INTRODUCTION TO

EPIDEMIOLOGICAL METHODS
PREPARED BY-
AMEENA MEHABOOB
NAZARETH COLLEGE OF PHARMACY OTHERA
TOPICS
Types of epidemiology studies.

Standard measures in epidemiological studies.

Measures of disease frequency.

Measures of association.

Study designs in epidemiology studies.

Intervention studies.

Controlled clinical trials.

Errors in Epidemiological studies.

Validity and Reliability.

Bias and confounding.

 Epidemiology is “the study of the distribution and determinants of health
related states or events in specified populations and the applications of this
study to the control of health problems”
 It is the rigorous and scientific study of occurrence, patterns, causes,
distribution and determinants of diseases in defined human populations.
 2 major areas of epidemiology-
1. Study of infectious diseases in large populations called epidemics.
2. Study of chronic diseases
 Epidemiological studies help diagnose health problems and provide a basis
for the care of people.
 Objectives of epidemiology include application of information or findings to
improve the structure, activities, programs and outcomes of public health
care and for that purpose develop health policies rooted in evidence.
TYPES OF EPIDEMIOLOGICAL STUDIES

 Two main categories of research design are observational

studies and experimental or intervention studies.
 In observational studies, the investigators stand apart from
events taking place in the study - simply observe and record.
 In experimental or intervention study, investigators introduce
an intervention and observe the events or outcome.
 Quasi
RCT
Experimental experiment
studies
(intervention)
Non RCT Field trial

Pharmacoepidemiologic
Community trial

DUR

studies
Case series

Descriptive
Case report
studies

Drug survey
Non
experimental
Cross sectional
studies (non
intervention)
Case control

Analytic studies Cohort

Cross sectional
I. OBSERVATIONAL STUDIES
Observational studies can be descriptive or analytical.

1. Descriptive studies-
 This is the first phase of an epidemiological study.
 Observations are done on various aspects of illness among a population like time
of occurrence, symptoms, duration of illness, place of residence and individual
characteristics (age, education, occupation etc).
 This will lead to formulation of hypotheses regarding disease pattern, aetiology
and prognosis which can be tested later on using an analytical type of design.
 Census and clinical records are important sources of information in this type
of study.
 Case reports, case series, cross-sectional and ecologic studies come under this
category.
A. Drug Utilization Review (DUR)
 Also known as drug utilization evaluations.
 Authorized, structured, on going review of prescribing, dispensing and use of
medication.
 Help to promote rational use of medicines.
 3 categories-
i. Prospective- evaluation of patients therapy before medication is dispensed.
ii. Concurrent- on going monitoring of drug therapy during the course of
treatment
iii. Retrospective- review of therapy after the patient has received the
medication.
 DUR will help to find out inappropriate use of medicines and can also help to
find out irrational prescription writing styles.
B. Case reports:
 It is a careful detailed report of the profile of a single patient or case.
 Occurrence of an unusual case or rare event is studied as case report.
 One of the simplest method, weakest form of evidence for causation.
 Presentation of experience of a patient by his treating physician or other
health care professional.
 Case report focuses on an individual patient who is on drug use, but
experiences a particular outcome, generally ADR.
 Case reports are hypothesis generating reports.
 They bring forth an evidence that supports a hypothesis about drug
effective to be tested with other more effective and rigorous study
designs.
C. Case series:
 Also known as clinical series.
 It is a group or cluster of case reports generated by a single or group of
clinicians or hospitals or regulatory agencies like FDA or drug controller.
 It is a typical medical research study that tracks subjects with a known
exposure or examines their medical reports for exposure and outcome.
 A set of sequential case reports make a case series.
 Case series are collections of patients all of whom have a single exposure
and whose clinical outcomes are evaluated and described.
 No etiological relationship can be found out as there is no comparison group.
D. Descriptive cross sectional studies-
 Study in which the disease or condition and potentially related factors are
measured at a specific point of time for a defined population.
 It is a snapshot of frequency and characteristics of a condition in a population
at a particular point in time.
 This type of data can be used to assess prevalence of a condition in a
population.
2. ANALYTIC STUDIES
 Also known as explanatory studies.
 Compares an exposed group with a control group and are designed as hypothesis
testing studies.
 These are studies used to test hypotheses concerning the relationship between a
suspected risk factor and an outcome and to measure the magnitude of the
association and its statistical significance.
 The cardinal feature in an analytical study is the concern whether an exposure (risk
factor, independent variable or predictor variable) causes the outcome.
 Analytical studies look for causal associations.
 It includes two observational studies; cohort and case control studies
A. Cohort studies:
 These studies are also known as longitudinal studies or incidence studies.
 Cohort study is a study of groups of patients having some common drug exposure of interest.
 Cohort study assembles a group of persons without the disease of interest at the onset of study.
 They are followed over time and experiences of members of one cohort are compared with those in
other cohorts in order to describe outcome among people whose exposure status is known.
 It helps determine the development of disease in exposed and non exposed patients.
 Involves comparison of incidence of one or more outcome events among those who received a drug
with incidence of events of control group.
 The incidence of the disease can be measured and relative risk calculated.
 Relative risk is the incidence of disease in the exposed / Incidence of disease in those not exposed.
Prospective cohort study
• Best type of cohort study.
• Looks forward in time and allows maximum control over study definition and its
conduct.
• Very expensive.

