BIOSTATSTATISTICS AND EPIDEMIOLOGY

Course credit: 3 units (2 lectures, 1 laboratory)

Contact hours: 3 hours lecture, 2 hours laboratory
Ms. Katrine Paujana, RMT
Dr. Shela Tante, MD, PHSAE, RMT, RN

Topic: Medical Research and Study Designs

CI: Dr. Shela Tante, MD, PHSAE, RMT, RN

Look to link exposure and disease

What is Medical Research?
- What is the exposure?
 Medical Research may be regarded simply as the study of - Who are the exposed?
disease and health in human populations - What are the potential health effects?
 Here disease may be any adverse health outcome such as pre- - What approach will you take to study the relationship between exposure
term birth, it may not necessarily be a disease such as cancer. and effect?

Goals of Medical Research Basic Study Designs and their Application to Medical Research
 Describe the health status of populations by enumerating the Big Picture
occurrence of diseases, obtaining relative frequencies within
groups and discovering important trends  To prevent and control disease
 Explain the etiology of diseases by determining factors that
In a coordinated plan, look to
“cause” specific diseases or trends.
 Predict the number of disease occurrences and the distribution of  identify hypotheses on what is related to disease and may be “causing” it
health status within populations.  formally test these hypotheses
 Control the distributions of disease in the population by prevention
of new occurrences, eradication of existing cases, prolongation of - Study designs direct how the investigation is conducted
life for those with disease, or otherwise improving the health
status of afflicted persons. What designs exist to identify and investigate factors in disease?
Study Designs
Components of Medical Research
Measure Disease Frequency

- Quantity Disease

Assess distribution of disease

- Who is getting disease?
- Where is disease occurring?
- When is disease occurring?
 Formulation of hypotheses concerning causal and preventive factors

Identify determinants of disease

- Hypotheses are tested using epidemiologic studies

Types of Primary Studies

Timeframe of Studies
Descriptive Studies
Prospective Study
- describe occurrence of an outcome
 looks forward, looks to the future, examines future events, follows a
Analytic Studies
condition, concern or disease into the future
- describe the potential association between exposure and outcome

Basic Question in Analytic Medical Research

Are exposure and disease linked? Retrospective Study

 “To look back”, looks back in time to study events that have already
occurred

BMLS 2B Jacob Durana

 BIOSTATSTATISTICS AND EPIDEMIOLOGY
Course credit: 3 units (2 lectures, 1 laboratory)
Contact hours: 3 hours lecture, 2 hours laboratory
Ms. Katrine Paujana, RMT
Dr. Shela Tante, MD, PHSAE, RMT, RN

Disadvantages

 Cannot study cause and effect relationships

 Cannot assess disease frequency

Study Design Sequence

Hypothesis Formation Descriptive Studies

Analytical Studies – Study Designs

Analytic Epidemiology

Experimental Studies

 Randomized Controlled Clinical Trials (RCT)

 Community trials

Observational Studies

 Group data (i.e., we don’t have subject level info)

- Ecologic
 Individual data
Descriptive Studies - Cross-sectional
- Cohort
Case Reports - Case-control
- Case-crossover
 Detailed presentation of a single case or handful of cases
 Generally, report a new or unique finding
- e.g. previous undescribed disease
- e.g. unexpected link between diseases Experimental Studies
- e.g. unexpected new therapeutic effect  Treatment and/or exposures occur in a “controlled” environment
- e.g. adverse events  Planned research designs
 Clinical trials are the most well known experimental design. Clinical trials
Case Series
use randomly assigned data.
 Experience of a group of patients with a similar diagnosis  Community trials use non-random data
 Assesses prevalent disease
 Cases may be identified from a single or multiple sources
Observational Studies
 Generally report on new/unique condition
 Non-experimental
 May be only realistic design for rare disorders
 Observational because there is no individual intervention
Advantages  Treatment and/or exposures occur in a “non-controlled” environment
 Individuals can be observed prospectively, retrospectively, or currently (i.e.
 Useful for hypothesis generation cross-sectional)
 Informative for very rare disease with few established risk factors
 Characterizes averages for disorder Cross-sectional studies

BMLS 2B Jacob Durana

 BIOSTATSTATISTICS AND EPIDEMIOLOGY
Course credit: 3 units (2 lectures, 1 laboratory)
Contact hours: 3 hours lecture, 2 hours laboratory
Ms. Katrine Paujana, RMT
Dr. Shela Tante, MD, PHSAE, RMT, RN

