9.

Overview of Study Design - I

Dr. S M Raysul Haque

DPH, IUB
 Did
Did investigator
investigator assign
assign exposures?
exposures?

Yes No
Experimental study Observational study

Random allocation ? Comparison group ?

Yes No
Yes No
Analytic Descriptive
Randomize Non- study study
d controlled randomized
trial controlled trial
Direction?

Case- Cross-
Cohort
control sectional
study
study study
 Descriptive study

• Descriptive study is at the bottom of the research hierarchy, often represents the first
scientific toe in new areas of inquiry.

• A descriptive study is “concerned with and designed only to describe the existing
distribution of variables, without regard to causal or other hypothesis.”

• As there is no comparison group, descriptive study doesn’t allow assessment of

associations.
 Three key features
• Traditional descriptive epidemiology is concerned with describing the general
characteristics of the distribution of a disease, in relation to,
– Person
– Place and
– Time
Or
• Agent, host and environment
 The descriptive triad – or pentad?

• An alternative approach is to answer five basic “W” questions:

– Who?
– What?
– Why?
– When?
– Where?
– And an implicit sixth question, so what?
 WHO has the disease in question?

The description of characteristics of person must include age and sex.

• Other important variables might be religion, marital status, personality type, race,
socio-economic factors like education, income, or occupation.

• It can also include variables like type of medication use, types of food consumption
and other lifestyle variables.
 WHAT is the condition or disease being studied?

• Development of a clear, specific and measurable case definition is an essential step.

• If only stringent criteria is followed, targeting more severe cases of disease, milder or
earlier cases might be missed.

• The trade-off is better specificity or less false positive cases.

• E.g. AIDS cases

 WHY did the condition or disease arise?

• Descriptive studies often provide clues about the cause that can be pursued with
more sophisticated research designs.

• E.g. Clinical observation of blindness in newborn infants leads to underlying

association of high ambient oxygen concentrations in incubators

• Observation of cataract, heart defects and deafness in newborns leads association of

rubella during pregnancy
 WHEN is the condition common or rare?

Time provides important clues about health events.

• Outbreak of gastroenteritis soon after ingestion of staphylococcal toxin.

• Seasonal pattern of the disease e.g Dengue fever, pneumonia, influenza.

• Historical comparison e.g. Smallpox

• Cyclic trend e.g. Malaria
 WHERE is the disease rate is highest or lowest?
• The geography has had a huge effect on health and distribution of the disease.

• E.g. Areas with drinking water with high fluoride protects against dental caries.

• Study on migrants helps to distinguish possible roles of genetics and environment on

diseases, e.g. colon cancer

• International comparison, distribution e.g. Ebola virus, Swine Flu

• National-Within the countries e.g. Malaria

• Local-Urban and rural areas e.g. Helminthiasis

 So WHAT? – the implicit “W”

The sixth “W” relates to public health effect.

• Is it important?
• Is it serious?
• Are large numbers involved?
• Are its societal implications broad?
• Has it been studied before?
 Types of descriptive studies

There are two main types of descriptive studies, those that deal with
individuals and those that deal with population.

Individuals Populations

• Case-report • Ecological correlational

• Case-series report studies
• Cross-sectional studies
• Surveillance
 Case report and case series

• Most basic type of descriptive study of individuals.

• It describes the profile of a patient or a group of patients with similar diagnosis.
• Usually the clinician finds an unusual feature, that may lead to formulation of a
hypothesis.
• It is an important interface between the clinical medicine and epidemiology.
 Case reports

• Most common
• Documents unusual medical occurrence
• Reporting of a rare tumour (benign hepatocellular adenoma) in women who had taken
oral contraceptives persuaded a large case-control study that confirmed strong
association
• Frisbee finger
• Tennis elbow
 Case series report

• A case series aggregates individual cases with similar diagnosis in one report
• Span of short period
• Historical importance in epidemiology as often highlights on beginning or presence of
an epidemic
• Investigation on the activities of affected individuals can lead to formulation of
hypothesis
 Question on approach

• Mere documentation of an interesting medical occurrence?

OR

• Formulation of a hypothesis?

• Might depend upon collection of a case series rather than reliance on a single case
report
 Example

• Five Pneumocystis carinii pneumonia were reported in young men between 1980-81,
which usually occurs in immunosuppressed older cancer patients- Unusual !!!!!
• Same year Kaposi’s sarcoma reported in young adults-unusual!!!!

This led to revelation that

• They were immunosuppressive
• Investigation by CDC: A new disease AIDS
• Analytic studies revealed Risk factors and transmissible agent
 Pertinent questions for descriptive study

• WHO develops the disease with excessive frequency?