Retrospective cohort study

• It looks back on existing data.
• Generally data comes from medical records and computer database
• Advantage- low cost
• Disadvantage- bias
Cohort studies

Advantages Disadvantages

• Useful in hypothesis • Some studies require

testing large no of people for a
• Produce great deal of long period of time
useful information • Expensive
• Time consuming
B. Case-control studies:
 Simplest and most commonly used analytical strategy.
 Designed to establish causes of a disease by investigating association between
exposure to a risk factor and occurrence of a disease.
 It is a backward looking or retrospective study based on exposure histories of
cases and controls.
 Cases are compared with controls for the presence or absence of hypothesized
risk or exposure factors.
 Case control studies are often used in situations where the disease is a rare one
or has long latent period.
 Data are used to calculate an odds ratio, which is the ratio of odds of
developing the disease for exposed patients to odds of developing disease for
unexposed patients.
Advantages Disadvantages
The efficacy is high for study of rare
Susceptibility towards bias
or delayed outcomes

Relatively cheap Insufficient for rare exposure

Difficult to establish relationship

Quick
between exposure and disease

Inappropriate when outcome is not

Efficient
known.

Used to test hypothesis

Involve fewer participants

C. Cross sectional study
 Called instantaneous studies or prevalence studies.
 Studies of use of drugs at a specific point in time are conducted through
surveys, data base analysis or medication chart reviews.
 Helps in collection of data on a cross section of a population or a sample of it.
 Provides information about the situation that exists at a single time.
 Snapshot picture of the situation is obtained.
 Cross sectional studies have been used to compare drug use between states or
countries or regions.
 Advantages
• Short term, less expensive
• Provides wealth of data

