BIOSTATISTICS & EPIDEMIOLOGY BSMLS-2C

ARMAN ARQUILADA RMT 2025

TOPIC OUTLINE a study of phenomena

I. RESEARCH DESIGN in terms of
II. TYPOES OF DECRIPTIVE STUDY appearances of things,
DESIGNS or things as they
III. EPIDEMIOLOGY appear in our
IV. EPIDEMIOLOGIC STUDY DESIGNS experience, etc
V. HIERARYCHY OF STUDY DESIGNS (Wikipedia, et.al. )
VI. CORE ELEMENTS OF INVESTIGATING Ethnographic Ethnography is a
THE RELATIONSHIP BETWEEN DISEASE branch of anthropology
AND EXPOSURE IN OBSERVATIONAL and the systematic
STUDIES study of individual
i. Risk Ratio cultures. Ethnography
ii. Odds Ratio explores cultural
iii. POPULATION-BASED CASE-CONTROL phenomena from the
STUDY point of view of the
iv. HOSPITAL-BASED CASE-CONTROL subject of the study.
STUDY (Wikipedia)
Case Study an in-depth, detailed
examination of a
I. RESEARCH DESIGN
particular case within a
real-world context. For
➢ refers to the overall strategy utilized to carry out
example, case studies
research that defines a succinct and logical plan
in medicine may focus
to tackle established research question through
on an individual patient
the collection, interpretation, analysis, and
or ailment (wikipedia)
discussion of data. (Wikipedia)
Grounded Theory A research method
concerned with the
II. TYPES OF DESCRIPTIVE STUDY DESIGNS generation of theory,
which is “grounded” in
data that has been
QUANTITATIVE systematically
1. Descriptive Survey collected and
2. Descriptive Inferential analyzed. It is used to
uncover such things as
3. Descriptive Correlational social relationships and
4. Descriptive Analytical behaviors of groups,
QUALITATIVE known as social
process (https:/
1. Phenomenological /ebn.bmj.com/content/
2. Ethnographic 19/2/34)
3. Case Studies
4. Grounded Theory
III. EPIDEMIOLOGY
QUANTITATIVE
Descriptive Descriptive research is ➢ Branch of medical science that studies the
Survey used to describe distribution of disease in human populations and
characteristics of a the factors determining that distribution, chiefly by
population or phenomenon the use of statistics.
being studied. (Wikipedia) ➢ Unlike other medical disciplines, epidemiology
Descriptive Used to describe the concerns itself with groups of people rather than
Inferential results gathered using individual patients and is frequently retrospective,
different statistical analyses or historical, in nature.
to test a certain hypothesis. ➢ It developed out of the search for causes
of human diseases in the 19th century, and one
Descriptive Use to describe the
of its chief functions remains the identification of
Correlational existence of relationship
populations at high risk for a given disease so that
between two variables
the cause may be identified, and preventive
Descriptive Used to describe and
measures implemented.
Analytical analyze the cause and
➢ A variety of tools are used in the field of
effect of a certain
epidemiology. (Which will be discussed later on
phenomenon and derive at
the presentation).
a conclusion.
➢ The field of epidemiology is highly
interdisciplinary. In addition to its close ties to
statistics, particularly biostatistics, it relies heavily
QUALITATIVE on the concepts, knowledge, and theories of such
Phenomenological Phenomenology is the disciplines as biology, pathology,
philosophical study of and physiology in the health and biomedical
the structures of sciences as well as on the disciplines
experience and of anthropology, psychology, and sociology in
consciousness. This is the behavioral and social sciences.

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IV. EPDEMIOLOGIC STUDY DESIGNS o person (sex; age; occupation, etc.)

