ASSESSING

PREVENTIVE AND
THERAPEUTIC
MEASURES:
RANDOMIZED
TRIALS
CHAPTER 7
 UNPLANNED TRIAL
Been carried out many times when a
therapy thought to be the best
available has been in short supply and
has not been available for all the
patients who needed it.
 PLANNED TRIAL
Was described by the Scottish surgeon,
James Lind in 1747
He became interested in scurvy ― a
deficiency of Vitamin C, which killed
thousands of British seamen and conducted
an experiment and used lemon and oranges
as treatment
 RANDOMIZED
TRIALS PURPOSES:
Used for evaluating new drugs and other
treatments of disease, including new tests of new
health and medical care technology

Used to asses new programs for screening and

early detection, or new ways of organizing and
delivering health services
 DESIGN OF A
RANDOMIZED TRIAL
 SELECTION OF SUBJECTS
Criteria for determining who will or will not be
included in the study must be spelled out with great
precision, and in writing before the study is begun

There should be no element of subjective decision-

making on the part of the investigator in deciding
who is included or not included in the study
 ALLOCATING SUBJECTS TO
TREATMENT GROUPS
WITHOUT RANDOMIZATION
STUDIES WITHOUT COMPARISON
- First possible alternative is the case study or case series. In
this type of study, no comparison is made with an untreated group
or with a group that is receiving some other treatment

STUDIES WITH COMPARISON

 Historical Controls
 Simultaneous Nonrandomized Controls
 WHAT IS THE MAIN
PURPOSE OF
RANDOMIZATION?

The main purpose of randomization is

to prevent any potential biases on the
part of the investigators from influencing
the assignment of participants to different
treatment groups
 STRATIFIED
RANDOMIZATION

An assignment method that can be very helpful in

increasing the likelihood of comparability of the study
groups
In here, we first stratify our study population by each
variable that we consider important, and then
randomize participants to treatment groups within
each stratum
DATA COLLECTION ON
SUBJECTS

The data collected for

each of the study groups
be of the same quality
TREATMENT (ASSIGNED AND RECEIVED)

- We must know to which treatment group the

patient was assigned. In addition, we must know
which therapy the patient actually received.

OUTCOME
- The need for comparable measurements in all srudy
groups is particularly true for measurements of
outcome. Such measurements include both
improvement and any side effects
PROGNOSTIC PROFILE AT ENTRY

- If we know the risk factors for a bad outcome, we

want to verify that randomization has provided
reasonable similarity between the two groups in
terms of these factors.

MASKING (BLINDING)
--commonly used to reduce the risk of bias in clinical
trials with two or more study groups.
PLACEBO
- An inert substance that looks, tastes,
and smells like the active agent.
 CROSSOVER
Two Types:

 Planned Crossover – this type of design is very attractive

and useful provided that certain cautions are taken into
account

 Unplanned Crossover – may occur if someone in one

treatment group needs to be medically managed like the
second treatment group instead because of health concerns.
FACTORIAL DESIGN
 NONCOMPLIANCE
Patients may agree to be randomized, but following
randomization they may not comply with the assigned treatment.

This may be overt or covert: On the other hand, people may

overtly articulate their refusal to comply or may stop participating
in the study and these dropouts from the study. On the other
hand, people may just stop taking the agent assigned without
admitting this to the investigator or the study staff