Disadvantages
• No direct estimate of risk
• Scope for bias
D. Meta- analysis
 Defined as a systematic review that uses statistical techniques to integrate and
summarize results of included studies.
 Meta- analysis maybe conducted on several clinical trials in effort to obtain better
understanding of how well the treatment works.
 Meta analysis can be applied to RCT, case control or cohort studies.
 Meta analysis combines result of several studies that addresses a set of related
research hypotheses.
Advantages-
 Includes all studies
Disadvantages-
 Source of bias are not controlled
II. EXPERIMENTAL STUDIES
 Investigator determines through a controlled process the exposure for each
individual (clinical trial) or community (community trial) and then tracks the
individuals or communities over time to detect the effect of exposure.
 An experimental study may be controlled or non-controlled
 A controlled experiment can be randomized or non-randomized.
 In a non-randomized trial, we are not sure whether the difference observed in
the outcome is because of the intervention or because of the difference in the
characteristics of the two groups.
 With randomization, only chance determines the assignment of subjects to study
groups.
 A crossover study is a special design of controlled experimental study that is
sometimes used in clinical trials. In this design half the participants are assigned
to new treatment and then switch to standard treatment/placebo, while the
other half does the opposite.
A. RANDOMIZED CONTROL TRIALS
 In the clinical setting most randomized controlled trials are phase 3 trials for
efficacy (result under ideal trial conditions) and effectiveness (result under real
life clinical situations).
 Usually clinicians, have to choose between two or more drugs / therapeutic
regimens for treatment with an uncertainty or whether an intervention (or drug)
is efficacious or not.
 A Clinical trial conducted in a strictly randomised manner, using comparable
control is usually termed as randomised controlled trial.
 Here one group will be given- New treatment/intervention and the other, the
control will be given standard treatment/ Placebo and treated same as the
intervention group except that they don’t get the intervention under study.
 Importance of control group in RCT is because of following reasons
1. Regression to the mean-If a disease is at its extreme, subsequent
evaluation may show improvement which may be statistically significant,
but not occurred as a result of the intervention as such.
2. Unpredictable outcome- Clinical course of disease is usually varying and
the improvement or worsening may not be because of the treatment given.
3. Placebo effect- Placebo effect is the improvement due to the feeling of
being treated. Subjects in both intervention and control group may show
some improvement due to placebo effect. The effect observed in the
treatment group is a combination of real clinical effect and placebo effect
and changes in control group will help us to identify placebo effect from
treatment effect.
4. Hawthorne effect- The phenomenon where subjects tend to improve when
they know that they are being observed.
 Randomization is a key characteristic of RCTs.
 Randomization is the process by which every subject gets an equal
chance to be in the intervention or control group.
 At the end of the process the two groups are expected to be identical and
comparable in all aspects before intervention.
 This means that variables that may influence the outcome of clinical trial
are equally distributed among the two groups.
 Randomization eliminates selection bias and confounding bias, the
confounder being an external factor which has got independent
association to the exposure and the outcome under study. Randomization
is known as the heart of an RCT.
 It include two steps-
(i) the sequence generation
(ii) allocation concealment.
 TYPES OF RCT-
1. Parallel Group Trial
• Each participant is randomly assigned to a group, either the intervention arm or the control arm.
• These groups are followed for a fixed period of time parallel and the outcome will be measured.
2. Cross over Trial
• Trial group and control group will be crossed over their interventions after following for certain period of time.
• The advantage of this design is that all individuals will get an opportunity to be in both the arms.
• A person can act as his/ her own control in two different time point. A time lag is given between the two follow-up times to
completely cancel the effect of first intervention, known as washout period.
• The design is very powerful but can use only in limited occasions where the intervention will not completely cure or
permanently modify the clinical condition under study.

3. Factorial RCT:
• The effect of two or more independent interventions (factors) is studied in a single experiment.
• The major purpose of the research is to explore their effects independently but in same time points.
• Factorial design produce efficient experiments as it can also give the interaction between the interventions on a common
effect.
• Each participant is randomly assigned to a group that receives a particular combination of interventions or non
interventions.

4. Cluster RCT:
• Randomisation is not done for individuals rather it is done for groups or clusters from where the individual study
subjects are enrolled.
• Clusters (e.g., communities, hospitals, or other aggregates of people are randomized and all consenting persons enrolled
from the selected clusters to the selected groups
B. NON RANDOMIZED CONTROL TRIALS

Field trials
• Conducted on those high risk of conducting a disease
• Deals with subjects who are disease free.
• Involves evaluation of whether an agent or procedure reduces the risk of
developing a disease among those free from that condition at enrolment.
• More difficult to carry out.
• Risk of contracting a disease is small.
• Requires greater no. of subjects, longer period of time.

Community trials
• Research on social originating diseases.
• Field trials in which whole communities are the unit of allocation are called
community trials.
• These are done because many interventions are impossible at an individual
level.