Clinical study design

Clinical study design is the formulation of trials and experiments, as well as observational studies in medical, clinical and other types of research (e.g., epidemiological) involving human beings.^[1] The goal of a clinical study is to assess the safety, efficacy, and / or the mechanism of action of an investigational medicinal product, or new drug or device that is in development, but potentially not yet approved by a health authority (e.g. FDA).

Some of the considerations here are shared under the more general topic of design of experiments but there can be others, in particular related to patient confidentiality and ethics.

Outline of types of designs for clinical studies

Treatment studies

• Randomized controlled trial
  • Blind trial
  • Non-blind trial
• Adaptive clinical trial
• Nonrandomized trial (quasi-experiment)
  • Interrupted time series design (measures on a sample or a series of samples from the same population are obtained several times before and after a manipulated event or a naturally occurring event) - considered a type of quasi-experiment

Observational studies

• Cohort study
  • Prospective cohort
  • Retrospective cohort
  • Time series study
• Case-control study
  • Nested case-control study
• Cross-sectional study
  • Community survey (a type of cross-sectional study)
• Ecological study

Important considerations

When choosing a study design, many factors must be taken into account. Different types of studies are subject to different types of bias. For example, recall bias is likely to occur in cross-sectional or case-control studies where subjects are asked to recall exposure to risk factors. Subjects with the relevant condition (e.g. breast cancer) may be more likely to recall the relevant exposures that they had undergone (e.g. hormone replacement therapy) than subjects who don't have the condition.

The ecological fallacy may occur when conclusions about individuals are drawn from analyses conducted on grouped data. The nature of this type of analysis tends to overestimate the degree of association between variables.

Seasonal studies

Conducting studies in seasonal indications (such as allergies, Seasonal Affective Disorder, influenza, and others) can complicate a trial as patients must be enrolled quickly. Additionally, seasonal variations and weather patterns can affect a seasonal study.^[2][3]

Other terms

• The term retrospective study is sometimes used as another term for a case-control study. This use of the term "retrospective study" is misleading, however, and should be avoided because other research designs besides case-control studies are also retrospective in orientation.
• Superiority trials are designed to demonstrate that one treatment is more effective than another. This type of study design is often used to test the effectiveness of a treatment compared to placebo.
• Non-inferiority trials are designed to demonstrate that a treatment is at least not appreciably worse than another. This type of study design is often employed when comparing a new treatment to an established medical standard of care.
• Equivalence trials are designed to demonstrate that one treatment is as effective as another.
• When using "parallel groups", each patient receives one treatment; in a "crossover study", each patient receives several treatments.
• A longitudinal study assesses research subjects over two or more points in time; by contrast, a cross-sectional study assesses research subjects at only one point in time (so case-control, cohort, and randomized studies are not cross-sectional).

See also

• Conceptual framework
• Epidemiological methods
• Epidemiology
• Experimental control
• Meta-analysis
• Operationalization
• Academic clinical trials

References

1. Miquel Porta (2014) "A dictionary of epidemiology", 6th edn, New York: Oxford University Press. ISBN 9780199976737.
2. Yamin Khan and Sarah Tilly. "Flu, Season, Diseases Affect Trials". Applied Clinical Trials Online. Retrieved 26 February 2010.
3. Yamin Khan and Sarah Tilly. "Seasonality: The Clinical Trial Manager's Logistical Challenge" (PDF). published by: Pharm-Olam International (POI). Retrieved 26 April 2010. External link in |publisher= (help)

External links

• Epidemiologic.org Epidemiologic Inquiry online weblog for epidemiology researchers
• Epidemiology Forum An epidemiology discussion and forum community to foster debates and collaborations in epidemiology
• Some aspects of study design Tufts University web site
• Comparison of strength Description of study designs from the National Cancer Institute
• Political Science Research Design Handbook Truman State University website