o place (geographic subdivisions; type of
DESCRIPTIVE (Hypothesis Formulation) terrain, etc)
➢ Describe a disease or health condition/ o time (month; season, etc.)
phenomenon or intervention ➢ Also used in health systems research to describe
Types of Descriptive prevalence according to patterns of health
service utilization and compliance. KAP surveys
a. Case reports/Case series
may also be categorized under this category
b. Prevalence survey (cross-sectional or
cohort)
c. Ecologic study ECOLOGIC STUDIES
➢ unit of observation and unit of analysis is an
ANALYTIC (Risk/ protective factors identified) aggregate rather than individual persons
➢ Examine association (test of hypothesis) ➢ most practical design to use when exposure level
is relatively homogeneous in a population but
Types of Analytic differs between populations (ex., water quality) or
a. Observational - Examine association (test of when individual measurements of exposure are
hypothesis) impossible (ex., air pollution)
o Cross-sectional ➢ they are used to generate hypothesis, or as a
o Case-control quick method of examining associations
o Cohort ➢ Its most serious flaw is the risk of ecological
o Ecologic fallacy -- i.e., the characteristics of the
b. Experimental - Exposure variables are geographical unit are incorrectly attributed to
assigned individuals
o Clinical trials (RCT)
Types of Analytic Study Designs
o Field trials
o Community intervention trials 1. Cohort studies Observational
2. Case-control studies studies
V. HIERARYCHY OF STUDY DESIGNS 3. Cross-sectional studies
4. Experimental studies and clinical trials
Meta-Analysis
Experiments/Intervention studies
Cohort studies
Validity & Statistical

Case-Control studies
Power

Cross-sectional studies

Prevalence studies/surveys

Ecologic studies

Case Series

Case Reports

CASE STUDY/ CASE SERIES

➢ a simple descriptive account of interesting
characteristics observed in a person or group
(series) of subjects
➢ Unique cases that cannot be explained by
known diseases or syndromes
➢ Cases that show an important variation of a
disease or condition
➢ Cases that show unexpected events that may
yield new or useful information
➢ Cases in which one patient has two or more
unexpected diseases or disorders
DESCRIPTIVE CROSS-SECTIONAL STUDIES/
PREVALENCE SURVEYS
➢ Involves the collection of data on the occurrence
and distribution of the disease of interest in
populations
➢ To characterize the disease, the prevalence is
usually computed for specific categories of
variables related to:
2 TYPES OF COHORT STUDY
1) PROSPECTIVE COHORT STUDY
➢ Begins with the initial collection of all exposure
and independent variables from the subjects at
the time of data collection.
➢ At this time, subjects are also excluded from the
study if they already have an outcome of interest.
Then once this baseline information is collected,
the subjects are followed into the future.
Example:
If a researcher wants to study rates of heart diseases in
older age, they would choose and age group of youg
adults with similar characteristics who do not have heart
disease to use as their baseline.
2) RETROSPECTIVE COHORT STUDY
➢ Also begins with the initial collection of the
exposure and all independent variables from the

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subjects, but these variables are ascertained Without the c d

from a time in the subjects' past. disease (D-)
➢ Then, the outcome information on the subjects is
determined up to the current time that the data
are collected. RISK RATIO
Example:
𝒂+𝒃 𝒊𝒏𝒄𝒊𝒅𝒆𝒏𝒄𝒆 𝒓𝒂𝒕𝒆 𝒊𝒏 𝒕𝒉𝒆 𝒆𝒙𝒑𝒐𝒔𝒆𝒅 𝒈𝒓𝒐𝒖𝒑
They might look at a group of older adults with heart Risk ratio= 𝒐𝒓
𝒄+𝒅 𝒊𝒏𝒄𝒊𝒅𝒆𝒏𝒄𝒆 𝒓𝒂𝒕𝒆 𝒊𝒏 𝒕𝒉𝒆 𝒏𝒐𝒏 𝒆𝒙𝒑𝒐𝒔𝒆𝒅 𝒈𝒓𝒐𝒖𝒑
disease. Then they would analyze data about the group
members' medical history to see what factors could have
contributed.
Risk Ratio Value Interpretation
Risk ratio=1 Null value Same rate of
DISADVANTAGES OF A COHORT STUDY DESIGN: outcomes in both
groups compared. No
1. Length of time and cost relationship exist
2. Inability to keep track of the enrolled study subjects between the groups
during the entire follow-up period in a prospective being compared in the
cohort study. ratio
3. There is also the possibility that only a few (or none) Risk ratio>1 Positive Rate above the
of the subjects will get the outcome during the association fraction line is greater
designed follow-up period. than the rate below
the fraction line.
Subjects in the
CASE CONTROL STUDY exposed group are
➢ Observational Study in which investigators collect more likely to have
data in a group of subjects with the outcome of the outcome of
interest, and also collect data in a group of interest.
subjects without the outcome of interest. (With
outcome vs. Without Outcome) Risk ratio<1 Negative Rate above the
a. "Cases" = with outcome association fraction line is less
than the rate below
b. "Control" = without outcome the fraction line.
Subjects in the
exposed group are
DISADVANTAGES OF CASE-CONTROL: less likely to have the
1. The biggest disadvantage of a case control study is outcome of interest.
the concern of obtaining valid information. In other
words, information bias is a big problem for case
control designs.
ODDS RATIO
➢ is a measure association that provides the
Example:
strength and direction of the association between
Dietary recall is difficult if being asked about how much exposure and outcome in a population.
longer than two days in the past.
(𝒂)(𝒅)
Smoking details, such as how many times subjects have 𝑶𝒅𝒅𝒔 𝑹𝒂𝒕𝒊𝒐𝒏 =
tried to quit or the number if packs smoked per day over (𝒃)(𝒄)
several years are often difficult to remember as well.
Odds Ratio Value Interpretation
Odds ratio=1 Null value means that
exposure to
CROSS-SECTIONAL STUDY
property A
➢ Investigates the relationship between existing does not affect
exposure characteristics and existing outcome the odds of
information in a group of enrolled subjects. property B.
Odds ratio >1 Positive Increased
association occurrence in
DISADVANTAGES OF CROSS-SECTIONAL: an event
➢ The most problematic disadvantage, is that the Odds ratio<1 Negative Decreased
cross-sectional design does not allow association occurrence of
investigators to determine whether exposure an event
occurred before the outcome or not (protective
exposure)