• WHAT is the condition/disease being studied?
• WHY did the disease arise?
• WHEN is the disease common or rare?
• WHERE does the disease develop with excessive frequency?

• SO WHAT?
 CROSS-SECTIONAL SURVEY

• This is also known as prevalence survey

• It describes the health of population and provides a snapshot of the population at a
particular time
• Exposure and outcome (disease) status are assessed at the same time among
individuals in a well defined population
• “Case-control analogue of a population cohort study”
• Temporal sequence is often impossible to work out
 CROSS-SECTIONAL SURVEY

• The time window can be specific: any season or months of a year

• It also can be a fixed point in the course of events which may vary in from person to
person e.g.antenatal check up for pregnant women, pre-employment physical
examination, retirement etc.
• The subjects provide information at a single interview or examination
 Information source

• Census data
• Vital statistics record
• Employment health examinations
• Hospital medical records
• National survey e.g BINP,NNP etc
 Example

• National Nutrition Survey

• Bangladesh Integrated Nutrition Programme

Go door to door.
Ask questions. Know the status.
Prepare a report.
Go for policy implication.

E.g. Know the vitamin A status in the under five children

 Strength & Advantages
• A good survey data can be of great value for national health policy making by
– Assessing health status
– Assessing health care needs of population

• Less expensive
• Less time consuming
• Describes patterns of disease occurrence
• Helps in allocating resources
• Helps in formulating research questions
 Limitations

• It can only assess the relationship between the exposure and disease at a certain time
point.

• Not possible to identify whether the exposure preceded or resulted from the disease.
– Exception: long standing/ unchanged exposure such as sex or blood type

• It considers prevalence rather than incidence.

 Surveillance

• Formal definition, “the ongoing systematic collection, analysis, and interpretation of

health data essential to the planning, implementation, and evaluation of public health
practice, closely integrated with the timely dissemination of these data to those who
need to know.”

• Prevention and control integrated with feedbacks are the key objective
 Types of surveillance

• Passive surveillance relies on data generally gathered through traditional channels,

e.g. death certificates
• Active surveillance searches for cases, e.g. maternal deaths
• Eradication of smallpox was possible due to surveillance and containment
 Ecological correlational study

• Correlational studies look for associations between exposures and outcomes in

populations rather than in individuals

• The disease is described in relation to some factor or interest such as age, sex,
calendar time, food habits etc.

• Correlational studies can be a convenient initial search for hypothesis

 Example

• Different groups at a particular time point: As we have seen that the proportion of
smokers at different age group

• Different times for a particular group: In the elderly population the Chronic Heart
Disease has gone down over the years. It may be due to improved health care
facilities or by reduction of risk factors
 Correlation coefficient

• r is the descriptive measure in correlational studies, as long as there is a linear

relationship between exposure and disease.

• The linear relationship means that for every unit change in exposure there is
proportionate change in the disease frequency also.

• E.g. Death rates from coronary artery disease correlate with per capita sales of
cigarettes.
 Strength of correlational study

• This is the first step to investigate in the possible exposure disease relationship

• Quick and inexpensive

• Allows for comparison (with the already available data from different surveys) for
different geographic distribution
 Limitations of correlational study

• Inability to link exposure with disease in particular individuals

E.g. Anemia screening were correlated to mortality from malnutrition.

There is a strong positive correlation. More the numbers screened less is the mortality
from anemia.

Question: Does screening decreases mortality from PEM? Answer: NO! We in fact do
not know that the persons who were screened, actually experienced lower mortality
rates.
 Limitations of correlational study

• Inability to control of confounding

E.g. Having a balcony garden causes dengue.

The possibility is that the Aedes mosquito grow in the stagnant water in the flower
pot and the bite of Aedes causes Dengue.

It is not possible to separate the effects of such potential confoundings from the
correlational data.
 Limitations of correlational study

• Masks the actual relationship

- It represents the average exposure levels rather than the actual individual
levels.
- The exposure can be at times high, or it can be low and the relationship might
not be linear. It is possible that people with moderate anemia may die earlier
than those with mild anemia.
 Uses of descriptive studies

• Trend analysis
– Important to healthcare administrators
– Often provided by ongoing surveillances

• Planning
– Need for hospital beds
– Need for resource allocations

• Clues about cause

– Observation by clinicians, and subsequent development of hypotheses
 Limitations of descriptive studies

• Overstepping the data

– False inference of temporal relationship may cause public-health damage

– Investigators might draw causal inferences when none is possible

• E.g. oral contraceptive pill vs. ovarian cysts
• Electronic fetal monitoring