CHAPTER I

Introduction to Epidemiology and Biostatistics

Overview of the Lesson:

“Epidemiology is the study of the distribution and  determinants  of health-

related states or events in specified populations, and the  application  of this study to
the control of health problems. ” –

Epidemiology is the study of a scientific method of problem solving that helps “disease
detectives” understand how people get sick and die, who gets sick and dies, and how to avoid
getting sick. This technique has been around for centuries and is still valid today. Like all
scientific disciplines, the field of epidemiology has its own language. The terms that researchers
use to talk to each other are common words but may have unique meanings in epidemiology.

On the other hand, biostatistics is the development and application of statistical models
and theories of inferential, probability and computing and mathematics responsible for
interpreting the scientific data generated in the health sciences.

In this chapter, it will talk about the relation of epidemiology and biostatistics. We will
find out their importance in medical field.

Objectives:

At the end of the sessions, the pre – service teacher (PST) should be able to:

- Distinguish the relation of epidemiology and Biostatistics;

- Enumerate the importance and limitations of biostatistics;
- Identify the importance of biostatistics in allied health profession; and
- Define and Identify the different types of data and understand why we need to
classifying variables.

A. Introduction

A.1. EPIDEMIOLOGY

Epidemiology is a scientific discipline with sound methods of scientific inquiry at its

foundation. Epidemiology is data-driven and relies on a systematic and unbiased approach to
the collection, analysis, and interpretation of data. Basic epidemiologic methods tend to rely on
careful observation and use of valid comparison groups to assess whether what was observed,
such as the number of cases of disease in a particular area during a particular time period or
the frequency of an exposure among persons with disease, differs from what might be
expected. However, epidemiology also draws on methods from other scientific fields, including
biostatistics and informatics, with biologic, economic, social, and behavioral sciences.
 The word “epidemiology” literally means: epi = among, demos = people, logos = study
or studies conducted among human populations. Although the field of epidemiology was
originally focused on the study of infectious disease (contagious or transmittable diseases),
the scope has expanded to include chronic disease (long – lasting disease), and health-
related conditions (disease events that cause illness, death or disability). The current
definition of epidemiology is the study of the distribution (who has the problem) and
determinants (things that influence the problem) of health – related conditions in human
populations and the application of this method to the control of health problems.

The strength of epidemiology lies in its problem-solving methods. Depending on the

question to be investigated, several research techniques are available, including surveillance,
observation, and experiments.

A.2 ““Epidemiology is the study of the distribution and  determinants  of health-

related states or events in specified populations, and the  application  of this study to
the control of health problems “

DISTRIBUTION:

Epidemiology is concerned with the frequency and pattern of health events in a population:

Frequency refers not only to the number of health events such as the number of cases of
meningitis or diabetes in a population, but also to the relationship of that number to the size of
the population. The resulting rate allows epidemiologists to compare disease occurrence across
different populations.

Pattern refers to the occurrence of health-related events by time, place, and person. Time
patterns may be annual, seasonal, weekly, daily, hourly, weekday versus weekend, or any other
breakdown of time that may influence disease or injury occurrence. Place patterns include
geographic variation, urban/rural differences, and location of work sites or schools. Personal
characteristics include demographic factors which may be related to risk of illness, injury, or
disability such as age, sex, marital status, and socioeconomic status, as well as behaviors and
environmental exposures.

Characterizing health events by time, place, and person are activities of descriptive
epidemiology, discussed in more detail later in this lesson.

DETERMINANT:

Determinant: any factor, whether event, characteristic, or other definable entity, that brings
about a change in a health condition or other defined characteristic.
Epidemiology is also used to search for determinants, which are the causes and other factors
that influence the occurrence of disease and other health-related events. Epidemiologists
assume that illness does not occur randomly in a population, but happens only when the right
accumulation of risk factors or determinants exists in an individual. To search for these
determinants, epidemiologists use analytic epidemiology or epidemiologic studies to provide the
“Why” and “How” of such events. They assess whether groups with different rates of disease
differ in their demographic characteristics, genetic or immunologic make-up, behaviors,
environmental exposures, or other so-called potential risk factors. Ideally, the findings provide
sufficient evidence to direct prompt and effective public health control and prevention
measures.

