Measurement of long-term outcomes in observational and randomised controlled trials

Abstract

Background: Randomised controlled trials (RCTs) are the gold standard for evaluating treatment efficacy. However, the outcomes of RCTs often lack clinical utility and usually do not address real-world effectiveness.

Aims: To review how traditional RCTs may be triangulated with other methodologies such as observational studies and pragmatic trials by highlighting recently reported studies, outcomes used and their respective merits.

Method: Literature review focusing on drug treatment.

Results: Recently reported observational and some pragmatic studies show a degree of consistency in reported results and use outcomes that have face validity for clinicians.

Conclusions: No single experimental paradigm or outcome provides the necessary data to optimise treatment of mental illness in the clinical setting.