Scribd
Upload
20 views22 pages

4.3. Unit four Experimental study design

Uploaded by

barajaalalaa133
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PDF, TXT or read online on Scribd
20 views22 pages

4.3. Unit four Experimental study design

Uploaded by

barajaalalaa133
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
Available Formats
Download as PDF, TXT or read online on Scribd
You are on page 1/ 22

Unit four

Experimental/Interventional study
designs

June, 2017
Hawassa, Ethiopia

1
Session objectives
 Define experimental study designs
 Differentiate experimental study designs from other study
designs
 List types of experimental study designs
 Describe important issues in the design and conduct of
clinical trials

2
3
Definition
 An experiment is a set of observations, conducted under
controlled circumstances, in which the scientist manipulates
the conditions to ascertain what effect such manipulation has
on the observations.

 The main distinction from other types of analytic studies is


that individuals are allocated into experiment or control
group by the investigators.

4
Key Features of Experimental Design
 Investigator manipulates the condition under
study

 Always prospective

5
Intervention trails could be done for various
purposes
 Proof of concept trail: designed solely to produce
knowledge about cause and effect, does not test the efficacy
of the intervention in actual practice.

 Prevention trail: Interventions are to prevent disease and


study participants are persons without disease.

 Clinical trail: Interventions are treatment based on drugs


and study participants are persons with disease.

6
Classification of Intervention Studies: Based on
population
 A. clinical trial - usually performed in clinical setting
and the subjects are patients.

 B. Field trial - used in testing medicine for preventive


purpose and the subjects are healthy people. E.g. vaccine
trial

 C. Community trial- unit of the study is group of


people/community. E.g. fluoridation of water to prevent
dental caries.
7
Classification of Intervention Studies: Based on design
 A. Uncontrolled trial - no control group. control will
be past experience (history).

 B. Non-randomized controlled- there is control group


but allocation into either group is not randomized.

 C. Randomized controlled - there is control group


and allocation into either group is randomized.

8
Classification of Intervention Studies: Based on Trial
Objective
 A. Phase I - trail on small subjects to test a new drug
with small dosage to determine the toxic effect.

 B. Phase II - trial on small group to determine the


therapeutic effect.

 C. Phase III- study on large population - usually a


randomized control trial.

9
Issues in the design and conduct of clinical trials
 Selection of a study population

 Allocation of study groups

 Maintenance and assessment of compliance

 Ascertainment of outcome

10
1. Selection of a study population
 Reference population: The general group to whom
investigators expect the results of the particular trial to be
applicable.
 Represents the scope of the public health impact of the
intervention. And, it is related to the issue of generalizability.

 Experimental population: The actual group in which the


trial is conducted.
 It is preferable that if this group is not different from the
reference population for the sake of generalizability

11
Considerations for Selection of study population
 Must be sufficiently large to achieve the required sample
size for the trial

 Must produce sufficient number of endpoints

 Likelihood of obtaining complete and accurate follow-up


information for the period of trial.

12
2. Allocation of study groups
 Allocation into either group must be done after
determining eligibility and getting consent. It is always
advantageous to do the allocation at random.

 Advantages:

 Treatment groups will not be known by the researcher.

 "On average" the study group will be comparable; i.e.,


known and unknown potential confounders will be
equally distributed between the two groups.
13
3. Enhancing Compliance during Follow-up
 Selection of study population that are both
interested and reliable.

 Frequent contacts with individuals

 Use incentives, such as providing medical


information

14
Assessment of compliance
 Self-report, the simplest and the only way to assess
behavioral modification and exercise programs.

 Pills count - ask participants to bring unused pills to each


clinic visit, this may eliminate inaccuracies due to poor
memory, it assumes that all the unreturned pills has been
ingested.

 Biochemical tests used to validate self-report , objective


but expensive and logistically difficult
15
4. Ascertainment of outcome
 Use uniform ascertainment of outcome for complete
follow-up period for all study subjects

 Maintain a high level of follow-up – Reduce the


proportion of outcomes that are not ascertained to the
minimum and comparable between the two groups

 Use of placebo and blinding to prevent bias in


identification and reporting of event of interest.

16
The Quality of “Gold Standard"
 Randomization

 Blinding

 Use of Placebo

17
Randomization
 Maximize the chance that both groups are the same at
baseline.

 Ideally, both groups are at the same stage of the natural


history of disease.

 Ideally, only one factor affecting the outcome of interest


would vary between the study groups.

18
Double -blinding
 Blinding prevents biases related to assignment,
assessment, or compliance.

 Eliminates the potential for observation bias:


– in reporting of side effects
– in assessing outcome

 Eliminates differential compliance or loss to follow-up.

19
Placebo
 Placebos are inert treatments intended to have no effect
other than the psychological benefit of offering treatment.
 Can only be used if there is no accepted treatment for the
condition under study.
 Use of placebo minimizes the bias in the ascertainment of
both subjective disease outcomes and side effects.
 It facilitates that both groups in the study gain equal attention.
 Placebo effect: tendency for individuals to report favourable
response to any therapy regardless of the physiologic efficacy
of what they received.

20
Major Problems Related to Intervention Studies
 Feasibility/practical problems related to
compliance

 Cost

 Ethics

21
22

You might also like