HEALTH – RELATED STATES OR EVENTS

Epidemiology was originally focused exclusively on epidemics of communicable diseases ( 3) but

was subsequently expanded to address endemic communicable diseases and non-
communicable infectious diseases. By the middle of the 20th Century, additional epidemiologic
methods had been developed and applied to chronic diseases, injuries, birth defects, maternal-
child health, occupational health, and environmental health. Then epidemiologists began to look
at behaviors related to health and well-being, such as amount of exercise and seat belt use.
Now, with the recent explosion in molecular methods, epidemiologists can make important
strides in examining genetic markers of disease risk. Indeed, the term health-related states or
events may be seen as anything that affects the well-being of a population. Nonetheless, many
epidemiologists still use the term “disease” as shorthand for the wide range of health-related
states and events that are studied.

SPECIFIED POPULATIONS

Although epidemiologists and direct health-care providers (clinicians) are both concerned with
occurrence and control of disease, they differ greatly in how they view “the patient.” The
clinician is concerned about the health of an individual; the epidemiologist is concerned about
the collective health of the people in a community or population. In other words, the clinician’s
“patient” is the individual; the epidemiologist’s “patient” is the community. Therefore, the
clinician and the epidemiologist have different responsibilities when faced with a person with
illness. For example, when a patient with diarrheal disease presents, both are interested in
establishing the correct diagnosis. However, while the clinician usually focuses on treating and
caring for the individual, the epidemiologist focuses on identifying the exposure or source that
caused the illness; the number of other persons who may have been similarly exposed; the
potential for further spread in the community; and interventions to prevent additional cases or
recurrences.

APPLICATION:

Epidemiology is not just “the study of” health in a population; it also involves applying the
knowledge gained by the studies to community-based practice. Like the practice of medicine,
the practice of epidemiology is both a science and an art. To make the proper diagnosis and
prescribe appropriate treatment for a patient, the clinician combines medical (scientific)
knowledge with experience, clinical judgment, and understanding of the patient. Similarly, the
epidemiologist uses the scientific methods of descriptive and analytic epidemiology as well as
experience, epidemiologic judgment, and understanding of local conditions in “diagnosing” the
health of a community and proposing appropriate, practical, and acceptable public health
interventions to control and prevent disease in the community.

A.4. TWO BROAD TYPES OF EPIDEMIOLOGY

 Descriptive Epidemiology
 o Examining the distribution of disease in a population, and observing the basic
features of its distribution

 Analytic Epidemiology
o Testing a hypothesis about the cause of disease by studying how exposures
relate to the disease

A.5. PURPOSE OF EPIDEMIOLOGY

To provide a basis for developing disease control and prevention measures for groups at risk.
This translates into developing measures to prevent or control disease.

A.6. USES OF EPIDEMIOLOGY

Epidemiology and the information generated by epidemiologic methods have been used in
many ways. Some common uses are described below.

Assessing the community’s health

Public health officials responsible for policy development, implementation, and evaluation use
epidemiologic information as a factual framework for decision making. To assess the health of a
population or community, relevant sources of data must be identified and analyzed by person,
place, and time (descriptive epidemiology).

 What are the actual and potential health problems in the community?
 Where are they occurring?
 Which populations are at increased risk?
 Which problems have declined over time?
 Which ones are increasing or have the potential to increase?
 How do these patterns relate to the level and distribution of public health services
available?

More detailed data may need to be collected and analyzed to determine whether health services
are available, accessible, effective, and efficient. For example, public health officials used
epidemiologic data and methods to identify baselines, to set health goals for the nation in 2000
and 2010, and to monitor progress toward these goals.

Making individual decisions

Many individuals may not realize that they use epidemiologic information to make daily
decisions affecting their health. When persons decide to quit smoking, climb the stairs rather
than wait for an elevator, eat a salad rather than a cheeseburger with fries for lunch, or use a
condom, they may be influenced, consciously or unconsciously, by epidemiologists’ assessment
of risk. Since World War II, epidemiologists have provided information related to all those
decisions. In the 1950s, epidemiologists reported the increased risk of lung cancer among
smokers. In the 1970s, epidemiologists documented the role of exercise and proper diet in
reducing the risk of heart disease. In the mid-1980s, epidemiologists identified the increased
risk of HIV infection associated with certain sexual and drug-related behaviors. These and
hundreds of other epidemiologic findings are directly relevant to the choices people make every
day, choices that affect their health over a lifetime.

