STUDY

DESIGNS
By Matenge Mutalange
Adopted from BM Hamooya
RECAP RESEARCH

■ Systematic investigation that is;

 systematic ,
 methodical
 ethical
Leading to new discoveries. More insights , better understanding and
creation of new knowledge
■ QUANTITIVE RESEARCH

vs

■ QUALITATIVE RESEARCH
Research designs

• A study design is a specific plan or protocol for conducting the

study, which allows the investigator to translate the conceptual
hypothesis into an operational one.

• Objective of the research determines the type of research design

OBJECTIVES

■ Is to understand the following study designs:

– Cross sectional studies
– Case control
– Cohort studies
– Clinical trials
CROSS-SECTIONAL STUDIES

■ Cross-sectional studies are conducted in order to

determine the prevalence of a particular condition
or outcome at a certain point in time.
■ They are also referred to as prevalence studies
■ Mostly they take the form of surveys
■ They are useful for investigating disease trends
over time, as well as for health service
planning.
■ In a cross-sectional study the measurements of exposure
and outcome are made at the same time.
■ In sudden outbreaks of disease, a cross-sectional study to
measure several exposures can be the most convenient
first step in investigating the cause
■ Data from cross-sectional studies are helpful in assessing
the health care needs of populations
■ Data from repeated cross-sectional surveys using
independent random samples with standardized
definitions and survey methods provide useful indications
of trends
■ Valid surveys need well-designed questionnaires, an
appropriate sample of sufficient size, and a good response
Advantages

■ Relatively quick and easy to conduct

■ Good for descriptive analyses and for generating

hypotheses

■ Good for investigating many exposures and

outcome variables
 Disadvantages
■ Cross-sectional surveys are an inadequate approach to the
study of rare conditions, diseases of short duration or rare
exposures, since it would be necessary to survey a very
large population to identify enough cases or people
exposed.
■ Being based on prevalent (existing) rather than incident
(new) cases, they are of limited value to investigate
etiological relationships.
■ It is difficult to establish the time sequence events. Since
exposure and disease status are assessed at a single point
in time, in many cases, it is not possible to determine
whether the exposure preceded or resulted from the
disease.
 Measures of disease
frequency and association

■ In cross-sectional study, the measure of disease

frequency is Prevalence

■ And the measures of association are:

1. Prevalence ratio
2. Odds ratio
 CASE-CONTROL STUDIES

■ Case–control studies begin by identifying people with the

disease being studied
■ Case-control studies provide a relatively simple way to
investigate causes of diseases, especially rare diseases
■ They include people with a outcome of interest and a
suitable control (comparison or reference) group of
people unaffected by the outcome
■ The study compares the occurrence of the possible cause
in cases and in controls
■ The investigators collect data on disease occurrence at one
point in time and exposures at a previous point in time.
■ Case-control studies are longitudinal unlike the cross-
sectional studies
■ A case-control study begins with the selection of cases;
these cases should represent all the cases in a specified
population group
■ Controls are people without the disease
■ The controls should represent people who would have
been designated study cases if they had developed the
disease
■ An important aspect of case-control studies is the
determination of the start and duration of exposure for
cases and controls
■ In the case-control design, the exposure status of the
cases is usually determined after the development of the
disease (retrospective data) and usually by direct
questioning of the affected person or a relative or friend
Example of a case control study
Measure of Frequency and
Association Case-Control Studies
■ Measure of exposure frequency
– Prevalence

■ •Measure of association
– Odds Ratio
Advantages of case-control
studies
■ are the most efficient design for rare diseases
■ require a much smaller study sample than cohort
studies
 Disadvantages of case-
control studies
■ Case-control studies do not yield an estimate of rate or
risk, as the denominator of these measures is not defined
■ Case-control studies may be subject to recall bias if
exposure is measured by interviews and if recall of
exposure differs between cases and controls
■ Choosing an appropriate source population is also difficult
and may contribute to selection bias
■ Illustrative Example: Toxic shock syndrome (case–control study)
■ Toxic shock syndrome (TSS) is an illness characterized by high fever,
vomiting, diarrhea, confusion, and an exfoliating skin rash. It is fatal in 3–
15% of cases and is caused by the exotoxin of a particular strain of
Staphylococcus. In late 1979 and early 1980, the Centers for Disease
Control received an unusual number of reports of TSS from state health
departments in Wisconsin, Minnesota, Illinois, Utah, and Idaho (CDC,
1980a; 1980b). The cases occurred almost exclusively in women of
childbearing age. Several case–control studies were completed in the
wake of these reports. One of the case–control studies evaluated prior
exposures to risk factors in 52 cases and 52 age- and sex-matched
controls. All of the 52 cases in this study had used tampons during their
menstrual periods coincident with the onset of illness. In contrast, 44
(85%) of the 52 control study subjects had used tampons during their
prior menstrual period. Thus, cases were more likely to be tampon users
than controls (Shands et al., 1980). In this same study, among the 44
case–control pairs in which both study subjects had used tampons, 42
(95%) of the 44 cases used tampons continuously throughout
menstruation. In contrast, 34 (77%) of 44 controls did similarly. Thus,
among the tampon users, cases were more likely to use tampons
COHORT STUDIES
■ Cohort studies begin by identifying disease-free individuals
■ The term cohort derives from the Latin word cohors,
meaning ‘‘an enclosure”
■ Study subjects are then classified according to risk factors
thought to be associated with future disease occurrence
■ In their simplest sense, cohort studies follow two groups of
individuals.
– One group is characterized by an exposure
– Second group characterized ‘‘nonexposed.’’
■ Study outcomes in individuals are ascertained over time,
tallied, and compared in the form of incidence rates or
incidence proportions
■ Cohort studies can be either experimental or observational
– Experimental Cohort studies
– Observational Cohort studies
■ Once enrolled in a cohort study, each study subject is
followed until:

