Chapter Two: CLINICAL TRIAL,

EPIDEMIOLOGY, STUDY DESIGNS

Saad Haddi
SAMPLING METHODS

• Statistical analysis is an essential technique that enables a medical

research practitioner to draw meaningful inference from their data
analysis. Improper application of study design and data analysis may
render insufficient and improper results and conclusion.
A Clinical trial
• A Clinical trial is a medical experiment on human subjects,
particularly in a clinical setup, such as to find the efficacy and safety of
a new therapeutic or diagnostic regimen (Medical treatment). A
clinical trial is an experiment testing medical treatments on human
subjects.
• The clinical investigator controls factors that contribute to variability
and bias
• such as the selection of subjects, application of the treatment,
evaluation of outcome, and methods of analysis. A clinical trial must
employ one or more intervention techniques.
Epidemiology
• Epidemiology is the study of factors that affect distribution and
determinants of disease or a health condition in a human population.
• Epidemiological studies measure the risk of illness or death in an
exposed population compared to that risk in an identical, unexposed
population (for example, a population the same age, sex, race and
social status as the exposed population).
• Epidemiology is the study of how often diseases occur in different
groups of people and why.
3.2 EXPERIMENTAL, OBSERVATIONAL AND
LONGITUDINAL STUDIES

• 3.2.1 EXPERIMENTAL AND OBSERVATIONAL STUDIES

• An experimental study is a study where the researcher has control over
most of the variables. Once the research problem has been formed, the
researcher organizes a study that will allow him to find answers to the
research problem. In this case, the researcher conducts the study in a
specific setting such as a laboratory where he can control the variables.
• An observational study is a study where the researcher merely observes the
subject without controlling any variables. These types of studies are mainly
used in the social sciences. In disciplines such as sociology, anthropology,
etc. observational studies are used to comprehend human behavior.
•
Conti
Three major types of epidemiologic studies

• Three major types of epidemiologic studies are cohort, case-control,

and cross-sectional studies. A cohort or longitudinal study follows a
defined group over time.
1. Cohort Study
• A cohort study is a type of observational study that follows a group of
participants over a period of time, examining how certain factors (like
exposure to a given risk factor) affect their health outcomes. The
individuals in the cohort have a characteristic or lived experience in
common, such as a birth year or geographic area..
Types of Cohort Study

• There are two types of Cohort study; Prospective and retrospective

In prospective cohort studies, data is collected over time to compare

the occurrence of the outcome of interest in those who were exposed
to the risk factor and those who were not.
Prospective cohort studies look forwards in time, to examine the
relationship between the exposure and the outcome.
Prospective Cohort Study

Smokers’ and non-

smokers
exposure to
Cancer
Cont..
• In retrospective cohort studies, your participants must already
possess the disease or health outcome being studied before joining.
The study is then focused on analyzing the health outcomes of those
who share the exposure to the risk factor over some time.
• Example
• You are interested in how mosquito-borne diseases spread. You
interview a cohort of people who have been hospitalized with severe
dengue fever, asking about the events surrounding their illness and
their medical history to study the spread of the disease
Cont…
Case-Control Study
• A study that compares patients who have a disease or outcome of interest
(cases) with patients who do not have the disease or outcome (controls),
and looks back retrospectively to compare how frequently the exposure to a
risk factor is present in each group to determine the relationship between the
risk factor and the disease.
• Example
You are examining the relationship between drinking water contamination and
the incidence of gastrointestinal illnesses like gastroenteritis. Here, the case
group would be individuals who have been diagnosed with a gastrointestinal
illness, while the control group would be individuals without such an illness
Cont…
Cross-Sectional Studies
• A cross-sectional study is a type of observational research that analyzes
data of variables collected at one given point in time across a sample
population or a pre-defined subset.
• Example
• You want to know how many families with children in Somaliland are
currently Poor so you can estimate how much money is required to
fund a free lunch program in public schools.
• Because all you need to know is the current number of poor families, a
cross-sectional study should provide you with all the data you require
Examples 2
• i. Scientists in healthcare may use cross-sectional research to
understand how children ages 2-12 across the Somaliland regions are
prone to vitamin A deficiency.
• ii. A medical study looking at the prevalence of cancer amongst a
defined population. The researcher can evaluate people of different
ages, ethnicities, geographical locations, and social backgrounds.
3.2.3 LONGITUDINAL STUDIES