Completing the clinical picture

When investigating a disease outbreak, epidemiologists rely on health-care providers and

laboratorians to establish the proper diagnosis of individual patients. But epidemiologists also
contribute to physicians’ understanding of the clinical picture and natural history of disease. For
example, in late 1989, a physician saw three patients with unexplained eosinophilia (an increase
in the number of a specific type of white blood cell called an eosinophil) and myalgias (severe
muscle pains). Although the physician could not make a definitive diagnosis, he notified public
health authorities. Within weeks, epidemiologists had identified enough other cases to
characterize the spectrum and course of the illness that came to be known as eosinophilia-
myalgia syndrome. More recently, epidemiologists, clinicians, and researchers around the world
have collaborated to characterize SARS, a disease caused by a new type of coronavirus that
emerged in China in late 2002. Epidemiology has also been instrumental in characterizing many
non-acute diseases, such as the numerous conditions associated with cigarette smoking — from
pulmonary and heart disease to lip, throat, and lung cancer.

Searching for causes

Much epidemiologic research is devoted to searching for causal factors that influence one’s risk
of disease. Ideally, the goal is to identify a cause so that appropriate public health action might
be taken. One can argue that epidemiology can never prove a causal relationship between an
exposure and a disease, since much of epidemiology is based on ecologic reasoning.
Nevertheless, epidemiology often provides enough information to support effective action.
Examples date from the removal of the handle from the Broad St. pump following John Snow’s
investigation of cholera in the Golden Square area of London in 1854, to the withdrawal of a
vaccine against rotavirus in 1999 after epidemiologists found that it increased the risk of
intussusception, a potentially life-threatening condition. Just as often, epidemiology and
laboratory science converge to provide the evidence needed to establish causation. For
example, epidemiologists were able to identify a variety of risk factors during an outbreak of
pneumonia among persons attending the American Legion Convention in Philadelphia in 1976,
even though the Legionnaires’ bacillus was not identified in the laboratory from lung tissue of a
person who had died from Legionnaires’ disease until almost 6 months later.

B. RATIONALE OF STUDYING BIOSTATISTICS

Biostatistics is the development and application of statistical models and theories of

inferential, probability and computing and mathematics responsible for interpreting the scientific
data generated in the health sciences. Statistical methods encompass designs of a wide range
of biological experiments. With the onset of pharmaceutical studies and clinical trials, it has
developed in the directions of clinical trial methodology, general linear models of various types,
survival analysis, longitudinal models, and health outcome research. It infers the experimental
data in the health sphere to understand countless pressing and crucial health and other
development issues like:

Application of biostatistics
Medicine Public Health/Health Large Data Future
economics and policy
Chronic diseases such Environmental health. Cost pattern
as Diabetes, AIDS, The relationship between identification
Cancer, Neurological genetics & the Health data mining
conditions, and environment. Smoking Medicare data. Illness behavioral
Palliative care cessation Prevention Brain imaging profiles
utilization Genomics

What are the roles of biostatistics in clinical research?

Many students studying medicine find it unnecessary in their initial years of study to learn
statistics.  But the reality is many profound doctors feel the need biostatistics since in the time
of their study it was not in the medical study curriculum.  The doctors need to study
biostatistics not only for researchers but also to enhance their reading capability of  medical
literature. Only with the introduction of evidence-based medicines as a cornerstone for medical
management, biostatistics came into vital prevalence.

What is clinical research?

All systematic researches carried out on human beings to generate data for discovering or
verifying the pharmacological, clinical or adverse effects with the objective of determining
efficacy and safety of the new drug are called clinical researches. They bring in the following
benefits to humankind:
 Assess the relative benefits of competing therapies
 Investigating proposed medical treatments
 Establishing optimal treatment combinations
 Improving knowledge of disease
 Developing diagnostic methods
 New treatments & medical devices

The role of biostatistics in clinical research methodology:

Guidelines of evidence-based medicine are squarely depended on meta-analysis.  It is, in turn,
is based on many randomized controlled clinical types of research. Only this will provide rock-
solid evidence to help doctors in making decisions on the best management options for
patients. A firm understanding of statistical methods only will facilitate the understanding of
the randomized clinical research methods and results. Hence biostatistics plays an important
role in evidence-based medicine, which is the base for doctors to recommend treatments for
patients now.

