DIPLOMA OF HEALTHCARE

MANAGEMENT

DHM 1223
EPIDEMIOLOGY

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 DHM 1223
Lecture by: LALITA
ANBARASEN

CHAPTER 6: RESEARCH
METHODS, STUDY
DEIGN AND ANALYSIS
STUDIES
In an observational exploratory study a variety of associations are examined. Such studies are
useful for identifying clues as to cause–effect relationships. In an observational analytic study
evaluation of associations between exposure and outcome variables begins with a specific a
priori hypothesis.

Case-Control Study Design

Evaluate diseases with long latency periods and evaluate one or more exposure variables
associated with a given outcome.

A case-control study involves grouping people as cases (persons experiencing a health-related

state or event) and controls and investigating whether the cases are more or less likely than the
controls to have had past experiences, lifestyle behaviors, or exposures.

In other words, what is it about their past that made them cases? The outcome is always
identified before the exposure.

Because a case-control study begins with the outcome and looks back at an antecedent variable
or variables, it is retrospective in nature.
Selection of Cases- Establishing the diagnostic criteria and definition of disease is the first step in
conducting a case-control study. A strict diagnostic criterion for the disease will ensure that
cases reflect as homogeneous a disease entity as possible.

Selection of Controls- To better ensure that a case-control study is valid and reliable, the control
subjects should look like the case subjects with the exception of not having the disease. This
means that controls need to be selected from the same population from which the cases were
drawn.

Exposure Status - After the cases and controls have been identified, ascertainment of exposure
status is performed. Information about exposure status can be obtained through medical
records, interviews, questionnaires, or surrogates such as spouses, siblings, or employers.
Information on exposure status should be collected in a similar manner between cases and
controls to avoid bias.
Case-Crossover Study Design
The case-crossover design is becoming increasingly common in environmental epidemiology. In
a case-crossover study, each case serves as his or her own control, and the value of a time-
dependent exposure in the period just before the outcome occurred is compared with its value
at one or more previous control periods of time.

Nested Case-Control Study Design

A nested case-control study (also called a case-cohort study) is a case-control study “nested”
within a cohort study. A sample of cases and noncases are selected, and their exposure status is
compared. For example, a nested case-control study of 362 cases and 1,805 matched controls
attempted to examine the association between occupational chemical exposures and prostate
cancer incidence.
Cohort Study Design

Cohort as a general term means a group or body of people. As time passes, the group moves
through different and successive time periods of life; as the group ages, changes can be seen in
the health and vital statistics of the group.

Health factors as well as deaths are tracked in cohorts. In analytic epidemiology, a cohort study
generally involves the study of persons who have been exposed and are followed over time with
selected health outcomes compared with another group who have not been exposed.

Cohorts of persons within a group can be studied as a group, either prospectively or

retrospectively. In a prospective cohort study, the predictor variable is measured before the
outcome has occurred. In a retrospective cohort study, a historical cohort is reconstructed with
data on the predictor variable (measured in the past) and data on the outcome collected
(measured in the past after some follow-up period). The defining distinction between a
prospective and retrospective cohort study is the time when the investigator initiates the study,
whether before or after the occurrence of the outcome.
Experimental Study Designs

In 1747, James Lind conducted an experimental study by identifying a group of 12 sailors with
similar symptoms of scurvy.1 He divided them into six groups of 2 and supplemented their
regular diet with a specific dietary intervention. The intervention that produced a noticeable
improvement in the sailors was citrus fruit.

Clinical trial

Used to evaluate the efficacy and safety of a new drug or a new medical procedure; a
prophylactic trial is used to evaluate preventive measures; and a therapeutic trial is used to
assess new treatment methods.
Phase 1 Clinical trial.

- Conducted to a small number of group of human volunteers (10-30)

over 2-12 months
- To test the safety of the drug
- To identify the side effect

Phase 2 Clinical trial.

- Involve more volunteers than phase 1

- Consists of two or more groups receiving new doses of the same drug
- Effective doses have same level of side effect
- If the result suggest efficacy and safety phase 3 trial will be conducted
Phase 3 Clinical trial.

•Most phase 3 clinical trials include a large number of patients, at least

several hundred.
•These studies are often done in many places across the country (or
even around the world) at the same time.
•Phase 3 clinical trials are more likely to be offered in local community
hospitals and doctor's offices.
•These studies tend to last longer than phase I and II studies.
•Placebos may be used in some phase III studies, but they’re never
used alone if there’s a treatment available that works. Sometimes, a
patient who is randomly assigned to the placebo for part of the study
will at some point be offered the standard treatment as well.
Phase 4 Clinical trial.

Doctors can prescribe a drug for their patients after the FDA approves it. But the FDA
may require the sponsor to keep studying that approved treatment.

In these clinical trials, doctors may check if the treatment benefits people as much as it
did earlier. They also look for more possible side effects.

In a Phase IV clinical trial, doctors might study the drug or treatment in different doses,
or with other drugs or treatments. Or they might study how it works if people take it at
different times.

Drug makers may do phase IV clinical trials even if the FDA does not ask them to. They
might do this to get FDA approval to use the drug in a new way. For example, they might
want to use it for another type of cancer.

Phase IV clinical trials can also check the safety of drugs or treatments being used now.
They do this to make sure drug makers report any new or serious side effects. The FDA
may take away a drug's approval if new research shows it is not as safe or effective as
earlier testing showed. Doctors cannot prescribe it any longer if this happens.
Randomization

Participants can be randomly assigned to more than just two study groups when the efficacy of
various levels of a treatment or combinations of treatments are being investigated.

An important feature of randomization is that it balances out the effect of confounding.

Assuming that smoking is a confounding factor, randomization of a sufficiently large number of

participants will produce a similar distribution of smokers (in terms of age started, duration, and
intensity of smoking) between the intervention and control groups.

Although it is possible to adjust for confounding factors at the analysis phase of a study, this
assumes that data on the suspected confounders were collected at the outset of the study.

Nevertheless, although our best thinking might identify many possible confounding factors,
there may be some not considered. Randomization has the advantage of controlling for both
known and unknown confounders. Thus, randomization of a sufficiently large number of
participants produces groups that are alike on average.
Blinding

Blinding is used in experimental studies to minimize potential bias from the placebo effect.

A placebo is a substance containing no medication or treatment given to satisfy a patient’s

expectation to get well.

In some experimental studies that involve drug treatments, the placebos given to the control
group are virtually indistinguishable (to blind the patients and providers, when possible) from
the true intervention, providing a comparative basis for determining the effect of the treatment
being investigated. The placebo effect is the effect on patient outcomes (improved or worsened)
that may occur because of the expectation by a patient (or provider) that a particular
intervention will have an effect. The placebo effect is independent of the true effect
(pharmacologic, surgical, etc.) of a particular intervention. Just as a patient may respond to the
intervention itself and not the specific therapeutic benefit of the intervention, an assessing
investigator, albeit honest, may believe in a certain intervention, and unconscious bias may arise
in the way the researcher evaluates those participants who receive the intervention.

Blinding patients in controlled clinical trials in order to minimize the placebo effect dates back
about 100 years.
In a single-blinded study, the participants are unaware of who is receiving the active treatment,
but investigators are aware.

In a double-blinded study, neither the participants nor the investigators know who is receiving
the active treatment.

In a triple-blinded study, not only are the treatment and research approaches kept a secret from
the participants and investigators, but the nature of the interventions are kept from the person
analyzing the data.
End of
the
chapter