 An “observational” design that surveys exposures and disease status at a
single point in time (a cross-section of the population)
Case – Control Studies
 an “observational” design comparing exposures in disease cases vs.
healthy controls from same population
 exposure data collected retrospectively
 most feasible design where disease outcomes are rare

Observational Studies and Timeframe

Case - Control Design

Cross-sectional Design

Case Control Study Strength and Limitation

Strengths

 Less expensive and time consuming

 Efficient for studying rare diseases

Limitations

 Inappropriate when disease outcome for a specific exposure is not known

at start of study
 Exposure measurements taken after disease occurrence
 Disease status can influence selection of subjects
Cross-sectional Studies
Hypothesis Testing: Case-Crossover Studies
 Often used to study conditions that are relatively frequent with long
duration of expression (nonfatal, chronic conditions)  Study of event triggers within an individual
 It measures prevalence, not incidence of disease  Still a Case and Control component, but information on both components
 Example: community surveys will come from the same individual who is ultimately a case.
 Not suitable for studying rare or highly fatal diseases or a disease with  Case component = hazard period which is the time period right before the
short duration of expression disease or event onset. For example, period of heavy lifting right before
an MI.
Disadvantages  Control component = control period which is a specified time interval other
than the hazard period.
- Weakest observational design, (it measures prevalence, not
For example, presence or absence of a period of heavy lifting prior to time
incidence of disease). Prevalent cases are survivors
right before MI.
- The temporal sequence of exposure and effect may be difficult or
impossible to determine
Epidemiologic Study Designs
- Usually don’t know when disease occurred
- Rare events a problem. Quickly emerging diseases are also
Cohort Studies
problem.
 an “observational” design comparing individuals with a known risk factor
Epidemiologic Study Designs or exposure with others without the risk factor or exposure

BMLS 2B Jacob Durana

 BIOSTATSTATISTICS AND EPIDEMIOLOGY
Course credit: 3 units (2 lectures, 1 laboratory)
Contact hours: 3 hours lecture, 2 hours laboratory
Ms. Katrine Paujana, RMT
Dr. Shela Tante, MD, PHSAE, RMT, RN

 looking for a difference in the risk (incidence) of a disease over time

 best observational design
 data usually collected prospectively

Cohort Design

Cohort Study
Strengths
Timeframe of Studies - Exposure status determined before disease detection
- Subjects selected before disease detection
Prospective Study - Can study several outcomes for each exposure

 looks forward, looks to the future, examines future events, follows a Limitations
condition, concern or disease into the future
- Expensive and time-consuming
- Inefficient for rare diseases or diseases with long latency
- Loss to follow-up

Experimental Studies
Prospective Cohort study
 Investigator can “control” the exposure
 Akin to laboratory experiments except living populations are the subjects
 Generally, involves random assignment to groups
 Clinical trials are the most well-known experimental design
 The ultimate step in testing causal hypotheses
 In an experiment, we are interested in the consequences of some
treatment on some outcome.
 The subjects in the study who actually receive the treatment of interest are
called the treatment group.
 The subjects in the study who receive no treatment or a different treatment
are called the comparison group or control group.

Epidemiologic Study Designs

Retrospective Study Randomized Controlled Trials (RCTs)
 “to look back”, looks back in time to study events that have already  a design with subjects randomly assigned to “treatment” and “comparison”
occurred groups
 provides most convincing evidence of relationship between exposure and
Retrospective Cohort study effect
 not possible to use RCTs to test effects of exposures that are expected to
be harmful, for ethical reasons
 the “gold standard” of research designs
 provides most convincing evidence of relationship between exposure and
effect
 trials of hormone replacement therapy in menopausal women found no
protection for heart disease, contradicting findings of prior observational
studies.

BMLS 2B Jacob Durana

 BIOSTATSTATISTICS AND EPIDEMIOLOGY
Course credit: 3 units (2 lectures, 1 laboratory)
Contact hours: 3 hours lecture, 2 hours laboratory
Ms. Katrine Paujana, RMT
Dr. Shela Tante, MD, PHSAE, RMT, RN

Experimental Design

Randomized Controlled Trials

Disadvantages

 Very expensive
 Not appropriate to answer certain types of questions
- it may be unethical, for example, to assign persons to
certain treatment or comparison groups

BMLS 2B Jacob Durana