VI. CORE ELEMENTS OF INVESTIGATING THE

COHORT STUDY
RELATIONSHIP BETWEEN DISEASE AND SAMPLING POPULATION SAMPLES TO BE DATA TO BE COLLECTED IN THE
EXPOSURE IN OBSERVATIONAL STUDIES SELECTED AT THE START STUDY
OF THE STUDY
Population Exposed Number of indiv
DISEASE EXPOSURE STATUS without individuals with the disease (a)
STATUS disease Number of indiv
EXPOSED (E+) NEXPOSED (E-) without the disease
With the a b (b)
disease(D+)

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Unexposed Number of indiv CASE CONTROL STUDY

individuals with the disease (c) SAMPLING POPULATION SAMPLES TO BE DATA TO BE COLLECTED IN THE
Number of indiv SELECTED AT THE STUDY
START OF THE STUDY
without the
1. Population Population Number of exposed
disease(d)
with the with the individual (a)
disease(cases) disease
Number of
unexposed
individual (b)
2. Population Population Number of exposed
without the without the individual (c)
disease disease
(control) Number of
unexposed
EXAMPLE: individual (d)
Cohort Study of Smoking and Lung cancer ❖ Exposure data will be collected retrospectively
Sampling Samples To Be Data To Be Collected In The through personal interviews and/or records review
Population Selected At The Start Study
Of The Study EXAMPLE:
Population Individuals who Number with Lung
Case-Control on Hypertension and Physical Activity
without are smoking cancer (a)
Level
Lung cigarrette Number without Lung
cancer cancer (b) SAMPLING SAMPLES TO BE DATA TO BE COLLECTED IN THE
Individuals who Number with Lung POPULATION SELECTED AT STUDY
are not smoking cancer (c) THE START OF
cigarrette Number without Lung THE STUDY
cancer (d) 1. Population Population Number with active
with with the lifestyle (a)
hypertension hypertension
ADVANTAGES:
(cases)
➢ May yield information on the incidence of the Number with sedentary
disease lifestyle(b)
➢ Possible to compute for the relative risk 2. Population Population Number with active
➢ The temporal relationship between exposure without without lifestyle (c)
and disease is clearly defined. hypertension hypertension
➢ The design is particularly efficient for (control) Number with sedentary
studies involving rare exposure factors. lifestyle (d)
➢ It is the strongest observational design
for establishing cause-effect relationships.
ADVANTAGES:
DISADVANTAGES
• Feasible when dealing with rare disease
➢ Time-consuming
➢ Often requires a large sample size • Requires a smaller sample size than a cohort
➢ Expensive study
➢ Not efficient for the study of rare diseases • Little problem with attrition
➢ Losses to follow-up may diminish validity DISADVANTAGES:
➢ Changes over time in diagnostic methods
may lead to biased results • Incidence rates and attributable risks cannot be
computed
• The temporal sequence between disease and
PROSPECTIVE COHORT STUDY exposure may be a problem
EXPOSURE OUTCOME • Big chance for bias in the selection of cases and
controls
Assessed at start of study Followed-up into the
• Difficult to obtain information on exposure if
future
therecall period is too long.
• Selective survival may bias the comparison.
RETROSPECTIVE COHORT STUDY
EXPOSURE OUTCOME POPULATION-BASED CASE-CONTROL STUDY
Assessed at some point in Outcome has already ➢ Cases and controls are sampled from defined
the past for which records occurred population
are available
ADVANTAGES:
Design is also used when ✓ Source population is better defined.
there is simultaneous ✓ It is easier to make certain that cases and control
exposure to a factor (ex., come from the same source population
natural and man-made ✓ The exposure histories of the controls are more likely
disasters) to reflect those of persons without the disease of
interest.
✓

