Skip to main content

Evidence-Based Medicine: Experimental Studies

What is an experimental study?

The hallmark of the experimental study is that the allocation or assignment of individuals is under control of investigator and thus can be randomized. The key is that the investigator controls the assignment of the exposure or of the treatment but otherwise symmetry of potential unknown confounders is maintained through randomization. Properly executed experimental studies provide the strongest empirical evidence. The randomization also provides a better foundation for statistical procedures than do observational studies.

Types of Experimental Studies

A prospective, analytical, experimental study using primary data generated in the clinical environment. Individuals similar at the beginning are randomly allocated to two or more treatment groups and the outcomes the groups are compared after sufficient follow-up time. Properly executed, the RCT is the strongest evidence of the clinical efficacy of preventive and therapeutic procedures in the clinical setting.

A prospective, analytical, experimental study using primary data generated in the clinical environment. Individuals with a chronic condition are randomly allocated to one of two treatment groups, and, after a sufficient treatment period and often a washout period, are switched to the other treatment for the same period. This design is susceptible to bias if carry over effects from the first treatment occur. An important variant is the "N of One" clinical trial in which alternative treatments for a chronically affected individual are administered in a random sequence and the individual is observed in a double blind fashion to determine which treatment is the best.

A prospective, analytical, experimental study using primary data generated in the laboratory environment. Laboratory studies are very powerful tools for doing basic research because all extraneous factors other than those of interest can be controlled or accounted for (e.g., age, gender, genetics, nutrition, environment, co-morbidity, strain of infectious agent). However, this control of other factors is also the weakness of this type of study. Animals in the clinical environment have a wide range of all these controlled factors as well as others that are unknown. If any interactions occur between these factors and the outcome of interest, which is usually the case, the laboratory results are not directly applicable to the clinical setting unless the impact of these interactions are also investigated.

Source Information