The Challenge of Assessing Treatment Effectiveness in a Real-World Setting

Real-world data derived from observational studies, particularly from administrative health care and insurance claims databases, are increasingly being used to evaluate treatment effectiveness. To control for potential biases, a number of analytical techniques have been developed. However, the procedures used can be far from intuitive, and this along with other methodological issues can make it challenging to assess whether reported results are real or artefactual. This commentary summarizes some of the issues associated with evaluating treatment effectiveness in the real-world setting, at the same time highlighting the important role observational studies can play.