ practical issues

• Subject recruitment, getting adequate individuals to enrol

into a study is not easy.

3. Cost 48
 Advantages of Intervention Studies
• GOLD STANDARD =

 Randomized

 placebo controlled

 blinded clinical trials

• The ability to assign exposure

• The ability to control confounding

• Findings can be replicated = Generalizability

49
The hierarchy of epidemiological research

50
• Study about study!

51