• Longitudinal studies are versatile, repeatable, and often use surveys

to collect data that is either qualitative or quantitative. Additionally, in
a longitudinal study, a survey creator does not interfere with survey
participants. Instead, the survey creator distributes questionnaires
over time to observe changes in participants, their behaviours, or
their attitudes.
• The purpose of using the same individuals or samples in the
longitudinal study is to observe any measurable change over time.
This ensures that you can account for the same variables of interest in
the duration of your study.
Example
• Consider a study conducted to understand the similarities or differences
between identical twins who are brought up together versus identical twins who
were not.
• The study observes several variables, but the constant is that the participants all
have an identical twin.
• Researchers, in this case, would want to observe these participants from
childhood to adulthood to understand how growing up in different
environments influences traits, habits, and personality.
• Over many years, researchers can see both sets of twins as they experience life
without intervention. Because the participants share the same genes, it is
assumed that any differences are due to environmental factors, but only
attentive study can bring them to a conclusion.
Example
• In the study examining the links between smoking and stomach
cancer, you have to wait several years to see any results since the
negative effects of smoking accumulate over decades
Exercise
• Can you give us examples of longitudinal studies?
Cross-sectional vs Longitudinal study
There are three major types of longitudinal
studies for future research:
• i. Panel study: A panel study involves a sample of people from a
bigger population and is conducted at specified intervals for a longer
period. One of the most important features of the panel study is that
data is repeatedly collected from the same sample at different points
in time. Most panel studies are designed for quantitative analysis,
though they are also used for collecting qualitative data and analysis.
Cont..
• ii. Cohort Study: A cohort study samples a cohort (a group of people
who typically experience a common event at a given point in time).
Medical researchers tend to conduct cohort studies. Some might
consider clinical trials similar to cohort studies. However, in cohort
studies, researchers merely observe participants without
intervention, unlike clinical trials in which participants undergo tests.
Cont..
• iii. Retrospective study: A retrospective study makes use of already
existing data, collected during previously conducted research with
similar methodology and variables. While conducting a retrospective
study, the researcher uses an administrative database, pre-existing
medical records, or one-to-one interviews. Longitudinal study is
useful in science and medicine as well as many other fields.
3.3 PREVALENCE, INCIDENCE AND
MORTALITY
Prevalence
• Prevalence is a measure of disease that allows us to determine a person's
likelihood of having a disease.
• Therefore, the number of prevalent cases is the total number of cases of
disease existing in a population. A prevalence rate is the total number of
cases of a disease existing in a population divided by the total
population.
• So, if a measurement of cancer is taken in a population of 40,000 people
and 1,200 were recently diagnosed with cancer and 3,500 are living with
cancer, then the prevalence of cancer is 0.118. (or 11,750 per 100,000
persons).
Exercise
• A community living in a Toon Village south of Hargeisa was infected a
Measles a few months ago. 330 cases of Measles were identified in
the village. If the population of the city is 1200, calculate the
prevalence of Measless.
Cont..
Incidence
• Incidence is a measure of disease that allows us to determine a person's
probability of being diagnosed with a disease during a given period of time.
• Therefore, incidence is the number of newly diagnosed cases of a disease.
• An incidence rate is the number of new cases of a disease divided by the
number of persons at risk for the disease.
• If, over the course of one year, five women are diagnosed with breast cancer,
out of a total female study population of 200 (who do not have breast cancer
at the beginning of the study period), then we would say the incidence of
breast cancer in this population was 0.025. (or 2,500 per 100,000 women-
years of study)
Exercise
• A community living in a Toon Village south of Hargeisa was infected
with TB a few months ago. 230 cases of TB were treated in the village.
If the city’s population is 3100, calculate the Incidence rate of TB.
Cont..
Mortality
• Mortality is another term for death. A mortality rate is the number of
deaths due to a disease divided by the total population. If there are
25 Polio deaths in one year in a population of 30,000, then the
mortality rate for that population is 83 per 100,000
Exercise
• A community living in a Toon Village south of Hargeisa was infected
with TB a few months ago. 2 Patients died from TB in the village. If the
city’s population is 3100, calculate the Mortality rate of TB.
3.4 SAMPLING AND COMMON SAMPLING METHODS

• The process by which samples are obtained from a population is

called sampling.
• There are two main types of sampling: probability and non-probability
sampling.
• The difference between the two types is whether or not the sampling
selection involves randomization.
• Randomization occurs when all members of the sampling frame have
an equal opportunity of being selected for the study. Following is a
discussion of probability and non-probability sampling and the
different types of each.
Cont..
• Probability Sampling – Uses randomization and takes steps to ensure all
members of a population have a chance of being selected. The different
types of probability sampling include simple random sampling, stratified
sampling, cluster sampling, and systematic random sampling
• Non-probability Sampling – Does not rely on the use of randomization
techniques to select members. This is typically done in studies where
randomization is not possible in order to obtain a representative sample.
Bias is more of a concern with this type of sampling. The different types
of non-probability sampling include convenience, purposive sampling,
quota sampling and snowball sampling.