The other roles of biostatistics in clinical research:

 It is crucial for any clinical research from design, conduct, analysis, and reporting in
terms of controlling and minimizing biases, confounding factors, and measuring random
errors.  
 They provide formal accounting for sources of variability n patients’ response to
treatment.
 It helps the clinical researcher to form reasonable and precise inferences from all
collected information to make the correct decisions during the presence of uncertainty.
 Since clinical research is critical for a new drug for its efficacy, it is helped by
biostatistics in drug development.  It helps in the following areas:
1. Protocol & study design (e.g. randomized trials)
2. Data management
3. Sample size calculation for dose response studies, microarray studies and
Bayesian approaches
4. Misclassification
5. Effect modification
6. confounding
7. Study implementation
8. Study monitoring for safety and efficacy
9. Data analysis
10. Data reporting

Roles and Importance of Biostatistics in Clinical Research:

1. Protocol Development
In the protocol development in clinical researches, the following are the roles and
responsibilities of the biostatistician
 Objectives:
Based on the objective of the scientific manuscript, biostatisticians have to provide a clear
specification of the hypothesis to be tested. In other words, they have to provide the
parameters to be tested. Also, they are responsible for selecting and defining endpoints in
clinical research.
 Study Design:
The study design of the biostatistician should provide the data needed to answer the objectives
like:
1. Defining procedures to minimize selection bias
2. In the case of RCT or Randomized Control Trial, define randomization procedures like
sequence generation and allocation concealment and the length to follow up and frequency
of contacts.
 Sample size:
1. Justifying the primary endpoint in terms of power or precision
2. Methods used to calculate the sample size should be consistent with the primary method
of data analysis and also appropriate for the design
3. Assumptions should be supported with proper historical data.
4. Justification in terms of feasibility
 Analysis plan summary:
1. The purpose of analyzing plan summary is to assure objectives to be achieved and to
justify design and data collection byways like
2. To provide the statistical methodology for the assessment of the primary objectives like
testing procedures & statistical hypothesis
3. As per the role of DSMB or the Data Safety Monitoring Board to discuss statistical
methods to be used in planned interim analyses
 Protocol Review:
It is pertinent for the LSB or the lead study biostatistician to review the full protocol for
checking the following
1. Clarity
2. Completeness
3. Consistency
4. Data quality issues
5. Feasibility
 Protocol writing:
To write randomization procedures, sample size, and analysis plan with the inputs from
objectives, endpoints study design, and allocation concealment.

2. Data Management:
1. CRF management with content and design
2. Dataset specification with annotation of CRFs and record layout
3. Validation with error checking specification and test data
3. Study Implementation
It involves sampling selection and implementation of randomizes procedures

4. Study Monitoring
It involves monitoring of quality and for safety and efficacy

5. Data Analysis:
 A detailed analysis plan writing with all hypotheses to be tested along with the
hierarchy of analysis
 It helps to prepare for reporting, manuscript writing, along with the validity and
creditability of results
6. Reports or Manuscript writing:
 Method section with statistical methodology and description of data with endpoints and
design
 Result section includes data presented in the form of a graph, tables, and others
 Discussion section with the appropriate interpretation of the results
Biostatistics has become crucial for the modern-day clinical researches to understand them
better and for various other uses.

C. THE FOUR SCALES OF MEASUREMENT

Data can be classified as being on one

of four scales: nominal, ordinal,
interval or ratio. Each level of
measurement has some important
properties that are useful to know. For
example, only the ratio scale has
meaningful zeros.

1. Nominal Scale. Nominal variables

(also called categorical variables) can be
placed into categories. They don’t
have a numeric value and so cannot
be added, subtracted, divided or
multiplied. They also have no order; if
they appear to have an order then you
probably have ordinal variables instead.

A pie chart displays groups of nominal variables

(i.e. categories).
 2. Ordinal Scale. The ordinal scale contains
things that you can place in order. For
example, hottest to coldest, lightest to
heaviest, richest to poorest. Basically, if you
can rank data by 1st, 2nd, 3rd place (and so
on), then you have data that’s on an ordinal
scale.
The ordinal scale classifies according to rank.

3. Interval Scale. An interval scale has

ordered numbers with meaningful divisions.
Temperature is on the interval scale: a difference of 10
degrees between 90 and 100 means the same as 10
degrees between 150 and 160. Compare that to high
school ranking (which is ordinal), where the difference
between 1st and 2nd might be .01 and between 10th
and 11th .5. If you have meaningful divisions, you have
something on the interval scale.

4. Ratio Scale. The ratio scale is exactly the same

as the interval scale with one major difference: zero is meaningful. For example, a
height of zero is meaningful (it means you don’t exist). Compare that to a temperature
of zero, which while it exists, it doesn’t mean anything in particular (although
admittedly, in the Celsius scale it’s the freezing point for water).

Weight is measured on the ratio scale