Thursday, December 13, 2018

Experimental Designs - Clinical Trials


The National Institutes of Health has defined a clinical trial as a research study in which one or more human participants are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.  In terms of strength of study design, the randomized clinical trial is considered the strongest design to evaluate the cause-and-effect relationship of an intervention.

Clinical trials can be classified as either therapeutic or preventive.  Therapeutic trials investigate the effect of a treatment (independent variable) on an outcome (dependent variable).

Preventive trials assess if a treatment (independent variable) is effective in reducing the risk of developing a condition or disease (dependent variable).

Various experimental designs exist and I will be discussing different designs in future posts.  As for now, I will be discussing different types of clinical trial designs.

The “gold standard” of experimental designs is the randomized clinical trial (RCT).  The RCT design is also referred to as a pretest-posttest control group design.  This design is used to compare two or more groups that are created by random assignment, where one group receives the experimental treatment and the other group(s) serve as a control or comparison.  The different groups are sometimes referred to as treatment arms.  All groups undergo pre-testing before receiving treatment and post-testing afterwards.  During pre- and post-testing, outcomes data are collected from the study participants.  If the experimental group experiences a greater change in outcomes between pre- and post-testing than the control group, one can infer that the treatment caused an effect.

The information in previous posts on sampling, validity of intervention study design, and threats to study validity help to explain how the RCT is a strong study design.  Below is a flowchart that illustrates a RCT.


http://www.consort-statement.org/consort-statement/flow-diagram


The posttest-only control group design is the same as the pretest-posttest control group, except pre-testing does not occur in the experimental group nor control/comparison group.  In some situations, pre-testing is not possible.

Factorial designs are clinical trials that test the effect of more than one treatment, where potential interactions between the treatments are evaluated.

Randomized block designs are clinical trials where investigators wish to control for a possible extraneous variable that may influence differences between groups.  In such cases, homogeneous blocks of study participants.  For example, investigators may think that gender could influence the differences in effects between groups.  Each gender group could be randomly assigned to either the experimental group or the control group.  If 24 participants are included in the study (12 male and 12 female), the males could randomly assigned to groups (6 males in each group) and the female could be randomly assigned to groups (6 females in each group).  Gender can now be considered an independent variable and differences between males and females can be evaluated.

For more information about clinical trial designs, click on the following link.

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