– the subject withdraws from the study

– the study ends, or

– the study outcome is experienced.

Types of cohort studies
■ Prospective cohort studies: are cohort studies
planned to observe events/outcomes that are yet to
occur
■ Retrospective cohort studies or historical cohort
studies: are cohort studies carried out using records of
events/outcomes that had occurred in the past.
■ Ambidirectional cohort studies: are cohort studies
that combine prospective data with retrospective data
Sources of Retrospective
Data
■ Data can be obtained from a variety of sources,
including:
– medical records,
– administrative data sources,
– vital records systems,
– surveillance systems, and
– employment records
■ In addition, we may interview study subjects or their
proxies about prior events to obtain retrospective
data
Measure of disease frequency and association
■ Measures of disease frequency
– Risk
– Rate
– Prevalence
■ Measures of association
– Rate ratio
– Risk ratio
– Rate difference
– Risk difference
– Odds ratio
Cohort Studies: Advantages
■ Exposure to postulated cause is assessed before
occurrence of disease

■ Possible to estimate all measures of incidence (risk, rate,

odds) and effect (Rate Difference and Relative Risk)

■ Possible to study several outcomes of one cause

■ Good for rare exposures

 Cohort Studies:
■Disadvantages
Requires large investments in time, human, and financial
resources

■ Requires large sample sizes

■ Not easy to reproduce (Re: consistency of the association)

■ Inefficient for rare outcomes

■ Loss to follow up: there are usually challenges with

dropouts
■ Illustrative Example: Oral contraceptive estrogen dose and
venous thromboembolism (cohort study)
■ A cohort study examined the incidence of venous thromboembolism
in 234 218 women using oral contraceptives with varying amounts of
estrogen (Gerstman et al., 1991). The study was restricted to women
taking combination oral contraceptives. Women taking formulations
containing less than 50μg of estrogen were classified as ‘‘low-dose
users.’’ Women taking formulation containing exactly 50μg of
estrogen were classified as ‘‘intermediate-dose users.’’ Women
taking formulations containing more than 50μg of estrogen were
classified as ‘‘high-dose users.’’ The experience of each study subject
was tracked for the occurrence of venous thromboembolism (VTE).
The rates of VTE in each group were
■ Low-dose users: 4.2 per 10 000 person-years
■ Intermediate-dose users: 7.0 per 10 000 person-years
■ High-doses users: 10.0 per 10 000 person-years
 Thus, progressively higher estrogen doses were associated with VTE
 CLINICAL TRIALS
■ A clinical trials is a prospective study comparing the
effect and value of an intervention against a control
in human subjects

■ Meinert (1986) indicated that a clinical trial is a

research activity that involves administration of a
test treatment to some experimental unit in order to
evaluate the treatment.

■ Meinert (1986) also defines a clinical trial as a

planned experimental designed to assess the
efficacy of a treatment in humans by comparing the
outcomes in a group of patients treated with the test
Why Clinical Trials?

■ Unless the treatment is overwhelmingly

successful, a statistically designed experiment is
the only way to conclusively establish (with high
probability) the superiority of a given treatment.
All other methods are subject to bias.
Key words in the definition of
clinical trials
1. Experimental unit: there should be some individuals
to be experimented on
2. Treatment: there should be
3. Evaluation of the treatment: there should be an
evaluation of the treatment
 Problems with clinical trial
■ Well-designed clinical trials often require large sample
sizes (hundred of patients)
■ Physician opinions are often formed based on experience
with a few patients and anecdotal evidence
■ Physicians can hold strong, opposing positions on which
treatment is “best.”
■ Patient compliance
Randomization
■ Randomization: is a process by which an investigator
randomly assigns exposure status
■ Purpose of randomization
■– To obtain a group which is comparable
■– To balance all known and unknown confounders
between the exposed and unexposed groups
■– To achieve baseline comparability
■– To strengthen causal inference
Types of Qualitative research
designs
■ Ethnography
■ Phenomenology
■ Grounded theory
Ethnography

■ Ethnography is a qualitative research design often used in social

sciences often employed for gathering empirical on human cultures
■ Data collection is done through participant observation, interviews,
questions
■ It focuses on the cultural of people
■ Advantages and disadavatages?
Phenomenology

■ Qualitative research design on lived experience

■ Its purpose is to describe essences of lived experinces
■ Advatanges and disadavtages?