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HOSPITAL-BASED CASE-CONTROL STUDY

➢ Investigator selects cases from persons with the ATTRIBUTE COHORT CASE-CONTROL CROSS-
disease of interest who are admitted to a SECTIONAL
particular hospital Sampling Population without 1.Populationn Population with
➢ controls are selected from persons admitted with Population the disease with the disease both disease
other conditions but with no evidence of the (cases) and exposure
disease of interest status unknown
ADVANTAGES: 2.Population at start of study
✓ Subjects are more accessible. without the
✓ Subjects tend to be more cooperative. disease
✓ Background characteristics of cases and controls (controls)
may be balanced.
✓ Easier to collect exposure information from Temporal 1.Prospective (for Retrospective Current and/ or
medical records and biologic experiments sequence prospective retrospective
cohort)
CROSS-SECTIONAL STUDY 2.Retrospective
SAMPLING SAMPLES TO BE DATA TO BE COLLECTED (for Retrospective
POPULATION SELECTED AT THE IN THE STUDY cohort)
START OF THE STUDY
Population Sample of Number of
with both individuals from exposed
disease and the target individuals with
exposure population with disease (a)
status both disease Number of Use Compares Compares Describes
unknown at and exposure exposed incidence rates in prevalence of association
start of study status unknown individuals exposed and exposure among between
at start of study without disease unexposed cases and exposure and
(b) controls disease
Population Sample of Number of
whose individuals from exposed
physical the target individuals with
activity levels population disease (c)
and status with whose physical Number of
respect to activity levels exposed Measure of Incidence of Cannot be Prevalence of
hypertension and status with individuals disease disease among computed disease among
are unknown respect to without disease frequency exposed and exposed and
at start of hypertension are (d) unexposed groups exposed
study unknown at
start of study

ADVANTAGES:
✓ Less time-consuming and less costly than
prospective studies
✓ They often serve as the starting-point in Measure of 1.Relative risk Odds ratio 1.Prevalence
prospective cohort studies for screening-out association (estimate of ratio (inexact
already existing conditions between relative risk) estimate of
✓ The design allows the measurement of risk, 2.Attributable risk
disease and relative risk)
although the estimate is not precise
exposure 2.Odds ratio
DISADVANTAGES:
✓ It does not enable the direct estimation of risk.
✓ Prone to bias from selective survival
✓ Often difficult to establish the temporal sequence
of exposure factor and the disease

Experiments
➢ they provide the best evidence for testing any
hypothesis to investigate possible cause-effect
relationships
➢ They resemble cohort studies in that they
require follow-up of subjects to determine
outcome
➢ Distinguishing feature:
o action, manipulation or intervention on
the part of the investigator

