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Evidence-Based Medicine: Observational Studies

What is an observational study?

The allocation or assignment of factors is not under control of investigator. In an observational study, the combinations are self-selected or are "experiments of nature". For those questions where it would be unethical to assign factors, investigators are limited to observational studies. Observational studies provide weaker empirical evidence than do experimental studies because of the potential for large confounding biases to be present when there is an unknown association between a factor and an outcome. The symmetry of unknown confounders cannot be maintained. The greatest value of these types of studies (e.g., case series, ecologic, case-control, cohort) is that they provide preliminary evidence that can be used as the basis for hypotheses in stronger experimental studies, such as randomized controlled trials.

Types of Observational Studies

A prospective, analytical, observational study, based on data, usually primary, from a follow-up period of a group in which some have had, have or will have the exposure of interest, to determine the association between that exposure and an outcome. Cohort studies are susceptible to bias by differential loss to follow-up, the lack of control over risk assignment and thus confounder symmetry, and the potential for zero time bias when the cohort is assembled. Because of their prospective nature, cohort studies are stronger than case-control studies when well executed but they also are more expensive. Because of their observational nature, cohort studies do not provide empirical evidence that is as strong as that provided by properly executed randomized controlled clinical trials.

A retrospective, analytical, observational study often based on secondary data in which the proportion of cases with a potential risk factor are compared to the proportion of controls (individuals without the disease) with the same risk factor. The common association measure for a case-control study is the odds ratio. These studies are commonly used for initial, inexpensive evaluation of risk factors and are particularly useful for rare conditions or for risk factors with long induction periods. Unfortunately, due to the potential for many forms of bias in this study type, case control studies provide relatively weak empirical evidence even when properly executed.

An observational analytical study based on aggregated secondary data. Aggregate data on risk factors and disease prevalence from different population groups is compared to identify associations. Because all data are aggregate at the group level, relationships at the individual level cannot be empirically determined but are rather inferred from the group level. Thus, because of the likelihood of an ecologic fallacy, this type of study provides weak empirical evidence.

A descriptive study of the relationship between diseases and other factors at one point in time (usually) in a defined population. Cross sectional studies lack any information on timing of exposure and outcome relationships and include only prevalent cases.

A descriptive, observational study of a series of cases, typically describing the manifestations, clinical course, and prognosis of a condition. A case series provides weak empirical evidence because of the lack of comparability unless the findings are dramatically different from expectations. Case series are best used as a source of hypotheses for investigation by stronger study designs, leading some to suggest that the case series should be regarded as clinicians talking to researchers. Unfortunately, the case series is the most common study type in the clinical literature.

Anecdotal evidence. A description of a single case, typically describing the manifestations, clinical course, and prognosis of that case. Due to the wide range of natural biologic variability in these aspects, a single case report provides little empirical evidence to the clinician. They do describe how others diagnosed and treated the condition and what the clinical outcome was.