Outcomes research differs in many ways from more traditionally performed clinical research. Whereas conventional “bench laboratory” experiments are designed to quantify short-term biological parameters for a given number of subjects, outcomes research employs strategies and methods to determine how disease and clinical interventions affect patient populations over time. Often, these studies are carried out in hopes of affecting public policy. In order to do so, study design must be rigorous, satisfying several criteria to ensure successful execution and valid results. For example, groups of equally distributed patients must be carefully selected, ethical and disease appropriate interventions must be implemented, and endpoints must be identified to accurately reflect how these study subjects are affected. At the same time, critical interpretation of results requires that potential biases be acknowledged and accounted for. Furthermore, as we will see, the assessment of children undergoing surgical treatment carries with it certain challenges unique to this population.

A brief history of outcomes research

From the earliest days of contemporary medical practice, observers have questioned the effects of various medical therapies. In fact, the ability to judge and assess the effects of clinical intervention is a necessary prerequisite to gauge the evolution of medical treatment. Codman was among the first physicians to stress the importance of formally assessing the results of clinical intervention. As a result, he is considered the father of Outcomes Research. An orthopedic surgeon in Boston during the 1920s and 1930s, Codman espoused his “End Result Idea.”