MAIN CHARACTERISTICS OF AN EXPERIMENT

✓ Pre and post-treatment measurements made

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✓ Presence of a control group
✓ Random selection of subjects from a reference
population
✓ Random assignments of subjects and treatments
to groups
✓ HIgh degree of control over extraneous variables
PHASES OF EXPERIMENT DESIGNS
a. Level 1 or Phase 0 - Conduct preliminary parts
b. Level 2 or Phase 1 - Conduct animal assays
c. Level 3 or Phase 2 - Conduct of preclinical to clinical
trial (phase 1)
d. Level 4 or Phase 3 - conduct of clinical trial (phase 2
and phase 3) and mass production of the product
Why Are Pre And Post-Treatment Measurements
Needed?
➢ To enable measurement of change result in form
the treatment
o Change is often used as indicator of
effectiveness
used for initial Phase 1 clinical trial of the
drug
CLINICAL TRIALS
➢ Experimental designs used by clinicians and
epidemiologists to evaluate drugs, medical
devices and clinical or health care procedures
➢ The most common form of a clinical trial is the
randomized, controlled, double blind clinical trial
PHASES OF CLINICAL TRIALS
PHASE 1 Perform initial human testing in a small
group of healthy volunteers (about 20-100)
Major goal is to determine if drug is safe in
humans
PHASE 2 Test in a small group of patients (about 100
– 500)

Why Is A Control Group Needed? Objective is to determine possible short-term

side effects and risks associated with the
• TO determin whether or not change occurs even drug; if it works according to expected
in the absence of the treatment or intervention mechanism
COMMON VARIATIONS OF EXPERIMENTAL/QUASI-
EXPT’L DESIGNS PHASE 3 Test in a large group of patients (about
1000-5000) to show safety and efficacy
Pre-test Treatment Post-test
Experimental T X TTTTT
PHASE 4 Post-marketing surveillance of drug to
1 1 2 3 4 5…

Group
determine long-term safety and reassess
Control T 1 - TTTTT 5…
1 2 3 4
effectiveness, acceptability and continued
Group
use under normal field settings

❖ Used when the effect of more than one treatment or Sample of Protocols:
intervention is being studied
1. Remember to secure permit from BAI (animal
Pre- Treatment Post- utilization permit), UP- Biotechnology (biosafety
test test clearance for use of organisms and for genetic
manipulation) and DENR (gratuitous permit for
Experimental Group T 1 X T 2 the use of endemic plants)
1
HEPATOPROTECTIVE HEPATO-CURATIVE
Experimental Group T 1 X T 2
STUDY STUDY
2
1. Determine for liver 1. Induce liver
—------------- T 1 X T 2
enzymes (baseline data/ pre- damage (CCl4,
test) paracetamol)
Experimental group T 1 X T 2

T 2. desired concentration of the 2. Determine for liver

herbal extracts is added of an enzymes (pre-test)
Control Group T 1 - T 2 inducer of hepatic damage
(CCl4, paracetamol) 3. Induction of
desired concentration
❖ Used when the effect of more than one treatment 3. Determine for liver of the herbal extracts
or intervention is being studied enzymes (post- test)
4. Determine for liver
PRE-CLINICAL STUDIES 4. Compare with Controls enzymes (post- test)
➢ Experiments done prior to testing drugs in (+): Essentiale Forte
humans for purposes of (-): Distilled water 5. Compare with
o Isolating and characterizing active Reference: Sylimarin (Liver Controls
compounds Aid) (+): Essentiale
Forte
o Testing of absorption, distribution,
6. Histopathologic Exam or (-): Distilled
metabolism excretion and toxicological
Cytochemistry water
properties (ADME/Tox)
Reference:
o Pharmacology and toxicology in animals
Sylimarin (Liver
o Establishing no observable adverse
Aid)
effect levels to determine dosage to be

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6. Histopathologic
Exam or
Cytochemistry
HYPOGLYCEMIC STUDIES:
1. Determine for sugar level (baseline)
2. Induce hyperglycemic agent (Alloxan)
3. Determine for sugar enzymes (pre-test)
4. Induction of desired concentration of the herbal
extracts
5. Determine for sugar enzymes (post- test)
6. Compare with Controls
(+): Insulin (DM type 1) or Metformin (DM type 2)
(-): Distilled water
Reference: Charantia
7. Histopathologic Exam or Cytochemistry

OTHER NOTES:
Inductors:
• Hypotension studies: Carrageenan
• Hypolipidemia studies: Thiocetamide
MIXED METHODS RESEARCH

• Uses a combination of quantitative and qualitative

approaches in the conduct of the study
• Usually used when the research has several
objectives requiring either different types of
research designs to address them, or different
types of respondents

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